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Fitusiran

Alnylam Pharmaceuticals, Inc.

Drug Names(s): ALN-APC, fitusiran-SAR439774, synthetic double-stranded siRNA oligonucleotide against antithrombin mRNA, ALN-AT3

Description: ALN-AT3 is an RNAi therapeutic for the treatment of hemophilia. Utilizing our proprietary GalNAc-siRNA conjugate delivery approach, ALN-AT3 is a completely new approach which resets the clotting cascade to stop bleeding by silencing antithrombin (AT), an endogenous inhibitor of thrombin generation, an optimal anticoagulant target.

AT, also known as antithrombin III and SERPINC1, is a liver-expressed plasma protein that acts as an important endogenous anticoagulant by inactivating factor Xa and thrombin. AT plays a key role in normal hemostasis, which has evolved to balance the need to control blood loss through clotting with the need to prevent pathologic thrombosis through anticoagulation.

Deal Structure: Genzyme and Alnylam
In January 2014, Genzyme, a Sanofi company, and Alnylam Pharmaceuticals announced that they have significantly expanded their strategic agreement to develop and commercialize treatments for rare genetic diseases. Genzyme will have significant rights to Alnylams portfolio of clinical and pre-clinical stage drug candidates. Alnylam will retain most product rights in North America and Western Europe, and will have significantly expanded development and commercial opportunities for its genetic medicine pipeline through Genzymes established global infrastructure in rare diseases.

The expanded relationship between Genzyme and Alnylam includes the following components:

First, Genzyme will obtain expanded rights to patisiran. Under the original agreement from 2012, Genzyme had rights to commercialize patisiran in Japan and the broader Asia-Pacific region. This disease has a disproportionately high prevalence in these territories. Under the...See full deal structure in Biomedtracker

Partners: Sanofi


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