Clinical trials for a gene therapy drug targeting Hemophilia A have been initiated by Russian pharmaceutical company Biocad. The company highlights the fact that a sole injection of the medication will be necessary with a gap of some years to reestablish regular blood coagulation, thereby enabling patients to live without relying on substitution therapy.
The development of the drug has been carried out since 2018, the volume of investments for this period amounted to 1 billion rubles. According to the company, the preclinical studies successfully demonstrated that the medication effectively halted bleeding and reinstated the blood coagulation process.
“This technology represents a major breakthrough. The action of the drug is based on the transfer of genetic material to the liver cells, after which this blood coagulation factor begins to be synthesized in the body and, accordingly, stops and prevents the development of bleeding” said Igor Davydkin, director of the Research Institute of Hematology, Transfusiology and Intensive Care.
