Beta-Thalassemia

On December 12, we hosted an R&D Day in New York City. Alnylam management and key opinion leaders discussed the latest progress as well as plans for the future development of our RNAi therapeutics pipeline. At this event, we announced our pipeline growth strategy for development and commercialization of RNAi therapeutics across three Strategic Therapeutic Areas (STArs): Genetic Medicines, Cardio-metabolic Disease, and Hepatic Infectious Disease. [spotlight-link icon="podcast" href="http://edge.media-server.com/m/p/njherf95" type=" "] Listen to the webcast replay[/spotlight-link] [spotlight-link icon="presentation" href="http://www.alnylam.com/web/assets/MASTER-RD-DAY-DECK_Capella.pdf" type="(12.3 MB PDF)"] View the complete presentation[/spotlight-link]

We presented new data from multiple clinical and pre-clinical studies at the 10th Annual Meeting of the Oligonucleotide Therapeutics Society (OTS), held October 12 – 15, 2014 in San Diego.  Among multiple presentations, we presented additional data from our Phase 1 trial with ALN-TTRsc showing...

We presented pre-clinical data from three programs within our “Alnylam 5x15” RNAi therapeutic pipeline: ALN-AT3 for the treatment of hemophilia and rare bleeding disorders (RBD), ALN-CC5 for the treatment of complement-mediated diseases, and ALN-TMP for the treatment of β-thalassemia and iron overload disorders. These data were presented at the 55th Annual Meeting of the American Society of Hematology (ASH) held December 7 – 10 in New Orleans. [spotlight-link icon="release" href="http://investors.alnylam.com/releasedetail.cfm?ReleaseID=812683" type=" "]Read our ALN-AT3 press release[/spotlight-link] [spotlight-link icon="release" href="http://investors.alnylam.com/releasedetail.cfm?ReleaseID=812394" type=" "]Read our ALN-CC5/ALN-TMP 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="http://www.alnylam.com/web/assets/ALNY-Keystone-smallRNA-2012-cERD-Poster.pdf" type="(1.18 MB PDF)"]View our  poster[/spotlight-link] [spotlight-link icon="release" href="http://investors.alnylam.com/releasedetail.cfm?ReleaseID=734324" 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="http://www.alnylam.com/web/assets/HPN-ALNY-ASH2011-Anemia.pdf" type="(680 KB PPT)"]View the refractory anemia presentation[/spotlight-link] [spotlight-link icon="presentation" href="http://www.alnylam.com/web/assets/APC-ALNY-ASHDec2011.pdf" type="(1.88 MB PDF)"]View the hemophilia poster[/spotlight-link] [spotlight-link icon="presentation" href="http://www.alnylam.com/web/assets/TMPRSS6-ALNY-ASHDec2011.pdf" type="(112 KB PDF)"]View the beta-thalassemia poster [/spotlight-link] [spotlight-link icon="presentation" href="http://www.alnylam.com/web/assets/EGLN-ALNY-ASHDec2011.pdf" type="(435 KB PDF)"]View the erythropoiesis poster[/spotlight-link] [spotlight-link icon="release" href="http://investors.alnylam.com/releasedetail.cfm?ReleaseID=734336" type=" "]Read our press release[/spotlight-link]

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