CGTLive’s Weekly Rewind – May 20, 2022

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. Final Analysis of Phase 3 HOPE-B Trial: Durability of EtranacogeneDezaparvovec and QoL Improvements

The trial met its primary efficacy end point, and participants reported improvements in quality-of-life factors such as outlook on the future and feelings about having hemophilia.

2. Florian Eichler, MD, on How Novel Gene Therapy Delivery Improved Targeting in GM2 Gangliosidosis

The director of the leukodystrophy service at Mass Gen discussed the phase 1/2 study of AXO-AAV-GM2.

3. At-Risk Genotypes for DMD Gene Therapy Identified

Trial sponsors Pfizer, Sarepta, Genethon, and Solid Biosciences organized a group of experts to investigate shared serious AEs.

4. Homology-Directed Repair-Developed TCR Therapy Shows Potential in Type 1 Diabetes

Investigators from Seattle Children’s Hospital found that engineered T-cell regulatory products demonstrated significant suppression of effector T cells.

5. Yara Abdou, MD, on the Advantages of CAR Macrophages in Solid Tumors

The assistant professor of oncology at UNC School of Medicine discussed the first-in-human study being conducted with CT-0508.