Be Biopharma Begins Cell Therapy Trial for Hemophilia B

CAMBRIDGE, MA — Be Biopharma, a clinical-stage company pioneering engineered cell therapy, announced today that it has treated the first participant in its Phase 1/2 clinical trial for BE-101. This new B Cell Medicine (BCM) is designed to treat moderately severe to severe hemophilia B. A key feature of the therapy is that it was administered without any preconditioning or immunosuppression, a significant advantage for cell therapy.

The BeCoMe-9 trial will assess the safety and preliminary efficacy of BE-101. The investigational cell therapy is a first-in-class BCM engineered to insert the human Factor IX (FIX) gene into primary human B cells. This aims to produce long-lasting and sustained FIX levels, offering durable protection against bleeding events for a condition that affects an estimated 40,000 people worldwide.

Dr. Joanne Smith-Farrell, President and CEO of Be Biopharma, called the event a “significant milestone” for both the company and the hemophilia B community. She noted that BE-101 demonstrates the power of BCMs to deliver groundbreaking in vivo biologics.

Dr. Mark Reding, Director of the Center for Bleeding and Clotting Disorders at the University of Minnesota, highlighted the unmet need in the field. He explained that many people with hemophilia B still experience regular bleeding events despite recent advancements in treatment. He believes that BE-101 could offer a crucial new therapeutic option by providing sustained FIX levels without the need for burdensome preconditioning regimens, which are often required for other cell therapy options.