Mitsubishi Tanabe Pharma is joining forces with researchers from Jichi Medical University to launch a program in Japan focused on developing a gene therapy product to treat patients with hemophilia B, the company announced.
The project, titled “R&D of gene therapy product for hemophilia B,” was selected by the Japan Agency for Medical Research and Development (AMED) to receive financial support thought the agency’s Cyclic Innovation for Clinical Empowerment (CiCLE) program.
The program will support creation of an environment that enables the research and development of new medicinal products to be used in clinical settings, and speed the practical application of pharmaceuticals.
Hemophilia B is an inherited disorder that results from genetic mutations in the gene that provides instructions for producing the blood clotting factor IX. Low levels or faulty versions of this protein result in patients’ blood being unable to clot and stop bleeding from injuries.
People with this condition need to receive frequent replacement therapies to ensure they are protected from life-threatening hemorrhages. Replacement therapy involves supplying the missing clotting factor to the patient from an external source.
Mitsubishi Tanabe’s new research project will focus on developing gene therapies that could overcome and reverse the effects of faulty coagulation factor IX in patients with hemophilia B.
The company’s strategy will be based on the use of safe and inactive virus particles, called adeno-associated virus (AAV) vector, to transport and deliver to cells a corrected and functional version of the missing coagulation factor. Upon administration, the genetic sequence will be incorporated into the patients’ cells, which will use their natural machinery to translate the correctly coded information into functional protein.
This gene therapy approach is believed to have the ability to provide, with a single administration, sufficient amounts of factor IX so patients will not need continued treatments with clotting factor replacement therapies.
In a first step, Mitsubishi Tanabe Pharma will work in collaboration with Jichi Medical University to develop this new gene therapy product for hemophilia B. Later, they expect to expand plans to develop new gene therapies that target other genetic disorders.