#  @asgct ASGCT ASGCT posts on YouTube about community, education, hosted, the world the most. They currently have [------] followers and [---] posts still getting attention that total [---] engagements in the last [--] hours. ### Engagements: [---] [#](/creator/youtube::UCqBoSnKcP997h96ExhjKPbw/interactions)  ### Mentions: [--] [#](/creator/youtube::UCqBoSnKcP997h96ExhjKPbw/posts_active)  ### Followers: [------] [#](/creator/youtube::UCqBoSnKcP997h96ExhjKPbw/followers)  ### CreatorRank: [---------] [#](/creator/youtube::UCqBoSnKcP997h96ExhjKPbw/influencer_rank)  ### Social Influence **Social category influence** [stocks](/list/stocks) [finance](/list/finance) [countries](/list/countries) [technology brands](/list/technology-brands) [cryptocurrencies](/list/cryptocurrencies) [musicians](/list/musicians) **Social topic influence** [community](/topic/community), [education](/topic/education), [hosted](/topic/hosted), [the world](/topic/the-world), [pfizer inc](/topic/pfizer-inc) #4, [gene](/topic/gene), [health](/topic/health), [aav](/topic/aav), [target](/topic/target), [future](/topic/future) **Top assets mentioned** [Frontline Ltd. (FRO)](/topic/$fro) [Pfizer, Inc. (PFE)](/topic/pfizer) ### Top Social Posts Top posts by engagements in the last [--] hours "Hemophilia and Gene Therapy Hemophilia is a genetic disease that prevents blood from clotting properly leading to prolonged internal and external bleeding. There are existing treatments for hemophilia that require lifelong administration while gene therapy may offer a one-time treatment to address the cause of the disease. The subtitling of these videos was funded in part by a donation from Pfizer Inc. hemophilia American Society of Gene and Cell Therapy gene therapy hemophilia American Society of Gene and Cell Therapy gene therapy" [YouTube Link](https://youtube.com/watch?v=FKwqW9KXvuc) 2019-11-11T18:16Z 13.5K followers, 48.3K engagements "Viral Vectors Overview Vectors are essentially vehicles designed to deliver therapeutic genetic material such as a working gene directly into a cell. The subtitling of these videos was funded in part by a donation from Pfizer Inc" [YouTube Link](https://youtube.com/watch?v=Q6qk6Wh6cXU) 2020-09-01T14:03Z 13.5K followers, 173.1K engagements "Community Quick Take: Genetic Reports + Whats Next A common experience for people diagnosed with a genetic condition is receiving a genetic test result which often comes with many questions emotions and unfamiliar terms. In this unit Kim Nye founder of the TESS Research Foundation and parent advocate is joined by genetic counselor Dr. Lauren Beretich to walk through whats in a genetic report and how this important information can help guide a familys next steps. Read more: https://patienteducation.asgct.org/research-clinical-trials/community-quick-takes/genetic-reports-whats-next" [YouTube Link](https://youtube.com/watch?v=a67iIG-jl1I) 2026-02-18T12:30Z 13.5K followers, [--] engagements "Basics of AAV Gene Therapy Basics of AAV Gene Therapy - Steven Gray Education Session from the American Society of Gene & Cell Therapy's 22nd Annual Meeting" [YouTube Link](https://youtube.com/watch?v=p-nwUpPZF70) 2019-11-27T18:19Z 13.5K followers, 81.5K engagements "Spinal Muscular Atrophy Gene therapy is a fitting approach for diseases caused by a single gene mutation like SMA. It targets the cause of disease by delivering a fully functioning copy of the gene into motor neuron cells. Lets learn more about how it works. The subtitling of these videos was funded in part by a donation from Pfizer Inc" [YouTube Link](https://youtube.com/watch?v=1W5xRCwmcRA) 2019-02-01T09:00Z 12.5K followers, 45.3K engagements "Around the World: Oncology Hematology and Cancer-Focused Cell and Gene Therapy in India This webinar explores Indias rapid advancements in CAR T therapies including homegrown innovations. This program highlights clinical successes increased accessibility and research leadership showcasing Indias growing impact on global cancer and hematology gene therapy landscapes" [YouTube Link](https://youtube.com/watch?v=21rRJxbHg_s) 2025-10-06T14:43Z 13.5K followers, [---] engagements "NIH and FDA Cooperations - Dr. Barrie J. Carter PhD" [YouTube Link](https://youtube.com/watch?v=2J_yILgbIZY) 2011-09-30T16:39Z 12.6K followers, [---] engagements "Increasing Equitable Access with Hayley Lam PhD At #ASGCT2024 Dr. Lam (CIRM) spoke during a symposium on developing CGTs with a DEI-forward approach. In this interview she talks about incorporating DEI into the review criteria for CIRM's funded programs how to measure DEI and what she'd like to see in the long term for patients. Meeting registrants can watch this session (and all others) through June [--] on the virtual platform (https://annualmeeting.app.swapcard.com/c/asgct) and the app Host: Lynnea Olivarez incoming Communications Commmittee Chair Show your support for ASGCT:" [YouTube Link](https://youtube.com/watch?v=2VWV6Zd0rf4) 2024-06-07T21:24Z 13.2K followers, [--] engagements "Gene Therapy for Cardiac Symptoms in Friedreichs Ataxia Gene Therapy for Cardiac Symptoms in Friedreichs Ataxia - Hlne Puccio Scientific Symposium from the American Society of Gene & Cell Therapy's 22nd Annual Meeting" [YouTube Link](https://youtube.com/watch?v=6AYJ-C8a-c0) 2019-11-27T19:48Z 13.2K followers, [---] engagements "Cell Therapy Development for Solid Tumors with AffyImmune's Sonal Gupta MD PhD - The Issue Sonal Gupta MD PhD SVP and Head of Clinical Development at Affyimmune Therapeutics talks with host Emily Walsh Martin PhD to explore the challenges of cell therapy clinical trials and how to move patients quickly and thoughtfully through the steps required to receive therapy in aggressive solid tumor cancers. But first "weve moved straight from pumpkin spice season into ASGCT Annual Meeting registration season" as Dr. Walsh Martin says. So hop online (https://annualmeeting.asgct.org/register) and join" [YouTube Link](https://youtube.com/watch?v=7YGVBX7V0k0) 2024-12-10T11:18Z 12.1K followers, [--] engagements "Gene Therapy Approaches (2022 Update) Gene therapy cell therapy and gene editing are fields of biomedical research with a similar goal in mind: To treat disease by targeting the cause of the disease. Learn more below about these different approaches along with common challenges risks and benefits" [YouTube Link](https://youtube.com/watch?v=8DqG1D5DVUw) 2022-12-14T22:14Z 12.8K followers, 17.9K engagements "Community Quick Takes: Manufacturing Matters The gene therapy journey from lab to patient depends on something many people dont seemanufacturing. Gain insights on the complex behind-the-scenes process of making AAV gene therapies and why it matters to the patient community. This unit was developed in partnership with the Bespoke Gene Therapy Consortium (BGTC). Listen to Quick Takes from experts in the community on the following questions: 00:00 - What is a plasmid and how does it help in making a gene therapy 04:24 - Why are quality checks so critical when making gene therapies and what kinds" [YouTube Link](https://youtube.com/watch?v=BfIrgjL50Ro) 2025-07-30T11:01Z 13.5K followers, [---] engagements "Developing Therapeutic Platforms with Dr. P.J. Brooks During this episode of Soundbites of the Annual Meeting we spoke with P.J. Brooks PhD who is Deputy Director of the Division of Rare Diseases Research Innovation part of the National Center for Advancing Translational Sciences (NCATS) at the National Institutes of Health (NIH). In his conversation with ASGCT's Communications Committee Chair Lynnea Olivarez Dr. Brooks talked about: The impact of developing cell and gene therapies as platforms to treat more than one disease Projects he's involved in that are working towards the platform" [YouTube Link](https://youtube.com/watch?v=GBvJk-CR6c4) 2025-07-09T10:24Z 13.5K followers, [--] engagements "CRISPR Tools for Gene Editing in the Brain CRISPR Tools for Gene Editing in the Brain - Jeremy Day Scientific Symposium from the American Society of Gene & Cell Therapy's 22nd Annual Meeting" [YouTube Link](https://youtube.com/watch?v=GjhQfO-9hT8) 2019-11-27T19:02Z 12K followers, 36K engagements "Giants of Gene Therapy Podcast [--] - Terry Flotte MD Terry Flotte MD discusses the early days and possible future of gene therapy the use of AAV to deliver GT to targeted sites in the body and his inspiration behind becoming a physician scientist and medical school dean. He spoke one-on-one with ASGCT President Hans-Peter Kiem MD PhD. Welcome to the sixth episode of Giants of Gene Therapy Dr. Flotte is the Provost Dean and Celia and Isaac Haidak Professor at UMass Chan Medical School with faculty appointments in the Department of Pediatrics and the Horae Gene Therapy Center. He is also a" [YouTube Link](https://youtube.com/watch?v=IaEs8IbzN_0) 2023-11-02T21:19Z 11.9K followers, [---] engagements "The When What How and Why of FDA Engagement with Niki Gallo - The Issue Today Niki Gallo principal consultant of regulatory affairs at Halloran Consulting joins host Emily Walsh Martin for a deep dive into timeline management pitfalls the nuances of INDs and the rapidly evolving FDA landscape. Niki brings to the program her extensive experience shepherding innovative therapeutics through the regulatory process and timely insights into the complexities of transitioning from the bench to the bedside. Whether youre from industry or academia todays conversation will help wrap all our heads around" [YouTube Link](https://youtube.com/watch?v=J7NdGWMgILI) 2025-03-11T10:25Z 13.5K followers, [---] engagements "CRISPR Editing and AAV Integration in the Brain with Drs. Beverly Davidson and Bryan SimpsonThe . Drs. Beverly Davidson and Bryan Simpson join Molecular Therapy EiC Dr. Roland Herzog to discuss their recent paper Targeted long-read sequencing captures CRISPR editing and AAV integration outcomes in the brain which was published in Molecular Therapy. ASGCT is proud to present this episode of the Molecular Therapy Podcast in partnership with our upcoming Advancing Gene and Cell Therapy for Cancer conference which will be held on October [--] and [--] in Philadelphia and online. This landmark hybrid" [YouTube Link](https://youtube.com/watch?v=M7UvjIi7Sxs) 2024-09-10T20:45Z 12.8K followers, [---] engagements "Empowering Patients 2025: A Cell and Gene Therapies Summit - Day [--] This is the Day [--] recording of the inaugural virtual event held on March [--] [----]. This event will provide timely education and engaging discussions on cell and gene therapies (CGTs). In alignment with ASGCTs strategic plan the goal is to empower advocates patients and caregivers who are vital in developing treatments. Attendees will hear from experts in the CGT field as well as patient advocacy group leaders. Agenda: Partners in Progress: The Value of Patients and Advocates Intro to Gene Therapy Approaches Review of Clinical" [YouTube Link](https://youtube.com/watch?v=N4JfTDF18Tk) 2025-04-21T17:51Z 13.5K followers, [---] engagements "Empowering Patients 2025: A Cell and Gene Therapies Summit - Day [--] This is the Day [--] recording of the inaugural virtual event held on March [--] [----]. This event will provide timely education and engaging discussions on cell and gene therapies (CGTs). In alignment with ASGCTs strategic plan the goal is to empower advocates patients and caregivers who are vital in developing treatments. Attendees will hear from experts in the CGT field as well as patient advocacy group leaders. Agenda: Morning Coffee with Dr. Peter Marks CGT Manufacturing Basics The Journey from Early-stage Trials to Approval" [YouTube Link](https://youtube.com/watch?v=P09DChWdQE0) 2025-04-21T18:22Z 13.5K followers, [---] engagements "Dr. Snehal Naik: "I Hope You'll Join the Conversation" at the Policy Summit Hear directly from Snehal Naik PhD Chair of ASGCT's Regulatory Affairs Committee about why she encourages people to join her at the Policy Summit. Learn more and register today: http://asgct.org/policysummit" [YouTube Link](https://youtube.com/watch?v=Pkvk82i-gWc) 2025-09-23T16:34Z 13.5K followers, [--] engagements "The Importance of Long-term Data with Dolan Sondhi PhD - The Issue On this episode of The Issue Host Emily Walsh Martin PhD is tackling her absolute favorite subject: the importance of long-term data Emily is joined by Dolan Sondhi PhD of Weill Cornell Medical College to discuss her recent paper Twenty-Year Survival Analysis of Adeno-Associated Virus Vector Serotype 2-Mediated Gene Therapy to the Central Nervous System for CLN2 Disease. We hope to see all of your long-term data at the ASGCT Annual Meeting the premier event in cell and gene therapy next month Join us in New Orleans May 13-17" [YouTube Link](https://youtube.com/watch?v=RzpZB6yb_Zs) 2025-04-14T10:31Z 13.5K followers, [--] engagements "Education Session [---] Updates on Gene Therapies for Non Maligant Diseases of the Immune System" [YouTube Link](https://youtube.com/watch?v=UAjko4HGiNs) 2017-09-26T21:44Z 12.4K followers, [---] engagements "Molecular Therapy Podcast: COVID-19 Diagnostics with Dr. Maxim Berezovski Join Drs. Paloma Giangrande (Editor-in-Chief of Molecular Therapy Nucleic Acids and Chief Technology Officer at Eleven Therapy) and Maxim Berezovski (Professor of Bioanalytical Chemistry at the University of Ottawa). They will discuss a recent article published in Molecular Therapy Nucleic Acids by Dr. Berezovski and colleagues titled Discovery of DNA aptamers targeting SARS-CoV-2 nucleocapsid protein and protein-binding epitopes for label-free COVID-19 diagnostics" [YouTube Link](https://youtube.com/watch?v=Vy9l3Hy7V84) 2024-02-13T11:05Z 10.6K followers, [--] engagements "Update on Phase I/II Clinical Trial for Ornithine Transcarbamylase (OTC) Deficiency Using AAV Update on Phase I/II Clinical Trial for Ornithine Transcarbamylase (OTC) Deficiency Using AAV-mediated Gene Delivery - Cary Harding Scientific Symposium from the American Society of Gene & Cell Therapy's 21st Annual Meeting" [YouTube Link](https://youtube.com/watch?v=YJANZE36ojk) 2018-12-07T21:35Z 13.1K followers, [----] engagements "My 40-year Journey to Develop RNA Based Therapies My 40-year Journey to Develop RNA Based Therapies - John Rossi Plenary Session from the American Society of Gene & Cell Therapy's 22nd Annual Meeting" [YouTube Link](https://youtube.com/watch?v=YY9NhDMjX2s) 2019-12-04T21:15Z 12K followers, [---] engagements "Giants of Gene Therapy Podcast [--] - Kathy High MD Kathy High MD shares her personal and scientific journey from hemophilia to retinal disease during her one-on-one conversation with American Society of Gene & Cell Therapy President Hans-Peter Kiem MD PhD in the second episode of ASGCT's Giants of Gene Therapy. Dr. High is the president of therapeutics at AskBio. Before that she was the co-founder president and head of research and development at Spark Therapeutics where she oversaw the development approval and launch of Luxturna the first AAV gene therapy approved for use in the United States." [YouTube Link](https://youtube.com/watch?v=_nCKhaehym4) 2023-11-02T21:18Z 12.6K followers, [---] engagements "Limb-Girdle Muscular Dystrophy and Gene Therapy One muscular dystrophy sub-group is called limb-girdle muscular dystrophy or LGMD. These diseases weaken the muscles of a persons shoulders upper arms hips and thighs. Each subtype of LGMD is caused by a different inherited faulty gene. The subtitling of these videos was funded in part by a donation from Pfizer Inc" [YouTube Link](https://youtube.com/watch?v=a1OitIarHYo) 2019-10-24T10:00Z 12.6K followers, 30.5K engagements "What's the Difference Between Gene Therapy Cell Therapy and Gene Editing Gene therapy cell therapy and gene editing are fields of biomedical research with similar goals in mind: To treat disease by changing our bodies at a microscopic level. Specifically these approaches aim to change our genetic makeup. However each one changes our genetic material in slightly different ways. What do these approaches have in common and what makes them different Lets dive in. The subtitling of these videos was funded in part by a donation from Pfizer Inc" [YouTube Link](https://youtube.com/watch?v=aO4-KFEz8NE) 2019-01-17T10:00Z 13K followers, 70.4K engagements "The Big Picture: Accountability in an Evolving Ecosystem ASGCT is excited to host a thought-provoking conversation moderated by Jodi Wolff PhD to further deconstruct issues of responsibility and obligation in the post-approval gene therapy landscape. This fireside chat will address topics related to data capture for long-term monitoring for durability and safety standardization to enable comparability to facilitate/identify next-generation therapies and tensions that exist in the evolving landscape. ASGCT Ethics Webinar Accountability Gene Therapy Data Capture ASGCT Ethics Webinar" [YouTube Link](https://youtube.com/watch?v=auuiHWRfqU0) 2025-03-31T20:24Z 13.5K followers, [--] engagements "Pluripotent Cell-Derived T and NK Cells as a Cornerstone Approach for Cancer Immunotherapy Pluripotent Cell-Derived T and NK Cells as a Cornerstone Approach for Off-the-Shelf Cancer Immunotherapy - Bob Valamehr Education Session from the American Society of Gene & Cell Therapy's 22nd Annual Meeting" [YouTube Link](https://youtube.com/watch?v=b-PFP2Ba9oc) 2019-12-05T17:46Z 12.5K followers, [----] engagements "cGMP and Building Cohesive Teams with Christopher Choi PhD Roswell Park Comprehensive Cancer Ce. Host Emily Walsh Martin PhD is joined by Chris Choi PhD Roswell Park Comprehensive Cancer Center's Senior Vice President of Industry Partnerships and Technical Director of the centers new Good Manufacturing Practice (cGMP) Facility. Dr. Choi is also an associate professor of oncology. This wide-ranging conversation covers cell therapy development and manufacturing and how the growing Roswell Park Center is aiming to support sponsors in the CAR T and cell therapy space. Emily and Chris also discuss" [YouTube Link](https://youtube.com/watch?v=cT2U0s8x9kE) 2024-11-12T11:19Z 11.9K followers, [--] engagements "Research Briefing: Cell & Gene Therapy Approaches for HIV The Cell & Gene Therapy Approaches for HIV briefing hosted by ASGCT on December [--] [----] explored the potential of cell and gene therapies to address the challenges of curing HIV. Featuring researchers policymakers and a patient advocate the event highlighted advancements in CAR T-cell and AAV gene therapies as well as the importance of continued investment and advocacy in this field" [YouTube Link](https://youtube.com/watch?v=dMbcGcwqspI) 2024-12-05T20:12Z 12K followers, [--] engagements "Giants of Gene Therapy Podcast [--] - Jennifer Doudna PhD Jennifer Doudna PhD shares her personal journey to co-inventing CRISPR-Cas9 for gene editing and the promise of her discovery during a one-on-one conversation with ASGCT President Hans-Peter Kiem MD PhD. Welcome to the fifth episode of Giants of Gene Therapy Dr. Doudna has been at UC Berkeley since [----]. She is a professor in the departments of Molecular and Cell Biology and Chemistry the Li Ka Shing Chancellors Professor of Biomedical Science. Shes also the president and co-founder of the Innovative Genomics Institute. In [----] Dr. Doudna" [YouTube Link](https://youtube.com/watch?v=ddMF8BJ2AbU) 2023-11-02T21:22Z 10K followers, [--] engagements "IRB and Patient Safety - [--] Things You Must Know to Start a Human Gene Therapy Trial IRB and Patient Safety - [--] Things You Must Know to Start a Human Gene Therapy Trial - Sagar Vaidya Scientific Symposium from the American Society of Gene & Cell Therapy's 22nd Annual Meeting" [YouTube Link](https://youtube.com/watch?v=fa6hWOAJxvs) 2019-12-04T20:28Z 13.2K followers, [--] engagements "The Business of Advocates Advancing CGTs: Different non-profit business models and advice to PAGs This a recorded presentation from the workshop titled "The Business of Advocates Advancing CGTs" that took place during AM25" [YouTube Link](https://youtube.com/watch?v=fheAAUrrins) 2025-07-23T14:01Z 13.5K followers, [--] engagements "Gene Therapy for Genetic Hearing Loss Genetic hearing loss happens when changes in certain genes affect how the hearing system develops and works. Learn more about the genetic factors contributing to hearing loss and the ongoing research on gene therapies aimed at improving outcomes for individuals who are deaf or hard of hearing" [YouTube Link](https://youtube.com/watch?v=fiKPI5MjsHs) 2024-12-16T16:28Z 13.1K followers, [----] engagements "Mavis Agbandje-McKenna Ph.D. [----] ASGCT Outstanding Achievement Award Lecture During the [----] ASGCT Annual Meeting Mavis Agbandje-McKenna gave a talk on structure-guided AAV vectorology" [YouTube Link](https://youtube.com/watch?v=hXOvE_9LBBQ) 2021-03-10T21:26Z 12.6K followers, [----] engagements "Scientific Symposium [---] Progress Towards Gene Therapy for Muscular Dystrophies Organized by the" [YouTube Link](https://youtube.com/watch?v=icBz6zkysZY) 2017-09-26T21:44Z 13.2K followers, [--] engagements "RNA Exon Editing with Ascidian Therapeutics' Drs. Robert Bell and Michael Ehlers - The Molecular . Join Dr. Paloma Giangrande Editor-in-Chief of Molecular Therapy Nucleic Acids as she discusses RNA exon editing with Ascidian Therapeutics' leadership: CSO Dr. Robert Bell and CEO Dr. Michael Ehlers. Their conversation explores Dr. Bell's recent MTNA publication "RNA Exon Editing: Splicing the Way to Treat Human Diseases." Last Call: Submit your abstracts for ASGCT's [----] Annual Meeting in New Orleans by January 31st 11:59 PM ET. Present at cell and gene therapy's premier event and get published" [YouTube Link](https://youtube.com/watch?v=kfNqQk4vvVQ) 2025-01-07T11:35Z 12.2K followers, [--] engagements "Next Generation Oncolytic Immunotherapy Next Generation Oncolytic Immunotherapy - Howard Kaufman Education Session from the American Society of Gene & Cell Therapy's 22nd Annual Meeting" [YouTube Link](https://youtube.com/watch?v=mSPjH8WODNE) 2019-12-04T21:18Z 13.2K followers, [----] engagements "Inside the Molecular Therapy Family of Journals Join us as past ASGCT President Paula Cannon Ph.D. and our Editors-in-Chief take you behind the scenes of ASGCTs field-leading Molecular Therapy family of journals. Discover how these prestigious publications have shaped the landscape of cell and gene therapy research for the past [--] years. Learn more: asgct.org/molecular-therapy" [YouTube Link](https://youtube.com/watch?v=n1Tcj_dru1A) 2025-09-09T15:02Z 13.5K followers, [---] engagements "Nuclease-free Genome Editing Confers a Hepatocellular Growth Advantage for Corrected Cells in MMA Nuclease-free Genome Editing Confers a Hepatocellular Growth Advantage for Corrected Cells in Methylmalonic Acidemia (MMA) - Randy Chandler Scientific Symposium from the American Society of Gene & Cell Therapy's 22nd Annual Meeting" [YouTube Link](https://youtube.com/watch?v=railXA88-Zg) 2019-12-04T21:24Z 12.3K followers, [---] engagements "Ethics: from Clinical Trials to Products - Jonathan Kimmelman Ethics: from Clinical Trials to Products - Jonathan Kimmelman Scientific Symposium from the American Society of Gene & Cell Therapy's 21st Annual Meeting May [--] 2018" [YouTube Link](https://youtube.com/watch?v=sKF_p6KzUsc) 2018-12-07T21:24Z 13.2K followers, [---] engagements "25 Years of Molecular Therapy with Drs. Beverly Davidson and Jeff Chamberlain This episode is the third in a series hosted by Molecular Therapy Editor-in-Chief Joseph Glorioso PhD that will cover comprehensive reviews of critical developments in the field of gene and cell therapy over the past [--] years. In this episode Dr. Glorioso will discuss the reviews Current trends in gene therapy to treat inherited disorders of the brain with author Beverly Davidson PhD The Childrens Hospital of Philadelphia and The road toward AAV-mediated gene therapy of Duchenne muscular dystrophy with author Jeff" [YouTube Link](https://youtube.com/watch?v=vdlSa3B_RiE) 2025-07-16T10:39Z 13.5K followers, [---] engagements "Retinal Progenitor Cells for Treatment of Retinitis Pigmentosa - Henry Klassen Retinal Progenitor Cells for Treatment of Retinitis Pigmentosa - Henry Klassen Scientific Symposium from the American Society of Gene & Cell Therapy's 21st Annual Meeting" [YouTube Link](https://youtube.com/watch?v=wHMnrtujvu0) 2018-12-07T21:24Z 12.7K followers, [----] engagements "CAR T cells CAR T cells - Stephen Gottschalk Education Session from the American Society of Gene & Cell Therapy's 22nd Annual Meeting" [YouTube Link](https://youtube.com/watch?v=xToxcsN2ZuA) 2019-11-27T18:22Z 12.6K followers, [---] engagements "Around the World: Australasian Gene and Cell Therapy Society (AGCTS) ASGCT and AGCTS hosted two virtual seminars organized as part of ASGCT's Around the World in collaboration with the Coalition of International Gene Therapy Societies. PROGRAM Back to Base-ics: Developing targeted base-editing approaches for NF single nucleotide substitutions Presented by Dr Alexandra ODonohue Alexandra is a Postdoctoral Research Fellow in the Bioengineering & Molecular Medicine laboratory at the Childrens Hospital at Westmead. She received her Bachelor of Medical Science in molecular biology and genetics in" [YouTube Link](https://youtube.com/watch?v=ytPs0qMfOU4) 2024-11-22T21:34Z 12K followers, [--] engagements "Lunch & Learn: Meet the Patients Join us as patientshighlight their stories from navigating the diagnosis process to learning about and receiving investigational treatment options.We'll hear about life after receiving a therapy and patients' outlooks for the future of their disease communities" [YouTube Link](https://youtube.com/watch?v=0F_IdBo4G5A) 2023-02-03T22:36Z 13.3K followers, [---] engagements "Update on Phase I/II Clinical Trial of Intracerebral Administration of rAAV2/5hNAGLU Vector Update on Phase I/II Clinical Trial of Intracerebral Administration of rAAV2/5hNAGLU Vector in Children with MPSIIIB - Marc Tardieu Scientific Symposium from the American Society of Gene & Cell Therapy's 21st Annual Meeting" [YouTube Link](https://youtube.com/watch?v=3aaKv_gcab4) 2019-01-16T22:24Z 13.2K followers, [---] engagements "Antisense Strategies Against Non-Liver Targets Antisense Strategies Against Non-Liver Targets - Brett Monia Scientific Symposium from the American Society of Gene & Cell Therapy's 22nd Annual Meeting" [YouTube Link](https://youtube.com/watch?v=3sl7_RJWqgY) 2019-11-27T18:07Z 13.3K followers, [---] engagements "Inducing Immune Tolerance with Liver-directed Expression by AAV - Dwight Koeberl Inducing Immune Tolerance with Liver-directed Expression by AAV - Dwight Koeberl Education Session from the American Society of Gene & Cell Therapy's 21st Annual Meeting" [YouTube Link](https://youtube.com/watch?v=ASFN2KSX_vc) 2018-12-07T21:24Z 13.3K followers, [---] engagements "CNS Gene