Pre-Clinical Research Findings

Pre-Clinical Research Findings

Alnylam Pharmaceuticals, Inc., a leading RNAi (gene silencing) therapeutics company, and Medtronic, Inc the world’s leading developer of medical devices, announced the presentation of new pre-clinical research findings from their Huntington disease program at the 2009 World Congress on Huntington’s Disease held September 12-15, 2009 in Vancouver, British Columbia. Together, the two companies are developing a drug-device combination for the treatment of Huntington’s disease. The drug, ALN-HTT, consists of an RNAi therapeutic targeting huntingtin, the gene responsible for Huntington’s disease, and is delivered to the central nervous system (CNS) using an implantable pump infusion system developed by Medtronic.

The newly presented pre-clinical research, performed in collaboration with Professor Don M. Gash’s laboratory at the University of Kentucky College of Medicine, demonstrate that a small interfering RNA (siRNA) targeting the huntingtin gene achieves broad distribution in the CNS following continuous direct CNS administration. Further, direct delivery to the CNS resulted in robust silencing of the huntingtin gene mRNA, extending previously presented pre-clinical data. This silencing effect was achieved at substantial distances from the infusion site, an important step towards translating this delivery approach from animal models to the larger human brain. Additional pre-clinical studies showed ALN-HTT to be well tolerated following continuous direct CNS administration over a period of approximately one month.