VO659 - ASO gene therapy for ataxias SCA1, SCA3 and Huntington's disease
- Márcio
- Apr 20, 2023
- 1 min read
Vico Therapeutics informs that the first dose was administered to a human patient in the clinical trial (Phase 1/2a) of the drug VO659 for ataxias SCA1 and SCA3 and for Huntington's Disease (HD).
VO659 is an RNA-based gene therapy, more precisely, an ASO (antisense oligonucleotide) therapy. It's the most advanced, and although this study will still take a few years, it has the potential to effectively cure ataxia (and several other genetic diseases). ASO therapy "silences" the mutated genes (ATXN1 in the case of SCA1, and ATXN3 in the case of SCA3) and thus prevents them from encoding the defective proteins that cause the disease, eliminating the problem at its root. The same process occurs for HD (Huntington's disease). Let's trust science!
[Post published on 04/20/2023 by Márcio Galvão]
