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ePsCas9
- Development stage
- Preclinical
- Lead developer
- AstraZeneca
- Modality
- mRNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, 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
- Systemic
01
Overview
ePsCas9 is an engineered variant of the PsCas9 Type II-B family enzyme, developed as a highly efficient and precise CRISPR-Cas tool for therapeutic genome editing. It is designed for in vivo editing, delivered via mRNA formulated with lipid nanoparticles. This engineered enzyme demonstrates up to 20-fold increased activity across various targets while preserving safety advantages, including reduced off-targets and chromosomal translocations. It has shown high levels of editing in the Pcsk9 gene in mouse liver, indicating its potential for treating hypercholesterolemia.
- Brand names
- eSpOT-ON
- Other names
- engineered PsCas9
02
Targets
PCSK9 (Proprotein convertase subtilisin/kexin type 9)
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