CGTLive’s Weekly Rewind – August 19, 2022
Review top news and interview highlights from the week ending 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.
Expanding Access to Whole Genome Sequencing in Parkinson Research
May 12th 2025James Beck, PhD, chief scientific officer of the Parkinson’s Foundation, discussed the foundation’s efforts to integrate whole genome sequencing into PD GENEration and expand access across diverse global populations.
