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.