CAMBRIDGE, Mass. — Be Biopharma has dosed the first participant in its Phase 1/2 clinical trial evaluating BE-101, an experimental therapy for hemophilia B that represents the first engineered B cell medicine to reach human testing for the disease.
The ongoing trial, known as BeCoMe-9, is a first-in-human, multi-center dose escalation study assessing the safety and early efficacy of BE-101 in patients with moderately severe to severe hemophilia B. The condition affects roughly 40,000 individuals worldwide and is marked by a deficiency in clotting Factor IX (FIX), leading to frequent bleeding episodes.
Unlike gene therapies that require harsh preconditioning or immune suppression, BE-101 was administered without either. The company says preclinical studies suggest the therapy can engraft successfully without those interventions.
BE-101 is designed to insert the human FIX gene into B cells, potentially enabling long-lasting production of the clotting factor. The treatment is also intended to be titratable and re-dosable, offering flexibility that current therapies lack.
“This first dose in a human marks a major milestone for Be Bio and for patients with hemophilia B,” said Joanne Smith-Farrell, Ph.D., the company’s CEO. “BE-101 could not only reshape the treatment of this disease but also demonstrate the broader potential of B cell medicines.”
Dr. Mark Reding, Director of the Center for Bleeding and Clotting Disorders at the University of Minnesota and a trial investigator, noted that many patients still face regular infusions and persistent bleeding risks. “BE-101 offers the promise of sustained FIX levels and long-term protection, without preconditioning,” he said, calling it a potentially significant advance.
The trial will continue to enroll participants as Be Biopharma evaluates dosing levels and monitors outcomes related to both safety and therapeutic benefit.
