About Huntington Disease
Huntington’s disease is an autosomal dominant disorder, characterized by chorea (jerky involuntary movements especially affecting the shoulders, hips, and face), psychiatric illness, and cognitive decline. HD affects over 30,000 patients in the U.S. and 83,000-130,000 globally.
Because mutations in the Huntingtin gene (HTT) are causative, gene-silencing siRNA therapeutics should be disease-modifying, as has been established in HD mouse models. Intrathecal injection of ASO’s have been shown to lower the disease-causing Huntingtin protein in patients but less invasive intravenous or subcutaneous administration is precluded by the BBB. Ophidion’s OCCT-HTT siRNA therefore represents a first-in-class non-invasive HTT gene silencing approach which would be developed for intravenous use via the orphan disease designation (ODD) pathway.