Drug intelligence / Profile preview

CTX120

Development stage
Discontinued
Lead developer
CRISPR Therapeutics
Modality
CAR-T Cells → Engineered T Cells → Adoptive Cell Transfer → Cell Therapies, Gene Therapies
Administration
Intravenous
01

Overview

CTX120 is an allogeneic, gene-edited CAR-T cell therapy developed by CRISPR Therapeutics. It is derived from healthy donor T cells and engineered using CRISPR/Cas9 technology to express a chimeric antigen receptor (CAR) targeting B-cell maturation antigen (BCMA), which is highly expressed on multiple myeloma cells. The gene editing process also removes endogenous T-cell receptors and MHC class I molecules to reduce the risk of graft-versus-host disease and immune rejection. The therapy aims to induce immunologic cytotoxicity against BCMA-expressing malignant plasma cells in patients with relapsed or refractory multiple myeloma. CTX120 has received Orphan Drug designation for this indication.

02

Targets

BCMA (B-cell maturation antigen)

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