CGTLive®’s Weekly Rewind – March 22, 2024
Review top news and interview highlights from the week ending March 22, 2024.
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.
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.
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.
News and Expert Insights in Hemophilia Gene Therapy for Bleeding Disorders Awareness Month 2025
March 9th 2025In observance of Bleeding Disorders Awareness Month, held annually in March, catch up on some of the latest news and expert insights in gene therapy for hemophilia, which is among the most common of these disorders.
