Oligonucleotide based RNA modulating therapeutics

VICO Therapeutics is at the forefront of RNA modulation, specifically utilizing antisense oligonucleotides to address genetic neurological disorders. Their lead product, VO659, is designed to target CAG repeat expansions associated with polyglutamine diseases such as Spinocerebellar Ataxia (SCA) types 1 and 3, and Huntington’s disease. By preferentially binding to the expanded CAG repeats in mutant mRNA transcripts, VO659 inhibits the translation of toxic proteins, thereby aiming to reduce their levels and potentially halt or reverse disease progression. Preclinical studies have demonstrated significant reductions in mutant protein levels and improvements in motor function in relevant mouse models, highlighting the therapeutic potential of this approach.

The VICOMER™ platform further enhances VICO's capabilities in RNA modulation by offering multiple mechanisms of action, including translation inhibition, splicing modulation, RNA editing, and degradation. This platform is built on precision chemistry that allows for the optimization of efficacy and safety without making permanent changes to the DNA code. VICO's approach is disease-first, selecting the most appropriate RNA modulation strategy to correct specific genetic defects. Their innovative use of antisense oligonucleotides, which are biodegradable and highly sequence-specific, positions them as a leader in developing targeted therapies for complex genetic disorders.

Netherlands

Vico Therapeutics is committed to advancing RNA modulating therapies for patients with severe, genetic neurological disorders. Vico is initially targeting spinocerebellar ataxia types 3 and 1 and Huntington’s disease. Our VICOMER platform designs antisense oligonucleotides for various genetic defects by modulating or editing RNA.

VICO Therapeutics

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