Therapies with Todd Carter PhD Todd Carter PhD (CSO at Voyager Therapeutics) talks with incoming Communications Committee Chair Lynnea Olivarez about changes in the approach to developing gene therapies for CNS disorders like Huntington's disease. Show your support for ASGCT: https://asgct.org/membership/donate See omnystudio.com/listener (https://omnystudio.com/listener) for privacy information" [YouTube Link](https://youtube.com/watch?v=AiLsuezUmCM) 2024-06-07T21:24Z 13.3K followers, [--] engagements "Gene Therapy Basics Gene therapy has been studied for more than [--] years and can help stop or slow the effects of disease on the most basic level of the human bodyour genes. And to understand how it works well start at the basics. The subtitling of these videos was funded in part by a donation from Pfizer Inc" [YouTube Link](https://youtube.com/watch?v=BxEoX6TkitY) 2019-01-17T10:00Z 13.3K followers, 387.6K engagements "Scientific Symposium [---] Progress Towards Gene Therapy for Muscular Dystrophies Organized by the" [YouTube Link](https://youtube.com/watch?v=C7HW12YLelg) 2017-09-26T21:44Z 13.3K followers, [---] engagements "Gene Therapy for GM1 Gangliosidosis GM1 gangliosidosis (GM1) is a rare genetic disorder that progressively damages cells primarily in the brain and spinal cord and other tissues as well. Learn how gene therapy may target the cause of disease to slow or stop its progression along with helpful information on clinical trials and staying informed. The subtitling of these videos was funded in part by a donation from Pfizer Inc" [YouTube Link](https://youtube.com/watch?v=GJS7jps1MPA) 2021-07-28T18:07Z 13.4K followers, [----] engagements "Adenovirus-Based Vaccine for COVID-19 Adenoviruses have been used in gene therapy and have also been researched for use in vaccines for decades. An adenovirus-based vaccine was authorized for emergency use to fight COVID-19 in the U.S. along with the two mRNA vaccines. Learn more about how the adenovirus-based vaccine works. The subtitling of these videos was funded in part by a donation from Pfizer Inc" [YouTube Link](https://youtube.com/watch?v=GMoW-2HTVII) 2021-04-07T19:00Z 13.3K followers, 63.8K engagements "RNA-based Drugs Including Delivery of Antibodies and Vaccines Using mRNA RNA-based Drugs Including Delivery of Antibodies and Vaccines Using mRNA - Drew Weissman Scientific Symposium from the American Society of Gene & Cell Therapy's 22nd Annual Meeting" [YouTube Link](https://youtube.com/watch?v=GkL06JH883E) 2019-12-05T18:17Z 13.2K followers, [----] engagements "Gene Therapy for Inherited Retinal Diseases Inherited retinal disordersor IRDsare diseases that can cause severe vision loss or even blindness. Each one is caused by a gene that is not working properly which creates an opportunity for gene therapy to help slow or stop disease progression. The subtitling of these videos was funded in part by a donation from Pfizer Inc" [YouTube Link](https://youtube.com/watch?v=JuEnCDByQQY) 2019-03-01T15:07Z 13.2K followers, 44.4K engagements "Design Synthesis and Application of Antibody Nucleic Acid Analogs as Novel Biopharmaceuticals Design Synthesis and Application of Antibody Nucleic Acid Analogs as Novel Biopharmaceuticals - Christopher Alabi Education Session from the American Society of Gene & Cell Therapy's 22nd Annual Meeting" [YouTube Link](https://youtube.com/watch?v=KfwYBSLcw6o) 2019-11-27T19:04Z 13.4K followers, [---] engagements "Huntington's Disease: Can Gene Therapy Help Huntingtons disease is a genetic disorder caused by a breakdown of nerve cells in the brain. The disease affects an individuals ability to move their mood and how they think. Theres currently no cure for Huntingtons disease but there are types of gene therapy approaches that may offer hope for managing or slowing symptoms. The subtitling of these videos was funded in part by a donation from Pfizer Inc" [YouTube Link](https://youtube.com/watch?v=LD5iX7OzUJs) 2019-10-15T08:00Z 13.3K followers, 28.3K engagements "CALD and MLD Leukodystrophies are a set of rare genetic disorders that affect how the central nervous system functions. Since these disorders are all caused by a faulty genes gene therapy might be able to help. Leukodystrophies are genetic disorders that lead to the deterioration or malformation of the myelin sheath a fatty insulation that wraps around nerve axons and is essential for proper neuron function. When the myelin sheath is altered it makes it hard for our nervous system to send messages to different parts of our body which can impair walking speech and vision. The subtitling of" [YouTube Link](https://youtube.com/watch?v=MBghKIUHcTI) 2019-02-19T17:49Z 13.4K followers, [----] engagements "ASGCT: The Home of Cell and Gene Therapy The American Society of Gene & Cell Therapy (ASGCT) has been the primary hub for professionals working in cell and gene therapy for more than [--] years. The work of ASGCT and our members is now more important than ever with therapeutics moving from laboratories into clinics and ultimately the lives of patients. Hear from ASGCT's members and elected leadership exactly why this community is an essential part of their careers and why new scientists should invest their time in ASGCT" [YouTube Link](https://youtube.com/watch?v=Ow-6fneEfkQ) 2022-08-18T16:38Z 13.2K followers, [----] engagements "Design and Translational Application of HSV Gene Vectors in Nervous System Diseases Design and Translational Application of HSV Gene Vectors in Nervous System Diseases - Joseph Glorioso Education Session from the American Society of Gene & Cell Therapy's 21st Annual Meeting May [--] 2018" [YouTube Link](https://youtube.com/watch?v=Pr8SMMtVNR0) 2018-12-07T21:24Z 13.3K followers, [----] engagements "mRNA Vaccines for COVID-19 The use of messenger RNA (mRNA) in medical interventions has been researched for the last [--] years including in vaccines hoping to fight diseases such as cancer rabies Ebola and the Zika virus. That means the research was ready to be applied to this new disease COVID-19. With the recent authorization of two mRNA vaccines for emergency use to fight COVID-19 and more vaccine candidates in development we can build protection against the virus in our communities if enough people are vaccinated. Learn more at patienteducation.asgct.org. The subtitling of these videos was" [YouTube Link](https://youtube.com/watch?v=SR-9Bo2CaRQ) 2020-12-30T14:30Z 13.3K followers, 37.4K engagements "Nucleic Acid Delivery Systems for RNA Therapy and Gene Editing - Daniel Anderson Nucleic Acid Delivery Systems for RNA Therapy and Gene Editing - Daniel Anderson Scientific Symposium from the American Society of Gene & Cell Therapy's 21st Annual Meeting" [YouTube Link](https://youtube.com/watch?v=TW7n6jWjL0o) 2018-12-07T21:24Z 13.3K followers, [---] engagements "Fabry Disease and Gene Therapy Fabry disease is a rare genetic disorder caused by a lacking enzyme due to a faulty gene. Gene therapy aims to offer a one-time treatment to slow or ideally stop progression of the disease by introducing a working GLA gene to provide instructions to the cells to produce the deficient enzyme. The subtitling of these videos was funded in part by a donation from Pfizer Inc" [YouTube Link](https://youtube.com/watch?v=UqPP732iJ-8) 2020-09-21T19:28Z 13.3K followers, [----] engagements "HSPC Therapies - Engineering Cell Fitness: Enhancing HSPC Homing and Engraftment HSPC Therapies - Engineering Cell Fitness: Enhancing HSPC Homing and Engraftment - Hal Broxmeyer Scientific Symposium from the American Society of Gene & Cell Theray's 22nd Annual Meeting" [YouTube Link](https://youtube.com/watch?v=_-xyIcygkjI) 2019-12-04T19:51Z 13.3K followers, [---] engagements "Selective Targeting of Mutant Huntingtin Using Stereopure Oligonucleotides Selective Targeting of Mutant Huntingtin Using Stereopure Oligonucleotides as a Potential Therapeutic Approach for Huntingtons Disease - Michael Panzara Scientific Symposium from the American Society of Gene & Cell Therapy's 22nd Annual Meeting" [YouTube Link](https://youtube.com/watch?v=_GycCoWcPKQ) 2019-12-05T18:19Z 13.2K followers, [---] engagements "The Issue - Unlocking New Therapeutic Potential: Ensomas Platform for Rare Disease and Oncology Host Emily Walsh Martin welcomes Dr. Jim Burns CEO of Ensoma to discuss the company's novel technological approach to treating both rare diseases and solid tumor oncology. Dr. Burns provides a detailed overview of Ensoma's platform and its application in their ongoing clinical trials including the recently announced first patient infusion for chronic granulomatous disease. Learn how this initial trial is set to inform future therapeutic opportunities for indications with high unmet medical need." [YouTube Link](https://youtube.com/watch?v=aJ3zysz9JWg) 2025-12-16T11:15Z 13.5K followers, [--] engagements "Tay-Sachs and Sandhoff Disease (GM2) Tay-Sachs disease and Sandhoff disease are the two most common types of GM2 gangliosidosis (GM2) a group of rare fatal genetic disorders that progressively damage nerve cells primarily in the brain and spinal cord. Learn how gene therapy is being researched as a potential one-time treatment that could slow or stop disease progression for both diseases. The subtitling of these videos was funded in part by a donation from Pfizer Inc" [YouTube Link](https://youtube.com/watch?v=e-4bmTEBj2M) 2021-09-13T20:05Z 13.4K followers, 33.8K engagements "Automated and Decentralized CAR-T cell Manufacturing using Lentiviral vectors - Boro Dropulic Automated and Decentralized CAR-T cell Manufacturing using Lentiviral vectors: Bench to Commercialization Scientific Symposium from the American Society of Gene & Cell Therapy's 22nd Annual Meeting" [YouTube Link](https://youtube.com/watch?v=h6sGKIYOHsQ) 2019-11-27T18:10Z 13.3K followers, [----] engagements "Universal Gene-Edited CAR T-Cell Immunotherapy Universal Gene-Edited CAR T-Cell Immunotherapy - Philippe Duchateau Scientific Symposium from the American Society of Gene & Cell Therapy's 22nd Annual Meeting" [YouTube Link](https://youtube.com/watch?v=jH-5jFKYJq4) 2019-12-05T18:53Z 13.4K followers, [---] engagements "Utilizing Expedited Designations in Moving Toward Market Approval Utilizing Expedited Designations in Moving Toward Market Approval - Anne-Virginie Eggimann Scientific Symposium from the American Society of Gene & Cell Therapy's 22nd Annual Meeting" [YouTube Link](https://youtube.com/watch?v=jIdvllQaQ28) 2019-12-05T18:55Z 13.3K followers, [---] engagements "House Cancer Caucus Briefing: Immunotherapies Hear from a panel of ASGCT members who spoke to the House Cancer Caucus and Hill staff about CAR T and immunotherapies for cancer indications" [YouTube Link](https://youtube.com/watch?v=jRKeBoR69CM) 2023-01-26T15:32Z 13.5K followers, [---] engagements "Community Quick Takes: Access to Approved Cell and Gene Therapies Originally released in May [----] A person considering a gene or cell therapy treatment may experience barriers to accessing the treatment. Our experts will explore the various meanings of the term access. This includes the development of therapies eligibility to receive an FDA-approved treatment coverage provider referral and being able to navigate the logistics of treatment within a clinic. Each expert touches on aspects that shape the field and impact an individuals access to gene and cell treatments. Listen to Quick Takes" [YouTube Link](https://youtube.com/watch?v=jXV8KkBFpkA) 2025-04-11T17:28Z 13.5K followers, [---] engagements "Transthyretin Amyloidosis Transthyretin amyloidosis (ATTR) is a condition that gradually worsens over time and affects how certain organs work especially the heart and the nerves. This unit will explore the causes of ATTR amyloidosis and how new gene therapies are being developed to potentially slow or stop disease progression" [YouTube Link](https://youtube.com/watch?v=n3ZLh0w_rnc) 2024-12-16T16:28Z 13.4K followers, [----] engagements "CRISPR and Beyond with Vertex CSO David Altshuler MD PhDASGCT's The Issue Join host Emily Walsh Martin PhD and David Altshuler MD PhD Chief Scientific Officer of Vertex Pharmaceuticals. David came to Vertex after a lengthy career in medicine and academic research. David and Emily discuss how David and the team at Vertex think about innovation in drug development and how that impacts how they select programs and approaches for the diseases they focus on. They also cover what hed love to see happen with new cell and gene platform technologies being brought forward. Now is the time to register" [YouTube Link](https://youtube.com/watch?v=nBSUA1CdaGw) 2024-07-16T10:21Z 13.5K followers, [---] engagements "Around the World: Advanced Therapy Medicinal Production (ATMP) Research in South America Explore the latest developments in Advanced Therapy Medicinal Product (ATMP) research across South America including innovative production techniques and regional advancements driving the field forward" [YouTube Link](https://youtube.com/watch?v=nlRZdDyNrCI) 2024-12-03T16:00Z 13.4K followers, [---] engagements "Readministration of AAV vector in Pompe Disease - Manuela Corti Readministration of AAV vector in Pompe Disease - Manuela Corti Scientific Symposium from the American Society of Gene & Cell Therapy's 21st Annual Meeting" [YouTube Link](https://youtube.com/watch?v=wwxQ8g5rWlQ) 2018-12-07T21:24Z 13.3K followers, [---] engagements "Lunch & Learn: Gene Therapy [---] During this educational session experts in the field reviewed different gene therapy approaches disease applications and research processes and described the current state of the field" [YouTube Link](https://youtube.com/watch?v=x9u1ihTovaU) 2022-02-28T16:06Z 13.3K followers, 42.7K engagements "Education Session [---] The ABCs of AAV" [YouTube Link](https://youtube.com/watch?v=xhT7wJuJ1m8) 2017-09-26T21:44Z 13.4K followers, [----] engagements "Technological Frontiers in South American Gene and Cell Therapy As cell and gene therapies revolutionize modern medicine South America is emerging as a dynamic player on the global stage leveraging scientific innovation regulatory evolution and regional collaboration. Explore the technological frontiers shaping the development and deployment of advanced therapies across the continent" [YouTube Link](https://youtube.com/watch?v=xlS66b4HSt4) 2026-01-26T22:05Z 13.5K followers, [--] engagements "Why Leading Biotech Companies Choose ASGCT Inside the Industry's Premier Gene & Cell Therapy Event Discover why industry leaders make ASGCT's Annual Meeting a cornerstone of their business strategy. Three exhibitors and sponsors share their success stories and reveal the unique opportunities that make the ASGCT Annual Meeting invaluable for gene and cell therapy companies. 🎯 In this video: @Regeneron @GenScript @Bio-techne Hear directly from industry decision-makers Learn about ROI from exhibition investments Understand networking opportunities Explore why ASGCT stands out from other" [YouTube Link](https://youtube.com/watch?v=y-w00s-hHyM) 2024-10-25T20:17Z 13.3K followers, [---] engagements "mRNA Manufacturing mRNA Manufacturing - Anton McCaffrey Education Session from the American Society of Gene & Cell Therapy's 22nd Annual Meeting" [YouTube Link](https://youtube.com/watch?v=8j33dGRZ_S4) 2019-12-04T21:13Z 13.5K followers, 46.8K engagements "Around the World: Frontline of AAV Vectors for Gene Therapy Research in Japan Explore cutting-edge advancements in the use of AAV vectors for gene therapy research in Japan. This event brings together leading scientists researchers and professionals in the field to discuss innovative approaches challenges and breakthroughs in gene delivery systems specifically focusing on AAV technology. ASGCT Japan AAV Technology ASGCT Japan AAV Technology" [YouTube Link](https://youtube.com/watch?v=Ftr0NR0rWWQ) 2025-05-14T15:22Z 13.5K followers, [---] engagements "Improving AAV Production with Adam Schieferecke PhD + David Schaffer PhD Dr. Roland Herzog sits down with Drs. David Schaffer and Adam Schieferecke to discuss a recent article published in the February issue of Molecular Therapy by Drs. Schaffer and Schieferecke and colleagues titled Evolving membrane-associated accessory protein variants for improved adeno-associated virus production. Read the full article: https://www.cell.com/molecular-therapy-family/molecular-therapy/abstract/S1525-0016(23)00679-2 Dr. Roland Herzog: Editor-in-Chief of Molecular Therapy and Professor of Pediatrics at" [YouTube Link](https://youtube.com/watch?v=IDhImEftJX0) 2024-03-12T10:00Z 13.5K followers, [---] engagements "Elevating Your Research Career Dr. Anna Blakney on ASGCT Member Benefits In the fast-moving world of gene and cell therapy technical skills are only half the battle. Dr. Anna Blakney (Assistant Professor at UBC) explains why ASGCT is the essential home for professionals in the field. From accessing the latest research in Molecular Therapy to navigating the Career Center ASGCT membership offers a comprehensive suite of tools designed to benefit your career at every stage. 🔗 Become an ASGCT member today: asgct.org/become-a-member The American Society of Gene & Cell Therapy is the primary" [YouTube Link](https://youtube.com/watch?v=JmuRnnnTpfQ) 2026-02-09T17:44Z 13.5K followers, [--] engagements "Next-generation AAV Vectors Next-generation AAV Vectors - Dirk Grimm Education Session from the American Society of Gene & Cell Therapy's 22nd Annual Meeting" [YouTube Link](https://youtube.com/watch?v=MgpWCYiyZaA) 2019-12-04T21:20Z 13.5K followers, [----] engagements "Investigators guide to identifying and validating biomarkers for clinical trials This a recorded presentation from the session titled "To Biomarker Or Not To Biomarker Use Considerations For CGT Drug Development" Organized by the Patient Outreach Committee during AM25" [YouTube Link](https://youtube.com/watch?v=S1LRtC6oH_s) 2025-07-23T14:00Z 13.5K followers, [---] engagements ""Inspire and Be Inspired" Dr. Ruth Castellanos on the Power of the ASGCT Community "ASGCT has empowered me to become who I am right now." What does it mean to be a member of the American Society of Gene & Cell Therapy For Dr. Ruth Castellanos (Director of R&D at Kriya Therapeutics) it means more than just a professional affiliationits about finding direction and inspiration. Whether you are looking to be inspired by the latest science or hoping to inspire others with your work there is a place for you here. 🔗 Become an ASGCT member today: asgct.org/become-a-member The American Society of" [YouTube Link](https://youtube.com/watch?v=W8MC8jh52fU) 2026-02-05T16:58Z 13.5K followers, [--] engagements "Recent CMC Guidance for Cellular and Gene Therapies: BIO-ASGCT CGT Science Series Hear from Dr. Andrew Byrnes during the third installment of the BIO-ASGCT CGT Science Series" [YouTube Link](https://youtube.com/watch?v=dgXVt-DatdM) 2024-04-04T19:18Z 13.5K followers, [----] engagements "AAV Capsid Production with Drs. Mario Mietzsch and Robert McKenna - Molecular Therapy Podcast Dr. Daniel Stone speaks with Drs. Mario Mietzsch and Robert McKenna from the University of Florida to discuss a recent article published in the Biomanufacturing in Gene and Cell Therapy special issue of Molecular Therapy Methods & Clinical Development (https://www.sciencedirect.com/special-issue/10RZ8HRVPL2) by Drs. Mietzsch McKenna and colleagues titledProduction and characterization of an AAV1-VP3-only capsid: An analytical benchmark standard" [YouTube Link](https://youtube.com/watch?v=fMRbXtmjg44) 2024-06-18T10:08Z 13.5K followers, [---] engagements "Generation of Hematopoietic Stem and Progenitor Cells from iPSCs Generation of Hematopoietic Stem and Progenitor Cells from iPSCs - Andre Larochelle Education Session from the American Society of Gene & Cell Therapy's 22nd Annual Meeting" [YouTube Link](https://youtube.com/watch?v=fR661sBqlNM) 2019-11-27T19:52Z 13.5K followers, [----] engagements "The Issue: AAV Immunogenicity with Federico Mingozzi PhD Dr. Mingozzi has done seminal work throughout his career in AAV gene therapy and his [----] Molecular Therapy review of our current understanding of the immune reaction to these therapies is a must-read for anyone trying to get a handle on the very complicated landscape of immune reactions to viral vectors" [YouTube Link](https://youtube.com/watch?v=fkE4oi__PdA) 2024-02-19T17:42Z 13.5K followers, [---] engagements "Gene Editing Gene editing aims to be a one-time therapy that directly edits pieces of DNA within the cell. It's considered a type of gene therapy which is the use of genetic material to treat or prevent disease. The subtitling of these videos was funded in part by a donation from Pfizer Inc" [YouTube Link](https://youtube.com/watch?v=i_1kLfK1cP0) 2021-11-02T20:12Z 13.5K followers, 13.1K engagements "How Do Clinical Trials Work New therapies spend a lot of time being tested in labs and animals in preclinical studies before making it to patients. By the time the therapy enters human clinical trials it is better understood and ready for further development. Learn more information about the process of developing a treatment. The subtitling of these videos was funded in part by a donation from Pfizer Inc. gene therapy cell therapy clinical trials scientific development biotechnology gene editing crispr science research medical research gene therapy cell therapy clinical trials scientific" [YouTube Link](https://youtube.com/watch?v=kst1Msx9nOI) 2019-01-17T14:04Z 13.5K followers, 19K engagements "Engineered iPSC-derived Lymphocytes for Improved Cancer Therapy Engineered iPSC-derived Lymphocytes for Improved Cancer Therapy - Dan Kaufman Scientific Symposium from the American Society of Gene & Cell Therapy's 22nd Annual Meeting" [YouTube Link](https://youtube.com/watch?v=pGhFKbPd1h8) 2019-11-27T19:22Z 13.5K followers, [----] engagements "Community Quick Takes: State of the Field - Highlights and Approvals for CGT in [----] Originally released in February [----] The field of gene and cell therapy continues to grow and change with the approval of many new therapies this past year. There is much to highlight about the patient communities affected and what it means for future findings in treatments of rare and genetic conditions. Check out the quick takes below from Peter Marks MD PhD and Jeffrey Chamberlain PhD adddressing the following questions: - What were some of the field highlights for you this past year - What sort of" [YouTube Link](https://youtube.com/watch?v=wTOZz7ADBHg) 2025-04-11T17:21Z 13.5K followers, [--] engagements "Policy and Advocacy Shorts with Jennifer Wellman Jennifer Wellman Chief Development Officer at Akouos discusses ASGCT's role in addressing manufacturing and access challenges in gene therapy and shares her excitement about the field's rapid progress" [YouTube Link](https://youtube.com/watch?v=-V7hidTghs8) 2024-07-25T14:47Z 13.5K followers, [--] engagements "Welcome to ASGCT: Who We Are and What We Do Who are we at the American Society of Gene & Cell Therapy We're known for holding the world's premier gene therapy cell therapy and gene editing conference but our members are doing so much more. For thousands of researchers clinicians and patient advocates ASGCT is a community with which they can stay plugged into one of medicine's most exciting fields. Welcome to ASGCT. gene therapy gene editing crispr asgct medical research cell therapy CAR T-Cell gene therapy gene editing crispr asgct medical research cell therapy CAR T-Cell" [YouTube Link](https://youtube.com/watch?v=8xtLInvFLqo) 2018-07-24T20:32Z 13.5K followers, [----] engagements "What are Biomarkers A regulators guide to using biomarkers in an approved therapy This a recorded presentation from the session titled "To Biomarker Or Not To Biomarker Use Considerations For CGT Drug Development" Organized by the Patient Outreach Committee during AM25" [YouTube Link](https://youtube.com/watch?v=PBluQBHS7qI) 2025-07-23T14:00Z 13.5K followers, [--] engagements "25 Years of Molecular Therapy with Drs. Rayne Rouce and Joe Glorioso This episode is the fourth in a series hosted by Molecular TherapyEditor-in-Chief Joseph Glorioso PhD that will cover comprehensive reviews of critical developments in the field of gene and cell therapy over the past [--] years. In this episode Dr. Glorioso will discuss the review How to democratize cell and gene therapy: A global approach" [YouTube Link](https://youtube.com/watch?v=QI-jilbOgLQ) 2025-08-08T10:12Z 13.5K followers, [---] engagements "Immunogenicity of AAV Vectors and Transgenes - Roland Herzog Immunogenicity of AAV Vectors and Transgenes - Roland Herzog Education Session from the American Society of Gene & Cell Therapy's 21st Annual Meeting May [--] 2018" [YouTube Link](https://youtube.com/watch?v=SBhf_beSB1s) 2018-12-07T21:24Z 13.5K followers, [----] engagements "Development of an AAV-based Prophylactic Vaccine for Influenza Development of an AAV-based Prophylactic Vaccine for Influenza - Maria Limberis Scientific Symposium from the American Society of Gene & Cell Therapy's 22nd Annual Meeting" [YouTube Link](https://youtube.com/watch?v=WKkWypyy04c) 2019-11-27T19:06Z 13.5K followers, [---] engagements "Policy and Advocacy Shorts with Diane Berry PhD Diane Berry PhD Executive Vice President Chief Global Policy & Advocacy Officer at Sarepta Therapeutics highlights ASGCT's unique role in bringing together diverse stakeholders and providing evidence-based advocacy to advance policies in cell and gene therapy" [YouTube Link](https://youtube.com/watch?v=WPiJSOIm4AE) 2024-07-25T14:20Z 13.5K followers, [--] engagements "Community Quick Takes: Biomarker Breakdown Hear from Dr. Rebecca Ahrens-Nicklas a featured speaker in the ASGCT Annual Meeting session To Biomarker or Not To Biomarker Use Considerations for Cell and Gene Therapy Drug Development. Its a big question to answer but were providing some insights on how biomarkers may can help move cell and gene therapies forward and emphasizing the critical role of patient advocacy groups in shaping outcome measures. Listen to Quick Takes from experts in the community on the following questions: 01:25 - What exactly is a biomarker and how do they differ from" [YouTube Link](https://youtube.com/watch?v=_YNCuJZOUXs) 2025-06-24T17:00Z 13.5K followers, [---] engagements "Around the World: How U.S. Science is Impacting Cell and Gene Therapy This ASGCT session explores how U.S.