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.

Adstiladrin was approved in December 2022 and is now available for select patients under an Early Experience Program.

The chief research officer, Muscular Dystrophy Association, shared a message for Duchenne Action Month.

Novartis stated that the decision was made in light of a recommendation from an independent data monitoring committee.

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.

The company also announced its plans for a modified study protocol that may allow a roadway to approval.