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• 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. April 28, 2025 – 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) 28^th 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.

Investor Contact:
ir@be.bio

Media Contact:
media@be.bio

• Financing includes participation from new investor Nextech¹ along with existing investors Arch Venture Partners, Atlas Venture, RA Capital, Alta Partners, Longwood Fund, Bristol Myers Squibb, Takeda Ventures and Others
• Proceeds will Advance BE-101 Through Clinical Proof-of-Concept and Progress BE-102 to the Clinic

CAMBRIDGE, Mass., January 15, 2025 – Be Biopharma, Inc. (“Be Bio” or “the Company”), a leader in B Cell Medicines (BCMs), today announced the closing of its $92M Series C financing. The round includes participation from new investor Nextech with existing investors including ARCH Venture Partners, Atlas Venture, RA Capital Management, Alta Partners, Longwood Fund, Bristol Myers Squibb, and Takeda Ventures, among others.  In conjunction with the financing, Melissa McCracken, Ph.D. of Nextech will join Be Bio’s Board of Directors.

The proceeds from the Series C financing will be used to generate clinical proof-of-concept for BE-101 in the ongoing BeCoMe-9 Phase 1/2 clinical trial for people with hemophilia B and to advance BE-102 for the treatment of hypophosphatasia to the clinic. Both programs are built on Be Bio’s BCM product platform, creating durable, titratable, and redosable therapeutics that require no preconditioning and produce sustained and constant levels of therapeutic proteins.

“With this funding in hand, we are well-equipped to advance our two lead programs and solidify our position as a multi-program, clinical-stage company,” said Joanne Smith-Farrell, Ph.D., Chief Executive Officer of Be Bio.  “We are on track to demonstrate clinical proof-of-concept for BE-101 in Hemophilia B this year.  Additionally, BE-102 is poised to enter the clinic next year, potentially demonstrating BCMs’ potential to become a new gold standard in enzyme replacement by providing hypophosphatasia patients with a transformative new option.  I am grateful to our existing and new investors, who share our vision that BCMs can transform the treatment landscape for a wide range of diseases with potentially best-in-class medicines.”

“We are excited to partner with Be Biopharma at this pivotal stage,” stated Melissa McCracken, Ph.D., Partner at Nextech.  “Be Bio has established itself as a leader in this field with their lead programs having best-in-class potential. We believe BCMs, as a class, could revolutionize treatment paradigms across indications . We look forward to supporting their groundbreaking work and witnessing the transformative impact on patient care.”

About Nextech Invest

Nextech is a global, therapeutics-focused venture capital firm headquartered in Zurich, Switzerland with offices in Boston and San Francisco, USA. Nextech invests in the most promising drug discovery companies primarily in oncology and adjacent diseases, with the potential to create multiple medicines. Nextech’s portfolio is focused throughout the US and Europe with investments from company inceptions to public financings. For more information, please visit: www.nextechinvest.com

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 the reducing 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.  The US FDA cleared the BE-101 IND in May of 2024, and granted Fast Track designation in September of 2024.  The Phase 1/2 BeCoMe-9 Trial has been initiated and further details of the trial can be found at www.clinicaltrials.gov under NCT identifier: NCT06611436.

About BE-102

BE-102 is a first-in-class BCM that has been engineered using artificial intelligence-guided protein design to modify primary human B cells to produce ALP, an enzyme deficient in people living with HPP. A single infusion of BE-102 has the potential to express sustained therapeutic ALP with the flexibility to be titrated and/or re-dosed, if needed, and without the need for pre-conditioning. BE-102 has been selected as a Development Candidate and has the potential to transform the standard of care for people living with HPP.

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 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 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.

Investor Contact:
ir@be.bio

Media Contact:
media@be.bio

---

1. Nextech Invest Ltd, on behalf of one or more funds managed by it.

CAMBRIDGE, Mass. — Be Biopharma, Inc. (“Be Bio”), a clinical-stage company pioneering engineered B Cell Medicines (BCMs), today announced that Chief Executive Officer Joanne Smith-Farrell, Ph.D. will present at the 43^rd Annual J.P. Morgan Healthcare Conference being held at the Westin St. Francis in San Francisco, CA on Wednesday, January 15, 2025, at 3:30 pm PT.

