CGTLive’s Weekly Rewind – August 19, 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. Alexis Thompson, MD, MPH, on Beti-Cel's Approval in β-Thalassemia

The primary investigator and chief of hematology at Children’s Hospital of Philadelphia discussed beti-cel's approval.

2. FDA Approves Beti-Cel Gene Therapy for β-Thalassemia

ZYNTEGLO previously received a unanimous, favorable, vote from the FDA advisory committee.

3. Merit Cudkowicz, MD, on Evaluating Mesenchymal Cell Therapy for ALS

BrainStorm Cell Therapeutics has decided to submit a BLA for NurOwn in ALS following an erratum to a phase 3 trial.

4. Tisa-Cel Associated With Improved Outcomes at Higher Doses

Higher doses were associated with improved overall survival, event-free survival, and relapse-free survival.

5. Homology Trades in Gene Delivery for Gene Editing to Treat Phenylketonuria

The pheEDIT clinical trial initiated in June 2022.