CellGenTech

What CellGenTech does

CellGenTech, Inc. (セルジェンテック株式会社) is a privately held, clinical-stage Japanese bio-venture founded October 20, 2003 and headquartered in the Chiba University Inohana Innovation Plaza in Chiba-city, with a satellite Tokyo office opened in 2018. The company is built around the proprietary GMAC (Genetically Modified Adipocyte) platform — an ex-vivo gene therapy approach in which subcutaneous fat is harvested from the patient, adipocytes are isolated and expanded in a proprietary ceiling culture system, transduced with retroviral or lentiviral vectors carrying therapeutic genes, characterized under GCP-grade quality release, and re-implanted autologously to deliver sustained therapeutic-protein secretion. The end-to-end process from fat collection to administration takes approximately 21 days, and a non-frozen high-concentration preservation solution (Japanese Patent No. 7841789, registered March 30, 2026) supports a ready-to-use formulation. Multiple pipeline programs apply this platform: LCAT-GMAC for familial LCAT deficiency (world's first genetically modified human adipocyte implantation performed in February 2017), FVIII-GMAC and FIX-GMAC for hemophilia A and B respectively, GLA-GMAC for Fabry disease, plus earlier-stage research on BDNF-secreting adipocytes for neurodegenerative/retinal indications and insulin-secreting adipocytes for diabetes.

CellGenTech generates no commercial product revenue; its income base consists of government R&D grants from AMED, JST, NEDO, and METI and upfront/milestone/royalty economics from licensing arrangements. The company has executed exclusive co-development and commercialization agreements with DyDo Pharma for LCAT-GMAC in Japan (effective January 15, 2021) and with Kyorin Pharmaceutical for GLA-GMAC in Fabry disease in Japan (signed June 17, 2022). All clinical administration currently runs through Chiba University Hospital under Japan's Regenerative Medicine Safety Act, and the company has stated that future commercialization would proceed through licensing partners rather than direct distribution. A declared second growth vector is cell medicine CDMO services, anchored by the October 17, 2023 strategic investment from Fujimori Kogyo (now ZACROS Corporation), which explicitly cites plans to enter contract development and manufacturing for gene-modified human adipocytes.

The company's competitive position rests on an exclusive license from Eisai to foundational adipocyte-for-gene-therapy patents (US 7,820,438 B2; Japan 4879867), a joint patent with Chiba University on optimal adipocyte characteristics for gene transduction (PCT/JP2011/050919), and the new preservation-solution patent. Management combines a founder-CEO with prior gene therapy development experience at AnGes MG and large-pharma R&D veterans from Eisai, Astellas, and Seikagaku on the board, alongside an auditor with direct PMDA and OECD regulatory experience. CellGenTech operates with 11-50 employees, has raised equity through disclosed and undisclosed third-party share allotments with Nissui Pharmaceutical, Toho Holdings, DyDo, Kyorin, and ZACROS, and as of the input data has not completed an IPO or commercial launch.

Name

CellGenTech

Legal name

セルジェンテック株式会社

Website

https://cellgentech.com

Company type

Private

Founded year

2003

Operating status

Operating

Headcount range

11–50 employees

Short description

CellGenTech is a privately held, clinical-stage Japanese biotech developing ex-vivo gene therapies via its proprietary GMAC platform, which uses genetically modified patient-derived adipocytes to deliver sustained therapeutic proteins for rare genetic diseases including LCAT deficiency, hemophilia, and Fabry disease.

Ownership category

akta.pro rank

Product category

Gene Therapy & Regenerative Medicine

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

Lentiviral Vector Gene Therapy Developers (HLAAACAB)

akta.pro secondary industries

Gene Therapy for Rare/Monogenic Diseases (HLAAACAE), Cardiometabolic & Other Systemic Gene Therapies (HLAAACAI), Genetic & Genomic Rare Disease Therapeutics (HLAIAIAA)

Keywords

• Ex-vivo gene therapy
• Regenerative medicine
• Cell-based therapeutics
• Rare disease treatment
• Adipocyte gene delivery

Headquarters

HQ city

Chiba

HQ country

Japan

HQ region

Asia

Offices2 records

Markets served

GTM type

B2B

Offering type

Services

Cost components

Technology or R&D, Personnel, Operations, Marketing or Sales, Infrastructure

Revenue model

1. Licensing and Co-development Revenue: CellGenTech generates revenue through licensing agreements and co-development deals with pharmaceutical companies. Examples include DyDo Pharma (LCAT-GMAC, Japan) and Kyorin Pharmaceutical (GLA-GMAC for Fabry disease, Japan). These agreements grant exclusive co-development and commercialization rights in exchange for upfront payments, milestone payments, and royalties on future sales.
2. Contract Development and Manufacturing (CDMO): The company is developing capabilities for cell medicine product development and manufacturing (CDMO) services, as indicated by the Fujimori Kogyo (ZACROS) investment announcement which specifically references cell medicine product development and manufacturing contracting as a future business expansion.
3. Government Research Grants: CellGenTech receives government grants from AMED, JST, NEDO, and METI to fund R&D programs for various pipeline candidates including LCAT-GMAC, FVIII-GMAC, and platform technology development.

