CGTLive’s Weekly Rewind – February 16, 2024
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.
1. FDA Pushes Rocket’s LAD-I Gene Therapy PDUFA for CMC Review
Rocket submitted additional CMC data in response to FDA information requests.
2. Salvador Rico, MD, PhD, on A Multi-Pronged Approach to Tackling Dravet Syndrome
The chief medical officer of Encoded Therapeutics discussed ETX101 gene therapy and other initiatives in the company’s overall Dravet program.
3. European Commission Approves Exa-Cel for SCD and TDT
The approval follows the separate US approvals in December 2023 and January 2024.
4. Heather Lau, MD, MS, on Addressing Unmet Needs in MPSIIIA With Gene Therapy
The executive director of global clinical development at Ultragenyx Pharmaceutical discussed UX111, the company’s investigational gene therapy for MPSIIIA.
5. IND Cleared for First RNA Exon Editor Therapy, Trial to Initiate in Stargardt Disease
Ascidian Therapeutics presented positive preclinical data at the 2023 ASGCT meeting.
Newsletter
Stay at the forefront of cutting-edge science with CGT—your direct line to expert insights, breakthrough data, and real-time coverage of the latest advancements in cell and gene therapy.
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.
