StemCells, Inc.'s Technology Used to Create First Genetically Engineered Rat Derived From Rat Embryonic Stem Cells

StemCells, Inc.'s Technology Used to Create First Genetically Engineered Rat Derived From Rat Embryonic Stem Cells
PALO ALTO, Calif., Aug 31, 2010 (GlobeNewswire via COMTEX) --
StemCells, Inc. (Nasdaq: STEM | PowerRating) announced today that its technology was recently used by independent researchers to achieve the first genetically engineered rat derived from rat embryonic stem (ES) cells. This breakthrough, published this month in the international peer-reviewed journal Nature, opens the door to the types of genetic manipulations previously only possible in mice, and paves the way for modeling a broader range of human diseases with the rat. Both mice and rats are used as animal models of human disease; however certain aspects of the rat's physiology, behavior, and metabolism are closer to the human, making rats the preferred species for drug development and studying human disease. The creation of this first rat model using rat ES cells validates intellectual property owned by StemCells, which includes the rights to patents covering both rat ES and rat induced pluripotent stem (iPS) cells as well as genetically engineered rats derived from these cells.

StemCells' broad rat pluripotent stem cell intellectual property portfolio is based upon groundbreaking research led by prominent academic researchers at the University of Edinburgh, including Dr. Qi-Long Ying who was the first to succeed in deriving and culturing the true germline competent rat ES cells required for precise genetic engineering. In this newly published study, Dr. Qi-Long Ying and his colleagues at the Eli and Edythe Broad Center for Regenerative Medicine and Stem Cell Research at the University of Southern California demonstrated for the first time the creation of genetically modified rats using rat ES cells that have been gene targeted via homologous recombination, a method which involves adding DNA sequences to the cells to delete ('knock-out'), add ('knock-in') or otherwise modify genes of interest. This latest work resulted in the successful generation of knock-out rats missing the tumor suppressor gene p53 for use in studying cancer1, and serves as a proof-of-principle for creating genetically engineered rats using rat ES cells.

"The fact that we can now use rat ES cells to carry out genetic manipulation in this well characterized and widely studied species is truly revolutionary, and will lead to the creation of new, more sophisticated rat models that could advance the development of treatments for various human diseases," said Dr. Ann Tsukamoto, Executive Vice President of Research and Development at StemCells, Inc. "We intend to develop additional rat models with Dr. Ying for a broad range of diseases, and look forward to exploring licensing and development opportunities with third parties wishing to commercialize rat models derived from rat ES cells."

The rat ES cells used to generate the genetically engineered rats were cultured using a special '2i' inhibitor-based media formulation2 developed by Dr. Ying and his colleagues while at the University of Edinburgh. This proprietary 2i media is exclusively licensed to StemCells, Inc. and marketed as GS1-R(TM). GS1-R is part of the Company's SC Proven(R) line of specialty cell culture products, and is the first and only commercially available medium shown to enable the derivation and long-term maintenance of the true rat ES cells required for precise genetic manipulation3,4,5. For more information about GS1-R and the Company's other SC Proven(R) specialty cell culture products, interested parties are invited to visit


(1) Tong C, et al. Nature, doi:10.1038/nature09368, 2010.

(2) Smith A. Design principles of pluripotency. EMBO Molecular Medicine 1(5):251-4, 2009.

(3) Li P, et al. Cell 135(7): 1299-310, 2008.

(4) Buehr M, et al. Cell 135(7): 1287-98, 2008.

(5) Leitch H, et al. Development 137(14): 1-9, 2010.

About StemCells, Inc.

StemCells, Inc. is engaged in the research, development, and commercialization of stem cell therapeutics and tools for use in stem cell-based research and drug discovery. In its therapeutic product development programs, StemCells is targeting diseases of the central nervous system and liver. StemCells' lead product candidate, HuCNS-SC(R) cells (purified human neural stem cells), is in clinical development for the treatment of two fatal neurodegenerative disorders that primarily affect young children. StemCells also markets specialty cell culture products under the SC Proven(R)brand, and is developing stem cell-based assay platforms for use in pharmaceutical research, drug discovery and drug development. The Company has exclusive rights to approximately 55 issued or allowed U.S. patents and over 200 granted or allowed non-U.S. patents. Further information about StemCells is available at

The StemCells, Inc. logo is available at

Apart from statements of historical fact, the text of this press release constitutes forward-looking statements within the meaning of the U.S. securities laws, and is subject to the safe harbors created therein. These statements include, but are not limited to, statements regarding the prospects for creating additional rat models derived from rat ES cells and for out-licensing the Company's technology; the ability of GS1-R to enable the derivation and long-term maintenance of rat ES cells and facilitate efforts to genetically engineer more sophisticated disease models; the Company's ability to commercialize drug discovery and drug development tools; and the future business operations of the Company. These forward-looking statements speak only as of the date of this news release. The Company does not undertake to update any of these forward-looking statements to reflect events or circumstances that occur after the date hereof. Such statements reflect management's current views and are based on certain assumptions that may or may not ultimately prove valid. The Company's actual results may vary materially from those contemplated in such forward-looking statements due to risks and uncertainties to which the Company is subject, including those described under the heading "Risk Factors" in the Company's Annual Report on Form 10-K for the year ended December 31, 2009 and in its subsequent reports on Form 10-Q and Form 8-K.

This news release was distributed by GlobeNewswire,

SOURCE: StemCells, Inc.

CONTACT:  StemCells, Inc.

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