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CTX321
- Development stage
- Preclinical
- Lead developer
- CRISPR Therapeutics
- Modality
- CRISPR-Cas9 → CRISPR Systems → Programmable Nucleases → Gene Editing → Gene Therapies, Lipid-based Nanoparticles → Nanoparticles → Drug Delivery Systems
- Administration
- Intravenous
01
Overview
CTX321 is an investigational *in vivo* CRISPR-based gene editing therapy developed by CRISPR Therapeutics for the treatment of cardiovascular diseases associated with elevated lipoprotein(a) [Lp(a)]. The therapy utilizes CRISPR technology delivered via lipid nanoparticles (LNPs) to target and disrupt the *LPA* gene in the liver, which encodes apolipoprotein(a). By permanently reducing the production of Lp(a), CTX321 aims to mitigate the risk of atherosclerotic cardiovascular disease (ASCVD) and aortic stenosis. CTX321 was designed as a potentially more potent alternative to CTX320, showing approximately twofold greater editing efficiency in animal models.
02
Targets
LPA (Lysophosphatidic acid)
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