Vico Therapeutics

Vico Therapeutics is a clinical-stage genetic medicines company focused on developing antisense oligonucleotide (ASO) RNA modulating therapies for severe neurological diseases. The company's lead product candidate, VO659, is currently in Phase 1/2a clinical development for the treatment of spinocerebellar ataxia types three and one (SCA3 and SCA1) and Huntington's disease (HD). VO659 is designed to target the CAG repeat expansion that causes these polyglutamine diseases. It is the only ASO in clinical development that targets the underlying cause of all nine known polyglutamine diseases.

The company's platform designs fit-for-purpose ASOs by applying precision chemistry with mechanisms of action suited to target genetic neurological diseases. VO659 works through a dual mechanism of action, enabling potent target engagement across multiple disease models. In preclinical studies, VO659 demonstrated significant and dose-dependent reductions of mutant huntingtin protein (mHTT) and improvements in motor function in HD mouse models. It also showed allele-preferential reductions of mHTT in HD patient cell models. Similar reductions were observed for mutant ATXN1 and ATXN3 proteins in SCA1 and SCA3 models, respectively.

In April 2023, Vico Therapeutics initiated a Phase 1/2a clinical trial for VO659. This multi-center, open-label basket study is designed to assess the safety and tolerability of multiple ascending doses of VO659 administered intrathecally in participants with early manifest HD or mild to moderate SCA1 or SCA3. The study is expected to enroll approximately 71 participants.