- BE-101 is the First Engineered B Cell Medicine to Enter Clinical Trials for Hemophilia B
CAMBRIDGE, Mass., July 30, 2025 – Be Biopharma, Inc. (“Be Bio” or “the Company”), a clinical-stage company pioneering the discovery and development of engineered B Cell Medicines (BCMs), today announced the first participant was treated with BE-101 in the BeCoMe-9 Phase 1/2 clinical trial. BE-101 was administered without any preconditioning or immunosuppression, supported by preclinical evidence that BCMs engraft in the absence of these burdensome regimens.
BeCoMe-9 is a multi-center, first-in-human dose escalation Phase 1/2 clinical trial designed to assess the safety and preliminary efficacy of BE-101 in participants with moderately severe to severe hemophilia B, a bleeding disorder affecting approximately 40,000 people globally. BE-101 is a first-in-class BCM that is engineered to insert the human Factor IX (FIX) gene into primary human B cells for the treatment of hemophilia B. BE-101 aims to offer a transformative new therapeutic option by delivering long-lasting FIX levels, with the flexibility to be titratable and re-dosable, and without the need for preconditioning.
“Dosing the first participant in our first-in-human study of BE-101 marks a significant milestone for Be Bio and for people with hemophilia B,” said Joanne Smith-Farrell, Ph.D., President & Chief Executive Officer of Be Bio. “BE-101 is our first BCM to enter the clinic, and it holds the promise of transforming the treatment landscape for hemophilia B while demonstrating the power of BCMs to deliver groundbreaking in vivo biologics.”
“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.”
About the BeCoMe-9 Trial
BeCoMe-9 is a two-part, multi-center, first-in-human dose escalation Phase 1/2 clinical trial designed to assess the safety and preliminary efficacy of BE-101 in adult participants with moderately severe to severe hemophilia B. Part 1 of the trial is a dose escalation of BE-101 aiming to identify the dose required to achieve the desired FIX activity 28 days after infusion. Part 2 will be 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 1 and up to 6 in Part 2. After administration, participants will be monitored for safety and clinical activity for approximately 52 weeks post-IV administration of BE-101.
About BE-101
BE-101 is a first-in-class BCM that is engineered to insert the human Factor IX (FIX) gene into primary human B cells, allowing for continuous expression of active FIX for the treatment of hemophilia B. BE-101 has the potential to express sustained therapeutic FIX activity levels with a single infusion while having the flexibility to be titrated and/or re-dosed, and without the need for preconditioning. The potential to maintain therapeutic FIX activity levels while reducing the dosing frequency associated with current FIX replacement regimens could address the considerable infusion burden associated with current therapies and potentially drive reductions in the annualized bleeding rates and FIX usage. BE-101 has been granted Orphan Drug Designation and Fast Track Designation by the U.S. Food and Drug Administration. Further details of the trial can be found at www.clinicaltrials.gov under NCT identifier: NCT06611436.
About Hemophilia B
Hemophilia B is an X-linked recessive bleeding disorder that affects approximately 40,000 people globally. It is caused by mutations in the gene that encodes for the FIX protein, an essential enzyme in the coagulation cascade. This can lead to spontaneous bleeding as well as bleeding following injuries or surgery.1 People with hemophilia B bleed longer than other people. Bleeds can occur internally, into joints and muscles, or externally, from minor cuts, dental procedures or trauma.2 While an adeno-associated virus (AAV) vector-based gene therapy has been approved for some adults as a potential new option, the current standard of care and only treatment for children remains prophylactic administration of exogenous FIX derived from recombinant protein. The short biological half-life of FIX requires frequent infusions to maintain therapeutic levels.
About Engineered B Cell Medicines – A New Class of Cellular Medicines
The B cell is a powerful cell that produces thousands of proteins per cell per second at constant levels, and over decades. Precision genome editing can now be used to engineer B Cells that produce therapeutic proteins of interest, driving a new class of cellular medicines – Engineered B Cell Medicines (BCMs) – with the potential to be durable, allogeneic, re-dosable, and administered without pre-conditioning. The promise of BCMs could transform therapeutic biologics across protein classes, patient populations and therapeutic areas.
About Be Biopharma
Be Biopharma (“Be Bio”) is pioneering Engineered B Cell Medicines (BCMs) to dramatically improve the lives of people who are living with Hemophilia B and other genetic diseases, cancer, and other serious conditions. With eyes locked on the patient, our team of purpose-driven scientists, technologists, manufacturing experts and business builders collaborate to create a bold new class of cell therapies. Be Bio was founded in October 2020, and is backed by ARCH Venture Partners, Atlas Venture, RA Capital Management, Nextech, Alta Partners, Longwood Fund, Bristol Myers Squibb, Takeda Ventures, Seattle Children’s Research Institute, Pathway to Cures (the venture philanthropy fund for the National Bleeding Disorders Foundation) and others to re-imagine medicine based on the power of Engineered B cells. For more information, please visit us at Be.Bio and our LinkedIn page.
References
- What is Hemophilia? U.S. Centers for Disease Control and Prevention. Accessed November 9, 2023 https://www.cdc.gov/hemophilia/about/
- Hemophilia B. National Bleeding Disorders Foundation. Accessed November 9, 2023 https://www.hemophilia.org/bleeding-disorders-a-z/types/hemophilia-b
