CGTLive®’s Weekly Rewind – November 29, 2024

News
Article

Review top news and interview highlights from the week ending November 29, 2024.

CGTLive®’s Weekly Rewind

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. 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.



Recent Videos
Haydar Frangoul, MD, the medical director of pediatric hematology/oncology at Sarah Cannon Research Institute and Pediatric Transplant and Cellular Therapy Program at TriStar Centennial
Bhagirathbhai R. Dholaria, MD, an associate professor of medicine in malignant hematology & stem cell transplantation at Vanderbilt University Medical Center
Caroline Diorio, MD, FRCPC, FAAP, an attending physician at the Cancer Center at Children's Hospital of Philadelphia
R. Nolan Townsend; Sandi See Tai, MD; Kim G. Johnson, MD
Paul Melmeyer, MPP, the executive vice president of public policy & advocacy at MDA
John Brandsema, MD, a pediatric neurologist in the Division of Neurology at Children’s Hospital of Philadelphia
John Brandsema, MD, a pediatric neurologist in the Division of Neurology at Children’s Hospital of Philadelphia
Barry J. Byrne, MD, PhD, the chief medical advisor of Muscular Dystrophy Association (MDA) and a physician-scientist at the University of Florida
John Brandsema, MD, a pediatric neurologist in the Division of Neurology at Children’s Hospital of Philadelphia
Related Content
© 2024 MJH Life Sciences

All rights reserved.