CGTLive®’s Weekly Rewind – September 20, 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. Oncternal Therapeutics Axes B-Cell Lymphoma Trial for CAR-T Therapy ONCT-808

The company also discontinued its other clinical programs as well, which included small molecule and monoclonal antibody modalities.

2. From Grad Work to Gene Therapy Approval

Jeffrey Chamberlain, PhD, McCaw Endowed Chair of Muscular Dystrophy at University of Washington, discussed his research career with muscular dystrophies.

3. Rocket Pharmaceuticals Maxes Out Recruitment in Pivotal Trial for Danon Disease Gene Therapy RP-A501

The single-arm trial enrolled 12 male patients in total across the United States and European Union.

4. John Brandsema, MD, on Navigating the New Landscape of Care for DMD

The pediatric neurologist in the Division of Neurology at Children’s Hospital of Philadelphia discussed the complicated choice doctors, patients, and families now face with a wide range of treatment options available.

5. Poseida Therapeutics Garners FDA RMAT Designation for Allogeneic CAR-T P-BCMA-ALLO1 in R/R Multiple Myeloma

The product is currently being evaluated in a phase 1/1b clinical trial.