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CRISPR-LNP
- Development stage
- Preclinical
- Lead developer
- Editas Medicine
- Modality
- Gene Addition/Replacement → Gene Therapies, Gene Silencing → Gene Therapies, mRNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, CRISPR-Cas9 → CRISPR Systems → Programmable Nucleases → Gene Editing → Gene Therapies, Lipid-based Nanoparticles → Nanoparticles → Drug Delivery Systems
01
Overview
CRISPR-LNP refers to an in vivo gene editing therapeutic platform, specifically exemplified by a program developed by Editas Medicine and Genevant Sciences targeting the LDLR gene. This therapeutic utilizes lipid nanoparticles (LNPs) to deliver CRISPR nuclease mRNA and dual guide RNAs (gRNAs) to the liver. The mechanism involves deleting negative regulatory elements within the 3′ untranslated region (UTR) of the LDLR gene, which functionally upregulates LDLR mRNA and protein expression. This approach is intended as a one-time treatment for patients with heterozygous familial hypercholesterolemia (HeFH) and atherosclerotic cardiovascular disease (ASCVD) to achieve significant and sustained reductions in LDL cholesterol levels by increasing the liver's capacity to clear LDL from the blood.
- Other names
- in vivo CRISPR gene editing medicineLDLR 3′ UTR editor
02
Targets
LDLR (Low-density lipoprotein receptor)
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