Scott Schobel, MD, chief medical officer at Vico Therapeutics discussed the latest data testing an antisense therapy to treat Huntington’s disease and spinocerebellar ataxia type 1 (SCA1) and type 3 (SCA3).
Huntington’s disease manifests as a rare neurodegenerative disorder characterized by chorea movements, behavioral and psychiatric disturbances, and dementia. The gene involved is the huntingtin gene in which a CAG expansion occurs that results in neuronal damage, especially in the basal ganglia. SCA1 and SCA3 are rare neurological disorders, mostly involving the cerebellum, and the underlying genetic abnormalities also include CAG expansions.
VO659 represents an investigational antisense oligonucleotide therapy designed to target the CAG repeat expansion. The development of VO659 aims to treat Huntington’s disease, as well as SCA1 and SCA3. Recently, the company announced they have started enrolling patients for a phase 1/2 clinical trial. It is a multi-center, open-label basket study designed to assess the safety and tolerability of multiple ascending doses of VO659 administered intrathecally in participants with early manifest HD or mild to moderate SCA1 or SCA3. Approximately 71 participants are expected to be enrolled in the study.
To learn more about these and other neurological conditions, visit checkrare.com/diseases/neurology-nervous-system-diseases/
