Fitusiran: what if we rebalanced coagulation?

By Ana Espino | Published on April 17, 2026 | 4min read


Hemophilia is a genetic bleeding disorder caused by a deficiency in factor VIII (hemophilia A) or factor IX (hemophilia B), leading to spontaneous or prolonged bleeding. It primarily affects males due to its X-linked inheritance and represents a chronic condition that significantly impacts quality of life.

Current treatments are mainly based on replacement therapy with coagulation factors, administered either on demand or prophylactically. Although effective, these treatments have several major limitations: high cost, frequent intravenous injections that are burdensome for patients, and the development of neutralizing antibodies (inhibitors) that can reduce their effectiveness.