Today we are presenting new data from our ALN-AT3 program for the treatment of hemophilia and other bleeding disorders at the 54th American Society of Hematology (ASH) Annual Meeting in Atlanta. ALN-AT3 comprises part of our “Alnylam 5x15™” strategy, which is aimed at bringing innovative medicines to patients, with a focus on RNAi therapeutics toward genetically defined targets for diseases with very high unmet medical need. [spotlight-link icon="presentation" href="" type="(2.3 MB PDF)"] View our poster [/spotlight-link] [spotlight-link icon="release" href=""  type=" "] Read our press release[/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="" type="(2.2 MB PDF)"]View our presentation[/spotlight-link] [spotlight-link icon="presentation" href="" type="(2.8 MB PDF)"]View our poster[/spotlight-link] [spotlight-link icon="release" href="" type=" "]Read our press release[/spotlight-link]

At the 8th Annual Meeting of the Oligonucleotide Therapeutics Society, held in Boston October 28-31, 2012, we presented exciting data covering several of the company’s therapeutic programs and delivery platforms. THERAPEUTIC PROGRAMS Therapeutic research highlighted recent clinical and pre-clinical work from our programs in transthyretin-mediated amyloidosis (TTR), hemophilia, hypercholesterolemia, and liver cancer. Click here for presentations.

At the World Federation of Hemophilia World Congress in July 2012, scientists presented pre-clinical data with ALN-AT3, our development candidate targeting antithrombin (AT) for the treatment of hemophilia.  The new data demonstrate potent and durable AT knockdown with subcutaneous administration of ALN-AT3.  ALN-AT3 was shown to improve thrombin generation in hemophilia animal models. [spotlight-link icon="presentation" href="" type="(774 KB PDF)"]View our poster (slide format)[/spotlight-link] [spotlight-link icon="presentation" href="" type="(3.3 MB PDF)"]View our poster[/spotlight-link] [spotlight-link icon="release" href="" type=" "]View our  release[/spotlight-link]

At the annual ASCO meeting in June 2012, we presented data from our ALN-VSP Phase I extension study. Overall, the results demonstrated disease control lasting more than six months in the majority of patients treated on the extension study, including a complete response (CR) in an endometrial cancer patient who had multiple liver metastases. [spotlight-link icon="presentation" href="" type="(3.72 MB PDF)"]View our poster[/spotlight-link] [spotlight-link icon="release" href="" type=" "]View our  release[/spotlight-link]

Yesterday, scientists presented positive results from our Phase I clinical trial of ALN-PCS at the American Heart Association’s Arteriosclerosis, Thrombosis and Vascular Biology 2012 Scientific Sessions held in Chicago. Results showed that administration of a single dose of ALN-PCS, in the absence of concomitant lipid-lowering agents such as statins, resulted in statistically significant and durable reductions of PCSK9 plasma levels of up to 84% and lowering of LDL-C of up to 50%. [spotlight-link icon="presentation" href="" type="(572 KB PDF)"]View our  presentation[/spotlight-link] [spotlight-link icon="release" href="" type="(572 KB PDF)"]Read our press release[/spotlight-link]

Our scientists discovered a new method for monitoring RNAi activity in blood samples.  The results were presented at the Keystone Gene Silencing by small RNAs symposium (Vancouver, February 7-12).  This new method, circulating extracellular RNA detection (cERD), allows quantitative measurement of tissue-specific mRNA silencing from biological fluids including blood and cerebrospinal fluid (CSF). [spotlight-link icon="presentation" href="" type="(1.18 MB PDF)"]View our  poster[/spotlight-link] [spotlight-link icon="release" href="" type=""]Read our press release[/spotlight-link]

In December 2011, our scientists presented multiple posters and presentations at the 53rd American Society of Hematology (ASH) Annual Meeting and Exposition held in San Diego, CA.  We shared data from two programs within our “Alnylam 5x15” product pipeline, including pre-clinical data from  ALN-APC, an RNAi therapeutic targeting protein C for the treatment of hemophilia,... [spotlight-link icon="presentation" href="" type="(680 KB PPT)"]View the refractory anemia presentation[/spotlight-link] [spotlight-link icon="presentation" href="" type="(1.88 MB PDF)"]View the hemophilia poster[/spotlight-link] [spotlight-link icon="presentation" href="" type="(112 KB PDF)"]View the beta-thalassemia poster [/spotlight-link] [spotlight-link icon="presentation" href="" type="(435 KB PDF)"]View the erythropoiesis poster[/spotlight-link] [spotlight-link icon="release" href="" type=" "]Read our press release[/spotlight-link]


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