GeneTherapyLive’s Weekly Rewind – October 1, 2021

Welcome to GeneTherapyLive’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. Addressing Adverse Events in Gene Therapy

While gene therapies represent a revolutionary step forward in the potential treatment of many previously incurable diseases, the advent of these new therapies comes with a host of challenges. We reviewed recent serious adverse events leading up to the September FDA Advisory Committee Meeting.

2. Investigating BET Inhibitors to Improve CAR T Therapy: Joseph A. Fraietta, PhD

The director of the Solid Tumor Immunotherapy Lab at the University of Pennsylvania discussed JQ1 and other, more specific BET inhibitors he would like to study. He also touched on how these inhibitors could fit into the treatment landscape.

3. CRISPR Therapy Shows Some Benefit in Leber Congenital Amaurosis

Editas Medicine has announced initial positive data from their ongoing phase 1/2 BRILLIANCE trial of EDIT101, a CRISPR-based therapy, for the potential treatment of Leber congenital amaurosis 10 (LCA10), while a competing RNA-based therapy for LCA10 is currently being evaluated in a phase 2/3 trial.

4. Gene Therapy for Pompe Disease: Barry Byrne, MD, PhD

The director of the Powell Gene Therapy Center at the University of Florida discussed gene therapy programs being conducted for Pompe disease. He touched on his investigations into re-administration of intramuscular AAV9 in patients with late-onset Pompe disease and the center’s goals.

5. Developing Cell Therapies for Solid Tumors and Hematologic Malignancies

Sonny Hsiao, PhD, chief executive officer, president and cofounder, Acepodia, discussed the company’s cell therapy technologies and their recently presented positive interim data on lead program, ACE1702, at ESMO 2021.