VO659

VO659 is an investigational antisense oligonucleotide (ASO) therapy developed by Vico Therapeutics for the treatment of neurodegenerative diseases caused by CAG repeat expansions, including Huntington’s disease (HD), spinocerebellar ataxia type 1 (SCA1), and spinocerebellar ataxia type 3 (SCA3). It is designed to bind to expanded CAG repeats in mutant mRNA transcripts, leading to their degradation and a reduction in the production of toxic mutant proteins. This allele-preferential mechanism aims to lower levels of mutant huntingtin protein while sparing normal protein, potentially slowing or halting disease progression. VO659 is administered via intrathecal injection and has shown a significant reduction in mutant protein levels in early clinical trials. The drug has received Orphan Drug designation from both the FDA and EMA for Huntington’s disease[1][2][5][6][7].