Capella

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.

To view our corporate presentation, click here.

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




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Our flagship effort from the Alnylam 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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Alnylam scientists and collaborators presented new pre-clinical data from our ALN-TTR program at the XII International Symposium on Amyloidosis in Rome on April 18 – 21, 2010. These data demonstrated, for the first time, that treatment with an RNAi therapeutic can result in regression of pre-existing pathogenic TTR deposits in peripheral tissues.




 

The new pre-clinical studies, performed in collaboration with Dr. Maria Saraiva at the Institute for Molecular and Cellular Biology in Portugal, used a transgenic mouse model where the mutant human V30M TTR gene is over-expressed. Specifically, the new data demonstrated that administration of ALN-TTR01 in mature transgenic mice resulted in the regression of existing pathogenic mutant human V30M TTR deposits in tissues, including: dorsal root ganglia, sciatic nerve, stomach, and intestines. These effects in diseased tissues were mediated through silencing of the mutant human TTR expressed in the liver, resulting in regression of established peripheral TTR deposits. ALN-TTR01 administration resulted in a greater than 90% regression of measurable TTR deposits as compared with control-treated animals.

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