A Noninvasive Treatment Against Degenerating Brain Cells

Huntington’s Disease: OCCT-HTT siRNA

Ophidion is developing a first-in-class, systemically administered, disease-modifying treatment for Huntington’s disease (HD) that utilizes OCCT- delivered siRNA to suppress synthesis of both normal and mutant Huntingtin protein in the brain, an approach that has shown disease modifying benefit in HD animal models. Ophidion’s preclinical proof-of-concept studies have demonstrated suppression of wild-type and mutant human Huntingtin mRNA levels in several regions of the mouse brain.


Figure 1. Proof of Concept Study in Humanized Transgenic BACHD Mice: Sustained knockdown of 49% demonstrated for our lead formulation.

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.