Huntington's Disease

ALN-HTT: Huntington's Disease

ALN-HTT, an RNAi therapeutic for the treatment of Huntington's disease, is designed to silence the huntingtin gene, which is the cause of Huntington's when expressed as a toxic mutated protein.

In pre-clinical studies, ALN-HTT was well tolerated following administration to the brain and was shown to silence the huntingtin gene. Silencing the huntingtin gene also translated into a therapeutic effect in animal models, including improvement in motor behavior, which is a hallmark of this debilitating and fatal disease. The RNAi therapeutic reduced expression of mutant huntingtin in the brain and sustained a benefit in motor behavior for at least one week. In preliminary studies, the RNAi therapeutic was found to be well tolerated in the brain after direct CNS administration.

ALN-HTT is being developed in collaboration with Medtronic and CHDI Foundation. ALN-HTT is being developed for delivery to the central nervous system (CNS) using an implantable infusion system developed by Medtronic. CHDI is a not-for-profit virtual biotech company that is exclusively dedicated to rapidly discovering and developing therapies that slow the progression of Huntington's disease.

ALN-HTT Clinical Timeline