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

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We are developing “therapeutic gene transduced adipocyte-based medicine” for better treatment of hereditary diseases deficient in enzymes or protein factors.

Overview of “transduced adipocyte-based medicine” project

Overview of “transduced adipocyte-based medicine” project, supplying therapeutic protein(s) for various diseases

Overview of project

1. Contribution to the treatment of familial LCAT deficiency and Establishment of Proof of Concept 2. Expand the new therapy to rare diseases 3. Toward lifestyle-related diseases 4. Further applications beyond

1. Contribution to the treatment of familial LCAT deficiency and Establishment of Proof of Concept

Familial LCAT deficiency is a hereditary disease where the gene for the enzyme, LCAT is genetically deficient. LCAT has a function to increase so-called good cholesterol level. Familial LCAT deficiency is a rare disease accompanied with dysfunction in eye or kidney, and no therapy is available to date. The manufacturing process was established for LCAT gene transduced adipocyte-based medicine which can afford sustained release of LCAT to familial LCAT deficiency patients. The clinical research at Chiba University(collaborating institute) is scheduled and the application was submitted to MHLW in 2010. MHLW has fully reviewed and discussed important matters concerning safety and efficacy and MHLW approved conducting of the clinical research May. 2013 by MHLW. As the new regulation of the regenerative medicine was effective in 2014, repackaged the protocol of the clinical research for the class1 regenerative medicine at Chiba University Hospital and CMC and preclinical file to MHLW (Jun. 2016). The protocol of the clinical research and the data package had been reviewed and the conducting of the clinical research was approved by MHLW Aug. 8th 2016 .
First Patient In was conducted at Feb. 2017 and the promising result has gotten by the one time of the autologous transplantation of LCAT gene transduced adipocyte during 6months. We have established the Proof Concept of The Gene therapy and Regenerative Cell Medicine with Gene-transduced Adipocyte.

2. Expand the new therapy to rare diseases

There are many patients and their family who are suffered from various intractable diseases caused by inherited gene deficiency for indispensable enzyme or protein. We are continuing to expand our technology of transduced adipocytes for gene therapy to other rare and intractable diseases, such as lysosome disease and hemophilia. We conducted the feasibility studies of transduced adipocytes for gene therapy for lysosome disease and hemophilia by utilizing our technology, which are supported by Japanese Science and Technology Agency and evaluated the feasibility for these diseases.

3. Toward lifestyle-related diseases

New therapy of more smart insulin supply is awaited by many diabetic patients who have trouble to control blood glucose level because of the dysfunction of insulin secretion and/or other various disorders. We are developing the new insulin supplement therapy for severe diabetic patients by expanding our transduced adipocyte technology.

4. Further applications beyond

There have been many reports accumulated about suppressing intrinsic factor(s) which inhibits the onset and/or progression of arteriosclerotic disease, cancer or degenerative neuropathies. We are also continuing to apply our transduced adipocytes technology to those fields, developing the system for long term stable supply of therapeutic protein(s) to block the progression of those intractable diseases and to contribute to improve the QOL of the patients.