• Skip to primary navigation
  • Skip to main content
  • Skip to footer
Be Biopharma

Be Biopharma

Advancing our science with bold humanity

  • Investors & Media
    • News Releases
    • Publications & Presentations
  • Careers
  • Contact Us
  • Search
  • Our Company
    • About Be Bio
    • Life at Be Bio
    • Leadership
    • Board of Directors
    • Scientific Advisory Board
    • Investors
  • Our Science
    • About BCMs
    • Our Platform
    • Publications & Presentations
  • Patients & Caregivers

Press Releases

December 10, 2023 by mkendis

BE-101 is Company’s Lead Program in Hemophilia B; IND Filing Anticipated in Mid-2024

Be Bio Appoints Glenn Pierce, M.D., Ph.D., Hemophilia Physician-Researcher and Advocate, to its Scientific Advisory Board

Preclinical Study Findings Shared in Oral Presentation During 65th Annual Meeting of the American Society of Hematology

SAN DIEGO, December 10, 2023 – 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.”​

“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.

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.

Investor Contact:

ir@be.bio

Media Contact:

media@be.bio

References

1What is Hemophilia? U.S. Centers for Disease Control and Prevention. Accessed November 9, 2023

https://www.cdc.gov/ncbddd/hemophilia/facts.html

2Hemophilia B. National Bleeding Disorders Foundation. Accessed November 9, 2023

https://www.hemophilia.org/bleeding-disorders-a-z/types/hemophilia-b

Filed Under: Press Releases

November 22, 2023 by mkendis

November 22, 2023 09:00 AM Eastern Standard Time

CAMBRIDGE, Mass.–(BUSINESS WIRE)–Be Biopharma, Inc. (“Be Bio”), a company pioneering the discovery and development of Engineered B Cell Medicines (BeCMs), today announced that Chief Executive Officer Joanne Smith-Farrell, Ph.D., will present at the 35th Annual Piper Sandler Healthcare Conference being held at the Lotte New York Palace in New York, NY on Thursday, November 30, 2023.

35th Annual Piper Sandler Healthcare Conference
Presentation Date: Thursday, November 30, 2023
Presentation Time: 10:10 am ET

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.

Contacts

Investors:
ir@be.bio

Media:
media@be.bio

Filed Under: Press Releases

November 2, 2023 by rbracey

Study Selected for Oral Presentation During the American Society of Hematology 65th Annual Meeting

 

November 02, 2023 12:05 PM Eastern Daylight Time

CAMBRIDGE, Mass.–Be Biopharma, Inc. (“Be Bio”), a company pioneering the discovery and development of Engineered B Cell Medicines (BCMs) today announced that it will present new preclinical data in an oral presentation at the 65th Annual Meeting of the American Society of Hematology (ASH) taking place December 9-12, 2023, in San Diego, California. The research will describe the development of an ex vivo precision-engineered B Cell Medicine (BCM) that produces active and sustained levels of factor IX (FIX). The abstract is now available online in a special issue of the ASH official journal, Blood.

“We look forward to presenting our research at the ASH annual meeting, the pre-eminent global congress for scientific exchange in hematology,” said Dr. Rick Morgan, Chief Scientific Officer, Be Bio. “Our team of dedicated researchers is inspired by the potential of this bold new class of medicines.”

Details of the oral presentation are as follows:

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
Session Name: 703 Cellular Immunotherapies: Basic and Translational: Novel Approaches for Next Generation Cellular Immunotherapies
Session Date: Sunday, December 10, 2023
Presentation Time: 9:30 AM PST
Room: San Diego Convention Center, Room 6A
Publication Number: 463

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 Longwood Fund and 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

Investors:
ir@be.bio

Media:
media@be.bio

Filed Under: Press Releases

October 31, 2023 by rbracey

Findings From Study to be Presented at the 38th Annual Meeting of the Society for Immunotherapy of Cancer

 

October 31, 2023 12:01 PM Eastern Daylight Time

SAN DIEGO–(BUSINESS WIRE)–Be Biopharma, Inc. (“Be Bio”), a company pioneering the discovery and development of Engineered B Cell Medicines (BCMs), will present preclinical data demonstrating that a BCM-derived biologic can deliver steady-state plasma concentrations of a bispecific T cell engager at clinically relevant doses for treatment of relapsed/refractory acute lymphoblastic leukemia (ALL). Findings from the study, conducted jointly with researchers at Seattle Children’s Research Institute, will be presented during the Society for Immunotherapy of Cancer’s 38th Annual Meeting.

