Russian Ministry of Health has granted approval to Biocad to initiate a Phase III clinical trial for ANB-002, marking the introduction of the country’s first gene therapy for hemophilia B. This groundbreaking development focuses on restoring the body’s natural ability to produce factor IX independently. Unlike standard replacement therapy, which requires regular injections of the clotting factor, the gene therapy drug is administered once.
An expanded recruitment for the combined phase I-II CT is ongoing, with the optimal drug dosage already determined. In phase I-II CT, severe hemophilia B participants received the study drug in minimal, intermediate, and maximal doses. The option of using ANB-002 with concomitant glucocorticoid therapy was also studied. Now the phase I-II study has moved on to an additional stage. The efficacy and safety of the study drug will be further examined in current and new participants.
Furthermore, the recruitment strategy will include individuals who possess risk factors that may diminish the effectiveness of gene therapy. This includes participants with antibodies to AAV5 and those with a history of hepatitis B, provided their condition is currently inactive. They will be tested at the maximum dose both with and without concomitant therapy. Biocad reports that these changes significantly enhance accessibility to therapy for a broader range of individuals with hemophilia, many of whom may have previously been unable to receive gene therapy.