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EVX-HD

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: Intrathecal, Intratumoral

Overview

EVX-HD is an exosome-based gene editing therapeutic being developed by Evox Therapeutics for the treatment of Huntington's Disease. The program utilizes Evox's proprietary DeliverEX platform to package CRISPR/Cas9 components into exosomes for targeted delivery to the brain. The therapeutic mechanism involves the targeted editing of the MSH3 gene, which is a known genetic modifier of Huntington's Disease progression. By reducing MSH3 levels, the therapy aims to stabilize the CAG repeat expansions in the HTT gene, potentially slowing or halting the neurodegenerative process. It is currently in the preclinical stage of development.

Other names: Huntington's Disease program

Targets

MSH3 (DNA mismatch repair protein Msh3)

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