CGTLive®’s Weekly Rewind – March 29, 2024

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. FDA Deems Mesoblast’s Data on Remestemcel-L Cell Therapy Sufficient for BLA Submission in Pediatric SR-aGVHD

The company noted that it plans to submit the BLA in the second quarter of 2024.

2. John A. Charlson, MD, on Afami-Cel’s Upcoming Review for Synovial Sarcoma

The associate professor of medicine, Medical College of Wisconsin, discussed advantages of afami-cel and data from the pivotal SPEARHEAD-1 trial.

3. AOC Therapy Yields Strength Improvements in Myotonic Dystrophy

Avidity is currently enrolling in the phase 3 HARBOR trial, which it expects to initiate in the second quarter of 2024.

4. Vivien Sheehan, MD, PhD, on Choosing the Right Treatment Option for Patients With Sickle Cell Disease

The associate professor of pediatrics at Emory University discussed the need to empower patients and families to make their own treatment decisions.

5. SRP-9001 Improves DMD Disease Trajectory, But Falls Short on Primary End Point

In the phase 3 EMBARK trial, treatment with SRP-9001 improved secondary outcomes of time to rise, microdystrophin expression, and 10-meter walk/run.