FDA Clears for Commercial Use Exemplar Genetics’ Animal Model for Rare Neurodegenerative Disease

April 17, 2018

Rare Daily Staff

The U.S. Food and Drug Administration cleared Intrexon subsidiary Exemplar Genetics’ ExeGen ATM MiniSwine model for commercial use as a research model of ataxia telangiectasia, a rare, inherited, neurodegenerative disease.

Mutations in the ataxia telangiectasia gene 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, and immunological deficiencies. There is currently no cure for AT and treatment of patients is limited to supportive care designed to manage disease symptoms.

The ExeGen ATM model is the second Exemplar GE MiniSwine model reviewed and cleared by the FDA. The FDA previously approved for commercial use Exemplar’s ExeGen LDLR MiniSwine model for use in cardiovascular disease research.

ExeGen ATM is designed to enable translational research and better predictive efficacy as scientists seek to define disease mechanisms and develop novel therapeutics that address the pathologies of AT. The approval allows Exemplar to offer its investigational platform 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,” said Jill Weimer, 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 says its 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 percent 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 Swart, president and CEO 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.”

April 17, 2018
Photo: ExeGen ATM MiniSwine

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