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Applied Genetic Technologies Corporation (AGTC) was founded in January, 1999 by five scientific leaders in the use of viral vectors for Gene Therapy. They subsequently secured rights to a portfolio of strategically vital intellectual property in the application of Adeno-Associated Virus (AAV) to gene therapy. Since that time the Company has signed licenses to over 40 US and foreign patents, completed two rounds of venture financing, has five products in active development, and has signed a collaboration with a top five Biotech company. AGTC's most recent round of financing was completed in November 2003 and was led by Interwest Partners of Menlo Park, CA (www.interwest.com). Other investors included Skyline Ventures of Palo Alto, CA (www.skylineventures.com); Intersouth Partners of RTP, NC (www.intersouth.com); and MedImmune Ventures of Gaithersburg, MD (www.medimmune.com). The Company raised over $27M in this round to fund clinical development of its lead product and to complete scale-up of its manufacturing process. AGTC's lead product candidate is a treatment for Alpha One Antitrypsin Deficiency (Alpha One) an inherited cause of Emphysema. Alpha One is the most common potentially fatal hereditary disease of Western World adults; approximately 200,000 individuals are estimated to have Alpha One. Phase I Human Clinical Trials for an initial formulation of this treatment were completed at the University of Florida as an academically sponsored trial and demonstrated the safety of this treatment approach. A second AGTC sponsored Phase I trial with a higher potency version of the product is in progress. One of the biggest issues facing the viability of AAV gene therapy as a business model is the costly, inefficient and potentially inadequate cell-culture based production method. AGTC applied its expertise to develop a highly scalable production process for AAV. AGTC has exclusive rights to novel production methods that it adapted for production in large scale bioreactors. Bioreactors enable consistent large scale production of vector and easily support full scale clinical trials and market launch. Our strategy is to identify diseases which can be treated by delivery of a known gene and for which no other adequate treatments are available. Based on this we are also moving forward with four additional product candidates. Three of these products, like Alpha One, qualify for Orphan Drug Designation. These are treatments for Leber Congenital Amaurosis (LCA), X‑Linked Retinoschisis (XLRS), and Achromatopsia - all of which are inherited deficiencies that result in early blindness. Patients with these devastating diseases currently have no treatments available to them. The fifth product in development, while not an Orphan Disease, builds on AGTC's experience in ophthalmic indications. It is a treatment for Glaucoma patients whose disease is not controlled by the currently available treatments; approximately 10% of all cases. AGTC also jointly developed with Genzyme Corporation of Cambridge, MA (www.genzyme.com) a treatment for Age-Related Macular Degeneration. The team recently completed all the pre-clinical proof of concept and safety trials which show the treatment has high clinical potential. AGTC recently licensed all commercial rights to the treatment to Genzyme which will initiate the clinical trial process. AGTC facilities are located in Alachua, Florida, just twenty minutes from the University of Florida in the Progress Park. This fast growing business park is home to dozens of life science based companies as well as the University of Florida's Sid Martin Biotechnology Development Incubator (www.biotech.ufl.org).