At the University of Minnesota's Gene Therapy Center of Excellence (GT-COE), we are dedicated to changing the lives of patients with rare and life-threatening genetic diseases. With a legacy of pioneering advancements, the University of Minnesota has been at the forefront of understanding rare disorders and offering groundbreaking therapies. In 1968, our doctors performed the world’s first successful human allogeneic bone marrow transplant on a patient with severe combined immune deficiency. Then in 1983, we performed the first U.S. transplant for an inherited enzyme deficiency. Since then, we have continued to lead in the fields of cellular and gene therapy, providing cutting-edge treatments that target the root causes of these conditions and offering hope to patients and families who have long awaited viable treatment options. Minnesota is a leading site in the clinical trials of gene therapy for cerebral adrenoleukodystrophy, for ongoing gene therapy studies in Hurler syndrome and attenuated MPS I. In addition, we were the first center in the USA to offer gene therapy for metachromatic leukodystrophy.
Pioneering Expertise in Rare Disorders
In 2022, the National Organization for Rare Disorders (NORD) recognized the University of Minnesota/M Health Fairview Masonic Children’s Hospital as a Rare Disease Center of Excellence in its inaugural cohort. Our GT-COE is committed to advancing personalized medicine through genetics and cellular biology. We develop innovative therapies tailored to each patient's unique disorder, achieving a level of precision and effectiveness that was once unimaginable. By addressing the specific needs of each individual, we are redefining standards of care for rare diseases.
Our center is globally renowned for its expertise in treating a wide range of rare genetic disorders, including lysosomal storage disorders such as Hurler syndrome, Hunter syndrome, Maroteaux-Lamy syndrome, Krabbe disease, metachromatic leukodystrophy (MLD), and mannosidosis. We are one of the most experienced centers worldwide in using blood stem cell transplantation for adrenoleukodystrophy (ALD) and are home to the Adrenoleukodystrophy National Registry.
In addition to our work with lysosomal disorders, we serve as an international referral center for genetic disorders, including hemoglobinopathies such as sickle cell disease, thalassemia, and related conditions. Our deep understanding of these disorders allows us to anticipate and manage potential complications, providing patients with the best possible outcomes.
Our Facility
The University of Minnesota Medical Center is the core teaching hospital, specializing in organ and blood and marrow transplantation, heart disease, cancer, neurosciences, and behavioral sciences. Managed by Fairview Health Services, our facility includes an 18-bed inpatient unit at the M Health Fairview Masonic Children's Hospital, specifically for patients aged 0-25 years. This unit offers intensive care, including for patients with serious complications related to treatment, and is equipped with state-of-the-art air handling capabilities. It is staffed by clinical nurses specializing in blood and marrow transplantation (BMT) and the administration of gene therapy products. The outpatient clinic, located within the same building, operates Monday through Friday, 7:30 am to 5:00 pm, with weekend coverage available through the emergency department or inpatient unit. The clinic features nine examination rooms, 14 infusion beds/chairs, and a dedicated team including RNs, Medical Assistants, Advanced Practitioners, full-time phlebotomy staff, as well as a pharmacist and pharmacy technicians. There is extensive multidisciplinary provider support, including those with extensive experience in rare disorders, in providing exceptional care for patients in the inpatient and outpatient settings.
Established in 1995, the University of Minnesota's Molecular and Cellular Therapeutics facility produces Good Manufacturing Product (cGMP-grade) cell therapies for Phase I and Phase II human clinical trials. The MCT facility also houses the Fairview Cell Therapy Clinical Laboratory for minimally manipulated products and CD34 selection procedures, and it is adjacent to the Cancer Center Translational Laboratory, which supports cellular engineering for phase I-II clinical trials. The Translational Laboratory at the MCT also develops Regulatory T cell (Treg) products and iPSC-derived NK products.
Our Blood and Marrow Transplant and Cellular Therapy program adheres to rigorous regulatory standards and high-quality practices, as demonstrated by our continuous FACT Accreditation.