What Be Biopharma does

Be Biopharma is a clinical-stage biotechnology company founded in October 2020 by David Rawlings, M.D., and Richard James, Ph.D., from Seattle Children's Research Institute. The company is headquartered in Cambridge, Massachusetts, and operates as a privately held Delaware corporation with no parent company. As of 2025 it employs between 51 and 100 people and has raised over $356 million in cumulative investor commitments across a $52 million Series A (2020), a $130 million Series B (2022), an $82 million financing (2024), and a $92 million Series C (2025), backed by ARCH Venture Partners, Atlas Venture, RA Capital Management, Bristol Myers Squibb, Takeda Ventures, Alta Partners, Longwood Fund, Seattle Children's Research Institute, Pathway to Cures, and Nextech.

The company's product platform is Engineered B Cell Medicines (BCMs), which use CRISPR/Cas9 and AAV-mediated homology directed repair to insert therapeutic genes into primary human B cells. These engineered B cells are expanded ex vivo and differentiated into plasma cells that engraft in bone marrow without preconditioning and secrete therapeutic proteins at constant levels. The company's lead program, BE-101, is in a Phase 1/2 trial (BeCoMe-9) for hemophilia B and has received FDA Orphan Drug and Fast Track designations. A second program, BE-102, was developed using AI-guided protein design and is being advanced toward IND for hypophosphatasia. The company is also pursuing oncology applications of the BCM platform, including sustained delivery of bispecific T cell engagers. Manufacturing is supported by a strategic collaboration with Resilience.

Be Biopharma is pre-revenue and does not currently generate product sales. Its future revenue model is expected to rely on commercialization of BCM therapeutics upon regulatory approval, supported by clinical development partnerships and ongoing interactions with the FDA. Distribution is currently limited to clinical trial sites, with the BeCoMe-9 trial planning to enroll up to 24 participants across two parts monitored for approximately 52 weeks. The company's customer segments are patient populations with serious genetic diseases: hemophilia B patients (approximately 40,000 globally), hypophosphatasia patients (approximately 50,000 in the US and over 100,000 worldwide), and oncology patients who could benefit from sustained delivery of antitumor biologics.

Name

Be Biopharma

Legal name

Be Biopharma, Inc.

Website

https://be.bio

Company type

Private

Founded year

2020

Operating status

Operating

Headcount range

51–100 employees

Short description

Be Biopharma is a clinical-stage biotechnology company developing Engineered B Cell Medicines (BCMs), using CRISPR/Cas9 and AAV-mediated HDR to engineer B cells that durably secrete therapeutic proteins for patients with hemophilia B, hypophosphatasia, and cancer.

Ownership category

akta.pro rank

Product category

Cell Therapy / Biopharmaceuticals

NAICS

Biological Product (except Diagnostic) Manufacturing (325414), Research and Development in Biotechnology (except Nanobiotechnology) (541714)

SIC

Biological Products, (No Disgnostic Substances) (2836)

akta.pro primary industry

Blood Factors & Hemostasis Biologics (recombinant clotting factors, thrombolytics) (HLAAAAAL)

akta.pro secondary industries

Recombinant Protein Therapeutics (enzymes, hormones, growth factors) (HLAAAAAB), Hematology Specialty Pharmaceuticals (Non-malignant) (HLAIACAI)

Keywords

• Engineered B cell therapies
• Cell therapy platform
• Biopharmaceutical drug development
• Protein replacement therapy
• Rare disease therapeutics

Headquarters

HQ city

Cambridge

HQ country

United States

HQ region

North America

Offices1 record

Markets served

GTM type

B2B

Offering type

Hardware or Manufacturing

Cost components

Technology or R&D, Personnel, Operations, Supply Chain, Marketing or Sales

Revenue model

1. Biopharmaceutical Product Revenue: As a clinical-stage company, Be Bio does not yet generate revenue. The company is developing Engineered B Cell Medicines (BCMs) for genetic diseases (hemophilia B, hypophosphatasia) and cancer. Future revenue is expected from commercialization of BCM therapeutics upon regulatory approval.

Go-to-market motion1 record

Distribution channels1 record

Marketing channels6 records

Core offering

Be Biopharma develops Engineered B Cell Medicines (BCMs), a new class of cellular medicines in which primary human B cells are precision-engineered ex vivo using CRISPR/Cas9 and AAV-mediated homology directed repair to secrete therapeutic proteins continuously after engraftment as plasma cells in the bone marrow. The company's lead product candidate BE-101 is in Phase 1/2 clinical trials (BeCoMe-9) for hemophilia B, expressing human Factor IX; BE-102 is a preclinical development candidate for hypophosphatasia expressing tissue-nonspecific alkaline phosphatase.

Product overview

Be Biopharma is pioneering Engineered B Cell Medicines (BCMs), a new class of cellular medicines built on a programmable platform. The core BCM platform enables precise genome editing of a patient's own B cells to produce therapeutic proteins continuously. The company has two named product candidates: BE-101 (for hemophilia B, in Phase 1/2 clinical trials) and BE-102 (for hypophosphatasia, in preclinical development). Both programs leverage the BCM platform's ability to create durable, titratable, and re-dosable therapeutics without preconditioning.

