CGTLive®’s Weekly Rewind – May 2, 2025
Review top news and interview highlights from the week ending May 2, 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. FDA Approves Abeona Therapeutics' Epidermolysis Bullosa Gene Therapy Pz-Cel
The agency's decision was based on data from the phase 3 VIITAL study (NCT04227106).
2. Cassandra Gorsuch, PhD, on the Durability and Next Steps for the ARCUS Gene Editing Platform in DMD
The chief scientific officer at Precision Biosciences discussed the durable potential of ARCUS gene editing and the company’s next steps toward in-human trials for Duchenne muscular dystrophy.
3. The Evolving Landscape of Gene and Cell Therapies in Parkinson Disease: Pipeline Highlights
Emerging gene and cell therapies aim to slow Parkinson disease progression. Explore clinical updates on seven promising candidates in development.
4. Linda Marbán, PhD, on the Future of Capricor’s Cardiomyopathy Cell Therapy Deramiocel
The CEO of Capricor Therapeutics discussed future plans for the product after the promising data presented at MDA’s 2025 conference.
5. Exploring New Pathways in Muscular Dystrophy Research: From Fibrosis to Function
Matthew Alexander, PhD, a neuromuscular expert, discussed the evolving landscape of targeted therapies in muscular dystrophies, from fibrosis and glycosylation to combination strategies with gene therapy.
Conservative Management Following Treatment With DMD Gene Therapy Elevidys
May 19th 2025Ben Samelson-Jones, MD, PhD, the associate director of clinical in vivo gene therapy at Children’s Hospital of Philadelphia discussed a case he presented at the American Society of Gene and Cell Therapy’s 2025 meeting.
