Models will facilitate a better understanding of the disease and aid in therapeutic development.
CHDI Foundation, Inc. and Exemplar Genetics today announced a collaborative research agreement to create multiple miniature swine models of Huntington’s disease (HD), a devastating disorder for which there are currently no effective treatments.
Models that mimic this debilitating disease are anticipated to accelerate both the understanding of the disease as well as play a significant role in the development of potential therapeutics. The collaboration will generate two gene-targeted miniature swine models of HD; one that recapitulates the human disease phenotype, the other for use in developing gene-based therapies.
“We are excited to be partnering with CHDI in the development of these new models of Huntington’s disease,” said John Swart, Ph.D., president of Exemplar Genetics, a biotechnology enterprise focused on the development of gene-targeted models to enable advanced research of a number of chronic human diseases. “Our experience and that of our research partners is demonstrating that gene-targeted miniature swine models have some distinct advantages over the more commonly used rodent models. Our models mimic human disease, giving researchers a much clearer window into disease mechanisms and should result in accelerated therapeutic discovery.”
Exemplar currently has models of cystic fibrosis, cardiovascular disease, cancer, neurological disease and muscular dystrophy, has several more in its pipeline, and offers gene-targeted custom model development to groups looking to develop models for their own research purposes.
“We look forward to working with CHDI and their network of distinguished scientists to provide a vital tool in the advancement of research focused on finding effective treatments for this terrible disease,” noted Dr. Swart.
“We are delighted to have Exemplar as partners on this important project to develop new large animal models where the genetic mutation is generated in a context that is very similar to that found in the human disease,” said David Howland, Ph.D., Director, In Vivo Biology at CHDI. “We hope that such models will advance our understanding of premanifest stages of HD in particular and be useful for preclinical therapeutic studies, helping to speed those to the clinic.”
Huntington’s disease is a disorder passed down through families in which nerve cells in certain parts of the brain waste away, or degenerate.1 One in every 10,000 people will develop HD; currently, more than 250,000 people in the US are at risk of developing the disease.2
About Exemplar Genetics
Exemplar Genetics is a small biomedical research business that uses its patented technology to create gene targeted swine models of human disease that enable researchers to develop a better understanding of disease mechanisms and facilitate the discovery of new treatments and therapeutics. Exemplar Genetics works with universities, research institutes, disease foundations and biotechnology and pharmaceutical companies who utilize these models in their research programs to test their theories and products. Headquartered in Sioux Center, Iowa, Exemplar Genetics has operations in Iowa, South Dakota and Kentucky.
About CHDI Foundation, Inc.
CHDI Foundation, Inc. is a privately-funded, not-for-profit, biomedical research organization exclusively dedicated to rapidly discovering and developing therapies that slow the progression of Huntington’s disease. As a collaborative enabler, CHDI seeks to bring the right partners together to identify and address critical scientific issues and move drug candidates to clinical evaluation as quickly as possible. CHDI scientists work closely with a network of more than 600 researchers in academic and industrial laboratories around the world in the pursuit of these novel therapies, providing strategic scientific direction to ensure that common goals remain in focus.
1. PubMed Health, U.S. National Library of Medicine
2. Huntington’s Disease Society of America