CGTLive’s Weekly Rewind – March 31, 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.

The gene-edited cell therapy had previously been under a partial clinical hold restricting pediatric enrollment to the clinical trial.

The associate professor of neurology at Columbia University spoke about the importance of identifying patients with ALS-associated gene mutations as early as possible.

Most patients with RP treated with MCO-010 had clinically meaningful improvements in vision.

The Lillehei professor in stem cell and regenerative cardiovascular medicine at the University of Minnesota spoke about preclinical research with an iPS cell-derived product.

The Study Safety Committee has recommended that the trial proceed to enroll patients in the highest-dose cohort.