Alpha-1 Antitrypsin Deficiency

We presented clinical and non-clinical data in a series of posters and oral presentations at the 12th Annual Meeting of the Oligonucleotide Therapeutics Society (OTS), held September 25 - 28, 2016 in Montreal, Quebec. [spotlight-link icon="release" href="http://investors.alnylam.com/releasedetail.cfm?ReleaseID=991334" type=" "]Read our press release[/spotlight-link] [spotlight-link icon="presentation" href="http://www.alnylam.com/web/assets/OTS_ALN-AAT_Phase-1_092816_Final2.pdf" type="(670 KB PDF)"]View the ALN-AAT Phase 1/2 presentation[/spotlight-link] [spotlight-link icon="presentation" href="http://www.alnylam.com/web/assets/GalNAc-siRNA_Nonclinical_Review_092816.pdf" type="(2 MB PDF)"]View the poster on review of non-clinical safety data for GalNAc-siRNAs[/spotlight-link] [spotlight-link icon="presentation" href="http://www.alnylam.com/web/assets/GalNAc-siRNA_vs_ASO_092816.pdf" type="(2 MB PDF)"]View the poster on GalNAc-siRNA vs. GalNAc-ASO[/spotlight-link] [spotlight-link icon="presentation" href="http://www.alnylam.com/web/assets/Cholesterol-siRNA_Muscle_Delivery_092816.pdf" type="(620 KB PDF)"]View the poster on cholesterol conjugates for muscle delivery[/spotlight-link]  

We presented pre-clinical data on ALN-AAT, an investigational RNAi therapeutic targeting alpha-1 antitrypsin (AAT) for the treatment of AAT deficiency-associated liver disease. New data, presented at the Digestive Disease Week (DDW) meeting, held May 16 – 19, 2015, showed a robust knockdown of serum AAT of up to 93% in non-human primates (NHPs) with monthly subcutaneous dosing. This level of knockdown was highly durable, lasting for greater than 30 days following the final dose. Further, ALN-AAT was found to have a wide therapeutic index based on results from GLP toxicology studies. In addition, study results were reported from a transgenic mouse model of alpha-1 liver disease, where mice overexpress the human Z-AAT protein. [spotlight-link icon="release" href="http://investors.alnylam.com/releasedetail.cfm?ReleaseID=913507" type=" "] Read our press release[/spotlight-link] [spotlight-link icon="presentation" href="http://www.alnylam.com/web/assets/ALN-AAT_DDW_051715.pdf" type="(7 MB PDF)"] View the presentation [/spotlight-link]

We presented new pre-clinical data supporting the advancement of a Development Candidate (DC) for ALN-AAT, an RNAi therapeutic targeting alpha-1 antitrypsin (AAT) for the treatment of AAT deficiency-associated liver disease. Data were presented in a Late-Breaking Abstract Session at Digestive Disease Week (DDW), held May 3 – 6, 2014 in Chicago. As previously presented at the 64th Annual Meeting of the American Association for the Study of Liver Diseases (AASLD, “The Liver Meeting”) in November 2013 and as updated at DDW, studies were performed in transgenic mice overexpressing the human Z-AAT protein. Subcutaneous administration of a GalNAc-siRNA targeting AAT led to rapid, potent, dose-dependent, and durable knockdown of AAT of greater than 95%, 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=845522" type=" "] Read our press release[/spotlight-link] [spotlight-link icon="presentation" href="http://www.alnylam.com/web/assets/Alnylam-ALN-AATprogram-DDW-May2014.pdf" type="(1.4 MB PDF)"] View our presentation [/spotlight-link]

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]

At the Liver Meeting held this week in Boston, our scientists presented pre-clinical data from our ALN-AAT program for the treatment of liver disease associated with alpha-1 antitrypsin (AAT) deficiency. AAT deficiency is a rare, genetic condition characterized by misfolding of mutant AAT (“Z-AAT”) that causes lung and liver disease. [spotlight-link icon="presentation" href="http://www.alnylam.com/web/assets/ALNY-AASLD-AAT-Nov20121.pdf" type="(2.2 MB PDF)"]View our presentation[/spotlight-link] [spotlight-link icon="presentation" href="http://www.alnylam.com/web/assets/ALNY-AASLD-FibrosisPosterXPanel-Nov2012.pdf" type="(2.8 MB PDF)"]View our poster[/spotlight-link] [spotlight-link icon="release" href="http://investors.alnylam.com/releasedetail.cfm?ReleaseID=734084" type=" "]Read our press release[/spotlight-link]

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