-driven science is shaping global gene and cell therapy. Topics include the role of African genetic diversity in advancing precision medicine decentralized manufacturing to expand access and economic modeling to support gene therapy delivery in low-resource settings all aimed at promoting global health equity. Global Outreach ASGCT Manufacturing Global Outreach ASGCT Manufacturing" [YouTube Link](https://youtube.com/watch?v=cLnrhs-MOFo) 2025-09-19T15:01Z 13.5K followers, [---] engagements "Gene Editing for DMD Gene Editing for DMD - Rhonda Bassel-Duby Scientific Symposium from the American Society of Gene & Cell Therapy's 22nd Annual Meeting" [YouTube Link](https://youtube.com/watch?v=cndVe76w6DE) 2019-11-27T19:37Z 13.5K followers, 12.4K engagements "The Issue: Gene Editing Complexities with Tom McCauley PhD Episode [--] of The Issue on the ASGCT podcast network is an in-depth interview with Dr Tom McCauley CSO of Omega Therapeutics. Tom and Emily talk about his path from condensed matter physics to leading teams of scientists exploring the next generation of molecular therapeutics. They also dive into their mutual experience in the early days of in vivo gene editing and the challenges one faces with the complexities of these products particularly before there is much of a roadmap for development. Finally they dive into the recent public Ad" [YouTube Link](https://youtube.com/watch?v=d6Tu9tndpyw) 2024-01-16T12:00Z 13.5K followers, [---] engagements "Policy and Advocacy Shorts with Keith Wonnacott PhD Keith Wonnacott PhD Vice President of Regulatory Affairs at Lexeo Therapeutics shares insights on ASGCT's role in connecting academia and industry and discusses the evolution of regulatory approaches in cell and gene therapy" [YouTube Link](https://youtube.com/watch?v=eV-O0fle_AQ) 2024-07-25T14:31Z 13.5K followers, [--] engagements "Gene Therapy Basics (2022 Update) Gene therapy is the use of genetic material to treat or prevent disease. Learn more about the basics of gene therapy the potential for this growing field of research and the challenges and risks that still need to be investigated" [YouTube Link](https://youtube.com/watch?v=kAtd9X29SdQ) 2022-10-26T21:45Z 13.5K followers, 228.1K engagements "Patients Are the Center with Kimberly Haugstad MBA On this episode of Soundbites of the Annual Meeting we talked to Kimberly Haugstad CEO of RareRising and a longtime rare disease advocate who is also the parent of a child with severe hemophilia. In her conversation with ASGCT's Communications Committee Chair Lynnea Olivarez Kimberly discusses: why patients as the "lived experience experts" are so important to rare disease communications advice for scientists who would like to engage more directly with patient communities what's giving her hope right now Listen and subscribe to the ASGCT" [YouTube Link](https://youtube.com/watch?v=o_sDrc7WO8w) 2025-08-19T10:06Z 13.5K followers, [--] engagements "Building a Vision for Disease Prevention + Treatment with Dr. Mimi Lee On this episode of Soundbites of the Annual Meeting we talked to Mimi Lee MD PhD a physician-scientist and program manager at the Advanced Research Projects Agency for Health (ARPA-H). In her conversation with ASGCT's Communications Committee Chair Lynnea Olivarez Dr. Lee discusses: ARPA-H's unique approach to funding and structuring CGT development breaking down silos to improve health outcomes for all the role that CGT will play in prevention rather than just treatment of diseases Listen and subscribe to the ASGCT" [YouTube Link](https://youtube.com/watch?v=vmaQo_O6Y3s) 2025-08-04T10:19Z 13.5K followers, [--] engagements "Special Podcast Episode: What to Expect at ASGCT's Policy Summit with Chris Leidli + Francesca Cook Note: this episode is cross-posted with the Rx for Biotech podcast hosted by Chris Leidli. Francesca Cook is the Vice President Pricing and Market Access at REGENXBIO and past chair of ASGCT's Government Relations Committee. Shes attended and helped with ASGCTs annual Policy Summit since its inception five years ago. In this episode Cook shares her experience and advice around the eventcoming up Sept. 18-19 (virtually + in Washington DC) with its highest-profile line-up yet and more ways to" [YouTube Link](https://youtube.com/watch?v=wN5IVBgak6U) 2023-09-14T23:03Z 13.5K followers, [--] engagements "A Molecular Glue Degrader to Control CRISPR with Drs. Krishanu Saha and Namita Khajanchi Join Dr. Paloma Giangrande Editor-in-Chief of Molecular Therapy Nucleic Acids as she discusses a recently published MTNA article Controlling CRISPR-Cas9 genome editing in human cells using a molecular glue degrader (https://www.cell.com/molecular-therapy-family/nucleic-acids/fulltext/S2162-2531(25)00194-5_returnURL=https%3A%2F%2Flinkinghub.elsevier.com%2Fretrieve%2Fpii%2FS2162253125001945%3Fshowall%3Dtrue) with its authors Drs. Krishanu Saha and Namita Khajanchi (UW-Madison). If you enjoy this episode" [YouTube Link](https://youtube.com/watch?v=0DNFKV8ovUU) 2025-10-08T10:20Z 13.5K followers, [---] engagements "Cas9-CLIPT-mediated integration of large transgenes with Krishanu Saha Anna Tommasi and Dan Cap. Dr. Daniel Stone (Associate Editor-in-Chief Molecular Therapy Advances) sits down with Dr. Krishanu Saha Anna Tommasi and Dr. Dan Cappabianca from the University of Wisconsin-Madison. Join them as they discuss their teams recent publication Efficient nonviral integration of large transgenes into human T cells using Cas9-CLIPT and learn more about their novel method to improve the manufacturing of multifunctional genome-edited immune cell therapies. Music:'Electric Dreams' by Scott Buckley -" [YouTube Link](https://youtube.com/watch?v=0ITVpE_u-jQ) 2026-02-03T11:29Z 13.5K followers, [--] engagements "Next-Generation Cell & Gene Therapy Approaches for HIV On December [--] [----] the American Society of Gene & Cell Therapy (ASGCT) hosted a virtual Congressional briefing titled "Next-Generation Cell & Gene Therapy Approaches for HIV." The event brought together bipartisan Congressional staff leading researchers and patient advocates to explore how cell and gene therapies (CGTs) are opening the possibility of a cure for people living with HIV" [YouTube Link](https://youtube.com/watch?v=3MeoL5J2h0A) 2025-12-05T16:47Z 13.5K followers, [---] engagements "Reducing the Immunogenicity of AAV through Engineering the Vector - George Church Reducing the Immunogenicity of AAV through Engineering the Vector - George Church Scientific Symposium from the American Society of Gene & Cell Therapy's 21st Annual Meeting" [YouTube Link](https://youtube.com/watch?v=3YeyJDwEVMg) 2018-12-07T21:24Z 13.4K followers, [----] engagements "The Issue - Entrepreneurship Fundraising and the Future of Cell & Gene Therapy with Dr. Niki Paulk Host Dr. Emily Walsh Martin welcomes Dr. Niki Paulk CEO and Founder of Siren Biotechnology for a timely conversation on turning scientific promise into commercial success. Dr. Paulk offers perceptive insights into the complex world of biotech entrepreneurship covering essential fundraising strategies from Seed and Series Rounds to Community Rounds and beyond. This episode is a must-listen for both industry professionals and academic innovators. Music:Bright New Morning by Steven OBrien released" [YouTube Link](https://youtube.com/watch?v=6TphuUY4sMM) 2026-01-06T11:27Z 13.5K followers, [--] engagements "Lunch & Learn: How AAV Vectors Are Made We often hear that gene therapies are complex and require a lot of time and money to make. But what does that really mean Tune in to hear experts in the field help answer this question by walking us through the manufacturing process" [YouTube Link](https://youtube.com/watch?v=6sU9NAO4lX4) 2022-05-02T19:48Z 13.5K followers, 33.6K engagements "Lipid Nanoparticles with Drs. Daniel Stone Harrison Dulin and Keith R. Jerome - The Molecular T. Join Dr. Mohamed Abou-el-Enein Editor-in-Chief of Molecular Therapy Methods & Clinical Development as he discusses a recent article published in MTMCD with co-authors Drs. Daniel Stone Harrison Dulin and Keith R. Jerome from the Fred Hutchinson Cancer Center. The article Serum factors create species-specific barriers to hepatic gene transfer by lipid nanoparticles in liver-humanized mice" [YouTube Link](https://youtube.com/watch?v=7s2Jh7I6ZVI) 2025-09-10T10:36Z 13.5K followers, [---] engagements "Harnessing Naturaland Engineered Properties of iNKT cells for Adoptive Cancer Immunotherapy Harnessing Naturaland Engineered Properties of iNKT cells for Adoptive Cancer Immunotherapy - Leonid Metelitsa Education Session from the American Society of Gene & Cell Therapy's 22nd Annual Meeting" [YouTube Link](https://youtube.com/watch?v=8evBtUtgyeQ) 2019-11-27T20:00Z 13.5K followers, [---] engagements "25 Years of Molecular Therapy with Drs. Terry Flotte and Carl June In honor of Molecular Therapys 25th anniversary this episode is the first in a series hosted by Dr. Joseph Glorioso Editor-in-Chief of Molecular Therapy that will cover comprehensive reviews of critical developments in the field of gene and cell therapy over the past [--] years by expert authors. In this episode Dr. Glorioso will discuss two of these reviews Gene therapy then and now: A look back at changes in the field over the past [--] years with author Dr. Terry Flotte UMass Chan Medical School and From concept to cure: The" [YouTube Link](https://youtube.com/watch?v=DrNkt3AyZnQ) 2025-05-01T10:26Z 13.5K followers, [---] engagements "Ancestral AAV Capsid Reconstruction: Interrogating Viral Evolution to Inform Rational AAV Design Ancestral AAV Capsid Reconstruction: Interrogating Viral Evolution to Inform Rational AAV Design - Luk Vandenberghe Scientific Symposium from the American Society of Gene & Cell Therapy's 21st Annual Meeting May [--] 2018" [YouTube Link](https://youtube.com/watch?v=GrZ17whdmac) 2018-12-07T21:24Z 13.5K followers, [----] engagements "AAV Integration Roundtable During this virtual roundtable experts discussed the status of AAV integration research including analysis of recent clinical findings to develop recommendations for risk assessment" [YouTube Link](https://youtube.com/watch?v=L_4IuK3fNU0) 2021-08-20T14:59Z 13.5K followers, [----] engagements "Community Quick Takes: AAV Immune Response In this Community Quick Takes module we will explore a high-level summary of key topics from the recent ASGCT workshop Class Considerations on Immunogenicity for AAV Gene Therapy Products: Assessing Current Practice and New Data. Speakers from the event address fundamental questions about the immune system's response to AAV gene therapies how these responses can affect treatment outcomes and the methods being developed to manage immune reactions. Listen to Quick Takes from experts in the community on the following questions: 00:00 - What does the" [YouTube Link](https://youtube.com/watch?v=OB3-NpGkCnA) 2025-02-26T16:00Z 13.5K followers, [---] engagements "RNA interference gene therapy for Parkinsons disease with Kathy Steece-Collier Jeffrey Kordower. Molecular Therapy Editor-in-Chief Dr. Joseph Glorioso joins researchers Kathy Steece-Collier (Michigan State University) Jeffrey Kordower (Arizona State University) and Fredric Manfredsson (Barrow Neurological Institute) to discuss their groundbreaking work on RNA interference for Parkinsons disease. This episode focuses on their recent article titled "Disease-modifying multidimensional efficacy of putaminal CaV1.3-shRNA gene therapy in aged parkinsonism male and female macaques." Music:'Electric" [YouTube Link](https://youtube.com/watch?v=Oa4wcsSxi7k) 2026-01-08T11:19Z 13.5K followers, [---] engagements "Case study of how biomarkers may be limited in DMD This a recorded presentation from the session titled "To Biomarker Or Not To Biomarker Use Considerations For CGT Drug Development" Organized by the Patient Outreach Committee during AM25" [YouTube Link](https://youtube.com/watch?v=Os0k1l5-nEw) 2025-07-23T14:00Z 13.5K followers, [--] engagements "Generating CAR T cells in vivo with lentiviral vectors with Drs. Rachael Nimmo and Kyriacos Mitro. Editor-in-Chief of Molecular Therapy Dr. Joseph Glorioso is joined by Rachael Nimmo PhD former director of the Cell Technology Group at Oxford Biomedica and Kyriacos Mitrophanous PhD Chief Innovation Officer at Oxford Biomedica to discuss an article recently published in Molecular Therapyby Nimmo and colleagues titled Efficient in vivo generation of CAR T cells using a retargeted fourth-generation lentiviral vector" [YouTube Link](https://youtube.com/watch?v=PMSFHVP_os0) 2025-11-11T11:15Z 13.5K followers, [---] engagements "AAV Gene Therapy to CNS: Past Present and Future - Miguel Sena-Esteves Gene Therapy to CNS: Past Present and Future Miguel Sena-Esteves Education Session from the American Society of Gene & Cell Therapy's 21st Annual Meeting May [--] 2018" [YouTube Link](https://youtube.com/watch?v=Qbv3kfjZ9_w) 2018-12-07T21:24Z 13.5K followers, [----] engagements "Gene Therapy and Sickle Cell Disease Sickle cell disease is an inherited blood disorder that causes red blood cells to deform and become sickle (or crescent) shaped. Gene therapy and gene editing may offer a one-time treatment that addresses the genetic cause of sickle cell disease. The subtitling of these videos was funded in part by a donation from Pfizer Inc. sickle cell disease SCD gene therapy ASGCT American Society of Gene and Cell Therapy ASH ASH SCD ASH Research Collaborative ASH RC SCD Clinical Trials Network hematology hematology society ASH hematology sickle cell disease SCD gene" [YouTube Link](https://youtube.com/watch?v=Ro18vRnk6JI) 2020-09-17T21:50Z 13.5K followers, 57.8K engagements "Case study of how CSF biomarkers were approved by the FDA in MPS This a recorded presentation from the session titled "To Biomarker Or Not To Biomarker Use Considerations For CGT Drug Development" Organized by the Patient Outreach Committee during AM25" [YouTube Link](https://youtube.com/watch?v=SAoLYyodpzQ) 2025-07-23T14:00Z 13.5K followers, [--] engagements "AAV Engineering AAV Engineering - Junghae Suh Scientific Symposium from the American Society of Gene & Cell Therapy's 22nd Annual Meeting" [YouTube Link](https://youtube.com/watch?v=WhGHetJNHHc) 2019-11-27T18:00Z 13.5K followers, [----] engagements "AAV Vectors in the Eye - Shannon Boye AAV Vectors in the Eye Shannon Boye Education Session from the American Society of Gene & Cell Therapy's 21st Annual Meeting May [--] 2018" [YouTube Link](https://youtube.com/watch?v=Yf2FZtNizVk) 2018-12-07T21:24Z 13.5K followers, [----] engagements "Short and Long Read Sequencing for AAV Characterization: BIO-ASGCT CGT Science Series The BIO-ASGCT Cell and Gene Therapy (CGT) Science Series is a quarterly seminar series focused on scientific topics related to cell and gene therapy products. The CGT Science Series is intended to foster scientific exchange between BIO ASGCT and CBER review staff on a variety of topics that span the CGT product lifecycle. The inaugural seminar focuses on NGS characterization of AAV and was presented by Dr. Lauriel Earley. Dr. Lauriel Earley is a Senior Scientist with Shape Therapeutics who has been studying" [YouTube Link](https://youtube.com/watch?v=aHoh-OHezBc) 2023-04-05T21:25Z 13.5K followers, [----] engagements "AAV Vector Manufacturing and Analytics AAV Vector Manufacturing and Analytics - J. Fraser Wright Scientific Symposium from the American Society of Gene & Cell Therapy's 22nd Annual Meeting" [YouTube Link](https://youtube.com/watch?v=cz_D_etMR0E) 2019-11-27T18:06Z 13.5K followers, 16K engagements "The Ups and Downs of Conducting Groundbreaking Research with Dr. Kiran Musunuru "Certainly I've been anxious about data.I did an experiment I put a lot of time into it I'm hoping to get good results.we all know that feeling right The stakes are just so different here.this kid's life depends on whether this experiment worked." --Kiran Musunuru MD PhD One of the highlights of the ASGCT 28th Annual Meeting wasthe announcement on May [--] of a historic medical breakthrough (https://www.chop.edu/news/worlds-first-patient-treated-personalized-crispr-gene-editing-therapy-childrens-hospital) : a child" [YouTube Link](https://youtube.com/watch?v=g7zeR0RK49A) 2025-06-16T10:15Z 13.5K followers, [---] engagements "Community Quick Take: Gene Editing for Prion Disease Dr. Sonia Vallabh a speaker featured at ASGCT's Breakthroughs in Targeted In Vivo Gene Editing event shares her powerful personal journey to develop treatment for a rare neurologic condition prion disease. Learn what led her to research in vivo gene editing to target the disease which is showing promise but not without limitations. Listen to Quick Takes from experts in the community on the following questions (Time: [--] minutes): 02:17 Can you tell us a little bit about yourself and your journey 05:01 What is gene editing and how might it" [YouTube Link](https://youtube.com/watch?v=gXfZC-YsDZQ) 2025-11-19T13:30Z 13.5K followers, [---] engagements "Why ARPA-H's Dr. Mimi Lee Is Looking Forward to the Policy Summit Hear directly from speaker Mimi Lee MD PhD a program manager at the Advanced Research Projects Agency for Health (ARPA-H) about why she encourages people to join her at the Policy Summit. Learn more and register today: http://asgct.org/policysummit" [YouTube Link](https://youtube.com/watch?v=lY_4Dh2TyXo) 2025-09-18T21:25Z 13.5K followers, [---] engagements "Immune Responses to AAV-Based Gene Therapy Gene therapy aims to treat genetic conditions by delivering genetic material such as a working gene to target the underlying cause of disease. One of the most common ways to deliver these therapies uses adeno-associated viruses (AAVs). While AAV-based gene therapies have a strong safety record they can still trigger immune responses that affect how well the treatment works or whether someone is eligible to receive it. Learn more: https://patienteducation.asgct.org/understanding-cell-gene-therapy/viral-vectors/immune-responses-to-aav-based-gene-therapy" [YouTube Link](https://youtube.com/watch?v=m1GC3RZ1Y9E) 2025-11-21T03:21Z 13.5K followers, [---] engagements "Incorporating miRNAs into a taRNA vector platform with Drs. Tim Beissert and Ayegl Yildiz - The. Drs. Ayegl Yildiz and Tim Beissert join Dr. Paloma Giangrande to discuss a recent article published in Molecular Therapy Nucleic Acids by Drs. Yildiz. Beissert and colleagues titled Trans-amplifying RNA expressing functional miRNA mediates target gene suppression and simultaneous transgene expression. Are you registered for ASGCTs [----] Annual Meeting in New Orleans Join us May 13-17 for the premier event in our field and save $465 as an ASGCT member. Check out the full program plan your trip and" [YouTube Link](https://youtube.com/watch?v=n0UFQO_HcXI) 2025-03-04T11:28Z 13.5K followers, [---] engagements "25 Years of Molecular Therapy with Sheila Mikhail and Dr. Mark Kay In honor of Molecular Therapys 25th anniversary this episode is the second in a series hosted by Dr. Joseph Glorioso Editor-in-Chief of Molecular Therapy that will cover comprehensive reviews of critical developments in the field of gene and cell therapy over the past [--] years. In this episode Dr. Glorioso will discuss the reviews AAV vector development back to the future" [YouTube Link](https://youtube.com/watch?v=nbumrYTew2A) 2025-06-11T10:20Z 13.5K followers, [---] engagements "Building Your Biotech Community Benjamin McLeod on ASGCT Membership "You get the opportunity to meet a group of people who are actually inside of your industry." In the world of gene and cell therapy location shouldn't limit your potential. Benjamin McLeod (Founder of Convey Bio) discusses how ASGCT acts as a "built-in community" for professionals. No matter where you are on the map ASGCT brings the heart of the industry to you. 🔗 Join our global community: https://www.asgct.org/membership/become-a-member" [YouTube Link](https://youtube.com/watch?v=pSAVV8wea38) 2026-02-16T13:56Z 13.5K followers, [--] engagements "A Vaccine for Cocaine Addiction with Dr. Stephen Kaminsky On this episode of Soundbites of the Annual Meeting we talked to Stephen Kaminsky PhD professor of research in genetic medicine at Weill Cornell Medical College and lead author of a phase [--] clinical trial of a new vaccine for cocaine addiction. In his conversation with ASGCT's Communications Committee Chair Lynnea Olivarez Dr. Kaminsky discusses: How the vaccine works and how its approach is different from other treatments Safety results from the unblinded data Implications this vaccine could have for other substance use disorders" [YouTube Link](https://youtube.com/watch?v=q-IY9szYajw) 2025-07-22T10:07Z 13.5K followers, [---] engagements "Community Quick Takes: Preparing for Unknowns When the patient community talks about cell and gene therapies hope and uncertainty often go hand-in-hand. In this unit Dr. Amy Waldman gives a closer look at what lies ahead for someone participating in an investigational clinical trial or more importantly what still might not be known. Listen to Quick Takes from experts in the community on the following questions: 00:00 - Why arent all drugs given open label as opposed to a double-blind design 10:11 - What are some of the more realistic expectations that a person participating in a gene therapy" [YouTube Link](https://youtube.com/watch?v=wum8rQh-xk8) 2025-09-22T19:56Z 13.5K followers, [---] engagements "Community Quick Takes: Informed Consent Originally released in November [----] Listen to [--] Quick Takes from experts in the community on the following questions: -What are five tips for a patient or caregiver to be prepared for informed consent -What challenges do participants face while going through the consent process and what actions can researchers take to improve the process -How do we facilitate informed consent for approved therapies *_Want to dive deeper into this topic Visit our patient education site for the full blog:_* https://patienteducation.asgct.org/quick-takes/informed-consent" [YouTube Link](https://youtube.com/watch?v=ykFY4of1r-Q) 2025-04-11T17:20Z 13.5K followers, [--] engagements "AAV Vectors in the Liver - Ian Alexander AAV Vectors in the Liver - Ian Alexander Education Session from the American Society of Gene & Cell Therapy's 21st Annual Meeting May [--] 2018" [YouTube Link](https://youtube.com/watch?v=zLtZxpXm5V0) 2018-12-07T21:24Z 13.5K followers, [---] engagements "2025 Year in Review with the Molecular Therapy Family of Journals Join all four Editors-in-Chief of the Molecular Therapy family of journals for a special [----] Retrospective. Editors Dr. Glorioso Dr. Giangrande Dr. Abou-el-Enein and Dr. Cripe dive into the most exciting advancements in gene and cell therapy from the past year. Music:'Electric Dreams' by Scott Buckley - released under CC-BY 4.0.www.scottbuckley.com.au (https://www.scottbuckley.com.au/) Show your support for ASGCT: https://asgct.org/membership/donate See omnystudio.com/listener (https://omnystudio.com/listener) for privacy" [YouTube Link](https://youtube.com/watch?v=zs17-eRt_RI) 2025-12-09T15:30Z 13.5K followers, [---] engagements "Community Quick Take: Genetic Reports + Whats Next A common experience for people diagnosed with a genetic condition is receiving a genetic test result which often comes with many questions emotions and unfamiliar terms. In this unit Kim Nye founder of the TESS Research Foundation and parent advocate is joined by genetic counselor Dr. Lauren Beretich to walk through whats in a genetic report and how this important information can help guide a familys next steps. Read more: https://patienteducation.asgct.org/research-clinical-trials/community-quick-takes/genetic-reports-whats-next" [YouTube Link](https://youtube.com/watch?v=a67iIG-jl1I) 2026-02-18T12:30Z 13.5K followers, [--] engagements "Building Your Biotech Community Benjamin McLeod on ASGCT Membership "You get the opportunity to meet a group of people who are actually inside of your industry." In the world of gene and cell therapy location shouldn't limit your potential. Benjamin McLeod (Founder of Convey Bio) discusses how ASGCT acts as a "built-in community" for professionals. No matter where you are on the map ASGCT brings the heart of the industry to you. 🔗 Join our global community: https://www.asgct.org/membership/become-a-member" [YouTube Link](https://youtube.com/watch?v=pSAVV8wea38) 2026-02-16T13:56Z 13.5K followers, [--] engagements "The Issue - Evolving Patient Access to CGTs via Innovation in Medicaid Reimbursement Models with . In this episode Melissa Majerol Model Lead of the Cell and Gene Therapy Access Model at the Center for Medicare and Medicaid Innovation (CMMI) at CMS breaks down how the CMMI Access Model program aims to simplify and accelerate patient access to innovative cell and gene therapies starting with recently approved therapies in Sickle Cell disease. Listen in as we dive into the specifics of this new access model and its current status. Music:Bright New Morning by Steven OBrien released under CC-BY" [YouTube Link](https://youtube.com/watch?v=HYKtDgb71DQ) 2026-02-10T11:34Z 13.5K followers, [--] engagements "Elevating Your Research Career Dr. Anna Blakney on ASGCT Member Benefits In the fast-moving world of gene and cell therapy technical skills are only half the battle. Dr. Anna Blakney (Assistant Professor at UBC) explains why ASGCT is the essential home for professionals in the field. From accessing the latest research in Molecular Therapy to navigating the Career Center ASGCT membership offers a comprehensive suite of tools designed to benefit your career at every stage. 🔗 Become an ASGCT member today: asgct.org/become-a-member The American Society of Gene & Cell Therapy is the primary" [YouTube Link](https://youtube.com/watch?v=JmuRnnnTpfQ) 2026-02-09T17:44Z 13.5K followers, [--] engagements ""Inspire and Be Inspired" Dr. Ruth Castellanos on the Power of the ASGCT Community "ASGCT has empowered me to become who I am right now." What does it mean to be a member of the American Society of Gene & Cell Therapy For Dr. Ruth Castellanos (Director of R&D at Kriya Therapeutics) it means more than just a professional affiliationits about finding direction and inspiration. Whether you are looking to be inspired by the latest science or hoping to inspire others with your work there is a place for you here. 