CGTLive®’s Weekly Rewind – May 17, 2024

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. Liso-Cel/Breyanzi Gets Accelerated Approval for Relapsed/Refractory Follicular Lymphoma

The CAR T-cell therapy was also recently approved for treating chronic lymphocytic leukemia or small lymphocytic lymphoma.

2. Kevin Campbell, PhD, on the Challenges of Gene Therapy Approaches in Advanced Muscular Dystrophy

The Howard Hughes Investigator at the University of Iowa discussed his mouse model research into the pathophysiology of muscular dystrophy and how it relates to gene therapy approaches.

3. AADC Deficiency Gene Therapy up For FDA Priority Review

Eladocagene exuparvovec is approved in the United Kingdom and European Union under the name Upstaza.

4. Debora Mazzetti, MS, on Multitargeting MicroRNA in Glioblastoma

The research trainee at Brigham and Women’s Hospital discussed developing RNA constructs to aid with RNA interference.

5. Verve Therapeutics Doses First Patient in Phase 1b Trial for Gene Therapy VERVE-102

VERVE-102 is intended to reduce LDL-C in patients with heterozygous familial hypercholesterolemia or premature coronary artery disease.