We presented new pre-clinical data on ALN-AAT, an RNAi therapeutic targeting alpha-1 antitrypsin (AAT) in development
for the treatment of liver disease associated with AAT deficiency. These data were presented at the 64th
Annual Meeting of the American Association for the Study of Liver Diseases (AASLD, “The Liver Meeting”) held November 1 – 5, 2013. In a transgenic mouse model of Z-AAT protein over-expression, subcutaneous administration of a GalNAc-siRNA targeting AAT led to rapid, potent, dose-dependent, and durable knockdown of AAT of greater than 90%, as well as a significant reduction in fibrosis and the incidence of liver tumors.
[spotlight-link icon="release" href="http://investors.alnylam.com/releasedetail.cfm?ReleaseID=803892" type=" "] Read our press release[/spotlight-link]
[spotlight-link icon="presentation" href="http://www.alnylam.com/web/assets/ALNY-LiverMeeting-AATProgram-PosterXPanel-Nov2013.pdf" type="(1.3 MB PDF)"] View our poster [/spotlight-link]