🔗 Become an ASGCT member today: asgct.org/become-a-member The American Society of" [YouTube Link](https://youtube.com/watch?v=W8MC8jh52fU) 2026-02-05T16:58Z 13.5K followers, [--] engagements "Cas9-CLIPT-mediated integration of large transgenes with Krishanu Saha Anna Tommasi and Dan Cap. Dr. Daniel Stone (Associate Editor-in-Chief Molecular Therapy Advances) sits down with Dr. Krishanu Saha Anna Tommasi and Dr. Dan Cappabianca from the University of Wisconsin-Madison. Join them as they discuss their teams recent publication Efficient nonviral integration of large transgenes into human T cells using Cas9-CLIPT and learn more about their novel method to improve the manufacturing of multifunctional genome-edited immune cell therapies. Music:'Electric Dreams' by Scott Buckley -" [YouTube Link](https://youtube.com/watch?v=0ITVpE_u-jQ) 2026-02-03T11:29Z 13.5K followers, [--] engagements "Technological Frontiers in South American Gene and Cell Therapy As cell and gene therapies revolutionize modern medicine South America is emerging as a dynamic player on the global stage leveraging scientific innovation regulatory evolution and regional collaboration. Explore the technological frontiers shaping the development and deployment of advanced therapies across the continent" [YouTube Link](https://youtube.com/watch?v=xlS66b4HSt4) 2026-01-26T22:05Z 13.5K followers, [--] engagements "RNA interference gene therapy for Parkinsons disease with Kathy Steece-Collier Jeffrey Kordower. Molecular Therapy Editor-in-Chief Dr. Joseph Glorioso joins researchers Kathy Steece-Collier (Michigan State University) Jeffrey Kordower (Arizona State University) and Fredric Manfredsson (Barrow Neurological Institute) to discuss their groundbreaking work on RNA interference for Parkinsons disease. This episode focuses on their recent article titled "Disease-modifying multidimensional efficacy of putaminal CaV1.3-shRNA gene therapy in aged parkinsonism male and female macaques." Music:'Electric" [YouTube Link](https://youtube.com/watch?v=Oa4wcsSxi7k) 2026-01-08T11:19Z 13.5K followers, [---] engagements "The Issue - Entrepreneurship Fundraising and the Future of Cell & Gene Therapy with Dr. Niki Paulk Host Dr. Emily Walsh Martin welcomes Dr. Niki Paulk CEO and Founder of Siren Biotechnology for a timely conversation on turning scientific promise into commercial success. Dr. Paulk offers perceptive insights into the complex world of biotech entrepreneurship covering essential fundraising strategies from Seed and Series Rounds to Community Rounds and beyond. This episode is a must-listen for both industry professionals and academic innovators. Music:Bright New Morning by Steven OBrien released" [YouTube Link](https://youtube.com/watch?v=6TphuUY4sMM) 2026-01-06T11:27Z 13.5K followers, [--] engagements "The Issue - Unlocking New Therapeutic Potential: Ensomas Platform for Rare Disease and Oncology Host Emily Walsh Martin welcomes Dr. Jim Burns CEO of Ensoma to discuss the company's novel technological approach to treating both rare diseases and solid tumor oncology. Dr. Burns provides a detailed overview of Ensoma's platform and its application in their ongoing clinical trials including the recently announced first patient infusion for chronic granulomatous disease. Learn how this initial trial is set to inform future therapeutic opportunities for indications with high unmet medical need." [YouTube Link](https://youtube.com/watch?v=aJ3zysz9JWg) 2025-12-16T11:15Z 13.5K followers, [--] engagements "2025 Year in Review with the Molecular Therapy Family of Journals Join all four Editors-in-Chief of the Molecular Therapy family of journals for a special [----] Retrospective. Editors Dr. Glorioso Dr. Giangrande Dr. Abou-el-Enein and Dr. Cripe dive into the most exciting advancements in gene and cell therapy from the past year. Music:'Electric Dreams' by Scott Buckley - released under CC-BY 4.0.www.scottbuckley.com.au (https://www.scottbuckley.com.au/) Show your support for ASGCT: https://asgct.org/membership/donate See omnystudio.com/listener (https://omnystudio.com/listener) for privacy" [YouTube Link](https://youtube.com/watch?v=zs17-eRt_RI) 2025-12-09T15:30Z 13.5K followers, [---] engagements "Next-Generation Cell & Gene Therapy Approaches for HIV On December [--] [----] the American Society of Gene & Cell Therapy (ASGCT) hosted a virtual Congressional briefing titled "Next-Generation Cell & Gene Therapy Approaches for HIV." The event brought together bipartisan Congressional staff leading researchers and patient advocates to explore how cell and gene therapies (CGTs) are opening the possibility of a cure for people living with HIV" [YouTube Link](https://youtube.com/watch?v=3MeoL5J2h0A) 2025-12-05T16:47Z 13.5K followers, [---] engagements "Immune Responses to AAV-Based Gene Therapy Gene therapy aims to treat genetic conditions by delivering genetic material such as a working gene to target the underlying cause of disease. One of the most common ways to deliver these therapies uses adeno-associated viruses (AAVs). While AAV-based gene therapies have a strong safety record they can still trigger immune responses that affect how well the treatment works or whether someone is eligible to receive it. Learn more: https://patienteducation.asgct.org/understanding-cell-gene-therapy/viral-vectors/immune-responses-to-aav-based-gene-therapy" [YouTube Link](https://youtube.com/watch?v=m1GC3RZ1Y9E) 2025-11-21T03:21Z 13.5K followers, [---] engagements "Community Quick Take: Gene Editing for Prion Disease Dr. Sonia Vallabh a speaker featured at ASGCT's Breakthroughs in Targeted In Vivo Gene Editing event shares her powerful personal journey to develop treatment for a rare neurologic condition prion disease. Learn what led her to research in vivo gene editing to target the disease which is showing promise but not without limitations. Listen to Quick Takes from experts in the community on the following questions (Time: [--] minutes): 02:17 Can you tell us a little bit about yourself and your journey 05:01 What is gene editing and how might it" [YouTube Link](https://youtube.com/watch?v=gXfZC-YsDZQ) 2025-11-19T13:30Z 13.5K followers, [---] engagements "Generating CAR T cells in vivo with lentiviral vectors with Drs. Rachael Nimmo and Kyriacos Mitro. Editor-in-Chief of Molecular Therapy Dr. Joseph Glorioso is joined by Rachael Nimmo PhD former director of the Cell Technology Group at Oxford Biomedica and Kyriacos Mitrophanous PhD Chief Innovation Officer at Oxford Biomedica to discuss an article recently published in Molecular Therapyby Nimmo and colleagues titled Efficient in vivo generation of CAR T cells using a retargeted fourth-generation lentiviral vector" [YouTube Link](https://youtube.com/watch?v=PMSFHVP_os0) 2025-11-11T11:15Z 13.5K followers, [---] engagements "A Molecular Glue Degrader to Control CRISPR with Drs. Krishanu Saha and Namita Khajanchi Join Dr. Paloma Giangrande Editor-in-Chief of Molecular Therapy Nucleic Acids as she discusses a recently published MTNA article Controlling CRISPR-Cas9 genome editing in human cells using a molecular glue degrader (https://www.cell.com/molecular-therapy-family/nucleic-acids/fulltext/S2162-2531(25)00194-5_returnURL=https%3A%2F%2Flinkinghub.elsevier.com%2Fretrieve%2Fpii%2FS2162253125001945%3Fshowall%3Dtrue) with its authors Drs. Krishanu Saha and Namita Khajanchi (UW-Madison). If you enjoy this episode" [YouTube Link](https://youtube.com/watch?v=0DNFKV8ovUU) 2025-10-08T10:20Z 13.5K followers, [---] engagements "Around the World: Oncology Hematology and Cancer-Focused Cell and Gene Therapy in India This webinar explores Indias rapid advancements in CAR T therapies including homegrown innovations. This program highlights clinical successes increased accessibility and research leadership showcasing Indias growing impact on global cancer and hematology gene therapy landscapes" [YouTube Link](https://youtube.com/watch?v=21rRJxbHg_s) 2025-10-06T14:43Z 13.5K followers, [---] engagements "Dr. Snehal Naik: "I Hope You'll Join the Conversation" at the Policy Summit Hear directly from Snehal Naik PhD Chair of ASGCT's Regulatory Affairs Committee about why she encourages people to join her at the Policy Summit. Learn more and register today: http://asgct.org/policysummit" [YouTube Link](https://youtube.com/watch?v=Pkvk82i-gWc) 2025-09-23T16:34Z 13.5K followers, [--] engagements "Community Quick Takes: Preparing for Unknowns When the patient community talks about cell and gene therapies hope and uncertainty often go hand-in-hand. In this unit Dr. Amy Waldman gives a closer look at what lies ahead for someone participating in an investigational clinical trial or more importantly what still might not be known. Listen to Quick Takes from experts in the community on the following questions: 00:00 - Why arent all drugs given open label as opposed to a double-blind design 10:11 - What are some of the more realistic expectations that a person participating in a gene therapy" [YouTube Link](https://youtube.com/watch?v=wum8rQh-xk8) 2025-09-22T19:56Z 13.5K followers, [---] engagements "Around the World: How U.S. Science is Impacting Cell and Gene Therapy This ASGCT session explores how U.S.-driven science is shaping global gene and cell therapy. Topics include the role of African genetic diversity in advancing precision medicine decentralized manufacturing to expand access and economic modeling to support gene therapy delivery in low-resource settings all aimed at promoting global health equity. Global Outreach ASGCT Manufacturing Global Outreach ASGCT Manufacturing" [YouTube Link](https://youtube.com/watch?v=cLnrhs-MOFo) 2025-09-19T15:01Z 13.5K followers, [---] engagements "Why ARPA-H's Dr. Mimi Lee Is Looking Forward to the Policy Summit Hear directly from speaker Mimi Lee MD PhD a program manager at the Advanced Research Projects Agency for Health (ARPA-H) about why she encourages people to join her at the Policy Summit. Learn more and register today: http://asgct.org/policysummit" [YouTube Link](https://youtube.com/watch?v=lY_4Dh2TyXo) 2025-09-18T21:25Z 13.5K followers, [---] engagements "Lipid Nanoparticles with Drs. Daniel Stone Harrison Dulin and Keith R. Jerome - The Molecular T. Join Dr. Mohamed Abou-el-Enein Editor-in-Chief of Molecular Therapy Methods & Clinical Development as he discusses a recent article published in MTMCD with co-authors Drs. Daniel Stone Harrison Dulin and Keith R. Jerome from the Fred Hutchinson Cancer Center. The article Serum factors create species-specific barriers to hepatic gene transfer by lipid nanoparticles in liver-humanized mice" [YouTube Link](https://youtube.com/watch?v=7s2Jh7I6ZVI) 2025-09-10T10:36Z 13.5K followers, [---] engagements "Inside the Molecular Therapy Family of Journals Join us as past ASGCT President Paula Cannon Ph.D. and our Editors-in-Chief take you behind the scenes of ASGCTs field-leading Molecular Therapy family of journals. Discover how these prestigious publications have shaped the landscape of cell and gene therapy research for the past [--] years. Learn more: asgct.org/molecular-therapy" [YouTube Link](https://youtube.com/watch?v=n1Tcj_dru1A) 2025-09-09T15:02Z 13.5K followers, [---] engagements "Patients Are the Center with Kimberly Haugstad MBA On this episode of Soundbites of the Annual Meeting we talked to Kimberly Haugstad CEO of RareRising and a longtime rare disease advocate who is also the parent of a child with severe hemophilia. In her conversation with ASGCT's Communications Committee Chair Lynnea Olivarez Kimberly discusses: why patients as the "lived experience experts" are so important to rare disease communications advice for scientists who would like to engage more directly with patient communities what's giving her hope right now Listen and subscribe to the ASGCT" [YouTube Link](https://youtube.com/watch?v=o_sDrc7WO8w) 2025-08-19T10:06Z 13.5K followers, [--] engagements "25 Years of Molecular Therapy with Drs. Rayne Rouce and Joe Glorioso This episode is the fourth in a series hosted by Molecular TherapyEditor-in-Chief Joseph Glorioso PhD that will cover comprehensive reviews of critical developments in the field of gene and cell therapy over the past [--] years. In this episode Dr. Glorioso will discuss the review How to democratize cell and gene therapy: A global approach" [YouTube Link](https://youtube.com/watch?v=QI-jilbOgLQ) 2025-08-08T10:12Z 13.5K followers, [---] engagements "Building a Vision for Disease Prevention + Treatment with Dr. Mimi Lee On this episode of Soundbites of the Annual Meeting we talked to Mimi Lee MD PhD a physician-scientist and program manager at the Advanced Research Projects Agency for Health (ARPA-H). In her conversation with ASGCT's Communications Committee Chair Lynnea Olivarez Dr. Lee discusses: ARPA-H's unique approach to funding and structuring CGT development breaking down silos to improve health outcomes for all the role that CGT will play in prevention rather than just treatment of diseases Listen and subscribe to the ASGCT" [YouTube Link](https://youtube.com/watch?v=vmaQo_O6Y3s) 2025-08-04T10:19Z 13.5K followers, [--] engagements "Community Quick Takes: Manufacturing Matters The gene therapy journey from lab to patient depends on something many people dont seemanufacturing. Gain insights on the complex behind-the-scenes process of making AAV gene therapies and why it matters to the patient community. This unit was developed in partnership with the Bespoke Gene Therapy Consortium (BGTC). Listen to Quick Takes from experts in the community on the following questions: 00:00 - What is a plasmid and how does it help in making a gene therapy 04:24 - Why are quality checks so critical when making gene therapies and what kinds" [YouTube Link](https://youtube.com/watch?v=BfIrgjL50Ro) 2025-07-30T11:01Z 13.5K followers, [---] engagements "The Business of Advocates Advancing CGTs: Different non-profit business models and advice to PAGs This a recorded presentation from the workshop titled "The Business of Advocates Advancing CGTs" that took place during AM25" [YouTube Link](https://youtube.com/watch?v=fheAAUrrins) 2025-07-23T14:01Z 13.5K followers, [--] engagements "Investigators guide to identifying and validating biomarkers for clinical trials This a recorded presentation from the session titled "To Biomarker Or Not To Biomarker Use Considerations For CGT Drug Development" Organized by the Patient Outreach Committee during AM25" [YouTube Link](https://youtube.com/watch?v=S1LRtC6oH_s) 2025-07-23T14:00Z 13.5K followers, [---] engagements "Case study of how CSF biomarkers were approved by the FDA in MPS This a recorded presentation from the session titled "To Biomarker Or Not To Biomarker Use Considerations For CGT Drug Development" Organized by the Patient Outreach Committee during AM25" [YouTube Link](https://youtube.com/watch?v=SAoLYyodpzQ) 2025-07-23T14:00Z 13.5K followers, [--] engagements "What are Biomarkers A regulators guide to using biomarkers in an approved therapy This a recorded presentation from the session titled "To Biomarker Or Not To Biomarker Use Considerations For CGT Drug Development" Organized by the Patient Outreach Committee during AM25" [YouTube Link](https://youtube.com/watch?v=PBluQBHS7qI) 2025-07-23T14:00Z 13.5K followers, [--] engagements "Case study of how biomarkers may be limited in DMD This a recorded presentation from the session titled "To Biomarker Or Not To Biomarker Use Considerations For CGT Drug Development" Organized by the Patient Outreach Committee during AM25" [YouTube Link](https://youtube.com/watch?v=Os0k1l5-nEw) 2025-07-23T14:00Z 13.5K followers, [--] engagements "A Vaccine for Cocaine Addiction with Dr. Stephen Kaminsky On this episode of Soundbites of the Annual Meeting we talked to Stephen Kaminsky PhD professor of research in genetic medicine at Weill Cornell Medical College and lead author of a phase [--] clinical trial of a new vaccine for cocaine addiction. In his conversation with ASGCT's Communications Committee Chair Lynnea Olivarez Dr. Kaminsky discusses: How the vaccine works and how its approach is different from other treatments Safety results from the unblinded data Implications this vaccine could have for other substance use disorders" [YouTube Link](https://youtube.com/watch?v=q-IY9szYajw) 2025-07-22T10:07Z 13.5K followers, [---] engagements "25 Years of Molecular Therapy with Drs. Beverly Davidson and Jeff Chamberlain This episode is the third in a series hosted by Molecular Therapy Editor-in-Chief Joseph Glorioso PhD that will cover comprehensive reviews of critical developments in the field of gene and cell therapy over the past [--] years. In this episode Dr. Glorioso will discuss the reviews Current trends in gene therapy to treat inherited disorders of the brain with author Beverly Davidson PhD The Childrens Hospital of Philadelphia and The road toward AAV-mediated gene therapy of Duchenne muscular dystrophy with author Jeff" [YouTube Link](https://youtube.com/watch?v=vdlSa3B_RiE) 2025-07-16T10:39Z 13.5K followers, [---] engagements "Developing Therapeutic Platforms with Dr. P.J. Brooks During this episode of Soundbites of the Annual Meeting we spoke with P.J. Brooks PhD who is Deputy Director of the Division of Rare Diseases Research Innovation part of the National Center for Advancing Translational Sciences (NCATS) at the National Institutes of Health (NIH). In his conversation with ASGCT's Communications Committee Chair Lynnea Olivarez Dr. Brooks talked about: The impact of developing cell and gene therapies as platforms to treat more than one disease Projects he's involved in that are working towards the platform" [YouTube Link](https://youtube.com/watch?v=GBvJk-CR6c4) 2025-07-09T10:24Z 13.5K followers, [--] engagements "Community Quick Takes: Biomarker Breakdown Hear from Dr. Rebecca Ahrens-Nicklas a featured speaker in the ASGCT Annual Meeting session To Biomarker or Not To Biomarker Use Considerations for Cell and Gene Therapy Drug Development. Its a big question to answer but were providing some insights on how biomarkers may can help move cell and gene therapies forward and emphasizing the critical role of patient advocacy groups in shaping outcome measures. Listen to Quick Takes from experts in the community on the following questions: 01:25 - What exactly is a biomarker and how do they differ from" [YouTube Link](https://youtube.com/watch?v=_YNCuJZOUXs) 2025-06-24T17:00Z 13.5K followers, [---] engagements "The Ups and Downs of Conducting Groundbreaking Research with Dr. Kiran Musunuru "Certainly I've been anxious about data.I did an experiment I put a lot of time into it I'm hoping to get good results.we all know that feeling right The stakes are just so different here.this kid's life depends on whether this experiment worked." --Kiran Musunuru MD PhD One of the highlights of the ASGCT 28th Annual Meeting wasthe announcement on May [--] of a historic medical breakthrough (https://www.chop.edu/news/worlds-first-patient-treated-personalized-crispr-gene-editing-therapy-childrens-hospital) : a child" [YouTube Link](https://youtube.com/watch?v=g7zeR0RK49A) 2025-06-16T10:15Z 13.5K followers, [---] engagements "25 Years of Molecular Therapy with Sheila Mikhail and Dr. Mark Kay In honor of Molecular Therapys 25th anniversary this episode is the second in a series hosted by Dr. Joseph Glorioso Editor-in-Chief of Molecular Therapy that will cover comprehensive reviews of critical developments in the field of gene and cell therapy over the past [--] years. In this episode Dr. Glorioso will discuss the reviews AAV vector development back to the future" [YouTube Link](https://youtube.com/watch?v=nbumrYTew2A) 2025-06-11T10:20Z 13.5K followers, [---] engagements "Around the World: Frontline of AAV Vectors for Gene Therapy Research in Japan Explore cutting-edge advancements in the use of AAV vectors for gene therapy research in Japan. This event brings together leading scientists researchers and professionals in the field to discuss innovative approaches challenges and breakthroughs in gene delivery systems specifically focusing on AAV technology. ASGCT Japan AAV Technology ASGCT Japan AAV Technology" [YouTube Link](https://youtube.com/watch?v=Ftr0NR0rWWQ) 2025-05-14T15:22Z 13.5K followers, [---] engagements "25 Years of Molecular Therapy with Drs. Terry Flotte and Carl June In honor of Molecular Therapys 25th anniversary this episode is the first in a series hosted by Dr. Joseph Glorioso Editor-in-Chief of Molecular Therapy that will cover comprehensive reviews of critical developments in the field of gene and cell therapy over the past [--] years by expert authors. In this episode Dr. Glorioso will discuss two of these reviews Gene therapy then and now: A look back at changes in the field over the past [--] years with author Dr. Terry Flotte UMass Chan Medical School and From concept to cure: The" [YouTube Link](https://youtube.com/watch?v=DrNkt3AyZnQ) 2025-05-01T10:26Z 13.5K followers, [---] engagements "Empowering Patients 2025: A Cell and Gene Therapies Summit - Day [--] This is the Day [--] recording of the inaugural virtual event held on March [--] [----]. This event will provide timely education and engaging discussions on cell and gene therapies (CGTs). In alignment with ASGCTs strategic plan the goal is to empower advocates patients and caregivers who are vital in developing treatments. Attendees will hear from experts in the CGT field as well as patient advocacy group leaders. Agenda: Morning Coffee with Dr. Peter Marks CGT Manufacturing Basics The Journey from Early-stage Trials to Approval" [YouTube Link](https://youtube.com/watch?v=P09DChWdQE0) 2025-04-21T18:22Z 13.5K followers, [---] engagements "Empowering Patients 2025: A Cell and Gene Therapies Summit - Day [--] This is the Day [--] recording of the inaugural virtual event held on March [--] [----]. This event will provide timely education and engaging discussions on cell and gene therapies (CGTs). In alignment with ASGCTs strategic plan the goal is to empower advocates patients and caregivers who are vital in developing treatments. Attendees will hear from experts in the CGT field as well as patient advocacy group leaders. Agenda: Partners in Progress: The Value of Patients and Advocates Intro to Gene Therapy Approaches Review of Clinical" [YouTube Link](https://youtube.com/watch?v=N4JfTDF18Tk) 2025-04-21T17:51Z 13.5K followers, [---] engagements "The Importance of Long-term Data with Dolan Sondhi PhD - The Issue On this episode of The Issue Host Emily Walsh Martin PhD is tackling her absolute favorite subject: the importance of long-term data Emily is joined by Dolan Sondhi PhD of Weill Cornell Medical College to discuss her recent paper Twenty-Year Survival Analysis of Adeno-Associated Virus Vector Serotype 2-Mediated Gene Therapy to the Central Nervous System for CLN2 Disease. We hope to see all of your long-term data at the ASGCT Annual Meeting the premier event in cell and gene therapy next month Join us in New Orleans May 13-17" [YouTube Link](https://youtube.com/watch?v=RzpZB6yb_Zs) 2025-04-14T10:31Z 13.5K followers, [--] engagements "Community Quick Takes: Access to Approved Cell and Gene Therapies Originally released in May [----] A person considering a gene or cell therapy treatment may experience barriers to accessing the treatment. Our experts will explore the various meanings of the term access. This includes the development of therapies eligibility to receive an FDA-approved treatment coverage provider referral and being able to navigate the logistics of treatment within a clinic. Each expert touches on aspects that shape the field and impact an individuals access to gene and cell treatments. Listen to Quick Takes" [YouTube Link](https://youtube.com/watch?v=jXV8KkBFpkA) 2025-04-11T17:28Z 13.5K followers, [---] engagements "Community Quick Takes: State of the Field - Highlights and Approvals for CGT in [----] Originally released in February [----] The field of gene and cell therapy continues to grow and change with the approval of many new therapies this past year. There is much to highlight about the patient communities affected and what it means for future findings in treatments of rare and genetic conditions. Check out the quick takes below from Peter Marks MD PhD and Jeffrey Chamberlain PhD adddressing the following questions: - What were some of the field highlights for you this past year - What sort of" [YouTube Link](https://youtube.com/watch?v=wTOZz7ADBHg) 2025-04-11T17:21Z 13.5K followers, [--] engagements "Community Quick Takes: Informed Consent Originally released in November [----] Listen to [--] Quick Takes from experts in the community on the following questions: -What are five tips for a patient or caregiver to be prepared for informed consent -What challenges do participants face while going through the consent process and what actions can researchers take to improve the process -How do we facilitate informed consent for approved therapies *_Want to dive deeper into this topic Visit our patient education site for the full blog:_* https://patienteducation.asgct.org/quick-takes/informed-consent" [YouTube Link](https://youtube.com/watch?v=ykFY4of1r-Q) 2025-04-11T17:20Z 13.5K followers, [--] engagements "The Big Picture: Accountability in an Evolving Ecosystem ASGCT is excited to host a thought-provoking conversation moderated by Jodi Wolff PhD to further deconstruct issues of responsibility and obligation in the post-approval gene therapy landscape. This fireside chat will address topics related to data capture for long-term monitoring for durability and safety standardization to enable comparability to facilitate/identify next-generation therapies and tensions that exist in the evolving landscape. ASGCT Ethics Webinar Accountability Gene Therapy Data Capture ASGCT Ethics Webinar" [YouTube Link](https://youtube.com/watch?v=auuiHWRfqU0) 2025-03-31T20:24Z 13.5K followers, [--] engagements "The When What How and Why of FDA Engagement with Niki Gallo - The Issue Today Niki Gallo principal consultant of regulatory affairs at Halloran Consulting joins host Emily Walsh Martin for a deep dive into timeline management pitfalls the nuances of INDs and the rapidly evolving FDA landscape. Niki brings to the program her extensive experience shepherding innovative therapeutics through the regulatory process and timely insights into the complexities of transitioning from the bench to the bedside. Whether youre from industry or academia todays conversation will help wrap all our heads around" [YouTube Link](https://youtube.com/watch?v=J7NdGWMgILI) 2025-03-11T10:25Z 13.5K followers, [---] engagements "Incorporating miRNAs into a taRNA vector platform with Drs. Tim Beissert and Ayegl Yildiz - The. Drs. Ayegl Yildiz and Tim Beissert join Dr. Paloma Giangrande to discuss a recent article published in Molecular Therapy Nucleic Acids by Drs. Yildiz. Beissert and colleagues titled Trans-amplifying RNA expressing functional miRNA mediates target gene suppression and simultaneous transgene expression. Are you registered for ASGCTs [----] Annual Meeting in New Orleans Join us May 13-17 for the premier event in our field and save $465 as an ASGCT member. Check out the full program plan your trip and" [YouTube Link](https://youtube.com/watch?v=n0UFQO_HcXI) 2025-03-04T11:28Z 13.5K followers, [---] engagements Limited data mode. Full metrics available with subscription: lunarcrush.com/pricing
@asgct ASGCT
ASGCT posts on YouTube about community, education, hosted, the world the most. They currently have [------] followers and [---] posts still getting attention that total [---] engagements in the last [--] hours.
