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Alnylam Names Hemophilia Drug as Newest Pipeline Candidate


Alnylam Pharmaceuticals announced this week that it has added ALN-APC, an siRNA-based treatment for hemophilia, to its formal drug-development pipeline.

Earlier this year, Alnylam CEO John Maraganore said that the company would select two new drug programs before the end of the year, and was considering several diseases, including thalassemia, hemophilia, and hereditary angioedema (GSN 5/12/2011).

The newest program targets protein C, a natural anticoagulant pathway. The drug candidate is designed to boost levels of thrombin generation and reduce frequency of bleeding in hemophiliacs.

“With the opportunity to measure clinically meaningful biomarkers in a phase I trial [of ALN-APC], we expect to achieve early human data followed by execution on what we believe are clear and rapid development and commercialization paths,” Alnylam CMO Akshay Vaishnaw said in a statement. “We anticipate advancing this program into clinical development in 2013."

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