Clinical stage biotechnology Be Biopharma, Inc. recently announced that the first hemophilia B patient has been treated with a single intraveneous dose of their investigational cell therapy, BE-101. The ongoing BeCoMe-9 Phase 1/2 study is evaluating the safety and efficacy of BE-101 in adults with moderately severe to severe hemophilia B.
According to a Be Biopharma press release, the first part of their multicenter trial is a dose escalation of BE-101 meant to identify the requisite dose to reach the desired factor IX (FIX) activity 28 days after infusion. The second part will represent an expansion phase to further assess the safety and activity of BE-101 at the selected dose. Up to 24 participants will be enrolled across both parts, with up to 18 in part one and up to six in part two. Patients who eventually received the therapy will be monitored for safety and clinical activity for approximately 52 weeks after administration of the treatment.
BE-101 is engineered to insert the human factor IX (FIX) gene into primary human B cells and initiate the continuous production of therapeutic levels of the FIX protein. B cells are able to generate high concentrations of proteins regularly and over a long period, while CRISPR/Cas9 genome editing technology ensures that the FIX genes are engrafted with precision into the targeted cells.
“Despite recent advancements, many people living with hemophilia B require frequent infusions of FIX and continue to experience regular bleeding events that can lead to long-term consequences such as chronic pain and irreversible joint damage,” said Dr. Mark Reding, Director of the Center for Bleeding and Clotting Disorders at the University of Minnesota. “BE-101 has the potential to be an important new therapeutic option in hemophilia B by delivering sustained FIX levels and durable bleed protection, without the need for preconditioning, filling a significant unmet need.”
BE-101 was already been granted Orphan Drug Designation and Fast Track Designation by the U.S. Food and Drug Administration.
Source: Be Biopharma, Inc press release dated July 30, 2025
