Review top news and interview highlights from the week ending May 26, 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.
The investigational gene therapy, also known as SRP-9001, was recently backed by the agency’s advisory committee in a tight decision. Its new deadline for review is June 22, 2023.
The pediatric gastroenterologist and professor in residence at University of California at San Francisco discussed updated data from the phase 1/2 CAMPSIITE trial presented at ASGCT 2023.
All evaluable patients had improvements in natriuretic peptides, left ventricular volume, and NYHA class.
All evaluable patients had significant reductions in daily cornstarch intake at 1 year and at last visit during long-term follow-up.