Translational Research

Mission: Development and production of investigational biological therapeutic agents for use in Phase I and II clinical trials.

Novel cell- and gene-based therapies are being developed through dramatic advances in understanding stem cell replication, differentiation and tissue development, identification of numerous cytokines (proteins of the immune system produced mostly by white blood cells) and genes, and sophisticated cell separation and culture methods. The U.S. Food and Drug Administration (FDA) require that these investigational biological therapies be produced in accordance with current Good Manufacturing Practices (cGMP) when studied in clinical trials.

Development and production of novel biological therapies, including stem cell-based therapies, progenitor (precursors to cells) cell-based therapies, and genetic modification of stem cells, represent major research efforts of the University of Minnesota Stem Cell Institute. The complex and rigorous regulatory requirements, extensive training, experience, and physical facilities necessary for GMP production of biologicals (medicinal compounds prepared from living organisms) are, however, outside the scope and expertise of most individual investigators.

The Minnesota Molecular and Cellular Therapeutics (MMCT) Facility will house the Translational Research Laboratory. The Laboratory will provide critical support for phase I and II clinical trials of advanced cell-, tissue-, and gene-based therapies by producing novel investigational biological agents under cGMPs. The MMCT Facility provides the environment and regulatory structure needed to meet FDA requirements specified in cGMP regulations. Advanced, innovative cell- and gene-based therapies that are being developed by University researchers often require adaptation and translational development, often exceeding the capacity of less flexible contract GMP manufacturers. The Translational Research Program will enable Stem Cell Institute investigators to carry out translational clinical trials that synthesize protein for novel biological therapies, which is fundamental to the overall goals of the institute and the university.

The Vector Production Program has the overall goal of producing and testing novel retroviral vectors (viruses that contain an enzyme necessary for production of DNA from RNA) for clinical gene transfer. Clinical application of gene therapy projects is made possible by the activities of the Gene Therapy Laboratory at the MMCT Facility, where clinical-grade vector is prepared. The laboratory provides expertise in construction and packaging of retroviral vectors (carriers of viruses that contain an enzyme necessary for production of DNA from RNA) and gland-associated virus carriers.

As a producer of experimental materials for clinical use, MMCT policies are made in accordance with the FDA guidelines as stated in the Code of Federal Regulations (CFR) and the Food, Drug, and Cosmetic Act. Compliance with and commitment to the CFR and the Food, Drug and Cosmetic Act are demonstrated through formal cGMP training, a controlled documentation system, and an overall quality assurance (QA) program.

The purpose of the QA program is to provide an overall organizational framework and way of functioning that will result in cellular products of consistent, predictable composition that are free of unwanted materials, that meet the expectations of physicians who administer the components, and that comply with regulatory requirements and external standards.