Engagements: [---] #
Mentions: [--] #
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CreatorRank: [---------] #
Social Influence
Social category influence stocks finance countries technology brands cryptocurrencies musicians
Social topic influence community, education, hosted, the world, pfizer inc #4, gene, health, aav, target, future
Top assets mentioned Frontline Ltd. (FRO) Pfizer, Inc. (PFE)
Top Social Posts
Top posts by engagements in the last [--] hours
"Hemophilia and Gene Therapy Hemophilia is a genetic disease that prevents blood from clotting properly leading to prolonged internal and external bleeding. There are existing treatments for hemophilia that require lifelong administration while gene therapy may offer a one-time treatment to address the cause of the disease. The subtitling of these videos was funded in part by a donation from Pfizer Inc. hemophilia American Society of Gene and Cell Therapy gene therapy hemophilia American Society of Gene and Cell Therapy gene therapy"
YouTube Link 2019-11-11T18:16Z 13.5K followers, 48.3K engagements
"Viral Vectors Overview Vectors are essentially vehicles designed to deliver therapeutic genetic material such as a working gene directly into a cell. The subtitling of these videos was funded in part by a donation from Pfizer Inc"
YouTube Link 2020-09-01T14:03Z 13.5K followers, 173.1K engagements
"Community Quick Take: Genetic Reports + Whats Next A common experience for people diagnosed with a genetic condition is receiving a genetic test result which often comes with many questions emotions and unfamiliar terms. In this unit Kim Nye founder of the TESS Research Foundation and parent advocate is joined by genetic counselor Dr. Lauren Beretich to walk through whats in a genetic report and how this important information can help guide a familys next steps. Read more: https://patienteducation.asgct.org/research-clinical-trials/community-quick-takes/genetic-reports-whats-next"
YouTube Link 2026-02-18T12:30Z 13.5K followers, [--] engagements
"Basics of AAV Gene Therapy Basics of AAV Gene Therapy - Steven Gray Education Session from the American Society of Gene & Cell Therapy's 22nd Annual Meeting"
YouTube Link 2019-11-27T18:19Z 13.5K followers, 81.5K engagements
"Spinal Muscular Atrophy Gene therapy is a fitting approach for diseases caused by a single gene mutation like SMA. It targets the cause of disease by delivering a fully functioning copy of the gene into motor neuron cells. Lets learn more about how it works. The subtitling of these videos was funded in part by a donation from Pfizer Inc"
YouTube Link 2019-02-01T09:00Z 12.5K followers, 45.3K engagements
"Around the World: Oncology Hematology and Cancer-Focused Cell and Gene Therapy in India This webinar explores Indias rapid advancements in CAR T therapies including homegrown innovations. This program highlights clinical successes increased accessibility and research leadership showcasing Indias growing impact on global cancer and hematology gene therapy landscapes"
YouTube Link 2025-10-06T14:43Z 13.5K followers, [---] engagements
"NIH and FDA Cooperations - Dr. Barrie J. Carter PhD"
YouTube Link 2011-09-30T16:39Z 12.6K followers, [---] engagements
"Increasing Equitable Access with Hayley Lam PhD At #ASGCT2024 Dr. Lam (CIRM) spoke during a symposium on developing CGTs with a DEI-forward approach. In this interview she talks about incorporating DEI into the review criteria for CIRM's funded programs how to measure DEI and what she'd like to see in the long term for patients. Meeting registrants can watch this session (and all others) through June [--] on the virtual platform (https://annualmeeting.app.swapcard.com/c/asgct) and the app Host: Lynnea Olivarez incoming Communications Commmittee Chair Show your support for ASGCT:"
YouTube Link 2024-06-07T21:24Z 13.2K followers, [--] engagements
"Gene Therapy for Cardiac Symptoms in Friedreichs Ataxia Gene Therapy for Cardiac Symptoms in Friedreichs Ataxia - Hlne Puccio Scientific Symposium from the American Society of Gene & Cell Therapy's 22nd Annual Meeting"
YouTube Link 2019-11-27T19:48Z 13.2K followers, [---] engagements
"Cell Therapy Development for Solid Tumors with AffyImmune's Sonal Gupta MD PhD - The Issue Sonal Gupta MD PhD SVP and Head of Clinical Development at Affyimmune Therapeutics talks with host Emily Walsh Martin PhD to explore the challenges of cell therapy clinical trials and how to move patients quickly and thoughtfully through the steps required to receive therapy in aggressive solid tumor cancers. But first "weve moved straight from pumpkin spice season into ASGCT Annual Meeting registration season" as Dr. Walsh Martin says. So hop online (https://annualmeeting.asgct.org/register) and join"
YouTube Link 2024-12-10T11:18Z 12.1K followers, [--] engagements
"Gene Therapy Approaches (2022 Update) Gene therapy cell therapy and gene editing are fields of biomedical research with a similar goal in mind: To treat disease by targeting the cause of the disease. Learn more below about these different approaches along with common challenges risks and benefits"
YouTube Link 2022-12-14T22:14Z 12.8K followers, 17.9K engagements
"Community Quick Takes: Manufacturing Matters The gene therapy journey from lab to patient depends on something many people dont seemanufacturing. Gain insights on the complex behind-the-scenes process of making AAV gene therapies and why it matters to the patient community. This unit was developed in partnership with the Bespoke Gene Therapy Consortium (BGTC). Listen to Quick Takes from experts in the community on the following questions: 00:00 - What is a plasmid and how does it help in making a gene therapy 04:24 - Why are quality checks so critical when making gene therapies and what kinds"
YouTube Link 2025-07-30T11:01Z 13.5K followers, [---] engagements
"Developing Therapeutic Platforms with Dr. P.J. Brooks During this episode of Soundbites of the Annual Meeting we spoke with P.J. Brooks PhD who is Deputy Director of the Division of Rare Diseases Research Innovation part of the National Center for Advancing Translational Sciences (NCATS) at the National Institutes of Health (NIH). In his conversation with ASGCT's Communications Committee Chair Lynnea Olivarez Dr. Brooks talked about: The impact of developing cell and gene therapies as platforms to treat more than one disease Projects he's involved in that are working towards the platform"
YouTube Link 2025-07-09T10:24Z 13.5K followers, [--] engagements
"CRISPR Tools for Gene Editing in the Brain CRISPR Tools for Gene Editing in the Brain - Jeremy Day Scientific Symposium from the American Society of Gene & Cell Therapy's 22nd Annual Meeting"
YouTube Link 2019-11-27T19:02Z 12K followers, 36K engagements
"Giants of Gene Therapy Podcast [--] - Terry Flotte MD Terry Flotte MD discusses the early days and possible future of gene therapy the use of AAV to deliver GT to targeted sites in the body and his inspiration behind becoming a physician scientist and medical school dean. He spoke one-on-one with ASGCT President Hans-Peter Kiem MD PhD. Welcome to the sixth episode of Giants of Gene Therapy Dr. Flotte is the Provost Dean and Celia and Isaac Haidak Professor at UMass Chan Medical School with faculty appointments in the Department of Pediatrics and the Horae Gene Therapy Center. He is also a"
YouTube Link 2023-11-02T21:19Z 11.9K followers, [---] engagements
"The When What How and Why of FDA Engagement with Niki Gallo - The Issue Today Niki Gallo principal consultant of regulatory affairs at Halloran Consulting joins host Emily Walsh Martin for a deep dive into timeline management pitfalls the nuances of INDs and the rapidly evolving FDA landscape. Niki brings to the program her extensive experience shepherding innovative therapeutics through the regulatory process and timely insights into the complexities of transitioning from the bench to the bedside. Whether youre from industry or academia todays conversation will help wrap all our heads around"
YouTube Link 2025-03-11T10:25Z 13.5K followers, [---] engagements
"CRISPR Editing and AAV Integration in the Brain with Drs. Beverly Davidson and Bryan SimpsonThe . Drs. Beverly Davidson and Bryan Simpson join Molecular Therapy EiC Dr. Roland Herzog to discuss their recent paper Targeted long-read sequencing captures CRISPR editing and AAV integration outcomes in the brain which was published in Molecular Therapy. ASGCT is proud to present this episode of the Molecular Therapy Podcast in partnership with our upcoming Advancing Gene and Cell Therapy for Cancer conference which will be held on October [--] and [--] in Philadelphia and online. This landmark hybrid"
YouTube Link 2024-09-10T20:45Z 12.8K followers, [---] engagements
"Empowering Patients 2025: A Cell and Gene Therapies Summit - Day [--] This is the Day [--] recording of the inaugural virtual event held on March [--] [----]. This event will provide timely education and engaging discussions on cell and gene therapies (CGTs). In alignment with ASGCTs strategic plan the goal is to empower advocates patients and caregivers who are vital in developing treatments. Attendees will hear from experts in the CGT field as well as patient advocacy group leaders. Agenda: Partners in Progress: The Value of Patients and Advocates Intro to Gene Therapy Approaches Review of Clinical"
YouTube Link 2025-04-21T17:51Z 13.5K followers, [---] engagements
"Empowering Patients 2025: A Cell and Gene Therapies Summit - Day [--] This is the Day [--] recording of the inaugural virtual event held on March [--] [----]. This event will provide timely education and engaging discussions on cell and gene therapies (CGTs). In alignment with ASGCTs strategic plan the goal is to empower advocates patients and caregivers who are vital in developing treatments. Attendees will hear from experts in the CGT field as well as patient advocacy group leaders. Agenda: Morning Coffee with Dr. Peter Marks CGT Manufacturing Basics The Journey from Early-stage Trials to Approval"
YouTube Link 2025-04-21T18:22Z 13.5K followers, [---] engagements
"Dr. Snehal Naik: "I Hope You'll Join the Conversation" at the Policy Summit Hear directly from Snehal Naik PhD Chair of ASGCT's Regulatory Affairs Committee about why she encourages people to join her at the Policy Summit. Learn more and register today: http://asgct.org/policysummit"
YouTube Link 2025-09-23T16:34Z 13.5K followers, [--] engagements
"The Importance of Long-term Data with Dolan Sondhi PhD - The Issue On this episode of The Issue Host Emily Walsh Martin PhD is tackling her absolute favorite subject: the importance of long-term data Emily is joined by Dolan Sondhi PhD of Weill Cornell Medical College to discuss her recent paper Twenty-Year Survival Analysis of Adeno-Associated Virus Vector Serotype 2-Mediated Gene Therapy to the Central Nervous System for CLN2 Disease. We hope to see all of your long-term data at the ASGCT Annual Meeting the premier event in cell and gene therapy next month Join us in New Orleans May 13-17"
YouTube Link 2025-04-14T10:31Z 13.5K followers, [--] engagements
"Education Session [---] Updates on Gene Therapies for Non Maligant Diseases of the Immune System"
YouTube Link 2017-09-26T21:44Z 12.4K followers, [---] engagements
"Molecular Therapy Podcast: COVID-19 Diagnostics with Dr. Maxim Berezovski Join Drs. Paloma Giangrande (Editor-in-Chief of Molecular Therapy Nucleic Acids and Chief Technology Officer at Eleven Therapy) and Maxim Berezovski (Professor of Bioanalytical Chemistry at the University of Ottawa). They will discuss a recent article published in Molecular Therapy Nucleic Acids by Dr. Berezovski and colleagues titled Discovery of DNA aptamers targeting SARS-CoV-2 nucleocapsid protein and protein-binding epitopes for label-free COVID-19 diagnostics"
YouTube Link 2024-02-13T11:05Z 10.6K followers, [--] engagements
"Update on Phase I/II Clinical Trial for Ornithine Transcarbamylase (OTC) Deficiency Using AAV Update on Phase I/II Clinical Trial for Ornithine Transcarbamylase (OTC) Deficiency Using AAV-mediated Gene Delivery - Cary Harding Scientific Symposium from the American Society of Gene & Cell Therapy's 21st Annual Meeting"
YouTube Link 2018-12-07T21:35Z 13.1K followers, [----] engagements
"My 40-year Journey to Develop RNA Based Therapies My 40-year Journey to Develop RNA Based Therapies - John Rossi Plenary Session from the American Society of Gene & Cell Therapy's 22nd Annual Meeting"
YouTube Link 2019-12-04T21:15Z 12K followers, [---] engagements
"Giants of Gene Therapy Podcast [--] - Kathy High MD Kathy High MD shares her personal and scientific journey from hemophilia to retinal disease during her one-on-one conversation with American Society of Gene & Cell Therapy President Hans-Peter Kiem MD PhD in the second episode of ASGCT's Giants of Gene Therapy. Dr. High is the president of therapeutics at AskBio. Before that she was the co-founder president and head of research and development at Spark Therapeutics where she oversaw the development approval and launch of Luxturna the first AAV gene therapy approved for use in the United States."
