CGTLive®’s Weekly Rewind – June 27, 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. Vertex’s Islet Cell Therapy Zimislecel (VX-880) Restores Endogenous Insulin Secretion in Type 1 Diabetes

The patients also showed cessation of severe hypoglycemia events and hit recommended glycemic control targets.

2. Tami John, MD, on Barriers to Access for Gene Therapy in β-Thalassemia

The clinical associate professor at Stanford Medicine also provided background about the current state of care in TDT.

3. FDA Indicates AdComm Not Currently Required for Capricor’s BLA for DMD Cardiomyopathy Cell Therapy Deramiocel

In a mid-cycle review meeting, the agency also noted that the review committee found no major deficiencies with the BLA package.

4. Exploring DARIC CAR T-Cells for Autoimmune Indications

Peter Cook, PhD, a senior research scientist at Seattle Children’s Research Institute, discussed preclinical work on using dimerizing agent-regulated immune-receptor complex T-cells to target plasma cells.

5. enGene Snags FDA RMAT Designation for Bladder Cancer Gene Therapy Detalimogene

The gene therapy is currently being evaluated in the phase 1/2 LEGEND clinical trial.