CGTLive®’s Weekly Rewind – March 7, 2025

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.