- Oral presentation to feature nonclinical data for BE-102 for the potential treatment of hypophosphatasia
- Poster to highlight potential of BCMs as an allogenic off-the-shelf therapy
CAMBRIDGE, Mass.–(BUSINESS WIRE)–Be Biopharma, Inc. (“Be Bio”), a clinical-stage company pioneering the discovery and development of engineered B Cell Medicines (BCMs), today announced that it will present at the American Society of Gene and Cell Therapy (ASGCT) 28th Annual Meeting being held in New Orleans, May 13-17, 2025.
Details regarding the Be Biopharma presentations at the conference are as follows:
Oral Presentation Title: Ex Vivo Gene Editing of Autologous B Cells Produce Sustained Levels of Tissue Nonspecific Alkaline Phosphatase In Vivo for the Potential Treatment of Hypophosphatasia
Presenter: Hanlan Liu, Ph.D., MBA, SVP, Head of Late Research and NCD, Be Biopharma
Date: May 17, 2025
Presentation Time: 10:45 – 11:00 AM CT
Session Title: B-Cell and Solid Organ Therapies
Session Time: 10:15 AM – 12:00 PM CT
Session Room: 278-282
The oral presentation will include nonclinical data for Be Biopharma’s BE-102 program, a novel B cell therapy developed as a potential treatment of Hypophosphatasia (HPP). HPP is caused by deficiency of tissue-nonspecific alkaline phosphatase (TNSALP) activity, resulting from pathogenic mutations in the ALPL gene, which leads to multi-systemic clinical complications including deficient bone mineralization. Enzyme replacement therapy (ERT) is the only approved treatment for HPP which requires frequent lifelong injections. ERT is only available for perinatal/infantile- and juvenile-onset forms of HPP and weekly injections are associated with common side effects that can significantly affect a patient’s quality of life. BE-102 was developed to address these limitations. BE-102 is manufactured from primary human B cells by isolating, activating, and precision engineering with CRISPR/Cas9 followed by AAV-mediated delivery of a DNA donor template for the insertion of human ALPL gene into the CCR5 locus (a safe harbor locus). In vitro pharmacology results demonstrate that BE-102 secretes active TNSALP, which is capable of rescuing calcium deposit inhibited by inorganic pyrophosphate (PPi), a substrate which accumulates in people with HPP. In vivo studies were conducted in immune-deficient NOG-hIL6 mice, confirming long-term engraftment and continuous production of active TNSALP in vivo following a single IV administration of BE-102. Be Biopharma’s in vitro and in vivo pharmacology and safety data established nonclinical proof-of-concept that BE-102 has the potential to be a disease-modifying therapy for people with HPP by providing long-lasting active TNSALP, with the flexibility to be titratable and redosable as needed.
Poster Title: Exploration of Allogeneic Shielding Strategies by Precise CRISPR/Cas9 Genome Engineering of Primary Human B Cells to Enable Off-the-shelf B Cell Medicines for Sustained Delivery of in vivo Biologics
Presenter: Xuqing Zhang, Ph.D., Director, Immunology, Be Biopharma
Date: May 13, 2025
Time: 6:00 – 7:30 PM CT
Session Title: Tuesday Poster Reception
Session Room: Poster Hall, Hall I2
Final Abstract Number: 777
The abstract highlights Be Biopharma’s approach using both in vitro and in vivo assays to determine the potential of B Cell Medicines (BCMs) as an allogeneic off-the-shelf cell therapy without additional engineering and ways to enhance hypoimmunogenicity via CRISPR/Cas9 genome engineering. The data demonstrates BCMs are immunologically stealthy and can be further engineered to evade host immune responses, opening the possibility of exploring them as off-the-shelf cellular therapies without HLA matching for broader patient access.
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, redosable, and administered without pre-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 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.
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