“These findings underscore the clinical potential of BCMs as an emerging platform for sustained delivery of antitumor biologics. Our platform enables production of a diverse set of therapeutic proteins, including those difficult to manufacture using traditional systems, and offers a promising approach for a new class of medicines with broad therapeutic utility,” said Dr. Rick Morgan, Chief Scientific Officer, Be Bio.

“Bispecific T cell engagers are highly effective in treating relapsed/refractory ALL; however, their short half-life requires continuous intravenous administration at high doses for four-week increments. This study demonstrates that engineered plasma cells can deliver a constant infusion of bispecific T cell engagers at a steady-state, clinically relevant plasma concentration, offering a potential therapeutic advance to decrease the burden of therapy for patients,” said Dr. Richard James, principal investigator at Seattle Children’s Research Institute.

The poster presentation details are as follows:

Saturday, November 4, 2023, 12:10 pm PDT

Session: Session 205a: Rapid Oral Abstract-Basic Science

Title: “Development of ex vivo precision gene engineered B cell medicines that produce highly active and sustained levels of transgenic anti-tumor biologics”

Lead Authors: Rick Morgan, Ph.D., and Richard G. James, Ph.D.

Presenter: Sean Arlauckas, Ph.D., Director, Oncology Research, Be Bio

Abstract #: 409

Bispecific T cell engagers, consisting of an anti-CD3 single-chain fragment variable (scFv) fused to an anti-tumor antigen scFv, are highly effective in the treatment of relapsed/refractory acute lymphoblastic leukemia (ALL). However, the short half-life of bispecific T cell engagers necessitates continuous intravenous administration at high doses for four weeks at a time. Given their ability to produce proteins at steady state, BCMs are particularly well-suited to overcome these pharmacokinetic shortcomings. To generate BCMs, B cells are engineered ex vivo to constitutively secrete transgenic biologics and then differentiated into plasma cells. These cells were chosen for their high antibody production capacity (thousands of Ig molecules/cell/sec) and ability to engraft without preconditioning, making them a highly attractive cell-based platform for continuous biologic delivery1.

About the Study

To demonstrate proof-of-concept, a transgene coding for a bispecific anti-CD3:CD19 scFv was integrated into a safe-harbor locus of primary human B cells via targeted gene knock in. The engineered B cells were then differentiated into plasma cells ex vivo. Assessment of in vivo anti-tumor activity of these BCMs was conducted in NSG mice harboring a patient-derived CD19-expressing xenograft (PDx). Mice were inoculated with a luciferized B-ALL PDx line and autologous T cells were delivered after tumor transfer. Significant reduction in tumor burden (bioluminescent flux, area under the curve) was observed over the course of the 17-day study in the anti-CD3:CD19 scFv cohort compared to controls, which was in concordance with heightened in vivo T cell activation. The ~1000 pg/mL bispecific T cell engagers detected in mouse plasma demonstrated that a BCM-derived biologic can meet or even exceed the steady-state plasma concentrations achieved with clinically relevant blinatumomab doses2.

Deidentified human PBMCs were acquired under informed consent from the Fred Hutch Specimen Processing and Research Cell Bank (protocol #3942).

No toxicities were identified in this study.

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 Longwood Fund and 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.

References:

  1. Hung KL, Meitlis I, Hale M, Chen C, Singh S, Jackson SW, Miao CH, Khan IF, Rawlings DJ, James RG. Engineering Protein-Secreting Plasma Cells by Homology-Directed Repair in Primary Human B Cells. Mol. Ther. 2018; 26:456-467.
  2. Franquiz MJ and Short NJ. Blinatumomab for the Treatment of Adult B-Cell Acute Lymphoblastic Leukemia: Toward a New Era of Targeted Immunotherapy. Biologics. 2020; 14: 23–34.

 

Contacts

Investors:
ir@be.bio

Media:
media@be.bio

Filed Under: Press Releases

October 25, 2023 by rbracey

Findings To Be Presented During the European Society of Gene & Cell Therapy 30 Annual Meeting

BRUSSELS–(BUSINESS WIRE)–Be Biopharma, Inc. (“Be Bio”), a company pioneering the discovery and development of Engineered B Cell Medicines (BCMs), will present research from preclinical studies during the European Society of Gene & Cell Therapy (ESGCT) 30th Annual Meeting. In an oral presentation on Friday, October 27, from 11:00 to 13:00 CEST, Be Bio demonstrates that BCMs are capable of producing a variety of therapeutic protein classes using precision gene editing. The data include durable in vivo production of the human clotting factor IX, prevention of tumor growth in a patient-derived xenograft model via a CD19xCD3 bispecific antibody, and engraftment without preconditioning in immunocompetent non-human primates (NHPs). Also being presented are findings from a preclinical study showing BCMs are capable of producing highly active acid sphingomyelinase, the gene defective in Niemann-Pick disease, and correct the disease phenotype in knockout cells.

