Biocad, a Russian biotechnology company, has filed registration documents for Russia’s first original gene therapy drug aimed at treating hemophilia B, a rare hereditary disorder that affects blood clotting. The drug received the international nonproprietary name (INN) arvenacogene dezaparvovec. The company’s investments in its creation and research will exceed 3 billion rubles.
«We are submitting the registration dossier under the conditional registration procedure, which is provided for by the EAEU rules. This procedure allows for faster access to therapy for rare or severe diseases, provided there is sufficient clinical research data», — the company’s press service said.
The company’s own investments in its creation and research will exceed 3 billion rubles, according to TASS. The investigational drug is intended for single-dose administration.
The registration is based on the results of a combined Phase I–II trial (ANB-002-1/ SAFRAN). Despite the registration, the company will continue to monitor the condition of the participants in the current trial for at least another five years. Recruitment of patients for the Phase III trial (ANB-002-2/ MAGNOLIA) continues.
The development of the drug began more than seven years ago with the creation of the Genext platform technology, which allows the production of recombinant AAV vectors. Work on the platform was launched in 2016, and in 2018, Biocad began developing gene therapy for hemophilia B. In 2024, the Ministry of Health of the Russian Federation granted Biocad permission to conduct a phase III clinical trial of the first domestic gene therapy ANB-002 for the treatment of hemophilia B.
