Genetically engineered mini-pig models of sickle cell disease (SCD) that closely mimic the condition in people will be created as part of a contract with Exemplar Genetics. The models could help researchers find new treatments for the disease.
The work will support the efforts of the National Institutes of Health’s (NIH) National Center for Advancing Translational Sciences (NCATS). The agency aims to reduce or remove bottlenecks in the research pipeline to speed new drugs, diagnostics, and medical devices to patients.
“We believe these models will be an excellent resource for the research community to help enable significant advancements in the understanding of sickle cell disease mechanisms, ultimately leading to new treatments,” John R. Swart, PhD, president of Exemplar Genetics, said in a press release. “The Leidos subcontract serves as further recognition that better models are needed and genetically engineered porcine models can fill that void.”
Genetically engineered miniswine are more similar to humans in physiology, anatomy, and size than mouse models. They have been analyzed in more than 30 peer-reviewed scientific publications.
Mouse models have been a mainstay of biomedical research since the 1980s, but they often fail to recreate the pathology of human disease, limiting their use. Ineffective animal models can hinder discovery and progress critical to developing new therapies, the release said. That could be particularly significant when trying to treat rare genetic diseases.
“The development of these models is critical as new treatment options are discovered and need to be evaluated. A humanized pig model of sickle cell disease will provide a large animal system for testing both gene editing and drug therapy approaches for treatment of this disease that affects so many families in the U.S. and worldwide,” said Tim Townes, PhD, director of the Stem Cell Institute at the University of Alabama at Birmingham.
Exemplar Genetics’ ExeGen low-density lipoprotein receptor (LDLR) miniswine model was cleared for use in research by the U.S. Food and Drug Administration (FDA) in April. Research has linked LDLR with a reduction in brain changes associated with Alzheimer’s disease (AD).