CGTLive®’s Weekly Rewind – November 29, 2024

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. Genethon’s Gene Therapy GNT0004 Stabilizes or Improves Motor Functions in Patients With Duchenne Muscular Dystrophy

All 3 patients who received the high dose showed stabilization of motor function, as assessed by the 34-point North Star Ambulatory Assessment.

2. Exploring CAR-T for Autoimmune Disease

Georg Schett, MD, vice president research and chair of internal medicine at the University of Erlangen – Nuremberg, discussed findings from 2 early studies of CD19 CAR T-cell therapy.

3. FDA Approves StemCyte’s Cord Blood Cell Therapy Regenecyte for Unrelated Donor Hematopoietic Progenitor Cell Transplantation Procedures

The FDA’s decision was based on data from 3 different sources.

4. Future Trends for Genomic Medicines

David Barrett, JD, the chief executive officer of ASGCT, discussed likely future trends extrapolated from the organization’s Q3 Landscape Report.

5. Cabaletta Bio’s CAR-T CABA-201 Effects Clinical Responses in Autoimmune Diseases

Clinical responses were reported in patients with lupus, myositis, and systemic sclerosis.