at the source
We are a leader in RNA modulation with antisense oligonucleotides focused on genetic neurological disorders
Our pipeline of programs includes VO659, currently being developed for multiple polyglutamine disorders, spinocerebellar ataxia type 3 and type 1 and Huntington’s disease. VO659 targets the CAG repeat expansion in an allele preferential manner, is reversible and transient in nature and does not change DNA code.
The VICOMERTM antisense oligonucleotide platform offers multiple different mechanisms of action, enabling us to design RNA modulating therapeutics to address specific genetic defects at the source.