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.
Will the CMS CGT Model Support Patient Access to Sickle Cell Disease CGTs?
July 25th 2024Cell and gene therapies can transform the paradigm of care for patients with chronic, complex conditions, but these therapies come at an up-front cost of several million dollars per treatment, complicating the pipeline of access to them.
