The main active ingredient of Vico is an experimental treatment of AON recurrences of expanded trinucleotides (CAGs) resulting in poisonous and abnormally long sections of polyglutamine (polyQ) in proteins that cause a number of neurodegenerative polyQ disorders such as singoebellar ataxia type 1 (SCA1), type 3 (SCA3). )) and Huntington’s disease (HD).
It has been shown to preferably reduce mutant polyQ grades compared to wild type proteins. This has been demonstrated in several mouse models established for SCA1, SCA3 and Huntington’s disease, with extensive and long-term distribution of the compound in the central nervous system. Based on this encouraging knowledge combined with the broad applicability of the main compound to many other brain disorders with major unmet medical needs, Vico is advancing this program towards a first human trial expected by the end of 2021.