Review top news and interview highlights from the week ending 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.
Evaluating Spinal Muscular Atrophy Gene Therapy Zolgensma in Heavier and Older Patients
March 16th 2024Sandra P. Reyna, MD, the chief scientific advisor and head of global medical engagement for SMA at Novartis, discussed data from the phase 3b SMART clinical trial (NCT04851873) that were presented at MDA's 2024 conference.