CGTLive’s Weekly Rewind – April 7, 2023

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. New Knowledge of DNA Virus Replication Process Could Inform Gene Therapy Development

Study authors Matthew Charman, PhD, and Matthew D. Weitzman, PhD, elaborated on the findings and implications of their research.

2. Madeleine Powys, MBBS, on Lessons Learned With Libmeldy

The locum consultant of pediatric BMT and leukemia at Royal Manchester Children’s Hospital discussed the center’s experience in delivering the approved gene therapy to children with MLD.

3. Astrocyte Cell Therapy Temporarily Slows ALS Decline

No treatment-emergent adverse events experienced were deemed related to AstroRx, although 9 serious TEAEs did occur.

4. Matthew Gornet, MD, on Treating Degenerative Disc Disease With Cell Therapy

The spine surgeon from The Orthopedic Center of St. Louis discussed recent data on IDCT from the phase 1/2 DGX-A01 study.

5. Vertex, CRISPR Complete Rolling BLAs for Exa-Cel in Sickle Cell Disease and Transfusion-dependent Beta Thalassemia

The rolling BLAs also included requests for priority review of the CRISPR/Cas9 gene-edited therapy, and is supported by data from the phase 3 CLIMB-111 and CLIMB-121 studies, as well as the long-term follow-up study CLIMB-131.