Russia is actively developing the first gene drug for hemophilia B. During the first and second phases of clinical trials, scientists found that most participants experienced an increase in the activity of blood coagulation factor IX, sufficient for the complete absence of bleeding.
The head of the Department of Hematology and Hemostasis Disorders of the National Medical Research Center for Hematology of the Ministry of Health, Nadezhda Zozulya, noted that the first patients received the investigational drug a year ago through a single intravenous administration.
And during this year, most of them no longer had to undergo replacement therapy. I think this is a great result.
According to her, the Ministry of Health has already issued permission for a phase III clinical trial. Scientists will evaluate the safety and efficacy of the investigational drug at the selected dose for at least five years after receiving therapy.
Hemophilia B is a hereditary disease associated with impaired blood clotting, causing frequent bleeding and bruising due to a mutation in the gene encoding factor IX.
Currently, people with hemophilia use intravenous replacement therapy drugs two to four times a week to maintain the activity of the deficient blood coagulation factor at no less than 1–3%. This therapy requires lifelong use.
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