© ISTOCK.COM/WILDPIXELIn the early 2000s, cystic fibrosis researchers were frustrated with available mouse models of the disease. The mice carried the mutant version of the cystic fibrosis transmembrane conductance regulator (CFTR) gene that causes the disease in people, but the animals didn’t develop the mucus-coated lungs, coughing, or breathing difficulty observed in human patients. Researchers at the University of Iowa (UI) thought they might have a solution—genetically modified miniature pigs. In September 2008, the team published their results: the mutant pigs had defective transmembrane chloride ion transport, the molecular hallmark of cystic fibrosis that leaves human patients’ skin tasting salty. Additionally, the newborn pigs shared human CF infants’ defects in the intestine, pancreas, and liver (Science, 321:1837-41, 2008). It was later found that, unlike the mice, the animals developed CF-like lung disease within two months of birth (Sci Trans Med, 2:29ra31, 2010).
Several factors aligned to make the mutant CFTR pigs possible. The pig was already a well-established medical research model, and its genome was being sequenced. It probably also didn’t hurt that the scientists were working in the U.S.’s top pork-producing state. Buoyed by their success in cystic fibrosis, the UI researchers thought pig models could help crack other human diseases for which mouse models had been inadequate. In collaboration with John Swart, then an executive director at Boehringer Ingelheim’s Iowa operations, they created a startup to commercialize the idea. Exemplar Genetics of Sioux Center, in northwestern Iowa, was born.
Iowa is “a really good place to emerge as a leader in biotech,” says Joe Hrdlicka, executive director of the Iowa Biotechnology Association, a nonprofit trade ...