CGTLive®’s Weekly Rewind – May 2, 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. 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.