CGTLive®’s Weekly Rewind – March 22, 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 Approves Orchard Therapeutics’ Gene-Edited Cell Therapy Arsa-Cel for Metachromatic Leukodystrophy

Arsa-cel, approved under the name Lenmeldy, is set to be priced at $4.25 million.

2. Robert J. Hopkin, MD, on Safely Delivering Gene Therapy for Fabry Disease

The associate professor of clinical pediatrics at Cincinnati Children's discussed the latest data from the phase 1/2 STAAR study of isaralgagene civaparvovec.

3. Phase 3 Trial Seeks to Continue Supporting Arsa-Cel Gene Therapy for MLD

The gene-edited cell therapy has been approved as Lenmeldy by the FDA.

4. Atul Malhotra, MD, PhD, on the Importance of Preclinical Work for Bringing Cell Therapies to Clinic

The head of the early neurodevelopment clinic at Monash Children's Hospital discussed preclinical work in the context of neonatal cell therapy research.

5. Patients With Hemophilia Biding Their Time for Gene Therapy

Many surveyed participants were content with current treatments or wary of gene therapy’s adverse events and durability.