Drug intelligence / Profile preview

CTX213

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

Overview

CTX213 is an allogeneic, gene-edited, stem cell-derived beta-cell replacement therapy currently in preclinical development by CRISPR Therapeutics for the treatment of Type 1 Diabetes Mellitus. The product consists of unencapsulated (deviceless) precursor islet cells derived from human stem cells that have been modified using CRISPR/Cas9 technology. These genetic edits are designed to make the cells immune-evasive, potentially allowing them to survive and function after transplantation without the need for chronic immunosuppressive drugs. CTX213 is engineered to produce insulin in a glucose-dependent manner, aiming to restore natural glycemic control in patients. It represents a next-generation candidate informed by clinical data from the CTX211 program.

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