CGTLive’s Weekly Rewind – May 26, 2023

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 Pushes Review of Sarepta’s Delandistrogene Moxeparvovec for Duchenne Muscular Dystrophy

The investigational gene therapy, also known as SRP-9001, was recently backed by the agency’s advisory committee in a tight decision. Its new deadline for review is June 22, 2023.

2. Paul Harmatz, MD, on Reducing GAGs in MPS Type 2 With RGX-121 Gene Therapy

The pediatric gastroenterologist and professor in residence at University of California at San Francisco discussed updated data from the phase 1/2 CAMPSIITE trial presented at ASGCT 2023.

3. Danon Disease Gene Therapy Improves Disease Biomarkers

All evaluable patients had improvements in natriuretic peptides, left ventricular volume, and NYHA class.

4. Carol Miao, PhD, on Exploring New Delivery Methods for Gene Replacement Therapy and Gene Editing

All evaluable patients had significant reductions in daily cornstarch intake at 1 year and at last visit during long-term follow-up.