Gene therapy outfit nabs $37.5M round for clinical trial work

Florida's fledgling biotech community has picked up boasting rights to a big venture round for one of its University of Florida spinouts.  Applied Genetic Technologies, one of a growing group of upstart gene therapy biotechs, nabbed a $37.5 million round from A-list investors to back a leading mid-stage orphan product and a slate of earlier-stage programs.

Applied Genetic has been developing its adeno-associated viral delivery platform for the past 11 years, putting a new gene therapy in the spotlight for alpha one antitrypsin deficiency, a potentially lethal respiratory ailment. Now in Phase II, the alpha-one product is designed to demonstrate how Applied Genetic can correct a genetic malfunction that spurs the disease in some 200,000 patients. And the company boats that its cell-culture based production method can more efficiently produce this and other products. Another program for age-related macular degeneration is partnered with Genzyme while investigators in the virtual company, which has 7 workers, are also at work on Leber Congenital Amaurosis, X Linked Retinoschisis and Achromatopsia.

"These gene therapy products that AGTC is working on are almost like miracles," David Day, director of technology licensing at the University of Florida, told The Gainesville Sun. "These are the kinds of things that change people's lives in a permanent way for the better. To get cures for diseases closer to market is great." Day added that the venture round also is likely to bring a buyout offer closer. And it's clear that some big outfits are very interested in what they're doing.

GSK's S.R. One joined this round as a new investor, along with Alta Partners and Osage University Partners. MedImmune Ventures, part of the AstraZeneca group, has been backing the company as well, along with Intersouth and InterWest. The company CEO tells the Sun the new money will help the company double its staff in Gainesville, FL.

Gene therapy has had more than its share of ups and downs over the years as safety issues and new technology hurdles repeatedly batted back leading therapies. But now a new generation of developers has come along, like bluebird bio (a 2012 Fierce 15 company), which believes that they have overcome the challenges and are ready to start delivering products to the market.

"We are strong believers in the business model of developing treatments for genetic disorders," said Ed Hurwitz, general partner of Alta Partners. "Based on encouraging clinical results from AGTC and others, we concluded that a large set of genetically defined diseases could be cured using AGTC's proprietary vectors and manufacturing technologies. The Series B financing is designed to move several of AGTC's programs through proof of concept as well as to allow the company to leverage its manufacturing and development infrastructure with partners to accelerate a broad portfolio of curative products."

- here's the press release
- read the story from The Gainesville Sun

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