IZumi Bio And Kyoto University Form Collaboration To Advance Induced Pluripotent Stem Cell Technology

Main Category: Stem Cell Research
Article Date: 15 Apr 2009 - 3:00 PDT

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iZumi Bio, Inc., and Kyoto University's Center for iPS Cell Research and Application (CiRA), today announced a collaboration to promote the basic research, development and application of induced pluripotent stem (iPS) cell technology - a form of cellular reprogramming which originated in Japan - with the goal of advancing drug discovery and enabling cell-based therapies.

"Stem cell research holds great promise for the creation of new therapies that could revolutionize the treatment of disorders such as Parkinson's disease, diabetes and muscular dystrophy. The discovery that iPS cell technology brings, that "stem cell-like" cells can be generated from a small amount of human skin rather than from embryos, opens a new door for stem cell research and its application to therapeutic discovery," said Al Gore, former Vice President of the United States and a partner at venture capital firm Kleiner Perkins Caufield and Byers. Noting that Science Magazine named cellular reprogramming the "breakthrough of the year" in 2008, Gore concluded, "The partnership between these two leading organizations is a critical step in furthering this research and turning stem cell research into therapeutic realities sooner."

Through the collaboration, iZumi and CiRA will exchange part of their representative human iPS cell lines derived by various methods. The two organizations also will conduct comparison and characterization studies independently but will share their results to determine which methods produce the most appropriate iPS cell lines for drug screening and development, and those most suitable for cell-based therapy.

"This collaboration, which is focused on the advancement of iPS cell technology, will help us attain our goal of making drug discovery and development faster, more efficient and informed in order to create new therapeutics for unmet medical needs. Our approach is based on a paradigm shift that puts the patient at the forefront of the drug discovery process. Using patient and disease-specific cells to develop assays and screening systems has the potential to change the way drug discovery has been conducted to date by shortening the time to clinic through the selection of drug candidates with a higher probability of success," said John P. Walker, chief executive officer of iZumi. "We will initially focus on three neurological disorders, including Parkinson's disease, spinal muscular atrophy and amyotrophic lateral sclerosis, because these conditions currently have limited therapeutic treatments, and because scientists have demonstrated the ability to differentiate the affected cell types in these disorders. Through our collaboration with The Gladstone Institute, we also are focusing on cardiovascular disorders, including calcific aortic valve disease."

iZumi and CiRA respectively offer the capability to take skin cells from a diseased patient with known genotypic and phenotypic information and reprogram the cells to behave similarly to human embryonic stem cells. These iPS cells can then be changed into various cell types in the body using directed differentiation. The differentiated cells are then transformed into cell-based, disease-specific assays to identify small molecules, biologics or other agents for drug discovery.

"We are very pleased to collaborate with iZumi, a major biotechnology venture in the United States, on the basic research to help advance this important technology. I expect that this collaboration will contribute to establishing an evaluation method for selecting safe iPS cell lines and accelerating the development of iPS cell technology globally." said Dr. Shinya Yamanaka, director of Center for iPS Research and Application (CiRA) at Kyoto University. "To realize clinical applications of iPS cell technology as soon as possible, CiRA will form partnerships with research institutions both in and outside of Japan to promote global collaboration."

In 2006, Dr. Yamanaka announced that his lab succeeded in inducing iPS cells in mouse by using four transgenes - Oct3/4, Sox2, Klf4 and c-Myc - delivered into fibroblasts via retroviral vectors, which became the world's first report of its kind. He also reported the generation of human iPS cells the following year. iPS cell technology has the potential to avoid ethical issues and the risks of immune rejection - major obstacles for embryonic stem cell use in the clinic. Dr. Yamanaka's seminal papers have led to an explosion of renewed interest and focus in stem cell research worldwide. Recognized as a pioneer in the field of iPS cell technology, he now conducts research activities mainly at CiRA.

About iZumi Bio

iZumi Bio is a South San Francisco-based biotechnology company initially focused on the industrialization of induced pluripotent stem cell (iPS) technology with the mission of creating new therapeutics through cellular reprogramming and directed differentiation of patient cells. Izumi was founded in 2007 and is backed by Kleiner Perkins Caufield and Byers and Highland Capital Partners. iZumi has a collaboration with The Gladstone Institute of Cardiovascular Disease and Deepak Srivastava, M.D., in the area of cardiovascular disease to identify potential targets that address unmet medical need.

iZumi's technology involves reprogramming somatic cells to a pluripotent state. Because iPS cells have stem-cell-like properties and have the potential to differentiate into any cell type, iPS cells are considered to be of great potential for disease research, preclinical drug testing and cell-based therapy. iZumi's approach places the patient at the forefront of the drug discovery process, which could reduce drug development time and increase the probability of success for drug candidates. iZumi plans to use cellular reprogramming, including its iPS technology, to find new molecular targets and develop proprietary therapeutic small molecule or biologic drugs for its own pipeline to treat specific diseases.

About Kyoto University's Center for iPS Cell Research and Application (CiRA)

CiRA was established at Kyoto University in January 2008, about two months after Dr. Shinya Yamanaka, a professor of the department of stem cell biology at Kyoto University's Institute for Frontier Medical Sciences, announced the generation of human iPS cells. CiRA is the world's first research organization that focuses on iPS cells. With Dr. Yamanaka as director, CiRA conducts consistent research activities from basic to clinical studies in order to realize medical applications of iPS cell technology.

In October 2008, Dr. Yamanaka's group reported for the first time a virus-free method for inducing pluripotency in differentiated cells by using a pair of plasmid vectors to introduce the four iPS genes into embryonic mouse fibroblasts as part of CiRA's efforts to establish safe and efficient methods to generate iPS cells. CiRA also generates iPS cells from individuals in a wide age range and various kinds of disease-specific iPS cells. The center has 11 principal investigators, including Dr. Yamanaka, and is trying to recruit more competent researchers both in and outside of Japan to expand and reinforce research activities. In February 2010, a new five-story research building will be completed, which will help accelerate studies for clinical applications. CiRA collaborates with foreign research organizations, forming two partnerships last year - one was made with the University of Toronto in October and the other was with Novocell, Inc., of the United States in December.

Source: Kyoto University

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