CGTLive®’s Weekly Rewind – October 24, 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. Sarepta Therapeutics’ Limb-Girdle Muscular Dystrophy Gene Therapy SRP-9003 Produces “Robust Expression” of β-SG in Phase 3 Trial

The primary end point of the study was met.

2. AAVantgarde’s Stargardt Gene Therapy AAVB-039 Garners FDA Orphan Drug Designation, Cleared for Trial in UK

The IND application enabling the launch of the CELESTE trial in the US was previously cleared by the FDA in July 2025.

3. CARsgen’s CAR-T Satri-Cel Significantly Reduces CA19-9 Levels in Patients With Pancreatic Ductal Adenocarcinoma

It was noted that the CA19-9 reductions in the 5 patients observed after infusion of satri-cel ranged from 51.3% to 96.1%.

4. AskBio’s Heart Failure Gene Therapy AB-1002 Shows Ability to Improve NYHA Class in Phase 1 Trial

In terms of safety, there were no AEs or serious AEs deemed related to AB-1002.

5. Patients Treated With AskBio’s Limb-Girdle Muscular Dystrophy Gene Therapy AB-1003 Free of Serious Adverse Events at Up to 52 Weeks

Headaches, falls, and nausea constituted the common treatment-emergent AEs, and were deemed mild-to-moderate in terms of severity.