CGTLive®’s Weekly Rewind – March 7, 2025
Review top news and interview highlights from the week ending March 7, 2025.
CGTLive®’s Weekly Rewind
Welcome to CGTLive®’s Weekly Rewind! We’ve compiled 5 highlights from this week’s coverage of advances in gene and cell therapies, including FDA actions, notable research, and interviews with experts across the field.
1. Neurotech’s Encapsulated Cell Therapy Encelto Approved by FDA for Macular Telangiectasia Type 2
Neurotech expects that the therapy will become available in the United States in June 2025.
2. David-Alexandre Gros, MD, on Addressing Unmet Needs in End Stage Kidney Disease With Genetically Modified Porcine Kidney Transplant
The chief executive officer of Eledon Pharmaceuticals discussed the company’s collaboration with EGenesis.
3. FDA Accepts BLA for Capricor’s DMD Cardiomyopathy Cell Therapy Deramiocel With Priority Review
The agency has set the PDUFA action date for the BLA as August 31, 2025.
4. Newfound Hope for Spinal Muscular Atrophy
Following up on Rare Disease Day, observed on February 28, Mary Schroth, MD, FAAP, FCCP, chief medical officer at Cure SMA, shared insights on the advances and promise for treating spinal muscular atrophy.
5. Ocugen’s Geographic Atrophy and Stargardt Gene Therapies Garner Positive ATMP Opinions from EMA
According to Ocugen, the EMA has granted OCU410 and OCU410ST Advanced Therapy Medicinal Product classification.
Roadblocks Faced by Gene Therapies for Muscular Dystrophies
March 14th 2025John Brandsema, MD, a pediatric neurologist in the Division of Neurology at Children’s Hospital of Philadelphia, offered insights into the obstacles the clinical community is facing around integrating gene therapies into clinical practice.
