CGTLive®’s Weekly Rewind – July 25, 2025

CGTLive®’s Weekly Rewind

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 Revokes Sarepta’s Platform Technology Designation for Muscular Dystrophy Gene Therapy Vector Following Patient Deaths

The agency also put several of the company’s clinical trials on hold.

2. Sven Moller-Tank, PhD, on Regeneron’s Precision Approach to AAV Targeting

The senior director at Regeneron discussed the company’s receptor-driven approach to AAV targeting, leveraging antibody engineering to enhance tissue specificity and reduce off-target effects.

3. First Patient Dosed in Phase 2/3 GARDian3 Trial of OCU410ST for Stargardt Disease

Ocugen initiates pivotal confirmatory trial for OCU410ST, its second modifier gene therapy candidate, in patients with Stargardt disease.

4. Alexey Danilov, MD, PhD, on CAR-T and Bispecific Antibody Sequencing for DLBCL

The professor in the Division of Lymphoma in the Department of Hematology & Hematopoietic Cell Transplantation at City of Hope discussed unanswered questions with regard to DLBCL treatment sequencing.

5. European Commission Approves Obe-Cel for Adults With R/R B-Cell Precursor Acute Lymphoblastic Leukemia

The therapy was approved based on the findings of the pivotal phase 1b/2 FELIX clinical trial.