Huntington’s Disease: OCCT-HTT siRNA
Ophidion is developing therapeutic interventions to treat Huntington’s disease (HD). Having demonstrated we can deliver siRNA into the brain for our cognition indications, we are leveraging the power of our platform technology to deliver siRNA to knockdown the expression of Huntingtin protein in the brain. Removing the Huntington’s gene (HTT, both wildtype and mutant) through gene-silencing in the brain has been shown to result in disease modification in animals.
Ophidion is pursuing a first-in-class systemically delivered siRNA gene silencing therapeutic to treat Huntington’s disease. Our preclinical studies for our OCCT-HTT siRNA product have established in-vitro proof of concept in HD, where we have demonstrated knockdown of mRNA levels that could translate to efficacy in HD animal models and humans.
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. Mutations in the huntingtin gene (HTT) is the causative agent of Huntington’s Disease pathology. In the United States, HD affects over 30,000 patients and 83,000-130,000 globally.
Because Huntington’s disease is a monogenic disease, there is great potential for gene silencing therapeutics to be disease modifying agents. Huntington’s disease in mouse models can be ameliorated using therapeutic siRNA, and intrathecal delivery of antisense oligonucleotides have demonstrated the ability to lower the disease causing Huntingtin protein in people; however, delivery via a less invasive route of administration, such as the IV route, is the main barrier for RNAi approaches in larger animals (including humans), where ongoing clinical trials are being conducted using intrathecal route of administration. Ophidion’s OCCT-HTT siRNA represents an orphan drug opportunity where the product can be delivered intravenously.