Scott Schobel, MD, Chief Medical Officer at Vico Therapeutics, discusses current treatment options for Huntington’s disease and spinocerebellar ataxia (SCA1 and SCA3).
Huntington’s disease is a rare neurodegenerative disorder characterized by choreatic movements, behavioral and psychiatric disturbances, and dementia. Manifestation of this disorder typically occurs between 30 and 50 years of age, with reported earlier cases The gene involved is the huntingtin gene in which a CAG expansion occurs that results in neuronal damage, especially in the basal ganglia.
Spinocerebellar ataxia is an inherited, progressive, neurodegenerative disease that mainly affects the cerebellum. To date, over 40 genetic SCAs have been classified, including SCA1 and SCA3. Both SCA1 and SCA3 have a CAG expansion associated with their pathophysiologies.
In the interview, Dr. Schobel highlighted the limited availability of treatment options for both Huntington’s disease and SCA. Vico Therapeutics is developing treatments that selectively target expanded CAG repeats in mutant mRNA, inhibiting mRNA translation. This thereby leads to the reduction of mutant protein in those two conditions. The therapy in development, VO659, is an antisense oligonucleotide investigational therapy designed to target the CAG repeat expansion that causes all nine known polyglutamine diseases including Huntington’s disease, SCA1, and SCA3.
To learn more about these and other neurological conditions, visit checkrare.com/diseases/neurology-nervous-system-diseases/