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.
News and Expert Insights in Hemophilia Gene Therapy for Bleeding Disorders Awareness Month 2025
March 9th 2025In observance of Bleeding Disorders Awareness Month, held annually in March, catch up on some of the latest news and expert insights in gene therapy for hemophilia, which is among the most common of these disorders.
