CGTLive’s Weekly Rewind – February 2, 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 Finalizes Sets of Guidelines for Both CAR-T Products and Genome Editing Products

According to Federal Register notices, the guidance documents are updated versions of drafts originally published in March 2022.

2. Barry J Byrne, MD, PhD, on the Potential of Directed Evolution to Improve AAV Gene Therapy

The chief medical advisor of the Muscular Dystrophy Association discussed how directed evolution may help develop AAV capsids better suited for treating neuromuscular diseases.

3. FDA Approves Kite’s Axi-Cel Manufacturing Change to Reduce Turnaround Time

Kyverna has also announced data on its proprietary CAR-T manufacturing process, Ingenui-T.

4. Alfonso Sabater, MD, PhD, on Further Research With Gene Therapy in Eye Care

The associate professor of clinical ophthalmology at University of Miami discussed the first topical application of gene therapy to the eye.

5. Sarepta Therapeutics' Phase 3 Study for Limb-Girdle Muscular Dystrophy Gene Therapy SRP-9003 Begins Screening Activities

The multinational, open-label EMERGENE study will seek to enroll 15 patients with LGMD2E/R4 in total.