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CTX130

Development stage
Phase 1
Lead developer
CRISPR Therapeutics
Modality
CRISPR-Cas9 → CRISPR Systems → Programmable Nucleases → Gene Editing → Gene Therapies, CAR-T Cells → Engineered T Cells → Adoptive Cell Transfer → Cell Therapies
Administration
Intravenous
01

Overview

CTX130 is a first-in-class, allogeneic, gene-edited chimeric antigen receptor (CAR) T cell therapy developed using CRISPR/Cas9 technology. It is designed to target the CD70 antigen, which is expressed on various tumor cells including those in T-cell lymphomas and renal cell carcinoma. The therapy uses healthy donor-derived T cells that are genetically modified to express an anti-CD70 CAR and to disrupt the TRAC and β2-microglobulin (β2M) genes, reducing risks of graft-versus-host disease and immune rejection. Additionally, endogenous CD70 expression is disrupted to prevent fratricide among therapeutic cells. CTX130 has shown promising safety and efficacy in early-phase clinical trials for relapsed or refractory T-cell malignancies and advanced clear cell renal cell carcinoma[1][5][6][7].

Other names
Allogeneic CRISPR/Cas9-mediated genetically modified CAR T cells targeting CD70 antigen
02

Targets

CD70 (Cluster of Differentiation 70)

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