CGTLive’s Weekly Rewind – 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.