GeneTherapyLive’s Weekly Rewind – October 22, 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. Access to Gene Therapies for Rare Diseases: Barry Byrne, MD, PhD

The director of the Powell Gene Therapy Center at the University of Florida discussed improving access to gene therapies for patients with rare diseases worldwide, as well as recent challenges with manufacturing shortages due to the COVID-19 pandemic.

2. HIV Gene Therapy Gets Go-Ahead for Early-Phase Trial

Excision BioTherapeutics has received FDA clearance for its investigational new drug application for EBT-101, an investigational CRISPR-based gene therapy for the potential treatment of human immunodeficiency virus (HIV).

3. CAR T Therapies for Hematologic Malignancies and Solid Tumors

André Choulika, PhD, chief executive officer and cofounder, Cellectis, discussed therapies the company is developing, which target a number of molecular targets for different indications, including CD19, CD20, CD22, and CD70 for hematologic malignancies and solid tumors.

4. Optogenetic Therapy Shows Efficacy in Autosomal Recessive Retinitis Pigmentosa

Nanoscope Therapeutics presented positive data from their phase 1/2 study (NCT04919473) of MCO-010 in patients with retinitis pigmentosa (RP) at the 2021 Annual Meeting of the American Society of Retina Specialists (ASRS), October 8-12.

5. Identifying Patient-Specific Tumor Targets

Jessica Baker Flechtner, PhD, chief scientific officer, Genocea, discussed the ATLAS platform. GEN-011, and the TiTAN trial. She stressed the company’s focus on identifying the right targets for each patient.