05.03.2024
The Clinical and Diagnostic Department of Hematology and Hemostasis Disorders of the NMRC for Hematology of the Ministry of Health of the Russian Federation is conducting a clinical trial of the ANB-002-1/ SAFRAN gene therapy drug in patients with severe hemophilia B. The drug was developed by the Russian biotechnology company BIOCAD.The studied drug is an adeno-associated viral vector (delivery system) containing the blood coagulation factor IX gene. The mechanism of action consists in delivering a functional copy of the gene to the cells of the patient's body. When administered intravenously, recombinant vectors containing the coagulation factor IX gene penetrate liver cells. This allows the body to independently produce factor IX, which is necessary for normal blood clotting.
The drug is administered by a single long-term intravenous infusion. It is assumed that the studied gene therapy drug will be administered once.
In the world of science, the development of gene therapy began in the 1980s. The first gene therapy drug for the treatment of hemophilia was registered in Europe in August 2022.