“The data we are presenting give further evidence that our precision B cell engineering platform enables the expression of diverse therapeutic proteins and represents a promising approach for a new class of medicines with potentially broad therapeutic utility,” said Rick Morgan, Ph.D., chief scientific officer, Be Biopharma. “These data highlight the therapeutic modularity and unique advantages of our BCM platform.”

Today’s gene and cell therapies are potentially transformative medicines which can treat previously intractable diseases. However, there are barriers to the broad adoption of these therapies as current platforms struggle with drawbacks including inability to titrate dose and reliance on toxic preconditioning, among other challenges. Terminally differentiated human plasma cells (PCs) derived from genetically engineered B cells — termed B Cell Medicines, or BCMs — potentially offer natural longevity1, capacity for high levels of protein secretion (thousands of Ig molecules/cell/sec)2, ability to engraft without preconditioning, and the ability to redose.

Study Summary: “Development of an ex vivo precision gene engineered B cell medicine platform and demonstration of engraftment without preconditioning in non-human primates”

BCMs are produced via a CRISPR/Cas9 engineering platform that achieves gene knockouts with greater than 90% efficiency as well as targeted HDR-mediated gene insertions at frequencies as high as 60%. A BCM prototype engineered to express firefly luciferase with a phenotype of >90% CD27+CD38+ was injected via IV into immunodeficient mice, demonstrating rapid bone-marrow-homing and durable engraftment (>100 days). Further illustrating the modularity of the BCM platform, B cells were engineered to produce either lysosomal storage disease (LSD) enzyme acid sphingomyelinase (ASM), clotting factor IX (FIX), or an anti-CD19/CD3 bispecific T cell engager. These examples demonstrated that BCMs can produce proteins with specific activity much higher than standard recombinant proteins (ASM), are stably expressed for over 4 months in vivo (FIX) and show efficacy in tumor treatment (anti-CD19/CD3 scFv). In order to assess engraftment in an immunocompetent model, an ex vivo method was developed to engineer, expand and differentiate non-human primate (NHP) peripheral blood B lymphocytes into PCs. Using zirconium-89-oxine cell radiolabeling and high sensitivity PET/CT tracking, ex vivo expanded and differentiated autologous PCs demonstrated rapid homing and engraftment in PC niches in unconditioned NHPs.

Details for the oral presentation of this study are as follows:

Title: “Development of an ex vivo precision gene engineered B cell medicine platform and demonstration of engraftment without preconditioning in non-human primates”

Lead Author: Hanlan Liu, Ph.D., MBA, Senior VP, Pipeline and Non-clinical Development, Be Biopharma

Presenter: Rick Morgan, Ph.D., Chief Scientific Officer, Be Biopharma

Oral Presentation #: OR-77

Date/Time: Friday, October 27, 11:00-13:00 CEST

Session: 11a: Gene editing: Towards clinical trials

 

The study was funded and conducted through a collaborative research and development agreement with the National Heart, Lung, and Blood Institute (NHLBI), part of the National Institutes of Health.

Disclaimer: The content is solely the responsibility of the authors and does not necessarily represent the official views of the National Institutes of Health.

Study Summary: “Development of an ex vivo precision gene engineered B cell medicine that produces highly active and sustained levels of acid sphingomyelinase for the treatment of Niemann-Pick disease”

Niemann-Pick disease (NPD) is an autosomal recessive lysosomal storage disease caused by acid sphingomyelinase (ASM) deficiency. Sphingomyelin accumulates in multiple organs leading to organ failure, and in the severest form causes death early in life. Currently, the only treatment which is approved in NPB, a subset of NPD, is an enzyme replacement therapy (ERT) requiring an intensive dose escalation phase followed by Q2W maintenance intravenous infusions. This study evaluated the potential of engineering B cells to produce therapeutic levels of ASM, thus offering a potential new treatment for NPD.

