New data from our conjugate delivery platform was presented at the XX International Roundtable on Nucleosides, Nucleotides and Nucleic Acids held in August 2012.  This delivery platform enables subcutaneous dose administration of RNAi therapeutics with a very wide therapeutic index.  We are integrating this approach...

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]

Today, scientists presented final results from our Phase I clinical trial of ALN-TTRo1 at the XIII International Symposium on Amyloidosis held in Groningen. Data from this study show that administration of ALN-TTR01 resulted in statistically significant reductions in serum TTR protein levels, including both wild-type and mutant TTR protein, in ATTR patients.  Knockdown of TTR, the disease-causing protein, was found to be dose dependent, rapid, and durable after just a single dose. [spotlight-link icon="presentation" href="" type="(842 KB PDF)"]View our  presentation[/spotlight-link] [spotlight-link icon="release" href="" type=" "]View our  release[/spotlight-link] [spotlight-link icon="podcast" href="" type="(27.7MB MP3)"]Listen to KOL, Dr. Hawkins discuss the ALN-TTR program (recorded Jan. 2012)[/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 presented an update on our RNAi delivery strategies at the AsiaTIDES conference this week.  The presentation focuses on our recent progress utilizing conjugates and lipid nanoparticles for systemic adminstration of RNAi therapeutics. [spotlight-link icon="presentation" href="" type="(2.4MB PDF)"]View our  presentation[/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]

In September 2011, we and our collaborators presented new data at the 7th Annual Meeting of the Oligonucleotide Therapeutics Society demonstrating substantial new advances in our efforts around systemic delivery of RNAi therapeutics. [spotlight-link icon="presentation" href="" type="(2.1 MB PPT)"]View the LNP Poster[/spotlight-link] [spotlight-link icon="presentation" href="" type="(3.1 MB PPT)"]View the Conjugate Poster[/spotlight-link] [spotlight-link icon="release" href="" type=" "]Read our press release[/spotlight-link]


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