CGTLive’s Weekly Rewind – February 23, 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. 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.