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Exemplar Genetics & Lovelace Biomedical receive award to advance Sickle Cell research

SIOUX CENTER, Iowa, July 8, 2019

National Center for Advancing Translational Sciences supports generation, characterization of a MiniSwine model of Sickle Cell Disease, to support life changing research for patients.

Exemplar Genetics, a wholly owned subsidiary of Intrexon Corporation (NYSE: XON) committed to enabling the study of life-threatening human diseases, today announced a collaboration with the Lovelace Biomedical to continue development of Exemplar Genetics porcine animal model for Sickle Cell Disease (SCD).

The support from the National Center for Advancing Translational Research (NCATS) along with the collaboration between Exemplar and Lovelace Biomedical will be used to generate live MiniSwine to assess viability, characterize and validate the model, including a natural history study. The group will also develop assay to measure pain in the model.

Exemplar's ExeGen® models have assisted in small molecule and gene therapy research and has resulted in approximately 100 peer reviewed publications. Exemplar, Lovelace Biomedical and NCATS will collaborate to generate a more powerful model for SCD adding to Exemplar's significant pipeline of existing models for heart disease, cancer, and various rare genetic diseases that are already available to researchers.

"The NCATS support of the Lovelace Biomedical proposal of gathering the experts necessary to characterize the Exemplar SCD model, displays the confidence they have in our technical capabilities and our ability to execute a program of this magnitude. We are excited to work with Exemplar and other providers in this project," says Jacob McDonald, Lovelace Biomedical.

Small animal models are extensively used in both disease research and therapeutic development, and the current market for murine models alone exceeds $1 billion. However, the failure of small-animal models to fully reproduce the human disease condition remains a significant barrier to defining disease mechanisms and impedes the development of safe and efficacious therapeutics and diagnostic tools. MiniSwine research models are more anatomically, physiologically, and genetically similar to humans than are mice with regard to anatomy, physiology and immune system responses. These characteristics enable their use as an ideal species to use for sickle cell research. Additionally, their human-like size also makes them a more suitable model for imaging studies.

"The rare disease research community remains in desperate need of better models to advance potential treatments," said Dr. Christopher Rogers, Chief Scientific Officer of Exemplar Genetics. "The potential utility of an animal model with improved recapitulation of human symptoms is expected to be widely embraced by the research community."

About Exemplar Genetics

Exemplar Genetics, a wholly owned subsidiary of Intrexon Corporation (NYSE: XON), enables discovery by providing models and services that aid scientists in the development of next-generation procedures, devices and therapeutics. Through its innovative models and AAALAC-certified facilities, Exemplar Genetics assists researchers in making advances in the discovery of human disease mechanisms, the optimization of novel diagnostics, and the development of new treatments. For more information, visit www.exemplargenetics.com.

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ExeGen® is a registered trademark of Exemplar Genetics. Other names may be trademarks of their respective owners.

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Some of the statements made in this press release are forward-looking statements. These forward-looking statements are based upon our current expectations and projections about future events and generally relate to our plans, objectives and expectations for the development of our business. Although management believes that the plans and objectives reflected in or suggested by these forward-looking statements are reasonable, all forward-looking statements involve risks and uncertainties and actual future results may be materially different from the plans, objectives and expectations expressed in this press release.

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