Go-to-market motion1 record

Distribution channels2 records

Marketing channels5 records

Core offering

CellGenTech develops cell-based gene therapy medicines using its proprietary GMAC (Genetically Modified Adipocytes) platform, an ex-vivo gene therapy approach that collects a patient's subcutaneous fat, isolates adipocytes, introduces therapeutic genes via retroviral or lentiviral vectors, and reimplants the modified cells to deliver sustained therapeutic proteins. The company's pipeline targets rare genetic disorders including familial LCAT deficiency, hemophilia A and B, and Fabry disease.

Product overview

CellGenTech is a bio-venture company developing cell-based gene therapy medicines using its proprietary GMAC (Genetically Modified Adipocytes) platform. The company operates as a single unified platform (GMAC) with multiple therapeutic pipeline products targeting rare genetic disorders. The core GMAC platform enables ex-vivo gene therapy where patient adipocytes are collected, gene-modified, and reimplanted for sustained therapeutic protein delivery. Pipeline products include LCAT-GMAC for familial LCAT deficiency, FVIII-GMAC for Hemophilia A, FIX-GMAC for Hemophilia B, GLA-GMAC for Fabry disease, BDNF-GMAC for neurological conditions, and Insulin gene-transduced adipocytes for diabetes. A supporting research product is the Non-Frozen Cell Preservation Solution for storing regenerative medicine products.

Differentiator

Problem solved

Functional benefit

Products and services

• GMAC Platform (Genetically Modified Adipocytes) A proprietary ex-vivo gene therapy platform that uses patient-derived adipocytes for sustained therapeutic protein delivery, involving subcutaneous fat collection, adipocyte isolation, gene transfer via viral vectors, and reimplantation.
• LCAT-GMAC LCAT gene-transduced adipocyte product for treatment of familial LCAT deficiency, providing sustained LCAT enzyme secretion to address cholesterol metabolism dysfunction, kidney damage, anemia, and vision problems.
• FVIII-GMAC Factor VIII gene-transduced adipocyte product for treatment of Hemophilia A, providing sustained clotting factor VIII delivery to address bleeding disorders.
• FIX-GMAC Factor IX gene-transduced adipocyte product for treatment of Hemophilia B (Christmas disease), providing sustained clotting factor IX delivery.
• GLA-GMAC GLA (alpha-galactosidase A) gene-transduced adipocyte product for treatment of Fabry disease, providing sustained enzyme replacement to address globotriaosylceramide (GB3) accumulation in organs.
• Non-Frozen Cell Preservation Solution A proprietary preservation solution for storing regenerative medicine products without freezing, enabling ready-to-use GMAC biogenesis after administration with several days of non-frozen storability.

Quantifiable outcome

• First-in-human LCAT-GMAC clinical study demonstrated safety and lipid metabolism improvement over 6-month observation period in familial LCAT deficiency patients (Chiba University, February 2017; results published in Heliyon 2022).
• +3 more outcomes

Named customers2 records

Segments3 records

Ideal customer profiles2 records

Technology focussed Yes

API detail

Has API

No

API docs

API detail

Core technology

AI maturity

App detail

Feature6 records

Partnerships

Five partnerships are on record, tiered core and minor.

