CGTLive’s Weekly Rewind – June 16, 2023

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. Genetic Medicine in DMD: End Points, Assessment, and Approvals

CGTLive takes a look at challenges that have beset the first gene therapy coming close to market for treating Duchenne muscular dystrophy in 2023.

2. Matthew Frank, MD, PhD, on Tackling Antigen Loss and Absence in Hematological Malignancies

The assistant professor of medicine in the Division of Blood and Marrow Transplantation and Cellular Therapy at Stanford University discussed potential methods for optimizing CAR-T efficacy that could be explored in the future.

3. Patient Death Prompts Pause in 2seventy bio's Trial for Acute Myeloid Leukemia CAR-T

Seattle Children’s and 2seventy bio, which are collaborating on the development of SC-DARIC33, are currently investigating the cause of the grade 5 serious adverse event.

4. Susan Bal, MD, on Favorable Safety of BMS-986393 in R/R Multiple Myeloma

The assistant professor of medicine at University of Alabama – Birmingham discussed new data from the first-in-human trial of the CAR T-cell therapy.

5. Point-of-Care CAR T-Cell Therapy Yields 100% ORR in R/R CLL With or Without Richter’s Transformation

The phase 1/2 Euplagia-1 trial is currently ongoing in Europe.