Capella—the Online Voice of Progress in RNAi

Alnylam welcomes you to Capella, the destination for updates on our progress in translating the science of RNAi into innovative medicines. For Alnylam, Capella is our online voice for communicating the scientific progress we are making as we work to develop innovative medicines for patients.

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.


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 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.


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 in our ‘Alnylam 5×15’ efforts with ALN-TTRsc for the treatment of ATTR, ALN-AT3 for the treatment of hemophilia, and potentially future programs.

At the Boston University School of Medicine, our scientists presented positive clinical results from our Phase I trial with ALN-TTR02 for the treatment of transthyretin-mediated amyloidosis (ATTR).  These new data show that we have achieved very robust knockdown of TTR, the disease causing protein, including up to 94% reduction of serum TTR, and a nearly 80% level of suppression sustained at one month with just a single dose.  ALN-TTR02 was found to be generally safe and well tolerated.

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.


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.


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.


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%.



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