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ARTICLE | Emerging Company Profile

Prolonging thrombin production

How ApcinteX’s modified serpin could treat both hemophilia A and B

June 2, 2017 5:34 PM UTC

ApcinteX Ltd. is betting that inhibiting an endogenous brake on thrombin production will generate a therapy for hemophilia A and B that can be used by patients who have developed neutralizing antibodies to factor replacement therapies and those who have pre-existing immunity to gene therapy vectors. And because the target is active only at injury sites, it should be safer than other alternatives to factor replacement now in the clinic.

Hemophilia is caused by mutations in the coagulation enzymes Factor VIII or Factor IX, which work together to increase thrombin levels and stop bleeding. Current prophylaxis entails factor replacement therapy. But about 15-20% of hemophilia A patients and 2-5% of hemophilia B patients develop neutralizing antibodies to the replacement factor, according to National Hemophilia Foundation data. ...

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Activated protein C