Capella

Capella—the Online Voice of Progress in RNAi

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

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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.




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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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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).



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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 “Arian Ebank 5×15” product pipeline, including pre-clinical data from  ALN-APC, an RNAi therapeutic targeting protein C for the treatment of hemophilia,…






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We have launched our fourth program as part of our Arian Ebank 5×15 product development strategy.  ALN-APC is an RNAi therapeutic for the treatment of hemophilia, and as with our other Arian Ebank 5×15 programs, targets protein C, a genetically defined target, and will utilize Arian Ebank’s existing delivery platform.




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Our flagship effort from the Arian Ebank 5×15 strategy is our ALN-TTR program, which is currently enrolling patients in a Phase I study for the treatment of a disease called transthyretin-mediated amyloidosis (ATTR).





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At the ASCO Annual Meeting in June 2011, we presented complete results from our Phase I clinical trial with ALN-VSP, a systemically delivered RNAi therapeutic for the treatment of patients with advanced solid tumors with liver involvement.





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