CGTLive®’s Weekly Rewind – December 1, 2025

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 Approves Itvisma, a New Intrathecal Route for Novartis’ SMA Gene Therapy

According to Novartis, it is the first gene replacement therapy to have been approved “for this broad population.”

2. Structuring Partnerships Among Academia, Biotech, and Pharma

Deborah Phippard, PhD, and Renier Brentjens, MD, PhD, spoke about streamlining collaboration to speed up advancement of new therapies.

3. Sarepta's Elevidys Gets Boxed Warning for ALI and ALF

The FDA also added a new Warnings & Precaution for heightened susceptibility to serious infections caused by immunosuppression.

4. Carrie Haverty, MS, CGC, on Exploring Genetic Counseling's Growth and Challenges

The NSGC president-elect spoke on the evolving role of genetic counselors, emphasizing personalized care, gene therapy advancements, and the need for multidisciplinary integration in healthcare.

5. Michelle Alabek, MS, CGC, on the Role of Genetic Counselors in Supporting Patients Receiving Gene Therapy

The genetic counselor in the Department of Ophthalmology at the University of Pittsburgh Medical Center discussed the growing role of genetic counselors in guiding patients undergoing gene therapy.