YouTube Link 2023-11-02T21:18Z 12.6K followers, [---] engagements
"Limb-Girdle Muscular Dystrophy and Gene Therapy One muscular dystrophy sub-group is called limb-girdle muscular dystrophy or LGMD. These diseases weaken the muscles of a persons shoulders upper arms hips and thighs. Each subtype of LGMD is caused by a different inherited faulty gene. The subtitling of these videos was funded in part by a donation from Pfizer Inc"
YouTube Link 2019-10-24T10:00Z 12.6K followers, 30.5K engagements
"What's the Difference Between Gene Therapy Cell Therapy and Gene Editing Gene therapy cell therapy and gene editing are fields of biomedical research with similar goals in mind: To treat disease by changing our bodies at a microscopic level. Specifically these approaches aim to change our genetic makeup. However each one changes our genetic material in slightly different ways. What do these approaches have in common and what makes them different Lets dive in. The subtitling of these videos was funded in part by a donation from Pfizer Inc"
YouTube Link 2019-01-17T10:00Z 13K followers, 70.4K engagements
"The Big Picture: Accountability in an Evolving Ecosystem ASGCT is excited to host a thought-provoking conversation moderated by Jodi Wolff PhD to further deconstruct issues of responsibility and obligation in the post-approval gene therapy landscape. This fireside chat will address topics related to data capture for long-term monitoring for durability and safety standardization to enable comparability to facilitate/identify next-generation therapies and tensions that exist in the evolving landscape. ASGCT Ethics Webinar Accountability Gene Therapy Data Capture ASGCT Ethics Webinar"
YouTube Link 2025-03-31T20:24Z 13.5K followers, [--] engagements
"Pluripotent Cell-Derived T and NK Cells as a Cornerstone Approach for Cancer Immunotherapy Pluripotent Cell-Derived T and NK Cells as a Cornerstone Approach for Off-the-Shelf Cancer Immunotherapy - Bob Valamehr Education Session from the American Society of Gene & Cell Therapy's 22nd Annual Meeting"
YouTube Link 2019-12-05T17:46Z 12.5K followers, [----] engagements
"cGMP and Building Cohesive Teams with Christopher Choi PhD Roswell Park Comprehensive Cancer Ce. Host Emily Walsh Martin PhD is joined by Chris Choi PhD Roswell Park Comprehensive Cancer Center's Senior Vice President of Industry Partnerships and Technical Director of the centers new Good Manufacturing Practice (cGMP) Facility. Dr. Choi is also an associate professor of oncology. This wide-ranging conversation covers cell therapy development and manufacturing and how the growing Roswell Park Center is aiming to support sponsors in the CAR T and cell therapy space. Emily and Chris also discuss"
YouTube Link 2024-11-12T11:19Z 11.9K followers, [--] engagements
"Research Briefing: Cell & Gene Therapy Approaches for HIV The Cell & Gene Therapy Approaches for HIV briefing hosted by ASGCT on December [--] [----] explored the potential of cell and gene therapies to address the challenges of curing HIV. Featuring researchers policymakers and a patient advocate the event highlighted advancements in CAR T-cell and AAV gene therapies as well as the importance of continued investment and advocacy in this field"
YouTube Link 2024-12-05T20:12Z 12K followers, [--] engagements
"Giants of Gene Therapy Podcast [--] - Jennifer Doudna PhD Jennifer Doudna PhD shares her personal journey to co-inventing CRISPR-Cas9 for gene editing and the promise of her discovery during a one-on-one conversation with ASGCT President Hans-Peter Kiem MD PhD. Welcome to the fifth episode of Giants of Gene Therapy Dr. Doudna has been at UC Berkeley since [----]. She is a professor in the departments of Molecular and Cell Biology and Chemistry the Li Ka Shing Chancellors Professor of Biomedical Science. Shes also the president and co-founder of the Innovative Genomics Institute. In [----] Dr. Doudna"
YouTube Link 2023-11-02T21:22Z 10K followers, [--] engagements
"IRB and Patient Safety - [--] Things You Must Know to Start a Human Gene Therapy Trial IRB and Patient Safety - [--] Things You Must Know to Start a Human Gene Therapy Trial - Sagar Vaidya Scientific Symposium from the American Society of Gene & Cell Therapy's 22nd Annual Meeting"
YouTube Link 2019-12-04T20:28Z 13.2K followers, [--] engagements
"The Business of Advocates Advancing CGTs: Different non-profit business models and advice to PAGs This a recorded presentation from the workshop titled "The Business of Advocates Advancing CGTs" that took place during AM25"
YouTube Link 2025-07-23T14:01Z 13.5K followers, [--] engagements
"Gene Therapy for Genetic Hearing Loss Genetic hearing loss happens when changes in certain genes affect how the hearing system develops and works. Learn more about the genetic factors contributing to hearing loss and the ongoing research on gene therapies aimed at improving outcomes for individuals who are deaf or hard of hearing"
YouTube Link 2024-12-16T16:28Z 13.1K followers, [----] engagements
"Mavis Agbandje-McKenna Ph.D. [----] ASGCT Outstanding Achievement Award Lecture During the [----] ASGCT Annual Meeting Mavis Agbandje-McKenna gave a talk on structure-guided AAV vectorology"
YouTube Link 2021-03-10T21:26Z 12.6K followers, [----] engagements
"Scientific Symposium [---] Progress Towards Gene Therapy for Muscular Dystrophies Organized by the"
YouTube Link 2017-09-26T21:44Z 13.2K followers, [--] engagements
"RNA Exon Editing with Ascidian Therapeutics' Drs. Robert Bell and Michael Ehlers - The Molecular . Join Dr. Paloma Giangrande Editor-in-Chief of Molecular Therapy Nucleic Acids as she discusses RNA exon editing with Ascidian Therapeutics' leadership: CSO Dr. Robert Bell and CEO Dr. Michael Ehlers. Their conversation explores Dr. Bell's recent MTNA publication "RNA Exon Editing: Splicing the Way to Treat Human Diseases." Last Call: Submit your abstracts for ASGCT's [----] Annual Meeting in New Orleans by January 31st 11:59 PM ET. Present at cell and gene therapy's premier event and get published"
YouTube Link 2025-01-07T11:35Z 12.2K followers, [--] engagements
"Next Generation Oncolytic Immunotherapy Next Generation Oncolytic Immunotherapy - Howard Kaufman Education Session from the American Society of Gene & Cell Therapy's 22nd Annual Meeting"
YouTube Link 2019-12-04T21:18Z 13.2K followers, [----] engagements
"Inside the Molecular Therapy Family of Journals Join us as past ASGCT President Paula Cannon Ph.D. and our Editors-in-Chief take you behind the scenes of ASGCTs field-leading Molecular Therapy family of journals. Discover how these prestigious publications have shaped the landscape of cell and gene therapy research for the past [--] years. Learn more: asgct.org/molecular-therapy"
YouTube Link 2025-09-09T15:02Z 13.5K followers, [---] engagements
"Nuclease-free Genome Editing Confers a Hepatocellular Growth Advantage for Corrected Cells in MMA Nuclease-free Genome Editing Confers a Hepatocellular Growth Advantage for Corrected Cells in Methylmalonic Acidemia (MMA) - Randy Chandler Scientific Symposium from the American Society of Gene & Cell Therapy's 22nd Annual Meeting"
YouTube Link 2019-12-04T21:24Z 12.3K followers, [---] engagements
"Ethics: from Clinical Trials to Products - Jonathan Kimmelman Ethics: from Clinical Trials to Products - Jonathan Kimmelman Scientific Symposium from the American Society of Gene & Cell Therapy's 21st Annual Meeting May [--] 2018"
YouTube Link 2018-12-07T21:24Z 13.2K followers, [---] engagements
"25 Years of Molecular Therapy with Drs. Beverly Davidson and Jeff Chamberlain This episode is the third in a series hosted by Molecular Therapy Editor-in-Chief Joseph Glorioso PhD that will cover comprehensive reviews of critical developments in the field of gene and cell therapy over the past [--] years. In this episode Dr. Glorioso will discuss the reviews Current trends in gene therapy to treat inherited disorders of the brain with author Beverly Davidson PhD The Childrens Hospital of Philadelphia and The road toward AAV-mediated gene therapy of Duchenne muscular dystrophy with author Jeff"
YouTube Link 2025-07-16T10:39Z 13.5K followers, [---] engagements
"Retinal Progenitor Cells for Treatment of Retinitis Pigmentosa - Henry Klassen Retinal Progenitor Cells for Treatment of Retinitis Pigmentosa - Henry Klassen Scientific Symposium from the American Society of Gene & Cell Therapy's 21st Annual Meeting"
YouTube Link 2018-12-07T21:24Z 12.7K followers, [----] engagements
"CAR T cells CAR T cells - Stephen Gottschalk Education Session from the American Society of Gene & Cell Therapy's 22nd Annual Meeting"
YouTube Link 2019-11-27T18:22Z 12.6K followers, [---] engagements
"Around the World: Australasian Gene and Cell Therapy Society (AGCTS) ASGCT and AGCTS hosted two virtual seminars organized as part of ASGCT's Around the World in collaboration with the Coalition of International Gene Therapy Societies. PROGRAM Back to Base-ics: Developing targeted base-editing approaches for NF single nucleotide substitutions Presented by Dr Alexandra ODonohue Alexandra is a Postdoctoral Research Fellow in the Bioengineering & Molecular Medicine laboratory at the Childrens Hospital at Westmead. She received her Bachelor of Medical Science in molecular biology and genetics in"
YouTube Link 2024-11-22T21:34Z 12K followers, [--] engagements
"Lunch & Learn: Meet the Patients Join us as patientshighlight their stories from navigating the diagnosis process to learning about and receiving investigational treatment options.We'll hear about life after receiving a therapy and patients' outlooks for the future of their disease communities"
YouTube Link 2023-02-03T22:36Z 13.3K followers, [---] engagements
"Update on Phase I/II Clinical Trial of Intracerebral Administration of rAAV2/5hNAGLU Vector Update on Phase I/II Clinical Trial of Intracerebral Administration of rAAV2/5hNAGLU Vector in Children with MPSIIIB - Marc Tardieu Scientific Symposium from the American Society of Gene & Cell Therapy's 21st Annual Meeting"
YouTube Link 2019-01-16T22:24Z 13.2K followers, [---] engagements
"Antisense Strategies Against Non-Liver Targets Antisense Strategies Against Non-Liver Targets - Brett Monia Scientific Symposium from the American Society of Gene & Cell Therapy's 22nd Annual Meeting"
YouTube Link 2019-11-27T18:07Z 13.3K followers, [---] engagements
"Inducing Immune Tolerance with Liver-directed Expression by AAV - Dwight Koeberl Inducing Immune Tolerance with Liver-directed Expression by AAV - Dwight Koeberl Education Session from the American Society of Gene & Cell Therapy's 21st Annual Meeting"
YouTube Link 2018-12-07T21:24Z 13.3K followers, [---] engagements
"CNS Gene Therapies with Todd Carter PhD Todd Carter PhD (CSO at Voyager Therapeutics) talks with incoming Communications Committee Chair Lynnea Olivarez about changes in the approach to developing gene therapies for CNS disorders like Huntington's disease. Show your support for ASGCT: https://asgct.org/membership/donate See omnystudio.com/listener (https://omnystudio.com/listener) for privacy information"
YouTube Link 2024-06-07T21:24Z 13.3K followers, [--] engagements
"Gene Therapy Basics Gene therapy has been studied for more than [--] years and can help stop or slow the effects of disease on the most basic level of the human bodyour genes. And to understand how it works well start at the basics. The subtitling of these videos was funded in part by a donation from Pfizer Inc"
YouTube Link 2019-01-17T10:00Z 13.3K followers, 387.6K engagements
"Scientific Symposium [---] Progress Towards Gene Therapy for Muscular Dystrophies Organized by the"
YouTube Link 2017-09-26T21:44Z 13.3K followers, [---] engagements
"Gene Therapy for GM1 Gangliosidosis GM1 gangliosidosis (GM1) is a rare genetic disorder that progressively damages cells primarily in the brain and spinal cord and other tissues as well. Learn how gene therapy may target the cause of disease to slow or stop its progression along with helpful information on clinical trials and staying informed. The subtitling of these videos was funded in part by a donation from Pfizer Inc"
YouTube Link 2021-07-28T18:07Z 13.4K followers, [----] engagements
"Adenovirus-Based Vaccine for COVID-19 Adenoviruses have been used in gene therapy and have also been researched for use in vaccines for decades. An adenovirus-based vaccine was authorized for emergency use to fight COVID-19 in the U.S. along with the two mRNA vaccines. Learn more about how the adenovirus-based vaccine works. The subtitling of these videos was funded in part by a donation from Pfizer Inc"
YouTube Link 2021-04-07T19:00Z 13.3K followers, 63.8K engagements
"RNA-based Drugs Including Delivery of Antibodies and Vaccines Using mRNA RNA-based Drugs Including Delivery of Antibodies and Vaccines Using mRNA - Drew Weissman Scientific Symposium from the American Society of Gene & Cell Therapy's 22nd Annual Meeting"
YouTube Link 2019-12-05T18:17Z 13.2K followers, [----] engagements
"Gene Therapy for Inherited Retinal Diseases Inherited retinal disordersor IRDsare diseases that can cause severe vision loss or even blindness. Each one is caused by a gene that is not working properly which creates an opportunity for gene therapy to help slow or stop disease progression. The subtitling of these videos was funded in part by a donation from Pfizer Inc"
YouTube Link 2019-03-01T15:07Z 13.2K followers, 44.4K engagements
"Design Synthesis and Application of Antibody Nucleic Acid Analogs as Novel Biopharmaceuticals Design Synthesis and Application of Antibody Nucleic Acid Analogs as Novel Biopharmaceuticals - Christopher Alabi Education Session from the American Society of Gene & Cell Therapy's 22nd Annual Meeting"
YouTube Link 2019-11-27T19:04Z 13.4K followers, [---] engagements
"Huntington's Disease: Can Gene Therapy Help Huntingtons disease is a genetic disorder caused by a breakdown of nerve cells in the brain. The disease affects an individuals ability to move their mood and how they think. Theres currently no cure for Huntingtons disease but there are types of gene therapy approaches that may offer hope for managing or slowing symptoms. The subtitling of these videos was funded in part by a donation from Pfizer Inc"
YouTube Link 2019-10-15T08:00Z 13.3K followers, 28.3K engagements
"CALD and MLD Leukodystrophies are a set of rare genetic disorders that affect how the central nervous system functions. Since these disorders are all caused by a faulty genes gene therapy might be able to help. Leukodystrophies are genetic disorders that lead to the deterioration or malformation of the myelin sheath a fatty insulation that wraps around nerve axons and is essential for proper neuron function. When the myelin sheath is altered it makes it hard for our nervous system to send messages to different parts of our body which can impair walking speech and vision. The subtitling of"
YouTube Link 2019-02-19T17:49Z 13.4K followers, [----] engagements
"ASGCT: The Home of Cell and Gene Therapy The American Society of Gene & Cell Therapy (ASGCT) has been the primary hub for professionals working in cell and gene therapy for more than [--] years. The work of ASGCT and our members is now more important than ever with therapeutics moving from laboratories into clinics and ultimately the lives of patients. Hear from ASGCT's members and elected leadership exactly why this community is an essential part of their careers and why new scientists should invest their time in ASGCT"
YouTube Link 2022-08-18T16:38Z 13.2K followers, [----] engagements
"Design and Translational Application of HSV Gene Vectors in Nervous System Diseases Design and Translational Application of HSV Gene Vectors in Nervous System Diseases - Joseph Glorioso Education Session from the American Society of Gene & Cell Therapy's 21st Annual Meeting May [--] 2018"
YouTube Link 2018-12-07T21:24Z 13.3K followers, [----] engagements
"mRNA Vaccines for COVID-19 The use of messenger RNA (mRNA) in medical interventions has been researched for the last [--] years including in vaccines hoping to fight diseases such as cancer rabies Ebola and the Zika virus. That means the research was ready to be applied to this new disease COVID-19. With the recent authorization of two mRNA vaccines for emergency use to fight COVID-19 and more vaccine candidates in development we can build protection against the virus in our communities if enough people are vaccinated. Learn more at patienteducation.asgct.org. The subtitling of these videos was"
YouTube Link 2020-12-30T14:30Z 13.3K followers, 37.4K engagements
"Nucleic Acid Delivery Systems for RNA Therapy and Gene Editing - Daniel Anderson Nucleic Acid Delivery Systems for RNA Therapy and Gene Editing - Daniel Anderson Scientific Symposium from the American Society of Gene & Cell Therapy's 21st Annual Meeting"
YouTube Link 2018-12-07T21:24Z 13.3K followers, [---] engagements
"Fabry Disease and Gene Therapy Fabry disease is a rare genetic disorder caused by a lacking enzyme due to a faulty gene. Gene therapy aims to offer a one-time treatment to slow or ideally stop progression of the disease by introducing a working GLA gene to provide instructions to the cells to produce the deficient enzyme. The subtitling of these videos was funded in part by a donation from Pfizer Inc"
YouTube Link 2020-09-21T19:28Z 13.3K followers, [----] engagements
"HSPC Therapies - Engineering Cell Fitness: Enhancing HSPC Homing and Engraftment HSPC Therapies - Engineering Cell Fitness: Enhancing HSPC Homing and Engraftment - Hal Broxmeyer Scientific Symposium from the American Society of Gene & Cell Theray's 22nd Annual Meeting"
YouTube Link 2019-12-04T19:51Z 13.3K followers, [---] engagements
"Selective Targeting of Mutant Huntingtin Using Stereopure Oligonucleotides Selective Targeting of Mutant Huntingtin Using Stereopure Oligonucleotides as a Potential Therapeutic Approach for Huntingtons Disease - Michael Panzara Scientific Symposium from the American Society of Gene & Cell Therapy's 22nd Annual Meeting"
YouTube Link 2019-12-05T18:19Z 13.2K followers, [---] engagements
"The Issue - Unlocking New Therapeutic Potential: Ensomas Platform for Rare Disease and Oncology Host Emily Walsh Martin welcomes Dr. Jim Burns CEO of Ensoma to discuss the company's novel technological approach to treating both rare diseases and solid tumor oncology. Dr. Burns provides a detailed overview of Ensoma's platform and its application in their ongoing clinical trials including the recently announced first patient infusion for chronic granulomatous disease. Learn how this initial trial is set to inform future therapeutic opportunities for indications with high unmet medical need."
YouTube Link 2025-12-16T11:15Z 13.5K followers, [--] engagements
"Tay-Sachs and Sandhoff Disease (GM2) Tay-Sachs disease and Sandhoff disease are the two most common types of GM2 gangliosidosis (GM2) a group of rare fatal genetic disorders that progressively damage nerve cells primarily in the brain and spinal cord. Learn how gene therapy is being researched as a potential one-time treatment that could slow or stop disease progression for both diseases. The subtitling of these videos was funded in part by a donation from Pfizer Inc"
YouTube Link 2021-09-13T20:05Z 13.4K followers, 33.8K engagements
"Automated and Decentralized CAR-T cell Manufacturing using Lentiviral vectors - Boro Dropulic Automated and Decentralized CAR-T cell Manufacturing using Lentiviral vectors: Bench to Commercialization Scientific Symposium from the American Society of Gene & Cell Therapy's 22nd Annual Meeting"
YouTube Link 2019-11-27T18:10Z 13.3K followers, [----] engagements
"Universal Gene-Edited CAR T-Cell Immunotherapy Universal Gene-Edited CAR T-Cell Immunotherapy - Philippe Duchateau Scientific Symposium from the American Society of Gene & Cell Therapy's 22nd Annual Meeting"
YouTube Link 2019-12-05T18:53Z 13.4K followers, [---] engagements
"Utilizing Expedited Designations in Moving Toward Market Approval Utilizing Expedited Designations in Moving Toward Market Approval - Anne-Virginie Eggimann Scientific Symposium from the American Society of Gene & Cell Therapy's 22nd Annual Meeting"
YouTube Link 2019-12-05T18:55Z 13.3K followers, [---] engagements
"House Cancer Caucus Briefing: Immunotherapies Hear from a panel of ASGCT members who spoke to the House Cancer Caucus and Hill staff about CAR T and immunotherapies for cancer indications"
YouTube Link 2023-01-26T15:32Z 13.5K followers, [---] engagements
"Community Quick Takes: Access to Approved Cell and Gene Therapies Originally released in May [----] A person considering a gene or cell therapy treatment may experience barriers to accessing the treatment. Our experts will explore the various meanings of the term access. This includes the development of therapies eligibility to receive an FDA-approved treatment coverage provider referral and being able to navigate the logistics of treatment within a clinic. Each expert touches on aspects that shape the field and impact an individuals access to gene and cell treatments. Listen to Quick Takes"
YouTube Link 2025-04-11T17:28Z 13.5K followers, [---] engagements
"Transthyretin Amyloidosis Transthyretin amyloidosis (ATTR) is a condition that gradually worsens over time and affects how certain organs work especially the heart and the nerves. This unit will explore the causes of ATTR amyloidosis and how new gene therapies are being developed to potentially slow or stop disease progression"
YouTube Link 2024-12-16T16:28Z 13.4K followers, [----] engagements
"CRISPR and Beyond with Vertex CSO David Altshuler MD PhDASGCT's The Issue Join host Emily Walsh Martin PhD and David Altshuler MD PhD Chief Scientific Officer of Vertex Pharmaceuticals. David came to Vertex after a lengthy career in medicine and academic research. David and Emily discuss how David and the team at Vertex think about innovation in drug development and how that impacts how they select programs and approaches for the diseases they focus on. They also cover what hed love to see happen with new cell and gene platform technologies being brought forward. Now is the time to register"
YouTube Link 2024-07-16T10:21Z 13.5K followers, [---] engagements
"Around the World: Advanced Therapy Medicinal Production (ATMP) Research in South America Explore the latest developments in Advanced Therapy Medicinal Product (ATMP) research across South America including innovative production techniques and regional advancements driving the field forward"
YouTube Link 2024-12-03T16:00Z 13.4K followers, [---] engagements
"Readministration of AAV vector in Pompe Disease - Manuela Corti Readministration of AAV vector in Pompe Disease - Manuela Corti Scientific Symposium from the American Society of Gene & Cell Therapy's 21st Annual Meeting"
YouTube Link 2018-12-07T21:24Z 13.3K followers, [---] engagements
"Lunch & Learn: Gene Therapy [---] During this educational session experts in the field reviewed different gene therapy approaches disease applications and research processes and described the current state of the field"
YouTube Link 2022-02-28T16:06Z 13.3K followers, 42.7K engagements
"Education Session [---] The ABCs of AAV"
YouTube Link 2017-09-26T21:44Z 13.4K followers, [----] engagements
"Technological Frontiers in South American Gene and Cell Therapy As cell and gene therapies revolutionize modern medicine South America is emerging as a dynamic player on the global stage leveraging scientific innovation regulatory evolution and regional collaboration. Explore the technological frontiers shaping the development and deployment of advanced therapies across the continent"
YouTube Link 2026-01-26T22:05Z 13.5K followers, [--] engagements
"Why Leading Biotech Companies Choose ASGCT Inside the Industry's Premier Gene & Cell Therapy Event Discover why industry leaders make ASGCT's Annual Meeting a cornerstone of their business strategy. Three exhibitors and sponsors share their success stories and reveal the unique opportunities that make the ASGCT Annual Meeting invaluable for gene and cell therapy companies. 🎯 In this video: @Regeneron @GenScript @Bio-techne Hear directly from industry decision-makers Learn about ROI from exhibition investments Understand networking opportunities Explore why ASGCT stands out from other"
YouTube Link 2024-10-25T20:17Z 13.3K followers, [---] engagements
"mRNA Manufacturing mRNA Manufacturing - Anton McCaffrey Education Session from the American Society of Gene & Cell Therapy's 22nd Annual Meeting"
YouTube Link 2019-12-04T21:13Z 13.5K followers, 46.8K engagements
"Around the World: Frontline of AAV Vectors for Gene Therapy Research in Japan Explore cutting-edge advancements in the use of AAV vectors for gene therapy research in Japan. This event brings together leading scientists researchers and professionals in the field to discuss innovative approaches challenges and breakthroughs in gene delivery systems specifically focusing on AAV technology. ASGCT Japan AAV Technology ASGCT Japan AAV Technology"
YouTube Link 2025-05-14T15:22Z 13.5K followers, [---] engagements
"Improving AAV Production with Adam Schieferecke PhD + David Schaffer PhD Dr. Roland Herzog sits down with Drs. David Schaffer and Adam Schieferecke to discuss a recent article published in the February issue of Molecular Therapy by Drs. Schaffer and Schieferecke and colleagues titled Evolving membrane-associated accessory protein variants for improved adeno-associated virus production. Read the full article: https://www.cell.com/molecular-therapy-family/molecular-therapy/abstract/S1525-0016(23)00679-2 Dr. Roland Herzog: Editor-in-Chief of Molecular Therapy and Professor of Pediatrics at"
YouTube Link 2024-03-12T10:00Z 13.5K followers, [---] engagements
"Elevating Your Research Career Dr. Anna Blakney on ASGCT Member Benefits In the fast-moving world of gene and cell therapy technical skills are only half the battle. Dr. Anna Blakney (Assistant Professor at UBC) explains why ASGCT is the essential home for professionals in the field. From accessing the latest research in Molecular Therapy to navigating the Career Center ASGCT membership offers a comprehensive suite of tools designed to benefit your career at every stage. 🔗 Become an ASGCT member today: asgct.org/become-a-member The American Society of Gene & Cell Therapy is the primary"
YouTube Link 2026-02-09T17:44Z 13.5K followers, [--] engagements
"Next-generation AAV Vectors Next-generation AAV Vectors - Dirk Grimm Education Session from the American Society of Gene & Cell Therapy's 22nd Annual Meeting"
YouTube Link 2019-12-04T21:20Z 13.5K followers, [----] engagements
"Investigators guide to identifying and validating biomarkers for clinical trials This a recorded presentation from the session titled "To Biomarker Or Not To Biomarker Use Considerations For CGT Drug Development" Organized by the Patient Outreach Committee during AM25"
YouTube Link 2025-07-23T14:00Z 13.5K followers, [---] engagements
""Inspire and Be Inspired" Dr. Ruth Castellanos on the Power of the ASGCT Community "ASGCT has empowered me to become who I am right now." What does it mean to be a member of the American Society of Gene & Cell Therapy For Dr. Ruth Castellanos (Director of R&D at Kriya Therapeutics) it means more than just a professional affiliationits about finding direction and inspiration. Whether you are looking to be inspired by the latest science or hoping to inspire others with your work there is a place for you here. 🔗 Become an ASGCT member today: asgct.org/become-a-member The American Society of"
YouTube Link 2026-02-05T16:58Z 13.5K followers, [--] engagements
"Recent CMC Guidance for Cellular and Gene Therapies: BIO-ASGCT CGT Science Series Hear from Dr. Andrew Byrnes during the third installment of the BIO-ASGCT CGT Science Series"
YouTube Link 2024-04-04T19:18Z 13.5K followers, [----] engagements
"AAV Capsid Production with Drs. Mario Mietzsch and Robert McKenna - Molecular Therapy Podcast Dr. Daniel Stone speaks with Drs. Mario Mietzsch and Robert McKenna from the University of Florida to discuss a recent article published in the Biomanufacturing in Gene and Cell Therapy special issue of Molecular Therapy Methods & Clinical Development (https://www.sciencedirect.com/special-issue/10RZ8HRVPL2) by Drs. Mietzsch McKenna and colleagues titledProduction and characterization of an AAV1-VP3-only capsid: An analytical benchmark standard"
YouTube Link 2024-06-18T10:08Z 13.5K followers, [---] engagements
"Generation of Hematopoietic Stem and Progenitor Cells from iPSCs Generation of Hematopoietic Stem and Progenitor Cells from iPSCs - Andre Larochelle Education Session from the American Society of Gene & Cell Therapy's 22nd Annual Meeting"
YouTube Link 2019-11-27T19:52Z 13.5K followers, [----] engagements
"The Issue: AAV Immunogenicity with Federico Mingozzi PhD Dr. Mingozzi has done seminal work throughout his career in AAV gene therapy and his [----] Molecular Therapy review of our current understanding of the immune reaction to these therapies is a must-read for anyone trying to get a handle on the very complicated landscape of immune reactions to viral vectors"
YouTube Link 2024-02-19T17:42Z 13.5K followers, [---] engagements
"Gene Editing Gene editing aims to be a one-time therapy that directly edits pieces of DNA within the cell. It's considered a type of gene therapy which is the use of genetic material to treat or prevent disease. The subtitling of these videos was funded in part by a donation from Pfizer Inc"
YouTube Link 2021-11-02T20:12Z 13.5K followers, 13.1K engagements
"How Do Clinical Trials Work New therapies spend a lot of time being tested in labs and animals in preclinical studies before making it to patients. By the time the therapy enters human clinical trials it is better understood and ready for further development. Learn more information about the process of developing a treatment. The subtitling of these videos was funded in part by a donation from Pfizer Inc. gene therapy cell therapy clinical trials scientific development biotechnology gene editing crispr science research medical research gene therapy cell therapy clinical trials scientific"
YouTube Link 2019-01-17T14:04Z 13.5K followers, 19K engagements
"Engineered iPSC-derived Lymphocytes for Improved Cancer Therapy Engineered iPSC-derived Lymphocytes for Improved Cancer Therapy - Dan Kaufman Scientific Symposium from the American Society of Gene & Cell Therapy's 22nd Annual Meeting"
YouTube Link 2019-11-27T19:22Z 13.5K followers, [----] engagements
"Community Quick Takes: State of the Field - Highlights and Approvals for CGT in [----] Originally released in February [----] The field of gene and cell therapy continues to grow and change with the approval of many new therapies this past year. There is much to highlight about the patient communities affected and what it means for future findings in treatments of rare and genetic conditions. Check out the quick takes below from Peter Marks MD PhD and Jeffrey Chamberlain PhD adddressing the following questions: - What were some of the field highlights for you this past year - What sort of"
