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PUBLIC RELEASE DATE:
3-Jan-2013

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Contact: Heather Buschman
hbuschman@sanfordburnham.org
858-795-5343
Sanford-Burnham Medical Research Institute
@SanfordBurnham

Sanford-Burnham and Intrexon Corporation establish collaboration to accelerate stem cell research

Combines Sanford-Burnham Medical Research Institute's renowned scientific team and Intrexon's proprietary discovery platforms to accelerate human induced pluripotent stem cell research

IMAGE: Evan Y. Snyder, M.D., Ph.D., Sanford-Burnham Medical Research Institute, is professor and director of the Stem Cell Research Center and the Stem Cell and Regenerative Biology Program.

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LA JOLLA, Calif., January 3, 2013 - Sanford-Burnham Medical Research Institute, a nonprofit research institution and one of the largest iPSC generators in the world, and Intrexon Corporation, a leading synthetic biology company, today announced a new collaboration to accelerate stem cell research. Under the agreement, Sanford-Burnham will gain access to sophisticated proprietary cellular selection and gene regulation technologies that are not currently on the market, including Intrexon's Laser-Enabled Analysis and Processing (LEAP™) instrument and RheoSwitch Therapeutic System® (RTS®). As part of the agreement, Intrexon may obtain commercial and intellectual property rights resulting from technological advances made under the collaboration.

"I'm looking forward to merging and melding our expertise," said Evan Y. Snyder, M.D., Ph.D., professor and director of Sanford-Burnham's Stem Cell Research Center and Stem Cell and Regenerative Biology Program. "We'll bring our iPSC and gene therapy expertise to the table. Likewise, our colleagues at Intrexon will share their knowledge of how best to use the technologies. We envision we'll be meeting with them frequently and sharing insights to further advance the platforms for stem cell applications."

Sanford-Burnham is currently building the world's largest collection of human iPSCs generated from individual patients and healthy volunteers. The Stem Cell Research Center's expertise and resources are available to all Sanford-Burnham scientists, as well as other researchers at nonprofit and for-profit research organizations around the world.

LEAP™ for induced pluripotent stem cells

The LEAP™ instrument is an automated system that provides high-throughput cell imaging coupled with versatile laser-based cell processing. The instrument's applications include rapid and accurate in situ purification of adherent cells and cell colonies, features that are particularly useful when working with complex human iPSC cultures. The LEAP™ instrument enables scientists in Sanford-Burnham's Stem Cell Research Center to improve and accelerate their methods for generating human iPSCs and their differentiated progeny, which are used in the study of a variety of diseases. iPSCs are stem cells derived from adult cells--a research advance that garnered the 2012 Nobel Prize in Physiology or Medicine.

IMAGE: The Stem Cell Research Center at Sanford-Burnham Medical Research Institute provides resources and expertise to the entire scientific community.

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"Intrexon's LEAP™ instrument will allow us to isolate high-quality human iPSCs while eliminating non- or partially-reprogrammed cells or other undesirable cell types in the culture--a laborious process that previously took a trained technician a lot of time," explained Yang Liu, Ph.D., manager of Sanford-Burnham's Stem Cell Research Center. "Together with other automated equipment available in our facility, the new capabilities will free up valuable resources, allowing us to provide an even greater level of service to our internal and external users."

"We are big believers in iPSCs and their potential for use in new therapeutic modalities," said Fred Koller, Ph.D., vice president and executive director of the Intrexon Institute for Biomolecular Research. "It's exciting for us to use our technology collaboratively with Sanford-Burnham's team of premier scientists. We look forward to applying LEAP™, RTS® and other Intrexon tools in this stem cell research, and are proud to assist in the diverse medical advancements enabled by this collaborative effort with Sanford-Burnham."

Controlling gene expression with RTS®

RTS® technology, a proprietary biological "switch" that enables inducible controlled gene expression by administering an activator ligand, will give Sanford-Burnham scientists a new method to regulate when certain genes are turned on or off in cells. The system also provides more accurate delivery of new therapeutic candidates to specific tissues in animal models.

"We're interested in the RTS® technology because it will help us to turn genes on or off in stem cells that have been transplanted. For example, it can be used for therapeutic protein expression in stem cells that home to and help eradicate brain tumors," said Snyder.

"New cell-based therapies may someday result from our LEAP™ and RTS® technologies," Koller said. "Working with leaders in the field of academic stem cell research will leverage both parties' technologies to get there faster."

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About Sanford-Burnham Medical Research Institute

Sanford-Burnham Medical Research Institute is dedicated to discovering the fundamental molecular causes of disease and devising the innovative therapies of tomorrow. The Institute consistently ranks among the top five organizations worldwide for its scientific impact in the fields of biology and biochemistry (defined by citations per publication) and currently ranks third in the nation in NIH funding among all laboratory-based research institutes. Sanford-Burnham utilizes a unique, collaborative approach to medical research and has established major research programs in cancer, neurodegeneration, diabetes, and infectious, inflammatory, and childhood diseases. The Institute is especially known for its world-class capabilities in stem cell research and drug discovery technologies. Sanford-Burnham is a U.S.-based, non-profit public benefit corporation, with operations in San Diego (La Jolla), California and Orlando (Lake Nona), Florida. For more information, news, and events, please visit us at sanfordburnham.org.

About Intrexon Corporation

Intrexon Corporation is a privately held biotechnology company focused on the industrial engineering of synthetic biology. Intrexon is deploying its extensive capabilities to rapidly design and produce novel and enhanced biological products and processes across multiple industry sectors, including: human therapeutics, protein production, industrial products, agricultural biotechnology, and animal science. The company's advanced bioindustrial engineering platform enables Better DNA™ technology by combining revolutionary DNA control systems with corresponding advancements in modular transgene design, assembly, and optimization to enable unprecedented control over the function and output of living cells. More information about the Company is available at www.dna.com.



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