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.