Be Bio Announces New Preclinical Data Demonstrating its Novel Engineered B Cell Medicine (BCM), BE-101, Produces Active and Sustained Levels of Factor IX for the Treatment of Hemophilia B​
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Richard Morgan, Ph.D., Chief Scientific Officer, Be Biopharma (Photo: Business Wire)
SAN DIEGO--(BUSINESS WIRE)--Be Biopharma, Inc. ("Be Bio"), a company pioneering the discovery and development of Engineered B Cell Medicines (BCMs), today announced results from a new preclinical study demonstrating that the company's novel engineered BCM, BE-101, produces active and sustained levels of Factor IX (FIX) for the treatment of hemophilia B.​ These data were shared during an oral presentation at the 65th annual meeting of the American Society of Hematology taking place in San Diego, California. Also today, Be Bio announced plans to advance BE-101 as its lead program for the treatment of people with severe or moderately severe hemophilia B.​

In addition, the company announced the appointment of Glenn Pierce, M.D., Ph.D., globally renowned hemophilia physician-researcher, as a member of the Be Bio Scientific Advisory Board.

"The preclinical study findings presented today demonstrate that BE-101 can produce predictable, persistent FIX activity levels with the potential to prevent bleeding and joint damage in persons with hemophilia B," said Richard A. Morgan, Ph.D., chief scientific officer, Be Bio. "We are excited by these findings as we advance this novel BCM into clinical development to address enduring unmet medical needs for people living with hemophilia B. Our data demonstrate that a single dose of BE-101 allows for sustainable, steady-state FIX levels, making it a potentially transformative FIX replacement option for adults and children with hemophilia B."​

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"Despite several major advances in treatment options for people with hemophilia B, including extended-release recombinant factor IX biologics and the availability of a new gene therapy, significant unmet needs remain for people living with hemophilia B. Many people living with hemophilia B still experience bleeding episodes, irreversible joint damage and pain with current extended-release factor IX biologic therapy. A redosable treatment that can provide sustained therapeutic levels of FIX has the potential to prevent irreversible joint damage before it occurs, which could be a major therapeutic advance for adults and importantly, children living with hemophilia B," Dr. Pierce said.

"Our goal is to create a new, potentially transformative treatment option that will provide people with hemophilia B the freedom to live a normal life, free of limitations from hemophilia B. We look forward to filing our Investigational New Drug application for BE-101 in mid-2024​," said Joanne Smith-Farrell, Ph.D., chief executive officer, Be Bio. ​"As we announce our lead program in hemophilia B, we are delighted to welcome Dr. Pierce to our Scientific Advisory Board," Smith-Farrell said. "Dr. Pierce is a tireless patient advocate, hemophilia survivor, biotechnology industry leader, and deeply respected hematology thought leader. All of us at Be Bio are inspired by the opportunity to advance BE-101 as the first program in a bold new class of cell therapies with the potential to transform care for people living with serious disease."

Dr. Pierce has more than 30 years of experience in biopharmaceutical research and development and has been involved in the development and approval of six hemophilia therapies. He currently serves on the World Federation of Hemophilia (WFH) Vice President Medical and WFH USA Board of Directors and the U.S. Medical and Scientific Advisory Council of the National Bleeding Disorders Foundation. The co-author of more than 200 scientific papers, Dr. Pierce also served on the Blood Products Advisory Committee of the U.S. Food and Drug Administration and the Committee on Blood Safety and Availability of the US Department of Health and Human Services.

BE-101 Oral Presentation at American Society of Hematology

Poster Title:
"Development of an Ex Vivo Precision Gene Engineered B Cell Medicine That Produces Active and Sustained Levels of FIX for the Treatment of Hemophilia B"
Presentation #: 463
Oral Presentation Session: 703. Cellular Immunotherapies: Basic and Translational: Novel Approaches for Next Generation Cellular Immunotherapies
Date/Time: Sunday, December 10, 9:30am PT
Presenter: Richard A. Morgan, Ph.D., Chief Scientific Officer, Be Bio

Study Summary

In this study, primary human B cells were isolated and engineered by CRISPR/Cas9 genome editing followed by AAV-mediated homology directed repair (HDR) insertion of human FIX gene into the CCR5 safe harbor locus. The cells were then further expanded and differentiated toward the plasma cell lineage, resulting in FIX-producing BCMs. Approximately 50% targeted integration was achieved, as measured by droplet digital PCR (ddPCR). Engineered BCMs secreted up to 60 ng/1e6 cells/hour of FIX protein, approaching 40% of IgG secretion rate as measured by ELISA. Vitamin K-dependent activated partial thromboplastin time (aPTT), using the one stage clotting assay, demonstrated biological activity of BCM-produced FIX. Similarly, FIX-expressing BCMs exhibited vitamin K-dependent activity in vitro in the chromogenic assay. FIX-expressing BCMs were transferred into immunodeficient NOG-hIL6 mice, with stable FIX production demonstrated for >168 days in vivo. Redosability was demonstrated with increased engraftment (measured by human IgG levels) and a concomitant increase in plasma FIX. The safety of FIX-expressing BCMs has been characterized based on 28-day and 5-month in vivo studies in NOG-hIL6 mice (n=168 mice). Neither abnormal clinical observations nor mortality were observed in those studies. Biodistribution of the FIX-expressing BCMs was assessed using qPCR. The data confirmed the expected biodistribution of FIX-expressing BCMs in bone marrow tissue, where they engraft stably over time.

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About Hemophilia B

Hemophilia B is an X-linked recessive bleeding disorder that affects approximately 1:20,000 males. 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

Imagine what could "Be?" In nature, a single B cell engrafts in the bone marrow and can produce thousands of proteins per second at constant levels over decades. B cells are nature's exquisite medicine factories, manufacturing proteins to fight disease and maintain health. Unleashing the power of B cells is driving a new class of cellular medicines – Engineered B Cell Medicines (BCMs). BCMs have the potential to be durable, allogeneic, redosable and administered without toxic conditioning. The promise of BCMs could transform therapeutic biologics with broad application — 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 patients who are living with cancer, rare diseases 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 by B cell engineering pioneers David Rawlings, M.D., and Richard James, Ph.D., from Seattle Children's Research Institute. Be Bio is backed by ARCH Venture Partners, Atlas Venture, RA Capital Management, Alta Partners, Longwood Fund, Bristol Myers Squibb, Takeda Ventures, Seattle Children's Research Institute and others. Since our founding, Be Bio's investors have committed over $180 million to enable the Company to re-imagine medicine based on the power of B cell therapy. For more information, please visit us at Be.Bio and our LinkedIn page.


1What is Hemophilia? U.S. Centers for Disease Control and Prevention. Accessed November 9, 2023
2Hemophilia B. National Bleeding Disorders Foundation. Accessed November 9, 2023


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Release Summary

Be Bio presents new study showing active and sustained levels of Factor IX and announces plans to advance BE 101 as lead program for hemophilia B.

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