CGTLive’s Weekly Rewind – September 15, 2023
Review top news and interview highlights from the week ending September 15, 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. First Patient Receives Gene Therapy for BCG-Unresponsive Non-Muscle Invasive Bladder Cancer
Adstiladrin was approved in December 2022 and is now available for select patients under an Early Experience Program.
2. Sharon Hesterlee, PhD, on Unprecedented Progress in Muscular Dystrophies
The chief research officer, Muscular Dystrophy Association, shared a message for Duchenne Action Month.
3. Novartis Drops Development of Geographic Atrophy Gene Therapy GT005
Novartis stated that the decision was made in light of a recommendation from an independent data monitoring committee.
4. Juan Francisco Cabello, MD, on the History and Current State of Newborn Screening
The head of the Pediatric Neurology Fellowship Program at the University of Valparaíso in Chile discussed positive impact of newborn screening so far and work that still needs to be done.
5. BioCardia Likely to Miss the Mark in Phase 3 CardiAMP Heart Failure Cell Therapy Trial
The company also announced its plans for a modified study protocol that may allow a roadway to approval.
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.
