CGTLive®’s Weekly Rewind – June 21, 2024
Review top news and interview highlights from the week ending June 21, 2024.
CGTLive®’s Weekly Rewind
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. Choosing the Right Treatment Path for Sickle Cell Disease
In observance of World Sickle Cell Day, CGTLive brought together a variety of expert insights on navigating the rapidly expanding landscape of care for this inherited blood disorder.
2. Leigh Ramos-Platt, MD, on Sharing Experience With Gene Therapy Administration
The clinical professor of neurology and pediatrics at Keck School of Medicine of USC discussed her learning experience administering gene therapies for the first time.
3. Gene-Edited Exa-Cel Continues to Show Long-Term Benefit in Sickle Cell, Thalassemia
Data from up to 5 and 6 years of follow-up were presented at the 2024 EHA Congress.
4. Luke Roberts, MBBS, PhD, on Developing Gene Therapy for Congestive Heart Failure
The medical director of clinical development at AskBio discussed the progress of an early clinical trial of AB-1002.
5. Cabaletta Bio’s CAR-T CABA-201 Shows Initial Signs of Efficacy in Myositis and Lupus Trials
The data, from the first patients dosed in each trial, also continues to show safety.
Newsletter
Stay at the forefront of cutting-edge science with CGT—your direct line to expert insights, breakthrough data, and real-time coverage of the latest advancements in cell and gene therapy.
Jeffrey Chamberlain, PhD, on the Import of Early Intervention in DMD
July 10th 2025The McCaw Endowed Chair of Muscular Dystrophy at University of Washington, discussed how comprehensive care for DMD patients involves early diagnosis, steroid treatment, consideration of mutation-specific therapies, and more.
