Alnylam Pharmaceuticals, Inc. ALNY, a leading RNAi therapeutics
company, announced today that it is advancing its Development Candidate for
ALN-AS1, an RNAi therapeutic targeting aminolevulinate synthase-1 (ALAS-1) for
the treatment of hepatic porphyrias including acute intermittent porphyria
(AIP). The new pre-clinical research findings, presented at the 9^th Annual
Meeting of the Oligonucleotide Therapeutics Society being held October 6 – 8,
2013 in Naples, Italy, 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 (IND) 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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