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EVX-ALS
- Development stage
- Preclinical
- Lead developer
- Evox Therapeutics
- Modality
- 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
- Intrathecal
01
Overview
EVX-ALS is a preclinical-stage therapeutic candidate developed by Evox Therapeutics for the treatment of Amyotrophic Lateral Sclerosis (ALS). The program utilizes Evox's proprietary DeliverEX exosome platform to deliver a CRISPR/Cas9 gene-editing system designed to target the ATXN2 (Ataxin-2) gene. ATXN2 is a genetic modifier of ALS, and its reduction is hypothesized to decrease the aggregation of TDP-43, a protein central to the pathology of most ALS cases. By leveraging exosomes as a delivery vehicle, EVX-ALS aims to overcome the blood-brain barrier and other delivery challenges associated with large-molecule gene-editing tools in the central nervous system.
- Other names
- ALS program
02
Targets
ATXN2 (Ataxin-2)
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