CGTLive’s Weekly Rewind – February 3, 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. FDA Blocks 4D’s Gene Therapy Program for Fabry, Clears IND for Diabetic Macular Edema

The clinical hold comes a few weeks after the company announced it was stopping enrollment in the phase 1/2 clinical trial.

2. Raj Mehra, PhD, and Krishna Subramanian, PhD, on Striving For Disease-Modifying Therapies in Parkinson Disease

Mehra and Subramanian discussed preclinical research with the investigational gene therapy SLS-004.

3. Giant Axonal Neuropathy Gene Therapy Needs Double-blind Trial Before BLA Submission

The dosing of additional patients in a double-blind, placebo-controlled design will help support a BLA submission for TSHA-120.

4. Gerhard Ehninger, MD, on Managing Toxicities With Switchable CARs in AML

The professor at University Hospital Dresden discussed the positive safety profile of Unicar-T-CD123.

5. Remestemcel-L Cell Therapy Makes Comeback With Resubmitted BLA for Pediatric GvHD

Mesoblast is resubmitting its BLA with new CMC, survival, and mechanism of action data on the therapy and phase 3 trial.