CGTLive®’s Weekly Rewind – May 23, 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. Rocket’s PKP2-Arrhythmogenic Cardiomyopathy Gene Therapy RP-A601 Improves or Stabilizes Heart Function in Phase 1 Trial

The data presented came from 3 patients treated in the trial who had a follow-up time of up to 12 months.

2. Ben Samelson-Jones, MD, PhD, on Conservative Management Following Treatment With DMD Gene Therapy Elevidys

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.

3. Brian Kim, MBA, on Multiomic Single Cell Sequencing and the Ebbs and Flows of the Gene Therapy Field

The CEO of Mission Bio discussed the company’s single cell assay platform in the context of the broader developing field cell and gene therapy field.

4. Commercial Bottlenecks in Gene Therapy Development

Nathan Yozwiak, PhD, the head of research at the Gene and Cell Therapy Institute at Mass General Brigham, discussed how innovative research can be held back by financial considerations.

5. Global Cell and Gene Therapy: The Next Wave of Biotechnology Innovation

As CGT continues to evolve, innovation is set to expand the reach of these therapies, offering new solutions for treating a broader range of complex diseases and changing how we approach global patient care.