Applied Genetic Technologies Corporation (AGTC) is a clinical-stage gene therapy company developing products based on Adeno-Associated Virus vector (AAV). AAV provides long-term gene expression without toxicity or significant immune response. Products under development include treatments for inherited and acquired diseases, including Alpha-1 Antitrypsin Deficiency, Age- Related Macular Degeneration, Pompe Disease, and Leber's Congenital Amaurosis (LCA). Lead inventor Dr. Nicholas Muzyczka began working on the development of gene therapy vectors in the early 1970's, and twenty years later he became the founding Director of the UF Powell Gene Therapy Center; he serves on the NIH Recombinant Advisory Committee and the Board of Directors of the American Society for Gene Therapy. CEO Sue Washer has ten years of pharmaceutical management and research experience with Abbott Labs and Eli Lilly, and eleven years of senior management experience with entrepreneurial firms in the Gainesville area. She has worked with the UF Office of Technology Licensing to license technologies .
AGTC has received $1 million in public funding and $6 million in seed funding. The company netted over $20 million in its November 2003 financing round, led by Interwest Partners of Menlo Park, California joined by Skyline Ventures of Palo Alto, California, Intersouth Partners of Research Triangle Park, North Carolina, and MedImmune Ventures of Gaithersburg, Maryland.