Washington Editor

In its second round of financing, Applied Genetic Technologies Corp. raised $15.2 million to fund advancement of its gene therapy treatment for alpha-1 antitrypsin deficiency, an inherited form of emphysema.

Founded in September 2001 on the work of five research scientists from the University of Florida and the University of California, Applied Genetic Technologies Corp. (AGTC), of Gainesville, Fla., seeks to use its adeno-associated virus (AAV) technology as a gene therapy delivery mechanism.

The company selected alpha-1 antitrypsin deficiency (A1AD) as its first targeted disease because of its unique aspects, Susan Washer, AGTC's president and CEO, told BioWorld Today. "This disease is extremely well understood. Everybody understands exactly what's going on in the disease, what the clinical course of the disease is. And there's a protein replacement product on the market that is considered to be very efficacious, so to apply a new form of treatment like gene therapy to a disease that's this well-characterized offers some advantages."

AGTC's first financing, a $6 million investment made by PrimeBio Tech SAS, of Paris, helped the company complete preclinical studies in A1AD and to scale up its manufacturing levels to support both large clinical trials and a potential product launch.

Washer said the company expects to enter Phase I trials in 2004. She believes the $15.2 million raised in the Series A-1 round will last three years, and she does not expect to look for a development or commercialization partner. "Alpha-1 antitrypsin is a very focused market, and it qualifies for orphan drug protection in the U.S. We feel this is a product we can bring all the way through the process ourselves," she said.

A1AD affects about 100,000 people in the U.S. and another 100,000 in Western Europe, Washer said. Patients suffering from the disease often experience significant loss of daily function and shortened life spans.

The company believes a single gene therapy treatment will stabilize deterioration of lung function in individuals chronically affected. For newly diagnosed patients, treatment likely would lead to prevention of lung abnormalities and symptom-free normal life spans.

The proposed treatment is one of several human therapeutics under development by AGTC using its AAV technology and production methods, the company said.

The Series A-1 financing was led by InterWest Partners, of Menlo Park, Calif. Other participants were InterSouth Partners, of Research Triangle Park, N.C.; MedImmune Ventures Inc., of Gaithersburg, Md.; and Skyline Ventures, of Palo Alto, Calif.

AGTC employs eight people and operates out of the University of Florida's Sid Martin Biotechnology Development Incubator.