CGTLive®’s Weekly Rewind – April 26, 2024

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Review top news and interview highlights from the week ending April 26, 2024.

CGTLive®’s Weekly Rewind – April 26, 2024

CGTLive®’s Weekly Rewind – April 26, 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. FDA Issues CRL for Abeona's Epidermolysis Bullosa Gene Therapy Pz-Cel

The FDA is requesting more CMC information, specifically with regard to validation requirements for specific manufacturing and release testing methods.

2. Akshay Sharma, MBBS, on Overcoming Challenges for Administration of Ex-Vivo SCD Gene Therapy

The attending physician and assistant member of bone marrow transplantation and cellular therapy at St Jude Children’s Research Hospital also discussed hurdles to accessibility for SCD gene therapy.

3. One Big Step Away From BLA Submission for Sangamo’s Fabry Gene Therapy

The FDA is allowing a small, single-arm study to support a BLA submission of ST-290, if Sangamo can find help to continue developing the therapy.

4. Alexandra Gomez-Arteaga, MD, on Expanding Eligibility for Cell Transplant With Cell Therapy

The Assistant Professor of Medicine at Weill Cornell Medical College discussed the changing cell transplant landscape.

5. First Patient Treated With Benitec Biopharma’s Oculopharyngeal Muscular Dystrophy Gene Therapy Shows Swallowing Improvements

At the 90 day timepoint, the patient showed improvements in swallowing compared to their own natural history data, as measured by videofluoroscopic swallowing studies.

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Salvador Rico, MD, PhD, on Informing Clinical Trials With Natural History Studies in Dravet
Zheng-Yi Chen, DPhil, on International Collaboration on Clinical Trials
Maria Escolar, MD, the chief medical officer of Forge Biologics
Leigh Ramos-Platt, MD, on Allowing Access and Ensuring Preparation for Gene Therapies
John Murphy, PhD, the chief scientific officer of Arbor Biotechnologies
Erika Fullwood Augustine, MD, MS, the associate chief science officer of the Kennedy Krieger Institute
Maria Escolar, MD, the chief medical officer of Forge Biologics
Casey Maguire, PhD, associate professor of neurology and associate investigator of neurology, Harvard Medical School
Evan Weber, PhD, an assistant professor of pediatrics at Children's Hospital of Philadelphia
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