mkendis

February 1, 2024 by mkendis

San Diego, CA – Be Biopharma, Inc. (“Be Bio”), a company pioneering the development of engineered B Cell Medicines (BCMs), today announced that it will present at the 20^th Annual WORLDSymposium™ being held February 4-9, 2024, in San Diego, California.

Details regarding the Be Biopharma presentation at the conference 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 Neimann-Pick disease

Presenter: Monika Musial-Siwek, Ph.D., Director, Protein Sciences, Be Biopharma

Date: Thursday, February 8, 2024

Time: 1:00 – 2:00 PM PT

For more information, please visit the conference website worldsymposia.org.

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 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.

Investor Contact:

ir@be.bio

Media Contact:

media@be.bio

January 25, 2024 by mkendis

Data selected as Oral Presentation Supporting the Broad Therapeutic Utility of B Cell Medicines

Santa Fe, NM – Be Biopharma, Inc. (“Be Bio”), a company pioneering the development of engineered B Cell Medicines (BCMs), today announced an oral presentation and poster presentation at the Keystone Symposia on Emerging Cellular Therapies meeting, held from January 22 to 25, 2024 in Santa Fe, New Mexico. The data being presented support the utility of engineered B cell medicines (BCMs) across a broad range of applications. Together, the data demonstrate in vitro production and activity of several therapeutic proteins, as well as in vivo engraftment without preconditioning and activity in multiple model systems.

“Gene and cell therapies have transformative potential to treat previously intractable diseases, but have barriers to adoption such as lack of durability, inability to re-dose, and requirement of preconditioning for engraftment. Our BCM platform has the potential to address these barriers,” said Richard A. Morgan, Ph.D., Chief Scientific Officer, Be Bio. “The in vivo preclinical data presented today validates key attributes of our BCM platform – efficient protein production, editing and insertional efficiency, and durable protein production without preconditioning – while demonstrating its potential therapeutic benefits in genetic disease and cancer.”

Presentation Summary: Development of an Ex Vivo Precision Gene Engineered B Cell Medicine Platform that Produces Active and Sustained Levels of Therapeutic Proteins with Broad Utility

BCMs were produced via a precision genome engineering platform that achieves gene knockouts with greater than 90% efficiency and targeted HDR-mediated gene insertions at up to 60%. Modularity of the BCM platform was demonstrated across multiple models and proteins, including production of firefly luciferase, lysosomal storage disease enzyme acid sphingomyelinase, an anti-CD19/CD3 bispecific T cell engager, clotting factor IX, and fusion protein of tissue nonspecific alkaline phosphatase (ALP). These examples demonstrate that these BCMs can produce proteins with enzyme specific activity higher than recombinant proteins (ASM), show efficacy in tumor treatment (anti-CD19/CD3 scFv), and are stably expressed for at least 20 weeks in vivo (FIX). Engraftment in all models was achieved without preconditioning which broadens BCM clinical utility for patients for whom preconditioning toxicities are unacceptable or outweigh therapeutic benefit, and could facilitate additional rounds of treatment as needed. BCMs capable of expressing therapeutically relevant transgenes have the potential for broad and meaningful therapeutic utility in genetic diseases, cancer, and beyond.

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

Title: “Development of an Ex Vivo Precision Gene Engineered
B Cell Medicine Platform that Produces Active and Sustained Levels of Therapeutic Proteins with Broad Utility”

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

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

Date/Time: Tuesday, January 23, 2024, 5:00-7:00pm (MST)

Session: Strategies for Engineered Cell Therapies

Poster Summary: Development of an Ex Vivo Precision Gene Engineered B Cell Medicine Platform that Produces Active and Sustained Levels of Therapeutic Proteins with Broad Utility in Rare Diseases and Cancer

This study demonstrates production of BCMs via editing, expansion and differentiation of primary human B cells. Cells engineered to express firefly luciferase were engrafted into NOG-hIL6 mice and demonstrated in vivo persistence for 125 days. Data are also presented on BCMs engineered to produce FIX, which demonstrates activity via the chromogenic and aPTT assay. These BCMs were engrafted into NOG-hIL6 mice and demonstrate durable FIX secretion to 168 days. In addition to FIX, BCMs were engineered to produce an anti-CD19/CD3 bispecific T cell engager, which demonstrated potent activity in an in vivo PdX model of ALL, ALP, which corrected HPP-related bone mineralization deficits in vitro, and ASM, which demonstrated in vitro phenotypic correction in SMPD1 knockout cells. In addition, ex vivo-rhesus macaque plasma cells were generated and labeled with a radioactive tracer. These cells were administered without preconditioning and demonstrated homing to and engraftment in plasma cell niches in an autologous rhesus macaque.

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

Title: “Development of an Ex Vivo Precision Gene Engineered B Cell Medicine Platform that Produces Active and Sustained Levels of Therapeutic Proteins with Broad Utility in Rare Diseases and Cancer”

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

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

Poster #: 2

Poster Session: Wednesday, January 24, 2024, 7:30pm (MST)

About Engineered B Cell Medicines – A New Class of Cellular Medicines

About Be Biopharma

Investor Contact:

ir@be.bio

Media Contact:

media@be.bio

January 4, 2024 by mkendis

Angus Smith joins as Chief Financial Officer

Wing-Yen Wong, M.D., joins as interim Chief Medical Officer

Cambridge, Mass. – Be Biopharma, Inc. (“Be Bio”), a company pioneering the development of engineered B Cell Medicines (BCMs), today announced the appointment of Angus Smith as Chief Financial Officer (CFO) and Wing-Yen Wong, M.D., as interim Chief Medical Officer (CMO).

