CGTLive’s Weekly Rewind – May 19, 2023

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. Krystof Bankiewicz, MD, PhD, on Dialing Back Progression of Parkinson Disease With GDNF Gene Therapy

The professor at The Ohio State University and the University of California, San Francisco, discussed updated data from a phase 1 study of GDNF gene therapy in patients with Parkinson disease from the American Society of Gene and Cell Therapy 2023 Annual Meeting, in Los Angeles, California.

2. Reexamining the Outcome Measures Used in Ophthalmic Gene Therapy Trials

Bart P. Leroy, MD, PhD, head of the Department of Ophthalmology at the Center for Medical Genetics of Ghent University Hospital, spoke to the vital need to reevaluate the measures used to determine whether gene therapies for eye indications come to market.

3. CytoAgents’ Drug for Management of CAR-T-associated CRS Gets FDA IND Clearance

CTO1681, which is designed to lower NF-kB signaling without eliminating it altogether, was previously evaluated for safety in a phase 1 clinical trial in healthy adults.

4. TCR-Based T-Cell Therapy Cleared for Trials

The neoantigen-specific T-cell receptor therapy is the first to enter clinical trials out of a collaboration with Genentech. The collaboration is identifying and characterizing TCRs with the use of Adaptive’s TCR discovery platform.

5. Sleeping Beauty Engineered CAR-T Therapy CARCIK-CD19 Demonstrates Safety and Anti-Leukemia Activity in R/R B-ALL

Among 27 evaluated patients with relapsed/refractory B-cell acute lymphoblastic leukemia, 18 achieved a complete response in data presented at the American Society of Gene and Cell Therapy 2023 Annual Meeting, in Los Angeles, California.