YouTube Link 2025-04-11T17:21Z 13.5K followers, [--] engagements
"Policy and Advocacy Shorts with Jennifer Wellman Jennifer Wellman Chief Development Officer at Akouos discusses ASGCT's role in addressing manufacturing and access challenges in gene therapy and shares her excitement about the field's rapid progress"
YouTube Link 2024-07-25T14:47Z 13.5K followers, [--] engagements
"Welcome to ASGCT: Who We Are and What We Do Who are we at the American Society of Gene & Cell Therapy We're known for holding the world's premier gene therapy cell therapy and gene editing conference but our members are doing so much more. For thousands of researchers clinicians and patient advocates ASGCT is a community with which they can stay plugged into one of medicine's most exciting fields. Welcome to ASGCT. gene therapy gene editing crispr asgct medical research cell therapy CAR T-Cell gene therapy gene editing crispr asgct medical research cell therapy CAR T-Cell"
YouTube Link 2018-07-24T20:32Z 13.5K followers, [----] engagements
"What are Biomarkers A regulators guide to using biomarkers in an approved therapy This a recorded presentation from the session titled "To Biomarker Or Not To Biomarker Use Considerations For CGT Drug Development" Organized by the Patient Outreach Committee during AM25"
YouTube Link 2025-07-23T14:00Z 13.5K followers, [--] engagements
"25 Years of Molecular Therapy with Drs. Rayne Rouce and Joe Glorioso This episode is the fourth in a series hosted by Molecular TherapyEditor-in-Chief Joseph Glorioso PhD that will cover comprehensive reviews of critical developments in the field of gene and cell therapy over the past [--] years. In this episode Dr. Glorioso will discuss the review How to democratize cell and gene therapy: A global approach"
YouTube Link 2025-08-08T10:12Z 13.5K followers, [---] engagements
"Immunogenicity of AAV Vectors and Transgenes - Roland Herzog Immunogenicity of AAV Vectors and Transgenes - Roland Herzog Education Session from the American Society of Gene & Cell Therapy's 21st Annual Meeting May [--] 2018"
YouTube Link 2018-12-07T21:24Z 13.5K followers, [----] engagements
"Development of an AAV-based Prophylactic Vaccine for Influenza Development of an AAV-based Prophylactic Vaccine for Influenza - Maria Limberis Scientific Symposium from the American Society of Gene & Cell Therapy's 22nd Annual Meeting"
YouTube Link 2019-11-27T19:06Z 13.5K followers, [---] engagements
"Policy and Advocacy Shorts with Diane Berry PhD Diane Berry PhD Executive Vice President Chief Global Policy & Advocacy Officer at Sarepta Therapeutics highlights ASGCT's unique role in bringing together diverse stakeholders and providing evidence-based advocacy to advance policies in cell and gene therapy"
YouTube Link 2024-07-25T14:20Z 13.5K followers, [--] engagements
"Community Quick Takes: Biomarker Breakdown Hear from Dr. Rebecca Ahrens-Nicklas a featured speaker in the ASGCT Annual Meeting session To Biomarker or Not To Biomarker Use Considerations for Cell and Gene Therapy Drug Development. Its a big question to answer but were providing some insights on how biomarkers may can help move cell and gene therapies forward and emphasizing the critical role of patient advocacy groups in shaping outcome measures. Listen to Quick Takes from experts in the community on the following questions: 01:25 - What exactly is a biomarker and how do they differ from"
YouTube Link 2025-06-24T17:00Z 13.5K followers, [---] engagements
"Around the World: How U.S. Science is Impacting Cell and Gene Therapy This ASGCT session explores how U.S.-driven science is shaping global gene and cell therapy. Topics include the role of African genetic diversity in advancing precision medicine decentralized manufacturing to expand access and economic modeling to support gene therapy delivery in low-resource settings all aimed at promoting global health equity. Global Outreach ASGCT Manufacturing Global Outreach ASGCT Manufacturing"
YouTube Link 2025-09-19T15:01Z 13.5K followers, [---] engagements
"Gene Editing for DMD Gene Editing for DMD - Rhonda Bassel-Duby Scientific Symposium from the American Society of Gene & Cell Therapy's 22nd Annual Meeting"
YouTube Link 2019-11-27T19:37Z 13.5K followers, 12.4K engagements
"The Issue: Gene Editing Complexities with Tom McCauley PhD Episode [--] of The Issue on the ASGCT podcast network is an in-depth interview with Dr Tom McCauley CSO of Omega Therapeutics. Tom and Emily talk about his path from condensed matter physics to leading teams of scientists exploring the next generation of molecular therapeutics. They also dive into their mutual experience in the early days of in vivo gene editing and the challenges one faces with the complexities of these products particularly before there is much of a roadmap for development. Finally they dive into the recent public Ad"
YouTube Link 2024-01-16T12:00Z 13.5K followers, [---] engagements
"Policy and Advocacy Shorts with Keith Wonnacott PhD Keith Wonnacott PhD Vice President of Regulatory Affairs at Lexeo Therapeutics shares insights on ASGCT's role in connecting academia and industry and discusses the evolution of regulatory approaches in cell and gene therapy"
YouTube Link 2024-07-25T14:31Z 13.5K followers, [--] engagements
"Gene Therapy Basics (2022 Update) Gene therapy is the use of genetic material to treat or prevent disease. Learn more about the basics of gene therapy the potential for this growing field of research and the challenges and risks that still need to be investigated"
YouTube Link 2022-10-26T21:45Z 13.5K followers, 228.1K engagements
"Patients Are the Center with Kimberly Haugstad MBA On this episode of Soundbites of the Annual Meeting we talked to Kimberly Haugstad CEO of RareRising and a longtime rare disease advocate who is also the parent of a child with severe hemophilia. In her conversation with ASGCT's Communications Committee Chair Lynnea Olivarez Kimberly discusses: why patients as the "lived experience experts" are so important to rare disease communications advice for scientists who would like to engage more directly with patient communities what's giving her hope right now Listen and subscribe to the ASGCT"
YouTube Link 2025-08-19T10:06Z 13.5K followers, [--] engagements
"Building a Vision for Disease Prevention + Treatment with Dr. Mimi Lee On this episode of Soundbites of the Annual Meeting we talked to Mimi Lee MD PhD a physician-scientist and program manager at the Advanced Research Projects Agency for Health (ARPA-H). In her conversation with ASGCT's Communications Committee Chair Lynnea Olivarez Dr. Lee discusses: ARPA-H's unique approach to funding and structuring CGT development breaking down silos to improve health outcomes for all the role that CGT will play in prevention rather than just treatment of diseases Listen and subscribe to the ASGCT"
YouTube Link 2025-08-04T10:19Z 13.5K followers, [--] engagements
"Special Podcast Episode: What to Expect at ASGCT's Policy Summit with Chris Leidli + Francesca Cook Note: this episode is cross-posted with the Rx for Biotech podcast hosted by Chris Leidli. Francesca Cook is the Vice President Pricing and Market Access at REGENXBIO and past chair of ASGCT's Government Relations Committee. Shes attended and helped with ASGCTs annual Policy Summit since its inception five years ago. In this episode Cook shares her experience and advice around the eventcoming up Sept. 18-19 (virtually + in Washington DC) with its highest-profile line-up yet and more ways to"
YouTube Link 2023-09-14T23:03Z 13.5K followers, [--] engagements
"A Molecular Glue Degrader to Control CRISPR with Drs. Krishanu Saha and Namita Khajanchi Join Dr. Paloma Giangrande Editor-in-Chief of Molecular Therapy Nucleic Acids as she discusses a recently published MTNA article Controlling CRISPR-Cas9 genome editing in human cells using a molecular glue degrader (https://www.cell.com/molecular-therapy-family/nucleic-acids/fulltext/S2162-2531(25)00194-5_returnURL=https%3A%2F%2Flinkinghub.elsevier.com%2Fretrieve%2Fpii%2FS2162253125001945%3Fshowall%3Dtrue) with its authors Drs. Krishanu Saha and Namita Khajanchi (UW-Madison). If you enjoy this episode"
YouTube Link 2025-10-08T10:20Z 13.5K followers, [---] engagements
"Cas9-CLIPT-mediated integration of large transgenes with Krishanu Saha Anna Tommasi and Dan Cap. Dr. Daniel Stone (Associate Editor-in-Chief Molecular Therapy Advances) sits down with Dr. Krishanu Saha Anna Tommasi and Dr. Dan Cappabianca from the University of Wisconsin-Madison. Join them as they discuss their teams recent publication Efficient nonviral integration of large transgenes into human T cells using Cas9-CLIPT and learn more about their novel method to improve the manufacturing of multifunctional genome-edited immune cell therapies. Music:'Electric Dreams' by Scott Buckley -"
YouTube Link 2026-02-03T11:29Z 13.5K followers, [--] engagements
"Next-Generation Cell & Gene Therapy Approaches for HIV On December [--] [----] the American Society of Gene & Cell Therapy (ASGCT) hosted a virtual Congressional briefing titled "Next-Generation Cell & Gene Therapy Approaches for HIV." The event brought together bipartisan Congressional staff leading researchers and patient advocates to explore how cell and gene therapies (CGTs) are opening the possibility of a cure for people living with HIV"
YouTube Link 2025-12-05T16:47Z 13.5K followers, [---] engagements
"Reducing the Immunogenicity of AAV through Engineering the Vector - George Church Reducing the Immunogenicity of AAV through Engineering the Vector - George Church Scientific Symposium from the American Society of Gene & Cell Therapy's 21st Annual Meeting"
YouTube Link 2018-12-07T21:24Z 13.4K followers, [----] engagements
"The Issue - Entrepreneurship Fundraising and the Future of Cell & Gene Therapy with Dr. Niki Paulk Host Dr. Emily Walsh Martin welcomes Dr. Niki Paulk CEO and Founder of Siren Biotechnology for a timely conversation on turning scientific promise into commercial success. Dr. Paulk offers perceptive insights into the complex world of biotech entrepreneurship covering essential fundraising strategies from Seed and Series Rounds to Community Rounds and beyond. This episode is a must-listen for both industry professionals and academic innovators. Music:Bright New Morning by Steven OBrien released"
YouTube Link 2026-01-06T11:27Z 13.5K followers, [--] engagements
"Lunch & Learn: How AAV Vectors Are Made We often hear that gene therapies are complex and require a lot of time and money to make. But what does that really mean Tune in to hear experts in the field help answer this question by walking us through the manufacturing process"
YouTube Link 2022-05-02T19:48Z 13.5K followers, 33.6K engagements
"Lipid Nanoparticles with Drs. Daniel Stone Harrison Dulin and Keith R. Jerome - The Molecular T. Join Dr. Mohamed Abou-el-Enein Editor-in-Chief of Molecular Therapy Methods & Clinical Development as he discusses a recent article published in MTMCD with co-authors Drs. Daniel Stone Harrison Dulin and Keith R. Jerome from the Fred Hutchinson Cancer Center. The article Serum factors create species-specific barriers to hepatic gene transfer by lipid nanoparticles in liver-humanized mice"
YouTube Link 2025-09-10T10:36Z 13.5K followers, [---] engagements
"Harnessing Naturaland Engineered Properties of iNKT cells for Adoptive Cancer Immunotherapy Harnessing Naturaland Engineered Properties of iNKT cells for Adoptive Cancer Immunotherapy - Leonid Metelitsa Education Session from the American Society of Gene & Cell Therapy's 22nd Annual Meeting"
YouTube Link 2019-11-27T20:00Z 13.5K followers, [---] engagements
"25 Years of Molecular Therapy with Drs. Terry Flotte and Carl June In honor of Molecular Therapys 25th anniversary this episode is the first in a series hosted by Dr. Joseph Glorioso Editor-in-Chief of Molecular Therapy that will cover comprehensive reviews of critical developments in the field of gene and cell therapy over the past [--] years by expert authors. In this episode Dr. Glorioso will discuss two of these reviews Gene therapy then and now: A look back at changes in the field over the past [--] years with author Dr. Terry Flotte UMass Chan Medical School and From concept to cure: The"
YouTube Link 2025-05-01T10:26Z 13.5K followers, [---] engagements
"Ancestral AAV Capsid Reconstruction: Interrogating Viral Evolution to Inform Rational AAV Design Ancestral AAV Capsid Reconstruction: Interrogating Viral Evolution to Inform Rational AAV Design - Luk Vandenberghe Scientific Symposium from the American Society of Gene & Cell Therapy's 21st Annual Meeting May [--] 2018"
YouTube Link 2018-12-07T21:24Z 13.5K followers, [----] engagements
"AAV Integration Roundtable During this virtual roundtable experts discussed the status of AAV integration research including analysis of recent clinical findings to develop recommendations for risk assessment"
YouTube Link 2021-08-20T14:59Z 13.5K followers, [----] engagements
"Community Quick Takes: AAV Immune Response In this Community Quick Takes module we will explore a high-level summary of key topics from the recent ASGCT workshop Class Considerations on Immunogenicity for AAV Gene Therapy Products: Assessing Current Practice and New Data. Speakers from the event address fundamental questions about the immune system's response to AAV gene therapies how these responses can affect treatment outcomes and the methods being developed to manage immune reactions. Listen to Quick Takes from experts in the community on the following questions: 00:00 - What does the"
YouTube Link 2025-02-26T16:00Z 13.5K followers, [---] engagements
"RNA interference gene therapy for Parkinsons disease with Kathy Steece-Collier Jeffrey Kordower. Molecular Therapy Editor-in-Chief Dr. Joseph Glorioso joins researchers Kathy Steece-Collier (Michigan State University) Jeffrey Kordower (Arizona State University) and Fredric Manfredsson (Barrow Neurological Institute) to discuss their groundbreaking work on RNA interference for Parkinsons disease. This episode focuses on their recent article titled "Disease-modifying multidimensional efficacy of putaminal CaV1.3-shRNA gene therapy in aged parkinsonism male and female macaques." Music:'Electric"
YouTube Link 2026-01-08T11:19Z 13.5K followers, [---] engagements
"Case study of how biomarkers may be limited in DMD This a recorded presentation from the session titled "To Biomarker Or Not To Biomarker Use Considerations For CGT Drug Development" Organized by the Patient Outreach Committee during AM25"
YouTube Link 2025-07-23T14:00Z 13.5K followers, [--] engagements
"Generating CAR T cells in vivo with lentiviral vectors with Drs. Rachael Nimmo and Kyriacos Mitro. Editor-in-Chief of Molecular Therapy Dr. Joseph Glorioso is joined by Rachael Nimmo PhD former director of the Cell Technology Group at Oxford Biomedica and Kyriacos Mitrophanous PhD Chief Innovation Officer at Oxford Biomedica to discuss an article recently published in Molecular Therapyby Nimmo and colleagues titled Efficient in vivo generation of CAR T cells using a retargeted fourth-generation lentiviral vector"
YouTube Link 2025-11-11T11:15Z 13.5K followers, [---] engagements
"AAV Gene Therapy to CNS: Past Present and Future - Miguel Sena-Esteves Gene Therapy to CNS: Past Present and Future Miguel Sena-Esteves Education Session from the American Society of Gene & Cell Therapy's 21st Annual Meeting May [--] 2018"
YouTube Link 2018-12-07T21:24Z 13.5K followers, [----] engagements
"Gene Therapy and Sickle Cell Disease Sickle cell disease is an inherited blood disorder that causes red blood cells to deform and become sickle (or crescent) shaped. Gene therapy and gene editing may offer a one-time treatment that addresses the genetic cause of sickle cell disease. The subtitling of these videos was funded in part by a donation from Pfizer Inc. sickle cell disease SCD gene therapy ASGCT American Society of Gene and Cell Therapy ASH ASH SCD ASH Research Collaborative ASH RC SCD Clinical Trials Network hematology hematology society ASH hematology sickle cell disease SCD gene"
YouTube Link 2020-09-17T21:50Z 13.5K followers, 57.8K engagements
"Case study of how CSF biomarkers were approved by the FDA in MPS This a recorded presentation from the session titled "To Biomarker Or Not To Biomarker Use Considerations For CGT Drug Development" Organized by the Patient Outreach Committee during AM25"
YouTube Link 2025-07-23T14:00Z 13.5K followers, [--] engagements
"AAV Engineering AAV Engineering - Junghae Suh Scientific Symposium from the American Society of Gene & Cell Therapy's 22nd Annual Meeting"
YouTube Link 2019-11-27T18:00Z 13.5K followers, [----] engagements
"AAV Vectors in the Eye - Shannon Boye AAV Vectors in the Eye Shannon Boye Education Session from the American Society of Gene & Cell Therapy's 21st Annual Meeting May [--] 2018"
YouTube Link 2018-12-07T21:24Z 13.5K followers, [----] engagements
"Short and Long Read Sequencing for AAV Characterization: BIO-ASGCT CGT Science Series The BIO-ASGCT Cell and Gene Therapy (CGT) Science Series is a quarterly seminar series focused on scientific topics related to cell and gene therapy products. The CGT Science Series is intended to foster scientific exchange between BIO ASGCT and CBER review staff on a variety of topics that span the CGT product lifecycle. The inaugural seminar focuses on NGS characterization of AAV and was presented by Dr. Lauriel Earley. Dr. Lauriel Earley is a Senior Scientist with Shape Therapeutics who has been studying"
YouTube Link 2023-04-05T21:25Z 13.5K followers, [----] engagements
"AAV Vector Manufacturing and Analytics AAV Vector Manufacturing and Analytics - J. Fraser Wright Scientific Symposium from the American Society of Gene & Cell Therapy's 22nd Annual Meeting"
YouTube Link 2019-11-27T18:06Z 13.5K followers, 16K engagements
"The Ups and Downs of Conducting Groundbreaking Research with Dr. Kiran Musunuru "Certainly I've been anxious about data.I did an experiment I put a lot of time into it I'm hoping to get good results.we all know that feeling right The stakes are just so different here.this kid's life depends on whether this experiment worked." --Kiran Musunuru MD PhD One of the highlights of the ASGCT 28th Annual Meeting wasthe announcement on May [--] of a historic medical breakthrough (https://www.chop.edu/news/worlds-first-patient-treated-personalized-crispr-gene-editing-therapy-childrens-hospital) : a child"
YouTube Link 2025-06-16T10:15Z 13.5K followers, [---] engagements
"Community Quick Take: Gene Editing for Prion Disease Dr. Sonia Vallabh a speaker featured at ASGCT's Breakthroughs in Targeted In Vivo Gene Editing event shares her powerful personal journey to develop treatment for a rare neurologic condition prion disease. Learn what led her to research in vivo gene editing to target the disease which is showing promise but not without limitations. Listen to Quick Takes from experts in the community on the following questions (Time: [--] minutes): 02:17 Can you tell us a little bit about yourself and your journey 05:01 What is gene editing and how might it"
YouTube Link 2025-11-19T13:30Z 13.5K followers, [---] engagements
"Why ARPA-H's Dr. Mimi Lee Is Looking Forward to the Policy Summit Hear directly from speaker Mimi Lee MD PhD a program manager at the Advanced Research Projects Agency for Health (ARPA-H) about why she encourages people to join her at the Policy Summit. Learn more and register today: http://asgct.org/policysummit"
YouTube Link 2025-09-18T21:25Z 13.5K followers, [---] engagements
"Immune Responses to AAV-Based Gene Therapy Gene therapy aims to treat genetic conditions by delivering genetic material such as a working gene to target the underlying cause of disease. One of the most common ways to deliver these therapies uses adeno-associated viruses (AAVs). While AAV-based gene therapies have a strong safety record they can still trigger immune responses that affect how well the treatment works or whether someone is eligible to receive it. Learn more: https://patienteducation.asgct.org/understanding-cell-gene-therapy/viral-vectors/immune-responses-to-aav-based-gene-therapy"
YouTube Link 2025-11-21T03:21Z 13.5K followers, [---] engagements
"Incorporating miRNAs into a taRNA vector platform with Drs. Tim Beissert and Ayegl Yildiz - The. Drs. Ayegl Yildiz and Tim Beissert join Dr. Paloma Giangrande to discuss a recent article published in Molecular Therapy Nucleic Acids by Drs. Yildiz. Beissert and colleagues titled Trans-amplifying RNA expressing functional miRNA mediates target gene suppression and simultaneous transgene expression. Are you registered for ASGCTs [----] Annual Meeting in New Orleans Join us May 13-17 for the premier event in our field and save $465 as an ASGCT member. Check out the full program plan your trip and"
YouTube Link 2025-03-04T11:28Z 13.5K followers, [---] engagements
"25 Years of Molecular Therapy with Sheila Mikhail and Dr. Mark Kay In honor of Molecular Therapys 25th anniversary this episode is the second in a series hosted by Dr. Joseph Glorioso Editor-in-Chief of Molecular Therapy that will cover comprehensive reviews of critical developments in the field of gene and cell therapy over the past [--] years. In this episode Dr. Glorioso will discuss the reviews AAV vector development back to the future"
YouTube Link 2025-06-11T10:20Z 13.5K followers, [---] engagements
"Building Your Biotech Community Benjamin McLeod on ASGCT Membership "You get the opportunity to meet a group of people who are actually inside of your industry." In the world of gene and cell therapy location shouldn't limit your potential. Benjamin McLeod (Founder of Convey Bio) discusses how ASGCT acts as a "built-in community" for professionals. No matter where you are on the map ASGCT brings the heart of the industry to you. 🔗 Join our global community: https://www.asgct.org/membership/become-a-member"
YouTube Link 2026-02-16T13:56Z 13.5K followers, [--] engagements
"A Vaccine for Cocaine Addiction with Dr. Stephen Kaminsky On this episode of Soundbites of the Annual Meeting we talked to Stephen Kaminsky PhD professor of research in genetic medicine at Weill Cornell Medical College and lead author of a phase [--] clinical trial of a new vaccine for cocaine addiction. In his conversation with ASGCT's Communications Committee Chair Lynnea Olivarez Dr. Kaminsky discusses: How the vaccine works and how its approach is different from other treatments Safety results from the unblinded data Implications this vaccine could have for other substance use disorders"
YouTube Link 2025-07-22T10:07Z 13.5K followers, [---] engagements
"Community Quick Takes: Preparing for Unknowns When the patient community talks about cell and gene therapies hope and uncertainty often go hand-in-hand. In this unit Dr. Amy Waldman gives a closer look at what lies ahead for someone participating in an investigational clinical trial or more importantly what still might not be known. Listen to Quick Takes from experts in the community on the following questions: 00:00 - Why arent all drugs given open label as opposed to a double-blind design 10:11 - What are some of the more realistic expectations that a person participating in a gene therapy"
YouTube Link 2025-09-22T19:56Z 13.5K followers, [---] engagements
"Community Quick Takes: Informed Consent Originally released in November [----] Listen to [--] Quick Takes from experts in the community on the following questions: -What are five tips for a patient or caregiver to be prepared for informed consent -What challenges do participants face while going through the consent process and what actions can researchers take to improve the process -How do we facilitate informed consent for approved therapies Want to dive deeper into this topic Visit our patient education site for the full blog: https://patienteducation.asgct.org/quick-takes/informed-consent"
YouTube Link 2025-04-11T17:20Z 13.5K followers, [--] engagements
"AAV Vectors in the Liver - Ian Alexander AAV Vectors in the Liver - Ian Alexander Education Session from the American Society of Gene & Cell Therapy's 21st Annual Meeting May [--] 2018"
YouTube Link 2018-12-07T21:24Z 13.5K followers, [---] engagements
"2025 Year in Review with the Molecular Therapy Family of Journals Join all four Editors-in-Chief of the Molecular Therapy family of journals for a special [----] Retrospective. Editors Dr. Glorioso Dr. Giangrande Dr. Abou-el-Enein and Dr. Cripe dive into the most exciting advancements in gene and cell therapy from the past year. Music:'Electric Dreams' by Scott Buckley - released under CC-BY 4.0.www.scottbuckley.com.au (https://www.scottbuckley.com.au/) Show your support for ASGCT: https://asgct.org/membership/donate See omnystudio.com/listener (https://omnystudio.com/listener) for privacy"
YouTube Link 2025-12-09T15:30Z 13.5K followers, [---] engagements
"Community Quick Take: Genetic Reports + Whats Next A common experience for people diagnosed with a genetic condition is receiving a genetic test result which often comes with many questions emotions and unfamiliar terms. In this unit Kim Nye founder of the TESS Research Foundation and parent advocate is joined by genetic counselor Dr. Lauren Beretich to walk through whats in a genetic report and how this important information can help guide a familys next steps. Read more: https://patienteducation.asgct.org/research-clinical-trials/community-quick-takes/genetic-reports-whats-next"
YouTube Link 2026-02-18T12:30Z 13.5K followers, [--] engagements
"Building Your Biotech Community Benjamin McLeod on ASGCT Membership "You get the opportunity to meet a group of people who are actually inside of your industry." In the world of gene and cell therapy location shouldn't limit your potential. Benjamin McLeod (Founder of Convey Bio) discusses how ASGCT acts as a "built-in community" for professionals. No matter where you are on the map ASGCT brings the heart of the industry to you. 🔗 Join our global community: https://www.asgct.org/membership/become-a-member"
YouTube Link 2026-02-16T13:56Z 13.5K followers, [--] engagements
"The Issue - Evolving Patient Access to CGTs via Innovation in Medicaid Reimbursement Models with . In this episode Melissa Majerol Model Lead of the Cell and Gene Therapy Access Model at the Center for Medicare and Medicaid Innovation (CMMI) at CMS breaks down how the CMMI Access Model program aims to simplify and accelerate patient access to innovative cell and gene therapies starting with recently approved therapies in Sickle Cell disease. Listen in as we dive into the specifics of this new access model and its current status. Music:Bright New Morning by Steven OBrien released under CC-BY"
YouTube Link 2026-02-10T11:34Z 13.5K followers, [--] engagements
"Elevating Your Research Career Dr. Anna Blakney on ASGCT Member Benefits In the fast-moving world of gene and cell therapy technical skills are only half the battle. Dr. Anna Blakney (Assistant Professor at UBC) explains why ASGCT is the essential home for professionals in the field. From accessing the latest research in Molecular Therapy to navigating the Career Center ASGCT membership offers a comprehensive suite of tools designed to benefit your career at every stage. 🔗 Become an ASGCT member today: asgct.org/become-a-member The American Society of Gene & Cell Therapy is the primary"
YouTube Link 2026-02-09T17:44Z 13.5K followers, [--] engagements
""Inspire and Be Inspired" Dr. Ruth Castellanos on the Power of the ASGCT Community "ASGCT has empowered me to become who I am right now." What does it mean to be a member of the American Society of Gene & Cell Therapy For Dr. Ruth Castellanos (Director of R&D at Kriya Therapeutics) it means more than just a professional affiliationits about finding direction and inspiration. Whether you are looking to be inspired by the latest science or hoping to inspire others with your work there is a place for you here. 🔗 Become an ASGCT member today: asgct.org/become-a-member The American Society of"
YouTube Link 2026-02-05T16:58Z 13.5K followers, [--] engagements
"Cas9-CLIPT-mediated integration of large transgenes with Krishanu Saha Anna Tommasi and Dan Cap. Dr. Daniel Stone (Associate Editor-in-Chief Molecular Therapy Advances) sits down with Dr. Krishanu Saha Anna Tommasi and Dr. Dan Cappabianca from the University of Wisconsin-Madison. Join them as they discuss their teams recent publication Efficient nonviral integration of large transgenes into human T cells using Cas9-CLIPT and learn more about their novel method to improve the manufacturing of multifunctional genome-edited immune cell therapies. Music:'Electric Dreams' by Scott Buckley -"
YouTube Link 2026-02-03T11:29Z 13.5K followers, [--] engagements
"Technological Frontiers in South American Gene and Cell Therapy As cell and gene therapies revolutionize modern medicine South America is emerging as a dynamic player on the global stage leveraging scientific innovation regulatory evolution and regional collaboration. Explore the technological frontiers shaping the development and deployment of advanced therapies across the continent"
YouTube Link 2026-01-26T22:05Z 13.5K followers, [--] engagements
"RNA interference gene therapy for Parkinsons disease with Kathy Steece-Collier Jeffrey Kordower. Molecular Therapy Editor-in-Chief Dr. Joseph Glorioso joins researchers Kathy Steece-Collier (Michigan State University) Jeffrey Kordower (Arizona State University) and Fredric Manfredsson (Barrow Neurological Institute) to discuss their groundbreaking work on RNA interference for Parkinsons disease. This episode focuses on their recent article titled "Disease-modifying multidimensional efficacy of putaminal CaV1.3-shRNA gene therapy in aged parkinsonism male and female macaques." Music:'Electric"
YouTube Link 2026-01-08T11:19Z 13.5K followers, [---] engagements
"The Issue - Entrepreneurship Fundraising and the Future of Cell & Gene Therapy with Dr. Niki Paulk Host Dr. Emily Walsh Martin welcomes Dr. Niki Paulk CEO and Founder of Siren Biotechnology for a timely conversation on turning scientific promise into commercial success. Dr. Paulk offers perceptive insights into the complex world of biotech entrepreneurship covering essential fundraising strategies from Seed and Series Rounds to Community Rounds and beyond. This episode is a must-listen for both industry professionals and academic innovators. Music:Bright New Morning by Steven OBrien released"
YouTube Link 2026-01-06T11:27Z 13.5K followers, [--] engagements
"The Issue - Unlocking New Therapeutic Potential: Ensomas Platform for Rare Disease and Oncology Host Emily Walsh Martin welcomes Dr. Jim Burns CEO of Ensoma to discuss the company's novel technological approach to treating both rare diseases and solid tumor oncology. Dr. Burns provides a detailed overview of Ensoma's platform and its application in their ongoing clinical trials including the recently announced first patient infusion for chronic granulomatous disease. Learn how this initial trial is set to inform future therapeutic opportunities for indications with high unmet medical need."
