Drug intelligence / Profile preview

VCTX210

Development stage
Unknown
Lead developer
CRISPR Therapeutics
Modality
CRISPR-Cas9 → CRISPR Systems → Programmable Nucleases → Gene Editing → Gene Therapies, Stem Cell Therapies → Cell Therapies
Administration
Implant
01

Overview

VCTX210 is an investigational allogeneic, gene-edited, stem cell-derived therapy developed for the treatment of type 1 diabetes. The therapy was created through a strategic collaboration between CRISPR Therapeutics and ViaCyte (which was acquired by Vertex Pharmaceuticals in 2022). VCTX210 utilizes CRISPR/Cas9 technology to edit stem cell-derived pancreatic endoderm cells, making them "hypoimmune" or immune-evasive. This design is intended to allow the implanted cells to survive and function—producing insulin in response to blood glucose levels—without the need for chronic immunosuppression. A Phase 1 clinical trial for VCTX210 was initiated in early 2022. In February 2024, Vertex Pharmaceuticals opted out of the collaboration, resulting in the transfer of full ownership and control of the program back to CRISPR Therapeutics.

Brand names
VCTX210AVCTX-210AVCTX 210A
Other names
VCTX210AVCTX-210AVCTX 210A

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