CGTLive®’s Weekly Rewind – October 17, 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. BLA for Rocket’s LAD-I Gene Therapy Under Review by FDA Again

The FDA has set the PDUFA action date for the BLA at March 28, 2026.

2. Myrtelle’s Canavan Disease Gene Therapy Effects Significant Decrease in CSF NAA and Increases in Brain Myelin Volume

With regard to safety, MYR-101 was characterized as “well-tolerated” with a “favorable safety profile.”

3. FDA Picks Abeona’s XLRS Gene Therapy ABO-503 for RDEA Pilot Program

The RDEA program is meant to support use of novel end points for measuring efficacy in clinical studies for therapies aimed at treating rare diseases.

4. Patients Treated With Spur Therapeutics’ Gaucher Disease Gene Therapy FLT201 Maintain Clinical Benefit for Up to 2 Years

The efficacy data included 4 patients who had discontinued their SOC therapy in a timeframe of 4 to 11 weeks after being treated with FLT201.

5. Krystal Biotech’s Neurotrophic Keratitis Gene Therapy Viral Vector Secures FDA Platform Technology Designation

Krystal is the second company to have received the new FDA designation.