Alnylam Pharmaceuticals Presents New Data From Its Conjugate Delivery Platform, Enabling Subcutaneous Administration of RNAi Therapeutics With Wide Therapeutic Index
Published: Aug 06, 2012
CAMBRIDGE, Mass.--(BUSINESS WIRE)--Alnylam Pharmaceuticals, Inc.(Nasdaq: ALNY), a leading RNAi therapeutics company, is presenting new data today from its GalNAc-conjugate siRNA programs for the systemic delivery of RNAi therapeutics at the XX International Roundtable on Nucleosides, Nucleotides and Nucleic Acids being held August 5-9 in Montreal, Canada. GalNAc-conjugate siRNA are designed to achieve targeted delivery of RNAi therapeutics to hepatocyte cells of the liver through uptake by the asialoglycoprotein receptor. The research findings demonstrate potent and durable target gene silencing with subcutaneously administered GalNAc-conjugate siRNA from Alnylam’s ALN-TTRsc and ALN-AT3 pre-clinical programs. In addition, extensive safety evaluation studies demonstrate a wide therapeutic index for GalNAc-conjugate siRNA. ALN-TTRsc is an RNAi therapeutic targeting transthyretin (TTR) for the treatment of transthyretin-mediated amyloidosis (ATTR), and ALN-AT3 is an RNAi therapeutic targeting antithrombin (AT) for the treatment of hemophilia; both are key programs in the ‘Alnylam 5x15’ product development strategy.