Mutations in the F9 gene cause factor IX (FIX) deficiency, leading to severe bleeding in people with hemophilia B. Current therapies rely on frequent FIX infusions or newer agents that rebalance coagulation, but both approaches still require weekly to bimonthly dosing and often fail to entirely prevent breakthrough bleeding events, which remain common in patients. Therefore, new therapeutic strategies providing a predictable, durable, active and redosable source of FIX are urgently needed.

Be Biopharma Inc. has developed a CRISPR/Cas9-based precision B-cell gene therapy to deliver active tissue non-specific alkaline phosphatase (ALP) for the potential treatment of hypophosphatasia (HPP).

The FDA has granted orphan drug designation to Be Biopharma Inc.’s BE-101, a novel engineered B-cell medicine being developed for the treatment of hemophilia B.

Be Biopharma Inc. has announced the FDA’s clearance of its IND for BE-101, an autologous potentially first-in-class B-cell medicine in development for the treatment of hemophilia B.

Be Biopharma Inc. is looking to develop engineered B cells to treat a wide range of diseases. The new category of cellular medicine is based on the work of David Rawlings and Richard James, researchers at Seattle Children’s Research Institute and the University of Washington.