Bringing knowledge, experience and science together to discover, develop and deliver therapies for CNS disorders.
Vico was founded in Q4 2019 by the highly experienced entrepreneurs Luc Dochez and Josh Mandel-Brehm and seasoned scientists Dr. Judith van Deutekom and Dr. Gail Mandel with strong academic reputations and vast industry experience.
Together with a team of skilled scientists and drug development professionals, Vico’s focus is on development of RNA modulating therapies for rare neurological disorders, with initial focus on SCA – Spinocerebellar ataxia, HD – Huntington’s disease and RETT syndrome.
Spinocerebellar ataxias (SCAs) are a subgroup of hereditary ataxias, characterized by autosomal dominant mode of inheritance and progressive ataxia caused by degeneration of the cerebellum and often other connected brain regions. More than 30 types of SCAs have been identified to date (SCA1–SCA36), and the most common SCAs (types 1, 2, 3, 6 and 7) are caused by translated CAG trinucleotide repeat expansions that encode elongated polyglutamine (polyQ) stretches in the respective disease proteins. Presence of the elongated polyQ stretch confers pathogenic properties to the resulting protein, resulting in degeneration of specific neuronal subpopulations that differ between the different SCA types. For SCA1 and SCA3 disease manifestation includes ataxia of gait, stance, and limbs, dysarthria, and oculomotor abnormalities. To date, there are no disease-modifying therapies.
As the different polyQ proteins causing HD and several SCAs act through a dominant gain-of-function mechanism resulting in neurotoxicity, Vico’s lead product is designed to suppress these mutant proteins and slow or halt disease progression. A major strength of Vico’s approach is the broad applicability to different polyQ diseases and the selectivity for mutant proteins with preservation of normal protein function.
Huntington’s disease (HD) is a neurodegenerative disorder with a progressive and fatal course characterized by movement disorders, cognitive impairment, dementia and psychiatric manifestations including depression and psychosis. These symptoms result from the selective death and dysfunction of specific neuronal subpopulations within the central nervous system. The disease causing mutation is the expansion of a CAG-trinucleotide repeat in the coding region of exon 1 of the HTT gene, which results in a mutant huntingtin protein with an elongated polyglutamine (polyQ) stretch at its N-terminus. This expanded polyglutamine stretch confers a toxic gain-of-function to mutant protein forms, ultimately resulting in widespread neuronal death. Besides a toxic gain-of-function of mutant huntingtin, loss of the functions of normal huntingtin likely also plays a role in pathological mechanisms of HD. Today there is still a significant unmet need in disease-modifying therapies for the treatment and management of HD, with drugs that can alleviate some of the movement and psychiatric symptoms, but no curative treatments available.
Rett syndrome (RTT) is a neurodevelopmental disorder primarily affecting girls.
First symptoms include regression of early developmental milestones, such as speech and purposeful hand motions, followed by severe motor abnormalities, including respiration, and premature death, typically by age 40. Boys have an even more profound disease, usually succumbing before 2 years of age.
The underlying genetic defect is a mutation in the gene encoding the global transcriptional regulator, Methyl CpG Binding Protein 2 (MECP2), located on the X chromosome. MECP2 is a global transcriptional regulator and essential for normal nerve development and function. There is no cure for RTT, treatment is directed at improving symptoms.
Our therapeutic strategy is based on antisense oligonucleotide-mediated modulation of RNA to repair mutant MECP2 and reverse symptoms.
Learn, innovate, and develop is what we do.
All our employees have worked most of their professional careers on RNA-modulating therapeutics for rare diseases.
Our passion never dies in the mission to bring a therapy to the patients.