@asgct Avatar @asgct ASGCT

ASGCT posts on YouTube about dr, education, in the, pfizer inc the most. They currently have [------] followers and [---] posts still getting attention that total [---] engagements in the last [--] hours.

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Social Influence

Social category influence stocks 13.33% finance 6.67% countries 2.86% technology brands 2.86% musicians 0.95% cryptocurrencies 0.95%

Social topic influence dr 14.29%, education 11.43%, in the 9.52%, pfizer inc #5, community 8.57%, quick 5.71%, aav 5.71%, hosted 4.76%, gene 3.81%, target 3.81%

Top accounts mentioned or mentioned by @regeneron @genscript @biotechne

Top assets mentioned Frontline Ltd. (FRO)

Top Social Posts

Top posts by engagements in the last [--] hours

"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, 57.7K 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, 227.6K 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

"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, 172.9K 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

"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, 81.4K 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

"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

"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

"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

"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

"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

"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

"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

"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

"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

"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

"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

"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, 33.5K 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

"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, 48K 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

"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

"Duchenne Muscular Dystrophy and Gene Therapy The most common form of muscular dystrophy is called Duchenne. The disease is caused by a faulty gene that interferes with how muscle cells produce a protein called dystrophin. Its part of a vital protein complex that works to strengthen and protect muscle fibers. The subtitling of these videos was funded in part by a donation from Pfizer Inc. DMD Duchenne Muscular Dystrophy American Society of Gene and Cell Therapy Gene Therapy DMD Duchenne Muscular Dystrophy American Society of Gene and Cell Therapy Gene Therapy"
YouTube Link 2019-10-24T10:00Z 13.5K followers, 35.3K 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

"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

"Pluripotent Cell-Derived NK Cells as a Cornerstone Approach for Off-the-Shelf Cancer Immunotherapy Pluripotent Cell-Derived NK Cells as a Cornerstone Approach for Off-the-Shelf Cancer Immunotherapy - Bob Valamehr Scientific Symposium from the American Society of Gene & Cell Therapy's 21st Annual Meeting"
YouTube Link 2018-12-07T21:24Z 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

"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

"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

""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

"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

"Lunch & Learn: Gene Therapy for SMA Spinal Muscular Atrophy (SMA) is a rare genetic disease that affects the motor nerve cells in the spinal cord and is the leading genetic cause of infant mortality. During this education session we heard from experienced clinicians sharing insights on gene therapy options for SMA as well as a parent's perspective"
YouTube Link 2021-09-10T21:33Z 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, 15.9K 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

"Phase IIb Multiple Dose Non-Viral Cystic Fibrosis Gene Transfer Clinical Trial Phase IIb Multiple Dose Non-Viral Cystic Fibrosis Gene Transfer Clinical Trial - Stephen Hyde Scientific Symposium from the American Society of Gene & Cell Therapy's 22nd Annual Meeting"
YouTube Link 2019-12-04T21:55Z 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

"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

"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

"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

"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

"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

"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

"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

"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

"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

"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

"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

"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

"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

"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

"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

"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

"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

"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

"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

"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

"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 13.2K followers, 12.2K 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

"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

"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

"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

"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

"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

"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

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