CGTLive®’s Weekly Rewind – March 28, 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. Secretome Therapeutics Doses First Patient in Phase 1 Trial for Heart Failure Cell Therapy STM-01

According to Secretome, the first-in-human, open-label trial is the first trial to assess an allogeneic stem cell therapy product for HFpEF.

2. Natalie Goedeker, CPNP, on Handling Neuromuscular Gene Therapy at Real-World Sites

The codirector of pediatric neuromuscular clinical research at Washington University in St. Louis discussed the session she chaired at MDA’s 2025 conference.

3. Genetic and Gender Factors Linked to Fuchs Endothelial Corneal Dystrophy in University College London Study

These findings could support gene therapies targeting CTG18.1 that are currently in development.

4. David-Alexandre Gros, MD, on Using Tegoprubart to Support Genetically Modified Xenotransplant

The chief executive officer of Eledon Pharmaceuticals discussed the company’s investigational monoclonal antibody and its use along with EGenesis porcine kidney transplant product.

5. Sarepta’s DMD Gene Therapy Elevidys Demonstrates Manageable Safety Across 5-Year Timespan

In pooled data from 156 patients, there were no deaths or study discontinuations.