Alnylam Pharmaceuticals announced today that it is advancing its Development Candidate for ALN-AS1 for the treatment of hepatic porphyrias including acute intermittent porphyria. The new pre-clinical research findings show that subcutaneous administration of a GalNAc-siRNA targeting ALAS-1 leads to rapid, dose-dependent, and long-lasting knockdown of ALAS-1 mRNA and complete inhibition of the toxic intermediates that mediate the symptoms and pathology of AIP. Based on these findings, including results in non-human primate studies, the company has selected its ALN-AS1 Development Candidate and expects to file an Investigational New Drug application for this RNAi therapeutic in 2014. ALN-AS1 is part of the company’s “Alnylam 5x15” product development and commercialization strategy, in which the company aims to advance five genetic disease target programs into clinical development, including programs in late stages, by the end of 2015. In addition, the company presented new pre-clinical data with its proprietary, clinically validated GalNAc-siRNA conjugate delivery platform for subcutaneous delivery of RNAi therapeutics with a wide therapeutic index.
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