CGTLive’s Weekly Rewind – December 23, 2022

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. Running With Scissors: The FDA Moves to Tame Gene Therapy Drug Development

With several newly approved therapies hitting the market and an overflowing pipeline, the FDA has stepped in to help keep the burgeoning world of biotech on track.

2. Sham Mailankody, MBBS, on Updates on ALLO-715 CAR T in R/R Multiple Myeloma

The associate attending physician at Memorial Sloan Kettering Cancer Center discussed updated data presented at the ASH 2022 meeting.

3. Partial Hold Lifted on bluebird bio's Sickle Cell Disease Gene Therapy

The hold was originally placed in December 2021 and was related to a case of persistent, non-transfusion-dependent anemia in a pediatric patient.

4. Steven Pipe, MD, on Continuing Durability of Hemgenix in Hemophilia B

The professor of pediatric hematology/oncology at CS Mott Children’s Hospital discussed new follow-up data from the HOPE-B study presented at ASH 2022.

5. Gene Therapies Advance in Danon Disease, CLN2 Batten Disease

Both REGENXBIO and Rocket Pharmaceuticals have announced trial updates in their gene therapy programs for rare diseases.