CGTLive®’s Weekly Rewind – August 1, 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. Rocket’s PKP2-Arrhythmogenic Cardiomyopathy Gene Therapy RP-A601 Snags RMAT Designation

According to Rocket, the RMAT designation was granted based on results from a phase 1 clinical trial (NCT05885412).

2. ImmunoLogic, Episode 5: "Tumor Infiltrating Lymphocytes" With Michael T. Lotze, MD

In Episode 5 of ImmunoLogic, Michael T. Lotze, MD, discussed the evolution and future of tumor infiltrating lymphocyte (TIL) therapies.

3. FDA Gives Sarepta Green Light to Restart Shipment of DMD Gene Therapy Elevidys to Patients who are Ambulatory

The FDA also stated that it has concluded its investigation of the death an 8-year-old boy who was treated with Elevidys in Brazil.

4. FDA Accepts Atara Biotherapeutics’ Resubmission of BLA for Tabelecleucel

Notably, the BLA resubmission was accepted with priority review and the PDUFA target action date has been set for January 10, 2026.

5. Regeneron’s Future Plans for its AAV Targeting Approach

Sven Moller-Tank, PhD, a senior director at Regeneron, also discussed his thoughts on trends in the gene therapy field in general.