Review top news and interview highlights from the week ending February 16, 2024.
CGTLive’s Weekly Rewind – February 16, 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.
Rocket submitted additional CMC data in response to FDA information requests.
The chief medical officer of Encoded Therapeutics discussed ETX101 gene therapy and other initiatives in the company’s overall Dravet program.
The approval follows the separate US approvals in December 2023 and January 2024.
The executive director of global clinical development at Ultragenyx Pharmaceutical discussed UX111, the company’s investigational gene therapy for MPSIIIA.
Ascidian Therapeutics presented positive preclinical data at the 2023 ASGCT meeting.
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Jeffrey Chamberlain, PhD, on the Import of Early Intervention in DMD
July 10th 2025The McCaw Endowed Chair of Muscular Dystrophy at University of Washington, discussed how comprehensive care for DMD patients involves early diagnosis, steroid treatment, consideration of mutation-specific therapies, and more.