CGTLive’s Weekly Rewind – August 19, 2022

Article

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.

Recent Videos
Nirav Shah, MD, MSHP, associate professor of medicine, at the Medical College of Wisconsin
Bhagirathbhai R. Dholaria, MD, an associate professor of medicine in malignant hematology & stem cell transplantation at Vanderbilt University Medical Center
Reena Sharma, MD, an adult metabolic consultant at Salford Royal Hospital
Mark Hamilton, MD, PhD, a hematology-oncology and bone marrow transplant (BMT) cell therapy fellow at Stanford University
Barry J Byrne, MD, PhD, the chief medical advisor of MDA and a physician-scientist at the University of Florida
Barry J Byrne, MD, PhD, the chief medical advisor of MDA and a physician-scientist at the University of Florida
Sarah Larson, MD, the medical director of the Immune Effector Cell Therapy Program in the Division of Hematology/Oncology at David Geffen School of Medicine at University of California, Los Angeles (UCLA)
Manali Kamdar, MD, the associate professor of medicine–hematology and clinical director of lymphoma services at the University of Colorado
Manali Kamdar, MD, the associate professor of medicine–hematology and clinical director of lymphoma services at the University of Colorado
Related Content
© 2025 MJH Life Sciences

All rights reserved.