Drug intelligence / Profile preview

VCTX212

Development stage
Preclinical
Lead developer
Vertex
Modality
CRISPR-Cas9 → CRISPR Systems → Programmable Nucleases → Gene Editing → Gene Therapies, Stem Cell Therapies → Cell Therapies
Administration
Injectable
01

Overview

VCTX212 is an allogeneic, gene-edited, stem cell-derived cell therapy candidate designed for the treatment of Type 1 and Type 2 diabetes. Developed through a collaboration between CRISPR Therapeutics and ViaCyte (now part of Vertex Pharmaceuticals), the program utilizes CRISPR/Cas9 gene-editing technology to modify human stem cell-derived pancreatic islet cells. These modifications are intended to make the cells immune-evasive, allowing them to survive and function after implantation without the need for chronic immunosuppressive drugs. VCTX212 represents a follow-on or next-generation iteration of the VCTX210 and VCTX211 programs, aiming to provide a functional cure by replacing the insulin-producing capacity lost in diabetic patients.

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