GeneTherapyLive’s Weekly Rewind – November 12, 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. This week’s edition includes coverage of the 36th Annual Meeting of the Society for Immunotherapy of Cancer (SITC), November 10-14, 2021.

1. TCR T-Cell Therapy Shows Efficacy Across Solid Tumor Types

IMA203, an autologous, TCR-engineered T cell (TCR-T), PRAME-directed therapy, has shown efficacy across multiple types of solid cancers. The therapy was well-tolerated and all 12 evaluable patients achieved disease control, defined as stable disease or a partial response.

2. Developing Gene Therapies for Rare Diseases

The chief executive officer of Rocket Pharmaceuticals, Gaurav Shah, MD, discussed the company’s lead programs and future plans. He discussed programs in Fanconi anemia, LAD-1, and Danon disease, as well as the company’s future research and plans.

3. CAR T-Cell Therapy Plus Amplifying Vaccine Shows Initial Safety, Efficacy in Solid Tumors

BNT211, a CLDN6 chimeric antigen receptor (CAR) T-cell candidate, has shown signs of efficacy in solid tumors with a favorable safety profile, both alone and in combination with a CAR-T cell amplifying RNA vaccine (CARVac).

4. Gene Therapy Well-Tolerated in Fabry Disease

Isaralgagene civaparvovec (ST-920; Sangamo Therapeutics) has shown efficacy in treating Fabry disease, according to new data from the phase 1/2 STAAR clinical study (NCT04046224). The therapy was well-tolerated and above normal α-Gal A activity was maintained for up to a year after treatment.

5. Tmod CAR T-Cell Therapy Offers Precise Targeting of Solid Tumors

A2 Biotherapeutics has announced positive preclinical feasibility data on their mesothelin (MSLN) and carcinoembryonic antigen (CEA)-targeted Tmod chimeric antigen receptor (CAR) T-cell therapies, as well as data from their observational BASECAMP-1 study (NCT04981119).