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.

1. Lovo-cel BLA Submission Likely Delayed for Sickle Cell

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

2. Matthew B. Harms, MD, on the Necessity of Tracking Gene Mutations in ALS

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

3. Multi-Characteristic Opsin Optogenetic Therapy Meets Phase 2 Endpoint in Retinitis Pigmentosa

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

4. Rita Perlingeiro, PhD, on the Potential of iPS Cell-based Regenerative Medicine in Muscular Dystrophy

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.

5. First Patient With HPV16+ Solid Tumors in SQZ-AAC-HPV Trial Achieves CR

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