CGTLive®’s Weekly Rewind – September 13, 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. Allogeneic CAR-T Azer-Cel Achieves Complete Responses in Some Patients With Diffuse Large B-Cell Lymphoma

The cell therapy, originally developed by Precision BioSciences, is being evaluated for LBCL by Imugene.

2. John Brandsema, MD, on Balancing Risks and Rewards With Muscular Dystrophy Gene Therapy

The pediatric neurologist in the Division of Neurology at Children’s Hospital of Philadelphia discussed how Sarepta’s Elevidys has affected the landscape of care for DMD.

3. ProKidney Puts Focus on US Trial for T2D and CKD Cell Therapy Rilparencel

The company also reported that it is discontinuing the phase 3 REGEN-016 (PROACT 2) clinical trial evaluating rilparencel in Spain.

4. Barry J Byrne, MD, PhD, on Making Decisions in the Face of Rapidly Expanding Treatment Options

The chief medical advisor of Muscular Dystrophy Association (MDA) gave his view on treatment decision-making in the world of new options for DMD.

5. Solid Biosciences Tracking Long-Term Effects of Duchenne Muscular Dystrophy Gene Therapy in Phase 1/2 IGNITE-DMD Study

Following up on World Duchenne Awareness Day, observed annually on September 7 by the clinician and patient communities, CGTLive decided to take a closer look at this study.