CGTLive’s Weekly Rewind – January 26, 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. FDA Requests Black Box Warning Be Added for All Currently Approved CAR-T Therapies

The FDA has requested that black box warnings related to secondary cancer risks be added to all 6 CAR-T therapies currently on the market.

2. Barry J Byrne, MD, PhD, on Overcoming the Hurdles Presented by Immunity to AAV Vectors

The chief medical advisor of the Muscular Dystrophy Association discussed research he is working on with the goal of increasing eligibility for gene therapies and opening the door to redosing.

3. Janssen and Legend Biotech’s Carvykti to be Put in Front of FDA AdComm Regarding Multiple Myeloma Expanded Use BLA

The European Medicines Agency Committee for Advanced Therapies will likewise hold a SAG-O meeting regarding the Type II variation application for the CAR-T in the EU.

4. David Shyr, MD, on ASH 2023 Highlights, Further Research for Nula-Cel SCD Therapy

The clinical assistant professor at Stanford Medicine also shared his excitement on the recent approvals of lovo-cel and exa-cel.

5. OTOF Gene Therapy Restores Hearing in Children With Autosomal Recessive Deafness 9

Speech perception was also greatly improved in the children with hearing recovery.