• AMED (日本医療研究開発機構)coreStrategic or Co-development Partner · 5 October 2022Adopted for FY2022 development assistance project for non-clinical studies supporting IND filing for FVIII-GMAC (hemophilia A) gene therapy. AMED has supported multiple CellGenTech programs since 2015, including LCAT-GMAC development and broader regenerative medicine infrastructure projects.
• Kyorin Pharmaceutical (杏林製薬)coreStrategic or Co-development Partner · 17 June 2022Kyorin Pharmaceutical was granted exclusive co-development and in-licensing rights for GLA-GMAC (genetically modified adipocytes for Fabry disease) in Japan. Joint development and license agreement signed June 17, 2022. Both DyDo Pharma and Kyorin Pharmaceutical represent flagship co-development partnerships for CellGenTech's GMAC platform pipeline.
• DyDo Pharma (ダイドーファーマ)coreStrategic or Co-development Partner · 15 January 2021DyDo Pharma was granted exclusive co-development and co-commercialization rights for LCAT-GMAC for the treatment of familial LCAT deficiency in Japan. CellGenTech and DyDo Pharma entered into a licensing agreement effective January 15, 2021, covering joint development and exclusive sales rights for the LCAT-GMAC product in the Japanese territory.
• Takara Bio (タカラバイオ)minorStrategic or Co-development Partner · 31 May 2018Joint development agreement signed December 20, 2005 for gene transduction technology using RetroNectin for adipocyte-based gene therapy. Takara Bio contributed its RetroNectin method and lentiviral/retroviral vector technology, while CellGenTech contributed its adipocyte culture and preparation expertise. This foundational partnership established the technical basis for the GMAC platform.
• Chiba UniversitycoreStrategic or Co-development Partner · 1 February 2017CellGenTech and Chiba University's Department of Cell Therapy (led by Prof. Saito) developed the foundational GMAC technology through long-standing translational research collaboration. Chiba University Hospital conducted the world's first LCAT-GMAC clinical study (first patient in February 2017). Multiple joint patents filed (e.g., PCT/JP2011/050919 on cell population evaluation methods).

Scale indicators5 records

Recent moves8 records

Expansion highlights6 records

Emerging players

• Regenxbio: Clinical-stage gene therapy company developing AAV-based therapies with a focus on rare diseases including hemophilia A/B. Competes with CellGenTech's hemophilia franchise (FVIII-GMAC, FIX-GMAC) but uses a different delivery modality (in vivo AAV).
• Takara Bio: Japanese biotechnology company specializing in lentiviral/retroviral vector technology and cell engineering — directly comparable to CellGenTech's GMAC platform from a tools/enabling technology standpoint. Also a disclosed CellGenTech partner (RetroNectin joint development, 2005).
• Sangamo Therapeutics: Clinical-stage genomic medicine company developing in vivo genome editing and gene therapy for rare diseases including Fabry disease and hemophilia — directly overlapping with CellGenTech's GLA-GMAC and hemophilia programs but using a different modality (zinc finger nucleases vs. ex-vivo adipocyte transduction).
• CRISPR Therapeutics: Clinical-stage gene editing company developing CRISPR/Cas9-based therapies for rare genetic diseases including hemophilia. Indirect competitive threat to CellGenTech's gene therapy pipeline as gene editing technologies could displace ex-vivo cell-based approaches over time.

Direct peers

• bluebird bio: Gene therapy company developing lentiviral-based ex-vivo cell therapies for rare genetic diseases. Most directly comparable to CellGenTech in terms of ex-vivo cell modification approach and rare monogenic disease focus, though bluebird focuses on hematopoietic stem cells rather than adipocytes.
• uniQure: Clinical-stage gene therapy company developing AAV-based therapies for rare diseases including hemophilia. Comparable to CellGenTech as a rare-disease-focused gene therapy developer with a multi-indication pipeline strategy, though using a different delivery modality (in vivo AAV vs. CellGenTech's ex-vivo adipocyte).
• AnGes MG: Japanese clinical-stage gene therapy company developing nucleic acid and gene therapies for rare diseases. Highly comparable Japanese-domiciled peer in the cell/gene therapy space; notably, CellGenTech's CEO Masayuki Aso previously served at AnGes MG, indicating direct lineage and technology proximity.

Broad incumbents

• Spark Therapeutics (Roche): Gene therapy subsidiary of Roche focused on AAV-based therapies for rare inherited diseases, including hemophilia and inherited retinal diseases. Broad incumbent competing with CellGenTech's gene therapy pipeline in overlapping indications (hemophilia, retinitis pigmentosa/BDNF-GMAC).
• BioMarin Pharmaceutical: Established rare-disease biopharma with multiple approved enzyme replacement and gene therapies, including Roctavian (AAV5 gene therapy) for hemophilia A — directly competing with CellGenTech's FVIII-GMAC program in the same indication. Broad incumbent in rare genetic disease therapeutics.

Regional players

• Pluristem Therapeutics: Clinical-stage cell therapy company developing placenta-derived cell therapies for rare diseases. Comparable to CellGenTech as a cell-based therapeutics company targeting rare diseases, though using a different cell type (allogeneic placenta vs. autologous adipocytes) and primarily serving Western markets.

Market position

Strengths5 records

Weaknesses5 records

Competitive moat4 records

Key risks7 records

Key highlights7 records

Customer concentration

Revenue estimate

Valuation estimate

Number of profiles

Profiles8 records

Funding overview

Funding rounds3 records

Investors3 records

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M&A

Investments

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

Frequently asked questions about CellGenTech