CGTLive®’s Weekly Rewind – June 27, 2025
Review top news and interview highlights from the week ending 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.
Newsletter
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Jeffrey Chamberlain, PhD, on the Import of Early Intervention in DMD
July 10th 2025The 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.
