Pioneering RNA-based science to target the root cause of rare genetic neurological diseases
VO659: The first clinical candidate directly targeting the CAG repeat expansion mutation that causes all polyglutamine diseases
At Vico Therapeutics, we’re advancing antisense oligonucleotide (ASO) therapies to tackle rare genetic neurological diseases. With deep ASO expertise and a strong commitment to patients, we’re targeting the root causes of complex disorders like Huntington’s disease and spinocerebellar ataxias (SCA).
We’re not just treating symptoms at Vico, we are rewriting the potential for genetic intervention in neurological medicine.
About Us
Unlocking New Possibilities with the Power of Precision Genetic Targeting
Vico Therapeutics is a neurology genetic medicine company pioneering targeted treatments for devastating disorders. Our innovative antisense oligonucleotide (ASO) platform strikes at the root cause of these diseases – the CAG repeat expansion mutation – while carefully preserving essential protein function.
While other treatments for rare neurological diseases focus on symptom management, Vico is the only company working to modify the root cause of the disease. This precision approach brings new hope to patients with Huntington’s disease and spinocerebellar ataxias (SCA), conditions that have long awaited effective therapies.
Our Science
One Therapy, Multiple Targets: Targeting the Genetic Source of Neurodegenerative Disorders
Targets the Root Cause of Polyglutamine Diseases
Polyglutamine (polyQ) diseases are a group of nine devastating neurological disorders, including Huntington’s disease and various forms of spinocerebellar ataxia. While these conditions manifest differently, they share a common cause: CAG repeat expansion mutation in specific genes.
A Unified Approach to
Multiple Diseases
Our treatment targets this shared genetic driver - the CAG repeat expansion mutation itself. By focusing on this common mechanism, we’ve developed a potential therapeutic strategy that could address multiple polyQ disorders with a single approach.
Precision Through
Allele-Preferential Targeting
VO659, our lead candidate, employs an innovative allele-preferential approach. This precision targeting allows us to reduce disease-causing expanded CAG repeat expansion mutation while preserving essential normal gene function - a crucial advancement in treating these complex disorders.
Explore the science
that sets us apart.
Patients and Families
Transformative Therapy for Genetic Neurological Disorders: A Patient-Centered Mission
At the heart of our work lies an unwavering commitment to those most impacted by neurological disorders. We don’t just research treatments—we partner directly with patients, caregivers and advocacy communities to drive meaningful change.
Every breakthrough, every research decision, every therapeutic approach is designed with one fundamental goal: improving the lives of those living with neurodegenerative diseases. We believe true innovation emerges from deep collaboration and understanding.
