Targeting the Research and Development of Drugs for New Gene Therapy and Regenerative Medicine -CellGenTech, Inc.-

Research & Development

We focus on regenerative medicine and protein supplemental therapy, which are critical to improve patient's quality of life. We pursue safe and secure treatments utilize our proprietary technology of transduction of pre-adipocytes, especially for intractable diseases and orphan diseases.

Protein supplemental therapy by transduced adipocytes

Our platform technology is based on autotransplantation of transduced adipocytes which produce therapeutic proteins. The applications can be various in a wide range of diseases. Current lead pipeline targets reverse cholesterol transport enzyme in congenital metabolic disorder, which is in preclinical.
More programs including Factor VIII for Hemophilia A, α-Galactosidase for Fabry's Disease and Diabetes are on-going.

Particular Features

Solutions for rare diseases, intractable diseases, and lifestyle-related diseases

Our target is to provide a new therapy which offers not only therapeutic management of the disease but also achieves substantial improvement and maintenance of the patient's quality of life(QOL), releasing them from the burden caused by current therapy, if any.
In particular, focusing on the rare diseases and intractable diseases where therapeutic drugs are not yet available, we are making best effort to develop "transduced human adipocyte-based medicine" which is designed to release the therapeutic protein(s) stably in the patients and contribute to the benefit of those patients.

Development of the transduced human adipocyte-based medicine for intractable diseases

Many intractable diseases are caused by deficiency or shortage of dispensable enzyme or protein in patients. We are developing a "transduced adipocyte-based medicine" capable of supplying the protein(s) in need to the patients, by introducing the gene to the human adipocytes. We are continuing to strengthen our technology platform of "transduced adipocyte-based medicine" and also to conduct safety evaluation of our products for clinical use with most concern.

Intellectual property

Patent

1. "Primary cultured adipocytes for gene therapy"

Cellgentech Inc. obtained the exclusive right for clinical use from Eisai Co. Ltd.
The patent claims the innovative gene therapy where the therapeutic gene is introduced to the patient's own adipocytes ex vivo and after establishing the sustained secretion of the therapeutic protein, the adipocytes are implanted back to the patient.

Patent granted:
U.S. A. US 7,820,438 B2(2010)
Japan Patent No.4879867 (2011)

2. "Method for determination whether or not cell population is suitable for foreign gene transfer for the purpose of preparation of adipocytes for gene therapy application" (PCT/JP2011/050919)

Cellgentech, Inc. and Chiba University are co-applicants of this patent.
The patent claims the most suitable characteristics of the human adipocytes for use of ex vivo gene transduction and the method for determination of the eligibility.
We have established the manufacturing process based on the patent.