This will be the first drug in Russia for hemophilia gene therapy, aimed at “correcting” the damaged gene. Medical centers in Moscow, St. Petersburg, Ufa, Nizhny Novgorod, Samara, Chelyabinsk, Kemerovo, Novosibirsk, Syktyvkar, Kirov and other cities will be connected to the study.
The main objective of the research will be to evaluate the efficacy and safety of the drug (its “working” name is ANB-002). It is assumed that it will be administered to a sick person once, keeping the effect for at least several years.
ANB-002 has already successfully passed preclinical studies, demonstrating efficacy and safety in animals. BIOCAD’s scientific divisions are also working on the creation of a gene therapy drug for the treatment of hemophilia type A, whose preclinical trials continue today.
“Both developments are state-of-the-art drugs and are rAAV-based vectors that carry the genes for human blood coagulation factors: factor VIII in the case of hemophilia A or factor IX in the case of hemophilia B.
The peculiarity of the gene therapy approach is that a single administration of the drug, as a rule, makes it possible to achieve a stable therapeutic effect for many years, “explained Yulia Linkova, Vice President of Development and Clinical Research at BIOCAD.
The company has already announced the recruitment of volunteers to participate in clinical trials.
Hemophilia is one of the most common serious inherited diseases. In Russia, about three thousand people suffer from hemophilia (A and B). There are hundreds of thousands of people in the world. Until recently, there were no registered gene therapy drugs for the treatment of hemophilia, intended to eliminate the cause of the disease. The first drug of this type received registration in Europe in the summer of 2022.