GAINSVILLE, FL - Applied Genetic Technologies Corp., a clinical stage biotechnology company has closed on an $11,800,000 financing round led by InterWest Partners of Menlo Park, CA. Other participants in the round include Intersouth Partners and MedImmune Ventures Inc.

AGTC is focused on the research and development of novel therapeutics for patients with unmet medical needs utilizing the non-pathogenic adeno-associated virus (AAV).

In diseases caused by genetic mutations AAV is used to deliver a normal form of the gene to the patients, allowing their own body to produce a therapeutic protein.

The investment funds will be used by AGTC to complete the next phase in human clinical trials for its gene therapy treatments for Alpha-1 Antitrypsin Deficiency, a form of emphysema and Leber’s Congenital Amaurosis (LCA) a form of childhood blindness.

It will also fund preclinical development of gene therapy treatment for Achromatopsia, a form of daytime blindness.

Sue Washer, president and CEO of AGTC, said, “The clinical results stemming from the delivery of genes to correct inherited disorders are promising, especially for patients with genetic ocular diseases for which no treatment options are currently available.”

Alpha-1 is a hereditary defect that causes early onset emphysema in adults. Patients experience significant loss of daily function and shortened life spans.

Alpha-1 is the most common potentially lethal hereditary disease of American and Northern European adults affecting approximately one in 2500 persons.

There are approximately 130,000 symptomatic patients in the US and Europe. Previous human trials have shown the product to be well tolerated and can provide sustained expression of the therapeutic protein.

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