Evox Therapeutics ATXN2 ALS program

This is a preclinical gene editing therapeutic developed by **Evox Therapeutics** that targets the *ATXN2* gene for the treatment of **amyotrophic lateral sclerosis (ALS)** and **spinocerebellar ataxia type 2 (SCA2)**. The therapy utilizes Evox's proprietary **ExoEdit platform**, which encapsulates CRISPR-based genome editing ribonucleoproteins (RNPs) within engineered exosomes to deliver the therapeutic payload to the central nervous system. By leveraging naturally occurring vesicles, the system is designed to transport gene editing machinery across biological barriers, such as the blood-brain barrier, and into target cells. *ATXN2* is a significant disease modifier in ALS and the primary genetic driver of SCA2; reducing its expression or modifying the gene is intended to alleviate neurodegeneration. As of recent reports, the program is progressing toward CTA-enabling studies, which are projected to commence in 2026.

Other names

ExoEdit ATXN2 programEvox ALS Program (ATXN2 target)ATXN2-targeting exosome therapyATXN-2-targeting exosome therapyATXN 2-targeting exosome therapy