Drug intelligence / Profile preview

CTX321

Development stage
Preclinical
Lead developer
CRISPR Therapeutics
Modality
CRISPR-Cas9 → CRISPR Systems → Programmable Nucleases → Gene Editing → Gene Therapies, Gene Silencing → Gene Therapies, Gene Addition/Replacement → 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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