CGTLive®’s Weekly Rewind – January 3, 2025

CGTLive®’s Weekly Rewind

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.

1. Aurion Biotech’s Cell Therapy AURN001 Demonstrates Efficacy and Safety in Phase 1/2 ABA-1, CLARA Clinical Trial

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

2. Confirming the Safety of Pfizer's Hemophilia B Gene Therapy Beqvez

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.

3. Capricor Puts Rolling BLA for DMD Cardiomyopathy Cell Therapy Deramiocel in Front of the FDA

The company requested priority review for the BLA.

4. The Curative Potential of Liso-Cel in Third-Line R/R LBCL

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.

5. Leveling Up: Cell Therapy Research Expands Horizons in Autoimmune Disease

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