CGTLive’s Weekly Rewind – September 22, 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. Taysha Gene Therapies Drops Development of Giant Axonal Neuropathy Gene Therapy TSHA-120

The company is now prioritizing its gene therapy program for Rett syndrome.

2. Pat Furlong, BSN, RN, on the State of Gene Therapy in Muscular Dystrophy

The founding president and chief executive officer of Parent Project Muscular Dystrophy discussed recent and upcoming milestones in the field.

3. Research Identifies Cell Signatures of CAR T-Cells in Long Remission

Cells from patients with ALL treated in the CARPALL study were analyzed and sequenced.

4. Deborah Phippard, PhD, on the History and Current State of Gene Therapy for Neurological Indications

The chief scientific officer of Precision for Medicine discussed the previous and current delivery methods used for gene therapies and new innovations on the horizon.

5. Orchard Therapeutics’ BLA for Metachromatic Leukodystrophy Cell Therapy Arsa-Cel Accepted With Priority Review

The PDUFA data has been set for March 18, 2024.