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, 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, titratable and administered without pre-conditioning. The promise of BCMs could transform therapeutic biologics with broad application — across diverse protein classes, patient populations and therapeutic areas.

About Be Biopharma

Be Biopharma (“Be Bio”) is clinical-stage company pioneering Engineered B Cell Medicines (BCMs) to dramatically improve the lives of patients who are living with Hemophilia B, other genetic diseases, cancer, and other serious conditions. With eyes locked on the patient, Be Bio’s team of purpose-driven scientists, technologists, manufacturing experts and business builders collaborates 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, Alta Partners, Longwood Fund, Bristol Myers Squibb, Takeda Ventures, Seattle Children’s Research Institute and others. For more information, please visit us at Be.Bio and our LinkedIn page.

Investor Contact:
ir@be.bio

Media Contact:
media@be.bio

November 05, 2024 09:05 AM Eastern Standard Time

CAMBRIDGE, Mass.–(BUSINESS WIRE)–Be Biopharma, Inc. (“Be Bio”), a company pioneering the development of engineered B Cell Medicines (BCMs), today announced that an abstract relating to its BCM platform has been accepted for oral presentation at the 66th American Society of Hematology (ASH) Annual Meeting and Exposition to be held in San Diego, California from December 7 to 10, 2024. In addition, a Trials in Progress abstract related to the BeCoMe-9 Phase 1/2 clinical trial for BE-101 in hemophilia B has been accepted for online publication. Both abstracts were published today and are now available on the ASH website at www.hematology.org.

Details regarding the Be Biopharma oral presentation are as follows:

Abstract Title: A Versatile B Cell Engineering Platform Enables Development of B Cell Medicines for Sustained Delivery of Therapeutic Biologics (Abstract #88)
Session Name: 703. Cellular Immunotherapies other than CAR-T Cells: Basic and Translational: Novel Cell Platforms and Delivery Strategies
Date: Saturday, December 7, 2024
Presentation Time: 10:15 a.m. PT
Session Room: San Diego Convention Center, Ballroom 20AB

Details regarding the Be Biopharma online abstract are as follows:

Abstract Title: BeCoMe–9: A Phase 1/2 Dose Escalation and Expansion Study of BE-101 for the Treatment of Adults with Moderately Severe or Severe Hemophilia B (Abstract #5479)

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, 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, titratable 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-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 the reducing 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. The US FDA cleared the BE-101 IND in May of 2024, and granted Fast Track designation in September of 2024. The Phase 1/2 BeCoMe-9 Trial is open for enrollment and further details of the trial can be found at www.clinicaltrials.gov under NCT identifier: NCT06611436.

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, Alta Partners, Longwood Fund, Bristol Myers Squibb, Takeda Ventures, Seattle Children’s Research Institute 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.

Contacts

Investor Contact:
ir@be.bio

Media Contact:
media@be.bio

• Phase 1/2 BeCoMe-9 Trial Study Open for Enrollment and FDA Grants Fast Track Designation for BE-101, a Durable, Titratable, Redosable B Cell Medicine for Hemophilia B
• Second B Cell Medicine Development Candidate Nominated with BE-102 for Hypophosphatasia, A Large Genetic Disease with 50,000 Patients and High Unmet Medical Need
• $82 Million Financing from Arch Venture Partners, Atlas Venture, RA Capital, Alta Partners, Longwood Fund, Bristol Myers Squibb, Takeda Ventures and Others, to Advance BE-101 Through Clinical Proof of Concept and to Progress BE-102
• Addition of Senior Clinical and Commercial Leadership as Company Orients to Product Development

October 22, 2024 12:00 PM Eastern Daylight Time

CAMBRIDGE, Mass.–(BUSINESS WIRE)–Be Biopharma, Inc. (“Be Bio” or “the Company”), a leader in the discovery and development of engineered B Cell Medicines (BCMs), today announced key milestones alongside a new round of funding as its lead program, BE-101 for Hemophilia B, enters the clinic, and its second development candidate for Hypophosphatasia is unveiled. Both programs are built on Be Bio’s powerful and efficient BCM platform, which utilizes gene editing to engineer B cells to produce sustained levels of therapeutic proteins, resulting in durable, titratable, and redosable candidates that require no preconditioning and have the potential to become best-in-class genetic medicines.