In this study, primary human B cells were expanded, engineered by CRISPR/Cas9 genome editing with an AAV-delivered template for homology-directed repair (HDR) to insert a constitutive promoter followed by the ASM gene, SMPD1, into a safe harbor locus, which showed up to 30% HDR. BCMs were subsequently expanded and differentiated in vitro. Engineered BCMs secreted ASM up to ~150 ng/1e6 cells/24hr as measured by ELISA. HAP1 SMPD1 knockout cells exposed to supernatant from SMPD1-engineered BCMs restored ASM activity. Liquid chromatography–mass spectrometry analysis demonstrated that HAP1 SMPD1 knockout cells treated with supernatant from SMPD1-engineered BCMs mitigated the accumulation of lyso-sphingomyelin and that BCM-produced ASM was approximately 10-fold more active than recombinant ASM made from CHO cells.

Details for the poster presentation of this study are as follows:

Title: “Development of an ex vivo precision gene engineered B cell medicine that produces highly active and sustained levels of acid sphingomyelinase for the treatment of Niemann-Pick disease”

Lead Author: Monika Musial-Siwek, Ph.D., Director Protein Science, Be Biopharma

Co-Presenters: Timothy Mullen, Ph.D., Senior Scientist, Be Biopharma, Anja Hohmann, Ph.D., Senior Director, Cell Engineering, Be Biopharma

Poster: P579

Poster Session: October 25, 17:00-18:15, and October 26 from 20:30 to 21:30 CEST

About B Cells – 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 Longwood Fund and 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.

1 Landsverk et al (2017) J Exp Med
2 Hibi and Dosch (1986) J Immunol; Eyer et al (2017) Nat Biotech

Contacts

Investors: ir@be.bio
Media: media@be.bio

Filed Under: Press Releases

October 11, 2023 by rbracey

CAMBRIDGE, Mass.–Be Biopharma, Inc. (“Be Bio”), a company pioneering the discovery and development of Engineered B Cell Medicines (BeCMs), today announced that Chief Executive Officer Joanne Smith-Farrell, Ph.D., will present at the annual Cell & Gene Meeting on the Mesa to be held October 10-12 in Carlsbad, California, and livestreamed globally.

Details regarding the Be Biopharma presentation at the conference are as follows:

Event: 2023 Cell & Gene Meeting on the Mesa

Presentation: About B Cells: A New Class of Cellular Medicines

Date: October 11, 2023

Time: 4:15-6:00pm PT

Location: Park Hyatt Aviara Resort, 7100 Aviara Resort Dr., Carlsbad, California 92011

Dr. Smith-Farrell will also participate on October 12 in an interactive discussion about resourcing strategies to support advancing innovation while meeting the practical and economic considerations of R&D.

Organized by the Alliance for Regenerative Medicine, the Cell & Gene Meeting on the Mesa is a three-day conference featuring more than 120 dedicated company presentations by leading public and private companies, highlighting technical and clinical achievements over the past 12 months in the areas of cell therapy, gene therapy, gene editing, tissue engineering, and broader regenerative medicine technologies, as well as over 100 panelists and featured speakers.

Virtual attendance is available, which includes a livestream of the Be Biopharma presentation and the ability to view all conference sessions on-demand. Please visit https://meetingonthemesa.com for full information including registration. Complimentary attendance at this event is available for credentialed investors and members of the media only. Investors should contact Savannah Bryant at sbryant@alliancerm.org and interested media should contact Stephen Majors at smajors@alliancerm.org.

About B Cells – 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 (BeCMs). BeCMs have the potential to be durable, allogeneic, redosable and administered without toxic conditioning. The promise of BeCMs 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 (BeCMs) 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 Longwood Fund and 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.

Filed Under: Press Releases

  • « Go to Previous Page
  • Page 1
  • Page 2
  • Page 3
  • Page 4
  • Page 5
  • Page 6
  • Go to Next Page »

Footer

Be Biopharma
LinkedIn X

info@be.bio

Contact Us

Careers

  • Our Company
    • About Be Bio
    • Life at Be Bio
    • Leadership
    • Board of Directors
    • Scientific Advisory Board
    • Investors
  • Our Science
    • About BCMs
    • Our Platform
    • Publications & Presentations
  • Patients & Caregivers
  • Investors & Media
    • News Releases
    • Publications & Presentations

©2023, Be Biopharma

Privacy Policy

logo
  • Our Company
    • About Be Bio
    • Life at Be Bio
    • Leadership
    • Board of Directors
    • Scientific Advisory Board
    • Investors
  • Our Science
    • About BCMs
    • Our Platform
    • Publications & Presentations
  • Patients & Caregivers
  • Investors & Media
    • News Releases
    • Publications & Presentations
  • Careers
  • Contact Us