Mr. Smith brings more than 15 years of experience in the biotechnology and life sciences industry, having held C-level positions in several public companies, including his most recent role as the CFO of Affimed N.V. and co-President of the company’s U.S. subsidiary, where he was responsible for implementing a new business plan and for raising over $400M in capital to support clinical development efforts. Mr. Smith also brings nearly a decade of investment banking experience, providing strategic and financial advice across the healthcare sector. Dr. Wong brings over 35 years of experience as a practicing clinician and professor at Children’s Hospital of Los Angeles, and leading development efforts in several companies including BioMarin, Biogen, and Baxter. Most recently Dr. Wong was Group Vice-President, Head of Global Medical Affairs and Scientific Strategy at BioMarin where she was involved in the development of the most recently approved hemophilia gene therapy.

“We are thrilled to welcome Angus and Wing to Be Bio,” said Joanne Smith-Farrell, Ph.D., Chief Executive Officer. “We are rapidly transitioning from a research-based company to one focused on delivering innovative medicines to patients, with our first program in hemophilia B entering the clinic this year. These accomplished and experienced leaders will further strengthen our leadership team. Angus’s long track record of financial operating experience and fundraising will be important as we advance multiple programs to the clinic, while Wing’s expertise in drug development and as a clinical scientist is particularly important as we pioneer a new class of cellular medicines, Engineered B Cell Medicines (BCMs), to dramatically improve the lives of patients.”

Angus Smith

Mr. Smith brings more than 15 years of experience in the biotechnology and life sciences industry, including his most recent role as the CFO of Affimed N.V. and, prior to that, at Rockwell Medical. At Affimed, Mr. Smith led all finance, accounting, and investor relations functions, implemented a new business plan and financing strategy, and raised more than $400 million. Mr. Smith began his career in healthcare investment banking, serving most recently as a Director in the Healthcare Investment Banking Group at Cantor Fitzgerald. During his nearly decade-long investment banking tenure, Mr. Smith provided strategic and financial advice to life sciences and healthcare companies. He has worked on a substantial number of transactions across the healthcare sector with an aggregate transaction value of more than $15 billion.

Mr. Smith holds a B.A. in Mathematical Economics from Colgate University.

Wing-Yen Wong, M.D.

Dr. Wong brings over 35 years of experience as a practicing clinician and working in the biotechnology industry with a focus in the treatment and management of patients with hemophilia, sickle cell disease (SCD) and pediatric hematology/oncology. Most recently Dr. Wong was Group Vice-President, Head of Global Medical Affairs and Scientific Strategy at BioMarin and Vice President, Global Medical, Hematology and Immunology at Biogen. Prior to Biogen, Dr. Wong was the Head of Clinical Research and Global Senior Medical Director of Hemophilia/Hematology at Baxter Healthcare Corporation where she directed medical and clinical functions, including global medical support of licensed products, clinical development and new product approvals. During her tenure at Baxter, Dr. Wong successfully directed the BLA submission and launch of multiple hemophilia products including RIXUBIS®, FEIBA®, OBIZUR and ADVATE®. Prior to her work in industry, Dr. Wong spent over 15 years as a practicing clinician and professor at Children’s Hospital Los Angeles and the Los Angeles County (LAC) and University of Southern California (USC) Medical Center. As a clinician, Dr. Wong presented and published numerous studies and papers on her research in hemophilia and hematology and has served as a reviewer for medical journals including the American Journal of Hematology, Hemophilia and the Lancet. In 2003, Dr. Wong was appointed by the Secretary of Health and Human Services as a member of the Advisory Committee on Blood Safety and Availability.

Dr. Wong earned her M.D. at the USC, Keck School of Medicine and a BA at Occidental College. She served a Fellowship at the LAC and USC Medical Center in pediatric hematology-oncology and was appointed Associate Professor of Pediatrics at the University of Southern California.

About Engineered B Cell Medicines – A New Class of Cellular Medicines

About Be Biopharma

Investor Contact:

ir@be.bio

Media Contact:

media@be.bio

December 28, 2023 by mkendis

CAMBRIDGE, Mass.—Be Biopharma, Inc. (“Be Bio”), a company pioneering the discovery and development of Engineered B Cell Medicines (BCMs), today announced that Chief Executive Officer Joanne Smith-Farrell, Ph.D., will present at the 42nd Annual J.P. Morgan Healthcare Conference being held at the Westin St. Francis in San Francisco, CA on Wednesday, January 10, 2024.

42nd Annual J.P. Morgan Healthcare Conference
Presentation Date: Wednesday, January 10, 2024
Presentation Time: 8:30 am PT

About Engineered B Cell Medicines – A New Class of Cellular Medicines

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

Investor Contact:
ir@be.bio

Media Contact:
media@be.bio

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 65^th 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 65^th 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.²While 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

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

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

About Be Biopharma

Contacts

Investors:
ir@be.bio

Media:
media@be.bio

mkendis

Contacts

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