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CTX460

Development stage: Preclinical
Lead developer: CRISPR Therapeutics
Modality: Gene Addition/Replacement → Gene Therapies, Gene Editing → Gene Therapies, Gene Silencing → Gene Therapies
Administration: Intravenous

Overview

CTX460 is an investigational gene therapy candidate developed by CRISPR Therapeutics using the SyNTase editing platform. It targets the E342K mutation in the SERPINA1 gene, which is responsible for Alpha-1 Antitrypsin Deficiency (AATD). CTX460 is encapsulated in a proprietary lipid nanoparticle (LNP) and has shown promising preclinical results, including over 90% mRNA correction and a five-fold increase in total AAT levels in animal models[1][2][3]. The therapy is expected to enter clinical trials in mid-2026.

Targets

Serpin family A member 1 (SERPINA1) E342K genomic DNA locus

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