Recombinetics today announced a multi-year, exclusive collaborative research and development agreement with Children’s Minnesota, the seventh largest pediatric healthcare system in the country, to produce the first-ever large animal model of Phenylketonuria (PKU). PKU is one of the most common inborn errors of metabolism with a cumulative incidence of approximately 1:16,500 live births in the United States. There are currently no cures for this disease.
Recombinetics’ biomedical division, Surrogen, develops customized biomedical models of swine, which are genetically similar to humans. These models give researchers, like those at Children’s Minnesota, a better platform for evaluating the progression of PKU or diseases, and developing new treatment protocols like gene therapy. Currently, the main therapy available for PKU is dietary intervention, which is difficult to maintain through adulthood, and often fails to fully ameliorate disease-associated symptoms. To date, promising treatments developed in small animal models, such as mice, have failed to successfully translate for use in patients. If left untreated, PKU leads to growth failure, acquired microcephaly, hypopigmentation, and severe developmental disability in patients. Recombinetics is collaborating with Children’s to develop more options for patients with PKU that could lead to treatments for other inborn errors of metabolism.
“We’re eager to collaborate with Recombinetics on this first-ever large animal model of PKU, which will give us ways to study PKU not previously possible. With a pig model for PKU, we hope to greatly speed up the development of novel gene-edited approaches and gene therapy for pediatric patients,” said the project’s lead physician, Joseph Lillegard, MD, PhD of Children’s Minnesota and Mayo Clinic.
The project’s lead scientist, Dan Carlson, PhD, Senior Vice President of Research and Development of Recombinetics, says their goal in developing gene-edited biomedical pig models is to provide a pathway to finding novel therapies – and hopefully a cure for PKU. “Thousands of patients are living with this debilitating condition, and they and their families have no current line of sight on a cure. By working together with Dr. Lillegard and the innovative patient care team at Children’s Minnesota, we are developing a state-of-the-art model for PKU research to enable leading-edge treatments for patients.”