Gene therapy for SCA1 and SCA3 underway…

Best news of the day: the first patient received a dose of Vico Therapeutics' VO659 drug, for ataxias SCA1 and SCA3, and Huntington's disease. The study is being conducted in Europe with 71 patients. There's still a long way to go, but an important step has been taken. This is RNA-based therapy, using ASOs (antisense nucleotides) to silence mutated genes (the ATXN3 gene in the case of SCA3), preventing them from producing defective proteins that are toxic to neurons. In other words, it has the potential to cure ataxia (and several other neurological diseases). It's good to know that science is advancing.

https://vicotx.com/vico-therapeutics-announces-first-patient-dosed-in-phase-1-2a-clinical-trial-of-vo659-in-huntingtons-disease-and-spinocerebellar-ataxia-types-1-and-3/

[Post published on 04/04/2023 by Márcio Galvão]