GeneTherapyLive’s Weekly Rewind – September 24, 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. HIV Gene Therapy Gets Go-Ahead for Early-Phase Trial

The FDA has accepted the investigational new drug application for EBT-101, an investigational CRISPR-based gene therapy for the potential treatment of human immunodeficiency virus (HIV). Excision BioTherapeutics will initiate a phase 1/2 clinical trial later in 2021.

2. Challenging Paradigms in AMD: Gregory S. Hageman, PhD

The executive director of the Steele Center for Translational Medicine at the Moran Eye Center discussed his team’s research in the role of HTRA1 and touched on current avenues of research for the gene.

3. OpRegen's Continued Efficacy in Dry AMD Geographic Atrophy

Brian Culley, chief executive officer, Lineage Cell Therapeutics, discussed updated data from the phase 1/2 study of OpRegen that demonstrated anatomical and functional improvements in geographic atrophy (GA).

4. Selecting Cell Therapy Targets in Cancer

The chief executive and medical officers of Cytovia Therapeutics discussed the company's therapeutic targets, including GPC3 in solid tumors, CD38 in hematologic malignancies, and EGFR in renal cancer, as well as the company’s cell therapy scaffold.

5. Cilta-Cel Continues to Show Durable Efficacy in Multiple Myeloma

Updated data from the phase 1b/2 CARTITUDE-1 trial (NCT03548207), presented during the 2021 SOHO Annual Meeting, continue to demonstrate ciltacabtagene autoleucel's (cilta-cel) efficacy and safety in pretreated patients with multiple myeloma, with an overall response rate (ORR) of 97.9% at a median follow-up of 18 months.