CGTLive’s Weekly Rewind – February 23, 2024
Review top news and interview highlights from the week ending February 23, 2024.
CGTLive’s Weekly Rewind
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. Gene Editing Investigations Mature With heart-1 Trial for Familial Hypercholesterolemia
Verve Therapeutics plans to expand evaluations into the US after an IND clearance was delayed by a clinical hold.
2. N. Nora Bennani, MD, on Investigating VSV Therapy in Patients With R/R Multiple Myeloma, T-Cell Lymphoma
The chief medical officer of Encoded Therapeutics discussed ETX101 gene therapy and other initiatives in the company’s overall Dravet program.
3. IND Cleared for Solid Tumor Trial of ULBP6-Targeting Natural Killer (NK) Cell Activator
The ULBP6 target was discovered through 23andMe’s proprietary research platform of de-identified human genetic and phenotypic information.
4. Bruce Cree, MD, PhD, MAS, on How CAR-T for Autoimmune Disease May Impact Patients’ Lives
The clinical research director of the UCSF Multiple Sclerosis Center also discussed the high expense of producing individualized therapies like CAR-T.
5. Slow-and-Steady Is the Right Approach for Evaluating Cell Therapy for Autoimmune Diseases
Peter A. Merkel, MD, MPH, the chief of the Division of Rheumatology and a professor of medicine and professor of epidemiology at Penn Medicine, discussed clinical trial design considerations for this emerging field.
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.
