CGTLive’s Weekly Rewind – November 4, 2022


Review top news and interview highlights from the week ending November 4, 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. RDEB Cell Therapy Improves Wound Healing and Reduces Pain

Abeona has announced topline results from its phase 3 VIITAL study of EB-101.

2. REGENXBIO’s Gene Therapy Improves Diabetic Retinopathy Disease Severity

Updated data from the ALTITUTE trial were presented at the 55th ARS meeting in Pasadena, CA.

3. Paul Melmeyer on Regulatory Advocacy for Patients With Neuromuscular Diseases

The vice president of Public Policy & Advocacy of the Muscular Dystrophy Association discussed the organization’s goals and strategies in educating regulatory decision-makers.

4. MB-106, A CD20-CAR T, Yields 100% ORR in Waldenstrom Macroglobulinemia

Data from the first 2 patients were presented at the IWWM 2022 meeting.

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

In this feature, CGTLive highlights the PROVIDE trial of PR001 for Gaucher Awareness Month.

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Alfonso Sabater, MD, PhD, on Bringing Gene Therapy to Ophthalmology
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Thomas Crawford, MD, on Helping Patients With SMA Left Behind by Gene Therapy
J. Andrew Livingston, MD
Shankar Musunuri, PhD
Pat Furlong, BSN, RN, on Future Areas of Research in DMD
Michael Kelly, PhD, on Continuing Progress in Muscular Dystrophy in 2024
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