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EVX-SCA2
- 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-SCA2 is a preclinical-stage exosome-based gene editing therapy developed by Evox Therapeutics for the treatment of Spinocerebellar Ataxia Type 2 (SCA2). The program utilizes Evox's proprietary DeliverEX platform to deliver CRISPR/Cas9 components encapsulated within exosomes to target and edit the ATXN2 gene. By reducing the expression of the mutant ataxin-2 protein, which is characterized by toxic polyglutamine expansions, EVX-SCA2 aims to address the underlying genetic cause of the neurodegenerative disorder and potentially slow or halt disease progression.
- Other names
- Spinocerebellar Ataxia Type 2 program
02
Targets
ATXN2 (Ataxin-2)
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