Drug intelligence / Profile preview

CTX211

Development stage
Unknown
Lead developer
CRISPR Therapeutics
Modality
Gene Therapies, Pluripotent Stem Cells → Stem Cell Therapies → Cell Therapies
Administration
Implantation
01

Overview

CTX211 is an allogeneic, gene-edited, stem cell-derived investigational therapy developed for the treatment of type 1 diabetes. It is designed as an immune-evasive cell replacement therapy that enables patients to produce their own insulin in response to glucose. The product consists of stem cell-derived pancreatic beta-cell precursors that have been genetically modified using CRISPR/Cas9 technology to evade immune detection (hypoimmune) and enhance cell fitness. The gene edits include modifications such as B2M inhibition (to reduce MHC class I expression), HLA-E modulation (to provide protection from natural killer cells), and MANF modulation (potentially enhancing beta-cell survival). The goal is to provide a durable source of functional insulin-producing cells without the need for chronic immunosuppression[2][3][5][6][7].

Other names
CTX 211CTX211CTX-211VCTX 211VCTX211VCTX-211
02

Targets

MANF (Mesencephalic astrocyte-derived neurotrophic factor)HLA-E (Human leukocyte antigen-e)

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