Key recent milestones include:

• BE-101: Phase 1/2 BeCoMe-9 Trial Open for Patient Enrollment; Granted Fast Track Designation. In May 2024, Be Bio announced that BE-101, a first-in-class BCM, received clearance of its Investigational New Drug application. BE-101 is designed to produce constant levels of Factor IX in a durable, redosable, and titratable manner, without the need for preconditioning. BeCoMe-9 is a multi-center, first-in-human dose escalation study aimed at evaluating the safety and preliminary efficacy of BE-101 in adults with moderately severe to severe Hemophilia B. FDA has recently granted BE-101 Fast Track Designation.
• BE-102 Nominated as a Development Candidate for Hypophosphatasia (HPP): BE-102 has been selected as a development candidate for the treatment of Hypophosphatasia (HPP), a severe genetic disease with very high unmet medical need, affecting approximately 50,000 patients. HPP is characterized by loss of function mutations in the ALPL gene which lead to deficient alkaline phosphatase (ALP) activity, resulting in weakened and underdeveloped bones and teeth. The only approved therapy for HPP requires multiple injections per week and is limited to pediatric-onset forms of the disease. Data presented at the 2024 American Society for Bone and Mineral Research (ASBMR) Meeting showed that BE-102 has the potential to generate active and sustained levels of ALP in vivo.
• $82 Million Financing to Advance BE-101 to Clinical Proof of Concept and Progress BE-102: The Company has closed an $82 million financing, with backing from top venture capital firms and pharmaceutical companies, including ARCH Venture Partners, Atlas Venture, RA Capital Management, Alta Partners, Longwood Fund, Bristol Myers Squibb, and Takeda Ventures. The funds will be used to achieve clinical proof of concept for BE-101 and to advance the development of BE-102.
• Key Hires in Clinical Development and Commercial: The Company is excited to welcome Suha Patel as Senior Vice President of Commercial & Franchise Strategy and Kiran Patki, MD as Senior Vice President of Clinical Development. Ms. Patel joins from Roche/Genentech, where she led the successful launch of Hemlibra and held senior roles across marketing, medical marketing and sales. Dr. Patki joins from Rally Bio, where he served as Senior Vice President and Global Team Leader for asset development projects, with prior leadership experience at Alexion Pharmaceuticals. Concurrent with building out these later-stage functions, the Company will streamline its early research organization and related functions to focus resources on product development.

“Advancing two BCMs that harness the potential of this new modality represents an exciting step in transforming the treatment landscape for their respective indications with potentially best-in-class genetic medicines. With support from a top-tier syndicate of investors and pharmaceutical companies, we are eager to clinically demonstrate BE-101’s potential to provide a highly durable FIX replacement therapy for Hemophilia B patients. Additionally, we are excited to advance BE-102 to address the needs of a large patient population with few to no therapeutic options,” said Joanne Smith-Farrell, Ph.D., Chief Executive Officer of Be Bio. “As we build out the team to support product development, we are delighted to welcome Suha and Kiran, experienced leaders who bring deep development and commercial expertise to our team, further strengthening our leadership as we transition into a clinical-stage company.”

Dr. Smith-Farrell added: “Our innovative and highly effective research team is the driving force behind the creation of BE-101 and BE-102. They quickly brought the BCM platform to life, achieving IND clearance for BE-101 in just 2.5 years from the program’s inception, and advancing BE-102 to DC in only 15 months, showcasing the platform’s versatility and modularity. As we shift resources toward product development, some valued colleagues will be moving on. We are incredibly proud of their achievements and deeply grateful for their tireless and impactful work on behalf of Be Bio and the patients whose lives we hope these therapies will one day transform.”

