CGTLive’s Weekly Rewind – November 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 AdComm Favorable Towards Vertex and CRISPR’s Sickle Cell Gene-Edited Cell Therapy, but Recommends Postmarketing Research

The members of the AdComm were generally of the opinion that the off-target analyses conducted by Vertex were reasonably robust.

2. Maria Pia Morelli, MD, PhD, on Considerations for Cell Therapy in GI Cancers

The assistant professor at MD Anderson Cancer Center discussed factors to consider when trying to treat patients with gastrointestinal cancers with cell therapy.

3. ELEVIDYS Study Fails Primary End Point in DMD, Secondary End Points Show Statistically Significant Benefit

Despite the announcement, CEO Doug Ingram shared that the company is seeking a wider approval for delandistrogene moxeparvovec-rokl regardless of age or ambulatory status.

4. Brett Kopelan, MA, on the Future Treatment of Epidermolysis Bullosa With Advanced Therapeutics

In observance of Epidermolysis Bullosa Awareness Week, the executive director of debra of America discussed how a combination of advanced and topical approaches may represent the future of EB treatment.

5. CRISPR Therapy Well Tolerated, Detectable in First 3 Participants With HIV

Excision plans to escalate the trial of EBT-101 to the second dose level in the fourth quarter of 2023.