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Evox MSH3-targeting CRISPR exosome therapy
- Development stage
- Preclinical
- Lead developer
- Evox Therapeutics
- Modality
- CRISPR-Cas9 → CRISPR Systems → Programmable Nucleases → Gene Editing → Gene Therapies, Lipid-based Nanoparticles → Nanoparticles → Drug Delivery Systems
- Administration
- Intraparenchymal, Intracerebroventricular
01
Overview
This is a preclinical gene editing therapeutic developed by Evox Therapeutics for the treatment of Huntington's disease. Utilizing the proprietary ExoEdit platform, the therapy encapsulates CRISPR-Cas genome editing ribonucleoproteins within exosomes—naturally occurring extracellular vesicles—to facilitate delivery to the central nervous system. The program specifically targets the MSH3 gene, a known driver of somatic CAG repeat expansion in Huntington's disease, with the goal of modifying the gene to halt or slow disease progression. This modular platform approach is designed to enable rapid scaling across multiple genetic indications, including other trinucleotide repeat disorders.
- Other names
- HD Program (MSH3 target)ExoEdit MSH3 program
02
Targets
MSH3 (DNA mismatch repair protein Msh3)
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