CGTLive®’s Weekly Rewind – September 13, 2024

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Review top news and interview highlights from the week ending September 13, 2024.

CGTLive®’s Weekly Rewind

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.

Recent Videos
Paul Melmeyer, MPP, the executive vice president of public policy & advocacy at MDA
John Brandsema, MD, a pediatric neurologist in the Division of Neurology at Children’s Hospital of Philadelphia
John Brandsema, MD, a pediatric neurologist in the Division of Neurology at Children’s Hospital of Philadelphia
Barry J. Byrne, MD, PhD, the chief medical advisor of Muscular Dystrophy Association (MDA) and a physician-scientist at the University of Florida
John Brandsema, MD, a pediatric neurologist in the Division of Neurology at Children’s Hospital of Philadelphia
Chun-Yu Chen, PhD, a research scientist at Seattle Children’s Research Institute
William Chou, MD, on Targeting Progranulin With Gene Therapy for Frontotemporal Dementia
Alexandra Collin de l’Hortet, PhD, the head of therapeutics at Epic Bio
David Dimmock, MBBS, on Accelerating Therapy Discovery and Approval With AI David Dimmock, MBBS, on Accelerating Therapy Discovery and Approval With AI
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