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 the reducing 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. The US FDA cleared the BE-101 IND in May of 2024, and granted Fast Track designation in September of 2024. The Phase 1/2 BeCoMe-9 Trial has been initiated and 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. 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. The current standard of care remains prophylactic administration FIX replacement therapy with a dosing frequency that ranges from every week to every 2 weeks. The short biological half-life of FIX requires lifelong frequent infusions to maintain therapeutic levels.

About BE-102

BE-102 is a first-in-class BCM that has been engineered using artificial intelligence-guided protein design to modify primary human B cells to produce ALP, an enzyme deficient in people living with HPP. A single infusion of BE-102 has the potential to express sustained therapeutic ALP with the flexibility to be titrated and/or re-dosed, if needed, and without the need for pre-conditioning. BE-102 has been selected as a Development Candidate and has the potential to transform the standard of care for people living with HPP.

About Hypophosphatasia

HPP is a genetic disease caused by loss of function mutations in the ALPL gene, leading to a deficiency in ALP activity that is required for healthy mineralization of bones and teeth. Insufficient levels of ALP result in the inability of calcium and phosphate to mobilize from the blood, resulting in weakened and underdeveloped bones and teeth. HPP is estimated to affect up to 50,000 people. There is only one approved therapy which requires three to six times a week administration.

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 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, Alta Partners, Longwood Fund, Bristol Myers Squibb, Takeda Ventures, Seattle Children’s Research Institute 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.

Contacts

Investor:
ir@be.bio

Media:
media@be.bio

• New preclinical data demonstrate potential for precision-engineered B cell medicines (“BCMs”) to produce active and sustained levels of tissue-nonspecific alkaline phosphatase (“ALP”)
• Data presented at the American Society for Bone and Mineral Research 2024 Annual Meeting

September 30, 2024 09:00 AM Eastern Daylight Time

CAMBRIDGE, Mass.–(BUSINESS WIRE)–Be Biopharma, Inc. (“Be Bio”), a company pioneering the discovery and development of engineered B Cell Medicines (BCMs), today announced the presentation of preclinical data demonstrating the transformative power of BCMs to produce active alkaline phosphatase (ALP), highlighting BCMs as a potential treatment for Hypophosphatasia (HPP). Researchers coupled CRISPR/Cas9 precision gene engineering and artificial intelligence-guided protein design to modify primary human B cells to express ALP, an enzyme deficient in people living with HPP. The findings were presented during poster presentations at the American Society for Bone and Mineral Research (ASBMR) 2024 Annual Meeting (Thursday September 26^th through Saturday, September 28^th).

HPP is a rare genetic disease caused by loss of function mutations in the ALPL gene which leads to deficient ALP activity. People living with HPP can experience wide-ranging systemic complications, with impaired bone mineralization, such as rickets/osteomalacia, as the major clinical hallmark of disease. The only approved therapy for HPP is an enzyme replacement therapy, asfotase alfa, which requires multiple injections every week and is approved only for use in patients with pediatric-onset forms of HPP.

“This study builds on data presented earlier this year, demonstrating the ability of BCMs to produce sustained active levels of ALP in vivo,” said Rick Morgan, Chief Scientific Officer. “Our ALP-BCM is designed to address key unmet medical needs in HPP, delivering constant levels of active ALP, with a medicine that has the potential for once-yearly administration, does not require pre-conditioning, and can be re-dosed as necessary. These positive preclinical data support the potential of an ALP-BCM as a first-in-class medicine for people living with HPP.”

In this study, primary human B cells were expanded and precision engineered by CRISPR/Cas9 genome editing with AAV-mediated homology directed repair (HDR) to insert an ALP gene expression cassette into various loci, including CCR5 (a safe harbor locus). Guided by an artificial intelligence-based ALP protein structure design engine, protein constructs were optimized for activity and stability of ALP-Fc fusion proteins. Engineered BCMs secreted active ALP proteins (ALP-BCM) and demonstrated phenotypic correction in an in vitro bone mineralization model. Notably, ALP-BCMs engrafted in an immunodeficient mouse model and produced sustained levels of active ALP. These data underscore the potential of ALP-BCMs to address a significant unmet medical need as a potential HPP treatment. No adverse effects were observed during the in vivo study.

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 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.

Contacts

Investor Contact:
ir@be.bio

Media Contact:
media@be.bio