Drug intelligence / Profile preview

STX-1600

Development stage
Preclinical
Lead developer
Scribe Therapeutics
Modality
CRISPR-Cas9 → CRISPR Systems → Programmable Nucleases → Gene Editing → Gene Therapies, Classical Binding Small Molecules → Small Molecules, Gene Silencing → Gene Therapies, Covalent Small Molecules → Small Molecules, Gene Addition/Replacement → Gene Therapies
01

Overview

STX-1600 is an investigational epigenetic silencing therapy being developed by Scribe Therapeutics for the treatment of cardiovascular disease associated with elevated levels of Lipoprotein(a) [Lp(a)]. The drug utilizes Scribe's proprietary ELXR platform, which employs CRISPR-based technology to achieve durable, long-term silencing of the *LPA* gene without introducing double-stranded DNA breaks. By targeting the hepatic expression of the *LPA* gene, STX-1600 aims to reduce the synthesis and circulating levels of Lp(a), a genetically determined and independent risk factor for atherosclerosis, myocardial infarction, and calcific aortic valve stenosis. It is currently in the preclinical stage of development.

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