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 – the mutation that causes all polyglutamine diseases
About Us
Unlocking New Possibilities with Precision Genetic Targeting
At Vico Therapeutics, we’re pioneering targeted treatments for devastating neurological disorders. Our innovative antisense oligonucleotide (ASO) platform applies precision chemistry to target the source of these diseases, while carefully preserving essential protein function.
This precision approach brings new hope to patients with Huntington’s disease and spinocerebellar ataxias, conditions that have long awaited effective therapies.
Our Science
VO659: One Target, Multiple Neurodegenerative Diseases
VO659 is an ASO that directly targets the CAG repeat expansion and reduces mutant protein in an allele preferential manner.
Targets the Root Cause of Polyglutamine Diseases
Polyglutamine (polyQ) diseases are a group of devastating neurological disorders that are caused by a mutation producing a CAG repeat expansion. This mutation causes the formation of toxic protein believed to result in disease. VO659 is designed to bind directly to the CAG repeats, sterically block translation and reduce the mutant protein.
A Pipeline-In-A-Drug
The CAG repeat expansion causes nine polyQ diseases, including Huntington’s disease and various forms of spinocerebellar ataxia. By focusing on this common mutation, VO659 has the potential to address multiple polyQ disorders with a single therapy.
Precision Through
Allele-Preferential Targeting
VO659 employs an innovative allele-preferential approach. This precision targeting allows us to reduce the mutant protein while preserving essential normal gene function - a crucial advancement in treating these complex disorders.
Pipeline
We are dedicated to advancing innovative therapies with a focused pipeline of potential first-in-class investigational medicines. These are meticulously designed to address critical needs within Huntington’s disease, SCA1, SCA3, Rett syndrome and familial Alzheimer’s disease, aiming to transform patient outcomes.
Team
Leadership
Micah Mackison
Chief Executive Officer
Nicole Datson, PhD
Chief Scientific Officer
Scott Schobel, MD
Chief Medical Officer
Team
Board of Directors
Luc Dochez, PhD
(Chair and Founder)
Droia Ventures
Martijn Kleijwegt
EQT Life Sciences
Felice Verduyn-Van Weegen
EQT Life Sciences
Remi Droller
Kurma Partners
Jeroen Vangindertael, PhD
Ackerman’s van Haaren
Amy Schulman
Polaris Partners
Klaus Breiner, PhD
Pureos Bioventures
Josh Mandel-Brehm
(Co-Founder)
CAMP4 Therapeutics
Anders Hinsby, PhD
(Independent)
Muna Therapeutics
Micah Mackison
CEO, Vico Therapeutics
Catello Somma
(Board Observer)
Seroba Life Sciences