VNX-101

VNX-101 is an investigational gene therapy developed by Vironexis Biotherapeutics for the treatment of CD19-positive B-cell malignancies, primarily acute lymphoblastic leukemia (ALL). It utilizes an adeno-associated virus (AAV) vector to deliver a transgene encoding a secreted bispecific T-cell engager—a single-chain variable fragment (scFv) diabody that targets both CD19 on B-cells and CD3 on T-cells. Once administered intravenously, the AAV vector transduces liver cells, which then continuously secrete the bispecific protein into circulation. This protein binds to CD19 on malignant and benign B-cells and to CD3 on endogenous T-cells, redirecting T-cell cytotoxicity toward B-cells. The approach aims to provide continuous in vivo production of the therapeutic agent, potentially offering improved safety, efficacy, durability, and convenience compared with traditional CAR-T or bispecific antibody therapies. VNX-101 has received FDA Fast Track Designation and Rare Pediatric Disease Designation. Clinical development is ongoing in phase 1/2 trials for relapsed/refractory ALL as well as other B-cell malignancies[1][2][4][5][6].

Other names

Adeno-associated viral serotype rh74 vector encoding an anti-CD19-anti-CD3 bispecific protein (Vironexis)GP101GP-101GP 101