YouTube Link 2025-12-16T11:15Z 13.5K followers, [--] engagements
"2025 Year in Review with the Molecular Therapy Family of Journals Join all four Editors-in-Chief of the Molecular Therapy family of journals for a special [----] Retrospective. Editors Dr. Glorioso Dr. Giangrande Dr. Abou-el-Enein and Dr. Cripe dive into the most exciting advancements in gene and cell therapy from the past year. Music:'Electric Dreams' by Scott Buckley - released under CC-BY 4.0.www.scottbuckley.com.au (https://www.scottbuckley.com.au/) Show your support for ASGCT: https://asgct.org/membership/donate See omnystudio.com/listener (https://omnystudio.com/listener) for privacy"
YouTube Link 2025-12-09T15:30Z 13.5K followers, [---] engagements
"Next-Generation Cell & Gene Therapy Approaches for HIV On December [--] [----] the American Society of Gene & Cell Therapy (ASGCT) hosted a virtual Congressional briefing titled "Next-Generation Cell & Gene Therapy Approaches for HIV." The event brought together bipartisan Congressional staff leading researchers and patient advocates to explore how cell and gene therapies (CGTs) are opening the possibility of a cure for people living with HIV"
YouTube Link 2025-12-05T16:47Z 13.5K followers, [---] engagements
"Immune Responses to AAV-Based Gene Therapy Gene therapy aims to treat genetic conditions by delivering genetic material such as a working gene to target the underlying cause of disease. One of the most common ways to deliver these therapies uses adeno-associated viruses (AAVs). While AAV-based gene therapies have a strong safety record they can still trigger immune responses that affect how well the treatment works or whether someone is eligible to receive it. Learn more: https://patienteducation.asgct.org/understanding-cell-gene-therapy/viral-vectors/immune-responses-to-aav-based-gene-therapy"
YouTube Link 2025-11-21T03:21Z 13.5K followers, [---] engagements
"Community Quick Take: Gene Editing for Prion Disease Dr. Sonia Vallabh a speaker featured at ASGCT's Breakthroughs in Targeted In Vivo Gene Editing event shares her powerful personal journey to develop treatment for a rare neurologic condition prion disease. Learn what led her to research in vivo gene editing to target the disease which is showing promise but not without limitations. Listen to Quick Takes from experts in the community on the following questions (Time: [--] minutes): 02:17 Can you tell us a little bit about yourself and your journey 05:01 What is gene editing and how might it"
YouTube Link 2025-11-19T13:30Z 13.5K followers, [---] engagements
"Generating CAR T cells in vivo with lentiviral vectors with Drs. Rachael Nimmo and Kyriacos Mitro. Editor-in-Chief of Molecular Therapy Dr. Joseph Glorioso is joined by Rachael Nimmo PhD former director of the Cell Technology Group at Oxford Biomedica and Kyriacos Mitrophanous PhD Chief Innovation Officer at Oxford Biomedica to discuss an article recently published in Molecular Therapyby Nimmo and colleagues titled Efficient in vivo generation of CAR T cells using a retargeted fourth-generation lentiviral vector"
YouTube Link 2025-11-11T11:15Z 13.5K followers, [---] engagements
"A Molecular Glue Degrader to Control CRISPR with Drs. Krishanu Saha and Namita Khajanchi Join Dr. Paloma Giangrande Editor-in-Chief of Molecular Therapy Nucleic Acids as she discusses a recently published MTNA article Controlling CRISPR-Cas9 genome editing in human cells using a molecular glue degrader (https://www.cell.com/molecular-therapy-family/nucleic-acids/fulltext/S2162-2531(25)00194-5_returnURL=https%3A%2F%2Flinkinghub.elsevier.com%2Fretrieve%2Fpii%2FS2162253125001945%3Fshowall%3Dtrue) with its authors Drs. Krishanu Saha and Namita Khajanchi (UW-Madison). If you enjoy this episode"
YouTube Link 2025-10-08T10:20Z 13.5K followers, [---] engagements
"Around the World: Oncology Hematology and Cancer-Focused Cell and Gene Therapy in India This webinar explores Indias rapid advancements in CAR T therapies including homegrown innovations. This program highlights clinical successes increased accessibility and research leadership showcasing Indias growing impact on global cancer and hematology gene therapy landscapes"
YouTube Link 2025-10-06T14:43Z 13.5K followers, [---] engagements
"Dr. Snehal Naik: "I Hope You'll Join the Conversation" at the Policy Summit Hear directly from Snehal Naik PhD Chair of ASGCT's Regulatory Affairs Committee about why she encourages people to join her at the Policy Summit. Learn more and register today: http://asgct.org/policysummit"
YouTube Link 2025-09-23T16:34Z 13.5K followers, [--] engagements
"Community Quick Takes: Preparing for Unknowns When the patient community talks about cell and gene therapies hope and uncertainty often go hand-in-hand. In this unit Dr. Amy Waldman gives a closer look at what lies ahead for someone participating in an investigational clinical trial or more importantly what still might not be known. Listen to Quick Takes from experts in the community on the following questions: 00:00 - Why arent all drugs given open label as opposed to a double-blind design 10:11 - What are some of the more realistic expectations that a person participating in a gene therapy"
YouTube Link 2025-09-22T19:56Z 13.5K followers, [---] engagements
"Around the World: How U.S. Science is Impacting Cell and Gene Therapy This ASGCT session explores how U.S.-driven science is shaping global gene and cell therapy. Topics include the role of African genetic diversity in advancing precision medicine decentralized manufacturing to expand access and economic modeling to support gene therapy delivery in low-resource settings all aimed at promoting global health equity. Global Outreach ASGCT Manufacturing Global Outreach ASGCT Manufacturing"
YouTube Link 2025-09-19T15:01Z 13.5K followers, [---] engagements
"Why ARPA-H's Dr. Mimi Lee Is Looking Forward to the Policy Summit Hear directly from speaker Mimi Lee MD PhD a program manager at the Advanced Research Projects Agency for Health (ARPA-H) about why she encourages people to join her at the Policy Summit. Learn more and register today: http://asgct.org/policysummit"
YouTube Link 2025-09-18T21:25Z 13.5K followers, [---] engagements
"Lipid Nanoparticles with Drs. Daniel Stone Harrison Dulin and Keith R. Jerome - The Molecular T. Join Dr. Mohamed Abou-el-Enein Editor-in-Chief of Molecular Therapy Methods & Clinical Development as he discusses a recent article published in MTMCD with co-authors Drs. Daniel Stone Harrison Dulin and Keith R. Jerome from the Fred Hutchinson Cancer Center. The article Serum factors create species-specific barriers to hepatic gene transfer by lipid nanoparticles in liver-humanized mice"
YouTube Link 2025-09-10T10:36Z 13.5K followers, [---] engagements
"Inside the Molecular Therapy Family of Journals Join us as past ASGCT President Paula Cannon Ph.D. and our Editors-in-Chief take you behind the scenes of ASGCTs field-leading Molecular Therapy family of journals. Discover how these prestigious publications have shaped the landscape of cell and gene therapy research for the past [--] years. Learn more: asgct.org/molecular-therapy"
YouTube Link 2025-09-09T15:02Z 13.5K followers, [---] engagements
"Patients Are the Center with Kimberly Haugstad MBA On this episode of Soundbites of the Annual Meeting we talked to Kimberly Haugstad CEO of RareRising and a longtime rare disease advocate who is also the parent of a child with severe hemophilia. In her conversation with ASGCT's Communications Committee Chair Lynnea Olivarez Kimberly discusses: why patients as the "lived experience experts" are so important to rare disease communications advice for scientists who would like to engage more directly with patient communities what's giving her hope right now Listen and subscribe to the ASGCT"
YouTube Link 2025-08-19T10:06Z 13.5K followers, [--] engagements
"25 Years of Molecular Therapy with Drs. Rayne Rouce and Joe Glorioso This episode is the fourth in a series hosted by Molecular TherapyEditor-in-Chief Joseph Glorioso PhD that will cover comprehensive reviews of critical developments in the field of gene and cell therapy over the past [--] years. In this episode Dr. Glorioso will discuss the review How to democratize cell and gene therapy: A global approach"
YouTube Link 2025-08-08T10:12Z 13.5K followers, [---] engagements
"Building a Vision for Disease Prevention + Treatment with Dr. Mimi Lee On this episode of Soundbites of the Annual Meeting we talked to Mimi Lee MD PhD a physician-scientist and program manager at the Advanced Research Projects Agency for Health (ARPA-H). In her conversation with ASGCT's Communications Committee Chair Lynnea Olivarez Dr. Lee discusses: ARPA-H's unique approach to funding and structuring CGT development breaking down silos to improve health outcomes for all the role that CGT will play in prevention rather than just treatment of diseases Listen and subscribe to the ASGCT"
YouTube Link 2025-08-04T10:19Z 13.5K followers, [--] engagements
"Community Quick Takes: Manufacturing Matters The gene therapy journey from lab to patient depends on something many people dont seemanufacturing. Gain insights on the complex behind-the-scenes process of making AAV gene therapies and why it matters to the patient community. This unit was developed in partnership with the Bespoke Gene Therapy Consortium (BGTC). Listen to Quick Takes from experts in the community on the following questions: 00:00 - What is a plasmid and how does it help in making a gene therapy 04:24 - Why are quality checks so critical when making gene therapies and what kinds"
YouTube Link 2025-07-30T11:01Z 13.5K followers, [---] engagements
"The Business of Advocates Advancing CGTs: Different non-profit business models and advice to PAGs This a recorded presentation from the workshop titled "The Business of Advocates Advancing CGTs" that took place during AM25"
YouTube Link 2025-07-23T14:01Z 13.5K followers, [--] engagements
"Investigators guide to identifying and validating biomarkers for clinical trials This a recorded presentation from the session titled "To Biomarker Or Not To Biomarker Use Considerations For CGT Drug Development" Organized by the Patient Outreach Committee during AM25"
YouTube Link 2025-07-23T14:00Z 13.5K followers, [---] engagements
"Case study of how CSF biomarkers were approved by the FDA in MPS This a recorded presentation from the session titled "To Biomarker Or Not To Biomarker Use Considerations For CGT Drug Development" Organized by the Patient Outreach Committee during AM25"
YouTube Link 2025-07-23T14:00Z 13.5K followers, [--] engagements
"What are Biomarkers A regulators guide to using biomarkers in an approved therapy This a recorded presentation from the session titled "To Biomarker Or Not To Biomarker Use Considerations For CGT Drug Development" Organized by the Patient Outreach Committee during AM25"
YouTube Link 2025-07-23T14:00Z 13.5K followers, [--] engagements
"Case study of how biomarkers may be limited in DMD This a recorded presentation from the session titled "To Biomarker Or Not To Biomarker Use Considerations For CGT Drug Development" Organized by the Patient Outreach Committee during AM25"
YouTube Link 2025-07-23T14:00Z 13.5K followers, [--] engagements
"A Vaccine for Cocaine Addiction with Dr. Stephen Kaminsky On this episode of Soundbites of the Annual Meeting we talked to Stephen Kaminsky PhD professor of research in genetic medicine at Weill Cornell Medical College and lead author of a phase [--] clinical trial of a new vaccine for cocaine addiction. In his conversation with ASGCT's Communications Committee Chair Lynnea Olivarez Dr. Kaminsky discusses: How the vaccine works and how its approach is different from other treatments Safety results from the unblinded data Implications this vaccine could have for other substance use disorders"
YouTube Link 2025-07-22T10:07Z 13.5K followers, [---] engagements
"25 Years of Molecular Therapy with Drs. Beverly Davidson and Jeff Chamberlain This episode is the third in a series hosted by Molecular Therapy Editor-in-Chief Joseph Glorioso PhD that will cover comprehensive reviews of critical developments in the field of gene and cell therapy over the past [--] years. In this episode Dr. Glorioso will discuss the reviews Current trends in gene therapy to treat inherited disorders of the brain with author Beverly Davidson PhD The Childrens Hospital of Philadelphia and The road toward AAV-mediated gene therapy of Duchenne muscular dystrophy with author Jeff"
YouTube Link 2025-07-16T10:39Z 13.5K followers, [---] engagements
"Developing Therapeutic Platforms with Dr. P.J. Brooks During this episode of Soundbites of the Annual Meeting we spoke with P.J. Brooks PhD who is Deputy Director of the Division of Rare Diseases Research Innovation part of the National Center for Advancing Translational Sciences (NCATS) at the National Institutes of Health (NIH). In his conversation with ASGCT's Communications Committee Chair Lynnea Olivarez Dr. Brooks talked about: The impact of developing cell and gene therapies as platforms to treat more than one disease Projects he's involved in that are working towards the platform"
YouTube Link 2025-07-09T10:24Z 13.5K followers, [--] engagements
"Community Quick Takes: Biomarker Breakdown Hear from Dr. Rebecca Ahrens-Nicklas a featured speaker in the ASGCT Annual Meeting session To Biomarker or Not To Biomarker Use Considerations for Cell and Gene Therapy Drug Development. Its a big question to answer but were providing some insights on how biomarkers may can help move cell and gene therapies forward and emphasizing the critical role of patient advocacy groups in shaping outcome measures. Listen to Quick Takes from experts in the community on the following questions: 01:25 - What exactly is a biomarker and how do they differ from"
YouTube Link 2025-06-24T17:00Z 13.5K followers, [---] engagements
"The Ups and Downs of Conducting Groundbreaking Research with Dr. Kiran Musunuru "Certainly I've been anxious about data.I did an experiment I put a lot of time into it I'm hoping to get good results.we all know that feeling right The stakes are just so different here.this kid's life depends on whether this experiment worked." --Kiran Musunuru MD PhD One of the highlights of the ASGCT 28th Annual Meeting wasthe announcement on May [--] of a historic medical breakthrough (https://www.chop.edu/news/worlds-first-patient-treated-personalized-crispr-gene-editing-therapy-childrens-hospital) : a child"
YouTube Link 2025-06-16T10:15Z 13.5K followers, [---] engagements
"25 Years of Molecular Therapy with Sheila Mikhail and Dr. Mark Kay In honor of Molecular Therapys 25th anniversary this episode is the second in a series hosted by Dr. Joseph Glorioso Editor-in-Chief of Molecular Therapy that will cover comprehensive reviews of critical developments in the field of gene and cell therapy over the past [--] years. In this episode Dr. Glorioso will discuss the reviews AAV vector development back to the future"
YouTube Link 2025-06-11T10:20Z 13.5K followers, [---] engagements
"Around the World: Frontline of AAV Vectors for Gene Therapy Research in Japan Explore cutting-edge advancements in the use of AAV vectors for gene therapy research in Japan. This event brings together leading scientists researchers and professionals in the field to discuss innovative approaches challenges and breakthroughs in gene delivery systems specifically focusing on AAV technology. ASGCT Japan AAV Technology ASGCT Japan AAV Technology"
YouTube Link 2025-05-14T15:22Z 13.5K followers, [---] engagements
"25 Years of Molecular Therapy with Drs. Terry Flotte and Carl June In honor of Molecular Therapys 25th anniversary this episode is the first in a series hosted by Dr. Joseph Glorioso Editor-in-Chief of Molecular Therapy that will cover comprehensive reviews of critical developments in the field of gene and cell therapy over the past [--] years by expert authors. In this episode Dr. Glorioso will discuss two of these reviews Gene therapy then and now: A look back at changes in the field over the past [--] years with author Dr. Terry Flotte UMass Chan Medical School and From concept to cure: The"
YouTube Link 2025-05-01T10:26Z 13.5K followers, [---] engagements
"Empowering Patients 2025: A Cell and Gene Therapies Summit - Day [--] This is the Day [--] recording of the inaugural virtual event held on March [--] [----]. This event will provide timely education and engaging discussions on cell and gene therapies (CGTs). In alignment with ASGCTs strategic plan the goal is to empower advocates patients and caregivers who are vital in developing treatments. Attendees will hear from experts in the CGT field as well as patient advocacy group leaders. Agenda: Morning Coffee with Dr. Peter Marks CGT Manufacturing Basics The Journey from Early-stage Trials to Approval"
YouTube Link 2025-04-21T18:22Z 13.5K followers, [---] engagements
"Empowering Patients 2025: A Cell and Gene Therapies Summit - Day [--] This is the Day [--] recording of the inaugural virtual event held on March [--] [----]. This event will provide timely education and engaging discussions on cell and gene therapies (CGTs). In alignment with ASGCTs strategic plan the goal is to empower advocates patients and caregivers who are vital in developing treatments. Attendees will hear from experts in the CGT field as well as patient advocacy group leaders. Agenda: Partners in Progress: The Value of Patients and Advocates Intro to Gene Therapy Approaches Review of Clinical"
YouTube Link 2025-04-21T17:51Z 13.5K followers, [---] engagements
"The Importance of Long-term Data with Dolan Sondhi PhD - The Issue On this episode of The Issue Host Emily Walsh Martin PhD is tackling her absolute favorite subject: the importance of long-term data Emily is joined by Dolan Sondhi PhD of Weill Cornell Medical College to discuss her recent paper Twenty-Year Survival Analysis of Adeno-Associated Virus Vector Serotype 2-Mediated Gene Therapy to the Central Nervous System for CLN2 Disease. We hope to see all of your long-term data at the ASGCT Annual Meeting the premier event in cell and gene therapy next month Join us in New Orleans May 13-17"
YouTube Link 2025-04-14T10:31Z 13.5K followers, [--] engagements
"Community Quick Takes: Access to Approved Cell and Gene Therapies Originally released in May [----] A person considering a gene or cell therapy treatment may experience barriers to accessing the treatment. Our experts will explore the various meanings of the term access. This includes the development of therapies eligibility to receive an FDA-approved treatment coverage provider referral and being able to navigate the logistics of treatment within a clinic. Each expert touches on aspects that shape the field and impact an individuals access to gene and cell treatments. Listen to Quick Takes"
YouTube Link 2025-04-11T17:28Z 13.5K followers, [---] engagements
"Community Quick Takes: State of the Field - Highlights and Approvals for CGT in [----] Originally released in February [----] The field of gene and cell therapy continues to grow and change with the approval of many new therapies this past year. There is much to highlight about the patient communities affected and what it means for future findings in treatments of rare and genetic conditions. Check out the quick takes below from Peter Marks MD PhD and Jeffrey Chamberlain PhD adddressing the following questions: - What were some of the field highlights for you this past year - What sort of"
YouTube Link 2025-04-11T17:21Z 13.5K followers, [--] engagements
"Community Quick Takes: Informed Consent Originally released in November [----] Listen to [--] Quick Takes from experts in the community on the following questions: -What are five tips for a patient or caregiver to be prepared for informed consent -What challenges do participants face while going through the consent process and what actions can researchers take to improve the process -How do we facilitate informed consent for approved therapies Want to dive deeper into this topic Visit our patient education site for the full blog: https://patienteducation.asgct.org/quick-takes/informed-consent"
YouTube Link 2025-04-11T17:20Z 13.5K followers, [--] engagements
"The Big Picture: Accountability in an Evolving Ecosystem ASGCT is excited to host a thought-provoking conversation moderated by Jodi Wolff PhD to further deconstruct issues of responsibility and obligation in the post-approval gene therapy landscape. This fireside chat will address topics related to data capture for long-term monitoring for durability and safety standardization to enable comparability to facilitate/identify next-generation therapies and tensions that exist in the evolving landscape. ASGCT Ethics Webinar Accountability Gene Therapy Data Capture ASGCT Ethics Webinar"
YouTube Link 2025-03-31T20:24Z 13.5K followers, [--] engagements
"The When What How and Why of FDA Engagement with Niki Gallo - The Issue Today Niki Gallo principal consultant of regulatory affairs at Halloran Consulting joins host Emily Walsh Martin for a deep dive into timeline management pitfalls the nuances of INDs and the rapidly evolving FDA landscape. Niki brings to the program her extensive experience shepherding innovative therapeutics through the regulatory process and timely insights into the complexities of transitioning from the bench to the bedside. Whether youre from industry or academia todays conversation will help wrap all our heads around"
YouTube Link 2025-03-11T10:25Z 13.5K followers, [---] engagements
"Incorporating miRNAs into a taRNA vector platform with Drs. Tim Beissert and Ayegl Yildiz - The. Drs. Ayegl Yildiz and Tim Beissert join Dr. Paloma Giangrande to discuss a recent article published in Molecular Therapy Nucleic Acids by Drs. Yildiz. Beissert and colleagues titled Trans-amplifying RNA expressing functional miRNA mediates target gene suppression and simultaneous transgene expression. Are you registered for ASGCTs [----] Annual Meeting in New Orleans Join us May 13-17 for the premier event in our field and save $465 as an ASGCT member. Check out the full program plan your trip and"
YouTube Link 2025-03-04T11:28Z 13.5K followers, [---] engagements
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