Sioux Center-based Exemplar Genetics has recently been awarded a Phase II grant from the National Institutes of Health (NIH). The funding will be used to continue development the company’s pig model for ataxia-telangiectasia (A-T),an inherited, neurodegenerative disease that causes severe problems with mobility and coordination; patients with A-T also exhibit other clinical symptoms such as immunodeficiency, increased susceptibility to certain cancers, insulin resistant diabetes, and chromosomal instability.

Current treatment options for A-T are aimed at management of symptoms. However, since no therapy to treat the underlying defect is currently available, A-T remains a fatal disease.

The development of improved therapies for A-T is currently limited by the lack of an animal model that fully, and accurately, mimics the multi-systemic nature of this disease.  While several mouse models of A-T have been generated, they to fully replicate the complex human clinical symptoms seen, and also fail to develop the neurodegeneration that is the hallmark of human A-T. The pig model of A-T being developed by Exemplar may be a better model for the study of human disease since pigs more closely resemble humans in terms of their development, physiology and anatomy. The company’s pig model of AT will thus provide a valuable resource to stimulate discovery, therapeutic application, and the development of new diagnostic tools.

Exemplar Genetics has also successfully commercialized other pig models of human disease, including cystic fibrosis, and provides regulatory, model development, and animal housing and husbandry services.

For more information, visit: http://www.exemplargenetics.com/index.htm.

Reprinted from the Iowa SBIR/STTR newsletter.