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.
Sickle Cell Disease Gene Therapy Exa-Cel's Ability to Prevent VOCs
December 12th 2024Haydar Frangoul, MD, the medical director of pediatric hematology/oncology at Sarah Cannon Research Institute and Pediatric Transplant and Cellular Therapy Program at TriStar Centennial, discussed the latest data update from the CLIMB SCD-121 trial evaluating exa-cel.
10-Year Data Show Allogeneic Stem Cell Transplant Benefits for Sickle Cell Anemia
December 10th 2024A long-term follow-up to the DREPAGREFFE-1 trial suggest that children with sickle cell anemia may benefit long-term on risk of cerebral injury, cognitive functions, and quality of life over standard care transfusions.
Autologous HCT Shows No Benefit for Patients With MCL in First Complete Remission
December 10th 2024Among those who had undetectable minimal residual disease, autologous hematopoietic cell transplantation showed signs of benefit only for those who remained MRD-positive following induction therapy.
