CGTLive®’s Weekly Rewind – January 3, 2025

Welcome to CGTLive®’s Weekly Rewind! We’ve compiled 5 highlights from the past 2 week’s coverage of advances in gene and cell therapies, including FDA actions, notable research, and interviews with experts across the field.

The trial is evaluating AURN001 for the treatment of corneal edema secondary to corneal endothelial dysfunction.

Ben Samelson-Jones, MD, PhD, the associate director of clinical in vivo gene therapy at Children’s Hospital of Philadelphia, discussed follow-up data of up to 6 years with investigations of fidanacogene elaparvovec.

The company requested priority review for the BLA.

Manali Kamdar, MD, the associate professor of medicine–hematology and clinical director of lymphoma services at the University of Colorado, discussed 5-year follow-up data from the TRANSCEND-NHL-001 clinical trial.

Cell therapy, particularly CAR-T, is expanding into autoimmune diseases like lupus and multiple sclerosis. While promising, safety, efficacy, and broad applicability remain uncertain.