CGTLive’s Weekly Rewind – March 10, 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. Sangamo Joins Companies Halting Sickle Cell Gene-edited Cell Therapy Programs

The company halted its phase 3 plans for BIVV003 in favor of therapies for Fabry and immune rejection in kidney transplantation. The investigational zinc finger nuclease gene-edited cell therapy intended to treat sickle cell disease (SCD) will not be pursued beyond the necessities for completing the ongoing phase 1/2 PRECIZN-1 clinical trial (NCT03653247).

2. Matthew B. Harms, MD, on Gene Therapy in ALS, MDA 2023

The associate professor of neurology at Columbia University spoke about the session on amyotrophic lateral sclerosis (ALS) that he will be chairing at MDA’s 2023 Clinical and Scientific Conference, as well as the latest conversations surrounding genetic medicine developments in ALS.

3. FDA Extends Val-Rox PDUFA Date in Hemophilia A

Following the submission of a 3-year data analysis from the ongoing GENEr8-1 clinical trial (NCT03370913)—deemed a major amendment to the company's application—the FDA informed Biomarin this week that it has extended the review of valoctocogene roxaparvovec(val-rox; Roctavian) for the treatment of severe hemophilia A.

4. FDA Accepts BLA Resubmission for Remestemcel-L in Steroid-Refractory aGVHD

Mesoblast’s allogeneic off-the-shelf therapy showed long-term benefit in supporting data among children with steroid-refractory acute graft versus host disease. The FDA has set a PDUFA action date of August 2, 2023, for its review.

5. Jeffrey S. Chamberlain, PhD, on Regulating DMD Gene Therapy Clinical Trials

The professor and director of the Senator Paul D. Wellstone Muscular Dystrophy Specialized Research Center at the University of Washington School of Medicine discussed working with the FDA toward a first disease-modifying therapy approval in Duchenne muscular dystrophy.