Cellgentech provides an ex‑vivo gene‑therapy platform that harvests a patient’s own adipose tissue, introduces therapeutic genes into the cells (GMAC technology), expands them, and re‑implants them to produce missing proteins for rare metabolic and genetic disorders. The company also offers a patented high‑concentration, non‑freezing cell preservation solution that enables ambient‑temperature storage and transport, supporting both clinical use and contract‑manufacturing services for pharma and biotech partners.
Funding
Funding not disclosed
Founders
Product
Problem
Patients with rare genetic and metabolic disorders lack effective, disease‑modifying therapies, and existing cell‑based gene treatments require complex manufacturing, cold‑chain logistics, and specialized clinical infrastructure.
Solution
Cellgentech has built an ex‑vivo gene‑therapy platform that harvests a patient’s own adipose tissue, introduces therapeutic genes into the cells (GMAC technology), expands the modified cells, and re‑implants them to produce the missing protein in situ. The approach leverages autologous fat cells, which are abundant and easily accessed, enabling personalized treatment without immune rejection. In parallel, the company has patented a high‑concentration, non‑freezing cell preservation solution that maintains cell viability and function without cryogenic storage, simplifying logistics and supporting both clinical use and contract‑manufacturing (CDMO) services. Together, these technologies provide a scalable, regulatory‑compliant pathway for treating rare diseases such as familial LCAT deficiency, hemophilia A, Fabry disease, and other enzyme‑deficiency disorders.
Target Audience
Primary customers are pharmaceutical and biotech firms developing cell‑based gene therapies for rare diseases, as well as academic medical centers and hospitals seeking autologous regenerative treatments for metabolic and genetic disorders.
Features
- Autologous adipose‑derived cell platform (GMAC) that integrates therapeutic genes via viral or non‑viral vectors and expands cells ex‑vivo before autologous transplantation.
- Proven clinical‑grade manufacturing process meeting Japanese “first‑in‑human” regenerative‑medicine safety standards, with GMP‑qualified facilities for cell expansion and preservation.
- High‑concentration, non‑freezing cell preservation solution (patented, Japanese patent No. 7841789) that maintains cell viability at ambient temperature, eliminating the need for cryogenic storage and transport.
- Broad disease pipeline covering LCAT deficiency, hemophilia A, Fabry disease, and other rare metabolic disorders, with pre‑clinical and early‑phase clinical data published in peer‑reviewed journals.
- CDMO capability to produce gene‑modified adipose cells for third‑party partners, supported by strategic alliances with pharmaceutical companies (e.g., Kyowa, Fujifilm, etc.).
- Integrated regulatory support and clinical trial infrastructure through collaboration with Chiba University Hospital and participation in AMED‑funded projects.