Collaboration to Develop New Drug for Parkinson's

The formation of a research collaboration to apply proprietary RNAi-based therapeutic technology to develop a new drug for Parkinson's disease has been announced by Alnylam Pharmaceuticals (Cambridge, MA, USA) and the Mayo Clinic (Rochester, MN, USA).

Specifically, the collaboration will seek to develop a drug that suppresses the expression of the alpha-synuclein gene, found to be overexpressed in Parkinson's patients. Recently, Mayo Clinic researchers have found that simple overexpression of normal, wild-type apha-synuclein is sufficient to cause disease in a family with multiple affected members. Alpha-synuclein is found in various body tissues, primarily in the brain, where it may play a role in synaptic vesicle recycling.

Under the terms of the research agreement, Alnylam will identify, synthesize, and provide RNAi-based drug compounds targeted to alpha-synuclein gene expression. Mayo Clinic will test and select the RNAi compounds for their efficacy in a series of in vitro and in vivo studies.

"We anticipate that therapy to reduce alpha-synuclein gene expression will benefit not only Parkinson's disease patients who carry the rare gene mutation but also persons who carry common susceptibility variants of the gene, or persons who aggregate the alpha-synuclein protein via other genetic and nongenetic mechanisms,” said Demetrius Maraganore, M.D., professor of neurology at Mayo Clinic.





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