Applied Genetic Technologies Corporation (AGTC) was founded by five scientific leaders in the use of viral vectors for Gene Therapy and began operations in 2001. 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, raised over $45M in venture financing from blue chip investors and has five products in active development, one of which is partnered to Genzyme Corporation.

AGTC's most recent round of financing was completed in April 2009 and was led by Interwest Partners of Menlo Park, CA. Other investors included Intersouth Partners of RTP, NC and MedImmune Ventures of Gaithersburg, MD.

AGTC's lead product candidate is a treatment for Alpha One Antitrypsin Deficiency (Alpha-1). AGTC's treatment for Alpha-1, a debilitating respiratory disease caused by deficiencies in the tissue protective protein alpha one antitrypsin (AAT), has an outstanding safety profile in two Phase I human clinical trials conducted in Alpha-1 patients. The second trial also provided evidence of sustained expression of circulating protein in the top cohort of patients.

In July 2010 AGTC initiated a Phase II trial designed to further study safety in higher doses as well as evaluate efficacy as measured by circulating levels of AAT in patients. All patients are enrolled and the product has been well tolerated at all dosage levels. Alpha One is the most common potentially fatal hereditary disease of Western World adults; approximately 200,000 individuals are estimated to have Alpha One.

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 has developed a proprietary production system that is fully scalable, animal derived product free, and reviewed by US and European regulatory agencies. Further, the same basic system is applicable across the entire technology platform for all disease areas.

AGTC has exclusive rights to this novel production method 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. We have three additional products in development which, like Alpha One, qualify for Orphan Drug Designation. These are treatments for Leber Congenital Amaurosis (LCA), X Linked Retinoschisis (XLRS), and Achromatopsia (ACHM) – 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 was jointly developed with Genzyme Corporation of Cambridge, MA and is a treatment for Age-Related Macular Degeneration. AGTC recently licensed all commercial rights to the treatment to Genzyme which initiated a Phase I Clinical Trial in 2010.

AGTC facilities are located in Alachua, Florida, just twenty minutes from the University of Florida in the Progress Corporate 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 Incubator.