Alnylam Pharmaceuticals and partner Medtronic said this week that preclinical data indicates that an siRNA targeting the huntingtin gene, which is mutated in Huntington's disease, can be broadly distributed to the central nervous system when administered directly and continuously.
In 2005, the companies forged an alliance to develop RNAi-based therapies delivered using Medtronic's medical devices (see RNAi News, 2/11/2005). Among the indications being pursued under the partnership is Huntington's disease, an indication that Alnylam formally added to its pipeline in early 2008 (see RNAi News, 1/10/2008).
According to the companies, the newly released preclinical data also shows that direct delivery of huntingtin-targeted siRNAs induced "robust silencing" of the huntingtin mRNA, with the effect observed at "substantial distances from the infusion site."
Additional preclinical experiments showed that Alnylam's Huntington's disease drug ALN-HTT is well tolerated when delivered via continuous, direct infusion over a period of about one month.
"In aggregate, these new studies — which were performed in collaboration with the University of Kentucky, one of the leading academic institutions for research on direct CNS delivery for neurodegenerative diseases — support our continued and combined efforts with Medtronic to advance this important innovation to patients," Alnylam Vice President of Research, CNS, and Oncology Dinah Sah said in a statement.