CGTLive®’s Weekly Rewind – September 13, 2024
Review top news and interview highlights from the week ending September 13, 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. 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.
The Challenges of Gene Therapy Approaches in Advanced Muscular Dystrophy
September 13th 2024Kevin Campbell, PhD, a Howard Hughes Investigator at the University of Iowa, discussed his mouse model research into the pathophysiology of muscular dystrophy and how it relates to gene therapy approaches.
