Exemplar Genetics Rare Neurological Disease Model Cleared by FDA for Commercial Research Use

SIOUX CENTER, Iowa, April 16, 2018

Exemplar Genetics, a wholly owned subsidiary of Intrexon Corporation (NYSE: XON) committed to enabling the study of life-threatening human diseases, today announced the U.S. Food & Drug Administration (FDA) has exercised enforcement discretion in regard to the ExeGen®ATM MiniSwine model clearing it for commercial use as a research model. The ExeGen®ATM MiniSwine is genetically engineered (GE) to model ataxia telangiectasia (AT), a rare, inherited, predominantly neurological human disease. Exemplar has previously received approval for its ExeGen®LDLR MiniSwine model for use in cardiovascular disease research.

In humans, mutations in the ataxia telangiectasia mutated gene (ATM) lead to the multisystemic disorder AT. AT is principally considered a neurodegenerative disease and is characterized by cerebellar degeneration leading to profound motor impairment. Additional disease manifestations include fertility problems, thymic atrophy/hypoplasia, tumor development (mainly lymphoid organs), and immunological deficiencies. There is currently no cure for AT and treatment of patients is limited to supportive care designed to manage disease symptoms.

Following Exemplar’s previous approval of its ExeGen® LDLR MiniSwine model for use in cardiovascular disease research, the ExeGen® ATM model is the second GE MiniSwine model reviewed and cleared by the FDA. As a larger animal model, ExeGen® ATM is designed to enable superior translational research and better predictive efficacy as scientists seek to define disease mechanisms and develop novel therapeutics that address the pathologies of AT. As a result of this approval, Exemplar's powerful investigational platform can now be offered to researchers and drug developers.

“The ExeGen® ATM MiniSwine model has allowed us to study the disease in much greater depth than what was possible before,” commented Jill Weimer, Ph.D., Senior Director of Therapeutic Development for Sanford Research. “Through these discoveries we hope to improve our understanding of the underlying mechanisms of this disease and develop better methods to treat it.”

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. Exemplar's GE MiniSwine research models are more anatomically, physiologically, and genetically similar to humans than are mice and other small-animal disease models and are designed to provide improved predictive power to the preclinical stages of therapeutic development. With failure rates for therapeutic development programs remaining above 80% and the estimated average costs of successful development programs above $1 billion, the need for better and more predictive research tools remains significant.

“The orphan disease research community remains in desperate need of better models to advance potential treatments,” said John R. Swart, Ph.D., President and Chief Executive Officer of Exemplar Genetics. “The ExeGen® ATM MiniSwine model represents the first of several models we have developed to enable the advancement of those therapies. This approval should expand access to our AT model significantly.”

Through its suite of proprietary technologies, Exemplar has developed a comprehensive pipeline of MiniSwine models to enable medical research on numerous human health conditions, including heart disease, cancer, cystic fibrosis, cardiac arrhythmia, and neuromuscular/neurodegenerative disorders. The ability of Exemplar's models to more effectively recapitulate human disease has been well documented through studies led by contract research organizations as documented in more than 30 peer-reviewed publications.

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.


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

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