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.

To view our corporate presentation, click here.

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.


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).


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.


Arian Ebank 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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