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@Franca_ole "$RCKT announced that FDA accepted resubmission for Kresladi (Marne-cel) a GT for severe Leukocyte Adhesion Deficiency-I with a PDUFA date of 28.3.26. All endpoints were met and Kresladi was well tolerated. Rocket may receive a Rare Pediatric Disease Priority Review Voucher if approved"
X Link @Franca_ole 2025-10-14T16:15Z XXX followers, XXX engagements

"The #FDA today released a draft guidance document on innovative designs for clinical trials of cell and gene therapy products in small populations. Among other things these include Single Arm Trials Utilizing Participants as Their Own Control. $QURE $NTLA $BMRN $SGMO $NVS $CRSP $BEAM"
X Link @Franca_ole 2025-09-24T21:01Z XXX followers, 1054 engagements

".just back from Albania. XXX km on the bike with approx. 3700 meters of elevation gain across a wild country in simple accommodations. Finally we ended up in the Adriatic Sea on a coast that has been paved with lots of concrete . what a shame Not much has changed at $SGMO. only the Nobel Prize in Medicine might be of interest. I'll have to read up on that . or rather who can teach me about it"
X Link @Franca_ole 2025-10-12T12:32Z XXX followers, 1078 engagements

"Sales of cell and gene therapies (CGTs) are expected to grow strongly: for example GlobalData estimates that the market could grow from around $XXX billion in 2024 to over $XX billion by 2030. $EDIT $NVS $BEAM $SGMO $NTLA $VRTX $RHHBY $"
X Link @Franca_ole 2025-10-15T15:31Z XXX followers, XXX engagements

"Thermo Fisher operates several cell and gene therapy facilities globally and the San Francisco/Bay Area site is listed as part of its network for advanced therapies. $TMO also mentioned a unit called GA Custom Products and Services at its SSF site this could indicate customer-specific products possibly also for genetic or molecular biology applications. The proximity to the $SGMO plant in Brisbane is a given"
X Link @Franca_ole 2025-09-28T15:14Z XXX followers, 1573 engagements

"Sangamo Therapeutics Inc. is looking for a . Temporary Desktop Support Specialist $SGMO"
X Link @Franca_ole 2025-10-14T19:09Z XXX followers, 7740 engagements