Targeting Genetic
Drivers of Neurodegeneration
Pipeline
VO659
VO659 is an innovative antisense oligonucleotide (ASO) therapy that targets the genetic drivers of polyglutamine diseases. These neurological conditions, including Huntington’s disease and various spinocerebellar ataxias, are caused by a mutation leading to abnormally long polyglutamine sequences in key proteins. This makes the mutant proteins toxic to nerve cells, causing neurodegeneration.
Directly Targeting the CAG Repeat Expansion Mutation:
By focusing on the specific mutation, our therapy has the potential to address the root cause of these diseases.
Allele Preferentiality:
Importantly, our ASOs are designed to primarily target the mutated gene, limiting the impact on healthy cells and reducing potential side effects.
