CGTLive®’s Weekly Rewind – June 21, 2024

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.