Recombinetics and Mayo Clinic Announce the First Swine Model of Congenital Heart Failure

Mayo Clinic’s Todd and Karen Wanek Family Program for Hypoplastic Left Heart Syndrome (HLHS) and Recombinetics, Inc. announce the completion of collaborative research and development program to produce the first-of-its-kind large animal model of Dilated Cardiomyopathy (DCM). DCM is a major cause of heart failure and a significant source of mortality and morbidity for children and adults. There is no cure for DCM, and current pharmacologic therapies have limited effectiveness. DCM is the most common indication for heart transplantation.

Recombinetics’ biomedical division, Surrogen, develops customized biomedical models of disease in swine, which are genetically similar to humans. Surrogen’s swine model of DCM will give researchers and clinicians a better platform for characterizing the progression of the disease as well as enable evidence-based innovation of biologic, pharmaceutical, and device therapies directed towards preventing heart failure. Current progress towards developing effective treatments has been slow using available animal models because they do not accurately mimic disease progression and response to therapy in humans, and genetic heterogeneity of the disease results in a limited number of attractive molecular targets. The Surrogen model will serve as a reproducible, relevant, and reliable model for progression of biventricular heart failure from children to adult.

This collaboration leveraged the strengths of each institution. Surrogen, having expertise in gene editing created the model to mimic a genotype found in DCM patients, and Mayo Clinic, leaders in R & D surrounding pediatric heart failure, characterized the model using clinical grade procedure.  As a result of the team effort, Surrogen is prepared to launch the DCM model into the market.

“We expect this novel animal model will prove extremely valuable for evaluating new treatments for DCM and heart failure in a model that much more closely replicates human size and physiology,” said Timothy Nelson, MD, Ph.D. director of Mayo Clinic’s Todd and Karen Wanek Family Program for HLHS. The program, founded in 2010, is a collaborative network of specialists bonded by the vision of delaying or preventing heart failure for individuals affected by congenital heart defects, including hypoplastic left heart syndrome. The specialized team is addressing the various aspects of these defects by using research and clinical strategies ranging from basic science to diagnostic imaging to regenerative therapies.

The project’s lead scientist, Senior Vice President of Research and Development of Recombinetics, Dan Carlson, Ph.D., says “Our goal in developing gene-edited pig models is to provide a better pathway to finding novel therapies – and hopefully a cure for pediatric and adult patients with DCM and other cause of heart failure.”

Mayo Clinic and Dr. Timothy Nelson have a financial interest in the technology referenced in this news release. Mayo Clinic will use any revenue it receives to support its not-for-profit mission in patient care, education and research.

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