CGTLive’s Weekly Rewind – October 28, 2022

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Review top news and interview highlights from the week ending October 28, 2022.

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. Cystinosis Gene Therapy Trial in Full Swing

The trial is being conducted at the University of California – San Diego under the leadership of Stephanie Cherqui, PhD.

2. TJ Cradick, PhD, on A Potential Cure for HIV With EBT-101

The chief scientific officer of Excision BioTherapeutics discussed the phase 1/2 clinical trial of the CRISPR gene editing therapy.

3. PR001 Hopes to Address Underlying Mutations in Neuronopathic Type 2 Gaucher Disease

CGTLive highlights the PROVIDE trial of PR001 for Gaucher Awareness Month.

4. Aimee Donald, MBChB, PhD, on Assessing PR001 in Children With Type 2 Gaucher Disease

The pediatrician from Royal Manchester Children’s Hospital discussed progress with the PROVIDE trial of PR001.

5. JC Virus T-Cell Therapy to be Evaluated in Progressive Multifocal Leukoencephalopathy

The trial expects to enroll its first patient in the first half of 2023.

Recent Videos
David Dimmock, MBBS, on AI-Guided ASO Development for Ultra-Rare Diseases
Subhash Tripathi, PhD, on Generating In Vivo CARs With A2-CAR-CISC EngTreg Cells
Paul Y. Song, MD, the chairman and chief executive officer of NKGen
Paul Y. Song, MD, the chairman and chief executive officer of NKGen
Leigh Ramos-Platt, MD, on Looking Forward to Gene Therapy’s Growth
Paul Y. Song, MD, the chairman and chief executive officer of NKGen
Ignacio Mata, PhD, an associate professor of neurology at the Cleveland Clinic Lerner Institute
Subhash Tripathi, PhD, on Developing Safe, Specific Engineered Treg Cell Therapy
Ignacio Mata, PhD, an associate professor of neurology at the Cleveland Clinic Lerner Institute
Sharif Tabebordbar, PhD, on Improving In Vivo Gene Editing for DMD
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