CGTLive®’s Weekly Rewind – October 10, 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. Taysha Snags FDA Breakthrough Therapy Designation for Rett Syndrome Gene Therapy TSHA-102

Taysha also announced that the FDA has aligned with the company on the pivotal trial protocol and statistical analysis plan.

2. Rocket Pharmaceuticals Pulls BLA for Fanconi Anemia Gene Therapy RP-L102

The company cited strategic prioritization as the reason for its decision.

3. Orca Bio’s BLA for Hematologic Malignancy Cell Therapy Orca-T Accepted by FDA With Priority Review

The PDUFA target action date for the BLA has been set at April 6, 2026.

4. Avobis Bio's Crohn Disease Cell Therapy AVB-114 Garners FDA RMAT Designation

AVB-114 previously received fast track designation from the FDA in February of this year.

5. Neurogene Reaches Accord With FDA on Plan for Registrational Trial for Rett Syndrome Gene Therapy NGN-401

Neurogene also relayed new preclinical findings on the use of intracerebroventricular and intrathecal lumbar delivery of NGN-401.