Differentiator

Problem solved

Functional benefit

Products and services

• BE-101 BE-101 is a first-in-class Engineered B Cell Medicine (BCM) engineered using CRISPR/Cas9 and AAV-mediated HDR to insert the human Factor IX (FIX) gene into primary human B cells for the treatment of hemophilia B. BE-101 is designed to deliver continuous expression of active FIX with a single infusion, with potential to be titratable and re-dosable, without the need for preconditioning or immunosuppression.
• BE-102 BE-102 is a first-in-class Engineered B Cell Medicine engineered using AI-guided protein design to modify primary human B cells to produce tissue-nonspecific alkaline phosphatase (TNSALP/ALP) for the potential treatment of hypophosphatasia. BE-102 is designed to provide continuous secretion of therapeutic ALP from a single infusion without the need for preconditioning.

Quantifiable outcome

• BE-101 demonstrated stable FIX production for >168 days in preclinical studies
• +4 more outcomes

Segments3 records

Ideal customer profiles3 records

Technology focussed Yes

API detail

Has API

No

API docs

API detail

Core technology

AI maturity

App detail

Feature5 records

Partnerships

Three partnerships are on record, tiered strategic and core.

• ResiliencestrategicStrategic or Co-development Partner · 19 May 2022Strategic collaboration announced May 19, 2022 to manufacture engineered B cells, a new class of cellular medicines. Resilience provides manufacturing capabilities for Be Bio's BCM platform.
• Seattle Children's Research InstitutecoreStrategic or Co-development PartnerTechnology origin - Be Bio was founded by B cell engineering pioneers David Rawlings, M.D., and Richard James, Ph.D., from Seattle Children's Research Institute. The institute is an investor and research collaborator. Joint research conducted on bispecific T cell engagers for leukemia treatment.
• National Heart, Lung, and Blood Institute (NHLBI)strategicStrategic or Co-development PartnerCollaborative research and development agreement with NHLBI, part of the National Institutes of Health, to study engraftment of ex-vivo-generated plasma cells in non-human primates. Study published in Molecular Therapy journal.

Scale indicators6 records

Recent moves6 records

Expansion highlights7 records

Direct peers

• uniQure: Clinical-stage gene therapy company whose lead asset etranacogene dezaparvovec (Hemgenix) is the first approved gene therapy for hemophilia B. Competes head-to-head with BE-101 in hemophilia B but uses a one-time AAV approach rather than re-dosable engineered B cells.
• CRISPR Therapeutics: Leading CRISPR/Cas9-based gene editing company developing CTX001 (Casgevy) for sickle cell disease and beta-thalassemia. Uses similar gene editing modality as Be Bio's BCM platform and targets similar patient populations with durable genetic disease cures.
• Sangamo Therapeutics: Clinical-stage gene editing company developing zinc finger nuclease-based therapies, including programs for hemophilia A and B. Uses genome editing to produce therapeutic proteins from patient cells, conceptually similar to Be Bio's B cell engineering approach.

Broad incumbents

• Pfizer (Hemophilia Franchise): Markets BeneFIX (recombinant Factor IX) and acquired Global Blood Therapeutics. A major incumbent in hemophilia B treatment and a likely strategic acquirer/partner for cell therapy developers seeking commercial infrastructure.
• BioMarin Pharmaceutical: Developer of Roctavian, the first approved AAV gene therapy for hemophilia A, with active programs in hypophosphatasia (Voxzogo) and rare diseases directly overlapping Be Bio's BE-101 and BE-102 target indications.
• CSL Behring: Global leader in plasma-derived and recombinant coagulation therapies (Idelvion, Afstyla for hemophilia B; asfotase alfa/Strensiq for hypophosphatasia). Directly competes in both of Be Bio's lead indications with established patient and provider relationships.

Emerging players

• StrideBio: Emerging gene therapy company developing AAV-based treatments for rare diseases including hemophilia. Competes with Be Bio in the protein-replacement space but via one-time gene therapy rather than re-dosable cellular medicine.
• Generation Bio: Clinical-stage genetic medicines company developing non-viral lipid nanoparticle-based gene therapies for hemophilia and other rare diseases. Represents an alternative redosable gene therapy approach competing with Be Bio's redosability advantage.
• Sigilon Therapeutics: Clinical-stage cell therapy company developing engineered cell encapsulated therapies for rare diseases including hemophilia A and lysosomal storage disorders. Competes with Be Bio on protein replacement from engineered cells without preconditioning.
• Capstan Therapeutics: Emerging engineered cell therapy company focused on in vivo cell engineering using mRNA-LNP delivery. Operates in adjacent cell engineering space targeting similar protein-replacement and autoimmune indications as Be Bio.

Market position

Strengths5 records

Weaknesses5 records

Competitive moat4 records

Key risks5 records

Key highlights7 records

Customer concentration

• Xx.com/bebiopharma
• LinkedInlinkedin.com/company/be-biopharma-inc

Compliance2 records

Revenue estimate

Valuation estimate

Number of profiles

Profiles9 records

Funding overview

Funding rounds4 records

Investors8 records

Funding detail is available on the Subscription and Enterprise plan.Contact sales →

M&A

Investments

M&A and investment is available on the Subscription and Enterprise plan.Contact sales →

Frequently asked questions about Be Biopharma