Alnylam Files to Begin Phase I Trial of Hemophilia Drug | GenomeWeb

Alnylam Pharmaceuticals this week announced that it has filed a clinical trial application with UK regulators to begin human testing of an siRNA-based treatment for hemophilia A and B.

The drug, called ALN-AT3, is designed to silence antithrombin and is administered using the company's so-called GalNAc conjugates, which enable subcutaneous delivery.

Alnylam expects to start a phase I trial of the drug in the UK next year. That study is designed to evaluate single and multiple escalating doses of ALN-AT3 in up to 24 healthy volunteers.

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