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.
Alzheimer's & Brain Awareness Month 2025: Looking Back at Recent Progress in Cell and Gene Therapy
June 18th 2025In honor of Alzheimer's & Brain Awareness Month, observed annually in June, we took a look back at recent news and insights in cell and gene therapy for Alzheimer disease and other dementias.
Eugenio Galli, MD, PhD, on Reexamining Frailty as a Barrier to CAR-T Treatment
June 16th 2025The hematologist at the Hematology and Stem Cell Transplants Unit of Fondazione Policlinico Universitario A. Gemelli IRCCS in Rome discussed a study on the effect of comorbidities in patients receiving CAR-T.
