We presented new pre-clinical data supporting the selection of the ALN-AS1 Development Candidate for the treatment of hepatic porphyrias, including acute intermittent porphyria (AIP). The new research, presented at the 9th
Annual Meeting of the Oligonucleotide Therapeutics Society, held October 6 – 8, 2013 in Naples, Italy, showed that multi-dose administration of a GalNAc-siRNA targeting ALAS-1 led to rapid, dose-dependent, and long-lasting knockdown of the ALAS-1 mRNA in non-human primates, with an ED50
of approximately 1.25 mg/kg. Further, in a rat model of AIP, ALN-AS1 administration at doses as low as 2.5 mg/kg resulted in a complete blunting of phenobarbital-induced over-production of PBG and ALA, the toxic heme intermediates in AIP.
[spotlight-link icon="release" href="http://investors.alnylam.com/releasedetail.cfm?ReleaseID=795424" type=" "] Read our press release[/spotlight-link]
[spotlight-link icon="presentation" href="http://www.alnylam.com/web/assets/ALNY-ALN-AS1update-OTS2013.pdf" type="(1.1 MB PDF)"] View our ALN-AS1 presentation [/spotlight-link]
[spotlight-link icon="presentation" href="http://www.alnylam.com/web/assets/ALNY-ConjugateUpdate-OTS2013.pdf" type="(1.3 MB PDF)"] View our GalNAc-siRNA conjugate presentation [/spotlight-link]