Rare disease
Huntington's disease is a genetic inherited disorder characterized by progressive motor and cognitive impairment due to neuronal dysfunction and cell death. It is a neurodegenerative disease that leads to progressive motor, cognitive, and psychiatric symptoms.
Unmet need: There's currently no cure for Huntington's disease and no treatments that can stop the symptoms getting worse.
Preclinical evidence: Recent evidence indicates that EGF treatment improves motor behavior and cortical GABAergic function in the R6/2 mouse model of Huntington’s disease (Marottoli et al, Mol Neurobiol Nov 2019).
Our goal: Preclinical evaluation of EGF + xB3 in Huntington’s disease animal models to develop a potential disease-modifying treatment.
Regulatory path: Clear eligibility for orphan drug designation.
