Hemophilia

We presented new pre-clinical with ALN-AT3, an RNAi therapeutic targeting antithrombin (AT) for the treatment of hemophilia and rare bleeding disorders (RBD) at the XXIV Congress of the International Society of Thrombosis and Haemostasis (ISTH) being held June 29 – July 4. These data demonstrate that ALN-AT3 can normalize thrombin generation and improve hemostasis in hemophilia mice and fully correct thrombin generation in a non-human primate (NHP) hemophilia “inhibitor” model. [spotlight-link icon="presentation" href="http://www.alnylam.com/web/assets/ALN-AT3-preclin-ISTH-July2013.pdf" type="(900 KB PDF)"] View our presentation [/spotlight-link] [spotlight-link icon="release" href="http://investors.alnylam.com/releasedetail.cfm?ReleaseID=774899" type=" "] Read our press release[/spotlight-link]

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="http://www.alnylam.com/web/assets/ALNY-ASH-AT3posterXpanel-Dec2012-r.pdf" type="(2.3 MB PDF)"] View our poster [/spotlight-link] [spotlight-link icon="release" href="http://investors.alnylam.com/releasedetail.cfm?ReleaseID=734078"  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="http://www.alnylam.com/web/assets/ALNY-WFH2012-AT3-Poster-PPT.pdf" type="(774 KB PDF)"]View our poster (slide format)[/spotlight-link] [spotlight-link icon="presentation" href="http://www.alnylam.com/web/assets/ALNY-WFH-AT3poster-July2012.pdf" type="(3.3 MB PDF)"]View our poster[/spotlight-link] [spotlight-link icon="release" href="http://investors.alnylam.com/releasedetail.cfm?ReleaseID=734078" type=" "]View our  release[/spotlight-link]

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