Transgenic Animals to be Developed for Therapeutic and Diagnostic Applications ST. LOUIS and RICHMOND, Calif., July 9
ST. LOUIS and RICHMOND, Calif., July 9 /PRNewswire-FirstCall/ --
Sigma-Aldrich Corporation (Nasdaq: SIAL) and Sangamo BioSciences, Inc.
(Nasdaq: SGMO) today announced a research and license agreement to provide
Roche (SWX: ROG) with non-exclusive, worldwide rights for the use of its
proprietary zinc finger nuclease (ZFN) technology to develop cell-lines and
transgenic animals that have targeted modifications in a specified gene in a
specified species. Roche also has an option to obtain an exclusive, worldwide
license for the commercial use of such ZFN-generated transgenic animals in the
production of therapeutic and diagnostic products.
The research phase of the agreement will be conducted in collaboration
with both Sangamo and Sigma-Aldrich, Sangamo's exclusive licensee of ZFN
technology for high-value research reagents.
"There is growing appreciation of the value of ZFN technology as a rapid,
reliable and highly specific tool for modifying genes in eukaryotic cells and
whole organisms," said David Smoller, Ph.D., President of Sigma-Aldrich's
Research Biotech Business Unit. "We are excited to be working with Sangamo to
provide Roche with high-value ZFN reagents for the generation of transgenic
animals. ZFN technology promises to enable the generation of a variety of
transgenic models of human disease, expediting drug development and
production."
"Roche is a leading global healthcare company with an established
reputation of innovation. We are pleased to provide them with our ZFN
technology which provides a cutting-edge approach for disease research," said
Edward Lanphier, Sangamo's President and Chief Executive Officer. "The
frequency and precision of ZFN-mediated genome editing, in combination with
the ability to design ZFNs against potentially any gene, opens up the
possibility of more easily generated transgenic animals of any species."
Zinc finger DNA-binding proteins (ZFPs) are the dominant class of
naturally occurring transcription factors in organisms from yeast to humans.
Transcription factors, which are found in the nucleus of every cell, bind to
DNA to regulate gene expression. Though there are many kinds of transcription
factors, only ZFPs are amenable to engineering and precise targeting of a
particular gene or genes of interest. ZFNs are engineered forms of ZFPs that
also contain a nuclease component which can facilitate modification of a
target gene of interest.
"The power of ZFN technology was recently demonstrated in a study
published in Nature Biotechnology," said Philip Gregory, D.Phil., Sangamo's
Vice President for Research. "Using ZFNs in zebrafish, a widely recognized
system for human disease modeling and in vivo drug discovery, resulted in the
generation of fish in which the ZFN-targeted gene has been eliminated or
'knocked out'. Apart from the mouse, the majority of other animals including
zebrafish have historically lacked methods for precision targeted mutagenesis.
This publication demonstrates that ZFNs present a powerful solution to this
problem with application across virtually any species for any gene."
Terms of the Agreement
Under this agreement, Sangamo will provide a non-exclusive, worldwide
research license for the use of its proprietary ZFN technology in the
development of transgenic animals. In this phase Roche will pay research
fees, including research maintenance fees and research event payments. If
Roche elects to exercise its option for an exclusive commercial license it
will owe Sangamo an option license fee, additional payments upon the
achievement of certain clinical development events and royalties on sales of
therapeutic and diagnostic products developed by Roche using the ZFN-modified
animals.
About the Nature Biotechnology Publication
The work entitled, "Heritable Targeted Gene Disruption in Zebrafish Using
Designed Zinc Finger Nucleases" was carried out in collaboration with the
laboratory of Sharon Amacher, Ph.D., Associate Professor of Genetics, Genomics
and Development at the University of California, Berkeley. The paper and an
accompanying commentary appear in the June 2008 print issue of the magazine,
Nature Biotechnology, June 2008, vol 26, 6, pp702-708.
http://www.nature.com/nbt/journal/v26/n6/abs/nbt1398.html
About Sigma-Aldrich
Sigma-Aldrich is a leading Life Science and High Technology company. Our
biochemical and organic chemical products and kits are used in scientific and
genomic research, biotechnology, pharmaceutical development, the diagnosis of
disease and as key components in pharmaceutical and other high technology
manufacturing. We have customers in life science companies, university and
government institutions, hospitals, and in industry. Over one million
scientists and technologists use its products. Sigma-Aldrich operates in 36
countries and has 7,900 employees providing excellent service worldwide. We
are committed to Accelerating Customer Success through Leadership in Life
Science, High Technology and Service. For more information about
Sigma-Aldrich, please visit our award-winning Web site at
http://www.sigma-aldrich.com
About Sangamo BioSciences, Inc.
Sangamo BioSciences, Inc. is focused on the research and development of
novel DNA-binding proteins for therapeutic gene regulation and modification.
The most advanced ZFP Therapeutic(TM) development program is currently in
Phase 2 clinical trials for evaluation of safety and clinical effect in
patients with diabetic neuropathy. Phase 1 clinical trials are ongoing to
evaluate a ZFP Therapeutic for peripheral artery disease. Other therapeutic
development programs are focused on HIV/AIDS, neuropathic pain, cancer, nerve
regeneration and monogenic diseases. Sangamo's core competencies enable the
engineering of a class of DNA-binding proteins known as zinc finger DNA-
binding proteins (ZFPs). By engineering ZFPs that recognize a specific DNA
sequence Sangamo has created ZFP transcription factors (ZFP TF(TM)) that can
control gene expression and, consequently, cell function. Sangamo is also
developing sequence-specific ZFP Nucleases (ZFN(TM)) for therapeutic gene
modification as a treatment for a variety of monogenic diseases, such as X-
linked SKID and hemophilia, and for infectious diseases, such as HIV. Sangamo
has established strategic partnerships with companies outside of the human
therapeutic space including Dow AgroSciences, Sigma-Aldrich Corporation and
several companies applying its ZFP Technology to enhance the production of
protein pharmaceuticals. For more information about Sangamo, visit the
company's web site at http://www.sangamo.com/.
This press release may contain forward-looking statements based on
Sangamo's current expectations. These forward-looking statements include,
without limitation, references to the payment of fees and royalties under the
license agreement, development of transgenic species, and the application of
Sangamo's ZFP technology in the development of transgenic animals as models of
human disease and for drug development. Actual results may differ materially
from these forward-looking statements due to a number of factors, including
technological challenges, Sangamo's ability to develop commercially viable
products and technological developments by our competitors. See the company's
SEC filings, and in particular, the risk factors described in the company's
Annual Report on Form 10-K and its most recent Quarterly Report on Form 10-Q.
Sangamo assumes no obligation to update the forward-looking information
contained in this press release.
SOURCE Sangamo BioSciences, Inc.
