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SCA2-ASO
- Development stage
- Preclinical
- Lead developer
- Riboway Therapeutics
- Modality
- Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Modified DNA Oligonucleotides → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Single-strand DNA → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics
- Administration
- Intracerebroventricular, Intrathecal
01
Overview
Riboway Therapeutics is developing an antisense oligonucleotide (ASO) therapy targeting the ATXN2 RNA for the treatment of Spinocerebellar Ataxia Type 2 (SCA2). SCA2 is a rare, autosomal dominant neurodegenerative disorder caused by pathologic CAG repeat expansions in the ATXN2 gene, which leads to cerebellar dysfunction and progressive motor impairment. This investigational therapy utilizes Riboway's proprietary AI-enabled Smart ASO platform to bind to ATXN2 mRNA, thereby reducing the expression of the toxic ataxin-2 protein. Preclinical studies have indicated potency and safety advantages compared to competitor compounds. The program is currently in the IND-enabling stage of development.
- Other names
- ATXN2-ASOATXN-2-ASOATXN 2-ASORiboway SCA2 ASO
02
Targets
ATXN2 (Ataxin-2)
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