Taking Aim at Huntington’s Disease and Other Repeat Expansion Disorders

Huntington’s disease is a rare, inherited neurodegenerative disorder caused by a type of genetic mutation known as a trinucleotide repeat expansion, which leads to the production of a toxic protein that causes progressive brain cell loss. Vico Therapeutics is developing an experimental antisense oligonucleotide to treat the condition. Because the therapy targets the repeat expansion itself, rather than a specific gene, it may have applications across a broader set of so-called polyglutamine diseases of which Huntington’s is one. We spoke to Prarthana Khanna, vice president of corporate business development and strategy for Vico, about Huntington's disease, the company’s experimental ASO to target the disease, and why it has the potential to address multiple neurological diseases.