Recombinetics Inc., a leading gene editing company with platform technology applied to biomedicine and animal agriculture, today announced a first-of-its-kind study to improve outcomes for patients diagnosed with a rare disease known as Tuberous Sclerosis Complex (TSC).
Surrogen, a subsidiary of Recombinetics Inc., is focused on developing large animal models of human disease for use in medical research. This technology allows researchers to engineer exact patient mutations into the swine genome to develop biomedical models to study disease and find new therapies that are both safe and effective for patients.
TSC is a rare disease that occurs in 1 in 6,000 births. There are over 1 million individuals with TSC worldwide. TSC causes tumors that form in vital organs including the brain, eyes, heart, liver, lungs, and skin. In addition, autism, seizures and serious neurodevelopmental delay can occur. There are currently no treatments that can eliminate tumor cells in TSC, and no animal models that fully recapitulate the disease, creating a research bottleneck. Recombinetics Inc. will work with Boston’s Brigham and Women’s Hospital to advance research by creating reproducible and reliable animal models for pre-clinical studies, leading to new treatments for TSC.
According to Dr. Adrienne Watson, Recombinetics Inc. Vice President of Research and Development, “This new model of TSC will mirror the human disease genetically and is expected to show the same pathophysiology seen in human patients. This gives the research and therapeutics community an unprecedented opportunity to fully understand this disease and improve the lives of patients through the development of biomarkers, new surgical and imaging techniques, and new therapies to treat the many complications that patients with TSC face.”
“We couldn’t be more excited to be working with Brigham and Women’s Hospital to bring hope and healing to those who suffer from this insidious disease,” said Mark Platt, Recombinetics Inc.’s President and Chief Executive Officer. “The opportunity to study this disease, as well as many others, in large animal models with the exact human genetics is a game changer that will speed new therapeutic development at lower cost and lower patient risk.”
“Decades of experience has shown that mouse models of TSC fail to reproduce the fundamental pathology and tumor growth characteristics that occur in TSC. Hence, we are very excited and hopeful that this new swine model of TSC will reproduce the classic TSC lesions, enabling detailed studies of how they develop as well as assessment of novel therapeutic approaches,” said David J. Kwiatkowski, MD, PhD, and Elizabeth P. Henske, MD, both of Brigham and Women’s Hospital and Professors of Medicine at Harvard Medical School.
This project is supported by a gift from Cristy and Jim Wade of Texas, whose family is personally affected by TSC. The Wades state, “We are excited to help with this new line of research and are very hopeful this model will ultimately result in treatments to help those individuals and their families living with TSC.”
Founded in 2008, Recombinetics is a recognized global leader in the development, deployment, and commercialization of genetically engineered animals. Its four subsidiaries, Regenevida, Surrogen, Makana, and Acceligen, have delivered hundreds of animals to enable drug, device and therapeutic discovery, generate transplantable cells, tissues and organs, and provide improved health, well-being, and productivity of agricultural animal