Alnylam Pharmaceuticals, Inc. has announced the publication of a key study in the Proceedings of the National Academy of Sciences (PNAS) by Alnylam scientists and collaborators from the University of Massachusetts Medical School and Massachusetts General Hospital.
Results from the new research study demonstrate that chemically synthesized small interfering RNAs (siRNAs), the molecules that mediate RNAi, targeting the gene responsible for Huntington’s disease provide a therapeutic benefit in an animal model of the human disease.
“We are very encouraged by the findings published today and feel that they contribute to a growing body of work representing a strong rationale for the advancement of an RNAi therapeutic for the treatment of Huntington’s disease,” said Dinah Sah, Ph.D., Senior Director of Research at Alnylam.
“We believe these results reflect the progress that Alnylam and our collaborators are making in developing this promising therapeutic modality for a major neurodegenerative disease,” Sah added.
The new pre-clinical study showed that a single injection of an siRNA targeting huntingtin, the gene responsible for Huntington’s disease, resulted in improved symptoms of disease in an animal model. These improved effects included reduction in neuronal pathology and an improvement in motor behavior.
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.
“I am excited by these new data as there is a very significant need for novel therapeutics to treat patients with Huntington’s disease. Indeed, patients afflicted with this genetic disease are otherwise destined to an irreversible deterioration of neuronal function,” said Neil Aronin, M.D., Professor at the University of Massachusetts Medical School.
Mr. Aronin continued, “While more work is needed to advance this approach, the mechanism of action for RNAi therapeutics defines a promising therapeutic strategy that could slow or halt disease progression.”