CGTLive®’s Weekly Rewind – July 11, 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. Capricor’s BLA for DMD Cardiomyopathy Cell Therapy Deramiocel Slammed With CRL

In the CRL, the FDA stated that the statutory requirement for “substantial evidence of effectiveness” was not met by the BLA and that more clinical data will be needed.

2. Derek Jackson, BS, MA, on Evaluating Gene Therapy PCRX-201 for Osteoarthritis of the Knee

The vice president of cell & gene therapy product development at Pacira BioSciences discussed findings related to the immunogenicity of the therapy’s high-capacity adenovirus vector.

3. Cell Therapy’s Lot in Solid Tumors: Challenges and Opportunities

Several experts weighed in on the various strategies being explored to tackle solid tumors with cell therapy, expressing optimism despite the plethora of obstacles that remain.

4. Jeffrey Chamberlain, PhD, on the Import of Early Intervention in DMD

The McCaw Endowed Chair of Muscular Dystrophy at University of Washington, discussed how comprehensive care for DMD patients involves early diagnosis, steroid treatment, consideration of mutation-specific therapies, and more.

5. SineuGene Garners Orphan Drug Designation for ALS Gene Therapy

SNUG01 is intended to deliver a copy of the human TRIM72 gene to neurons via an rAAV9 vector.