Issue link: http://read.dmtmag.com/i/54910
genes to human cells, stimulating the patient's body into producing essential proteins for treating genetic diseases. AGTC participated in the Florida High Tech Corridor Council's (FHTCC) Matching Grants Research Program to further its research aimed at developing a treatment for a disease that causes daytime blindness in children. AGTC's research project showed proof of concept for its Achromatopsia gene therapy treatment. "Achromatopsia is an inherited disease that causes management changes in the Everglades, the technology has varied applications. The team's expertise has drawn interest from wildlife researchers around the country, and the Nova 2.1 sUAS will soon study natural environments from the skies over Idaho and Chesapeake Bay. "The collective knowledge this team has compiled on unmanned aerial platforms, payload design, fl ight mission planning and image georeferencing will have a far-reaching impact felt beyond ecological research," said Percival. young children to have daytime blindness, which limits their ability to go outside or even be in a lit room," said Sue Washer, president and CEO of AGTC. Using FHTCC's matching funds, AGTC worked with Dr. William W. Hauswirth at UF's Department of Ophthalmology to test its Achromatopsia treatment in mice. Through the success of those trials, AGTC then went on to test the gene treatment in dog models and Washer and her team put together a body of data helping move through the Food and Drug Administration approval process. "We're now raising funds to proceed to the human clinical trials portion of the project," Washer explained. Although continuing its Achromatopsia research, Virally Enhanced Vision When you hear the word "virus," what often comes to mind is a nasty agent that spreads diseases ranging from infl uenza to ebola. A 10-year-old research project that began in the labs of the University of Florida (UF) has demonstrated a method using a certain type of virus that could actually cure diseases caused by genetic defects. This gene therapy is driving the growth of Applied Genetic Technologies Corporation (AGTC) in Alachua, Fla., a graduate of UF's Sid Martin Biotech Incubator Program. Founded by UF scientists covering a range of specialties including molecular virology, biochemistry, genetics and immunology, AGTC is pioneering gene therapy through the use of Adeno-Associated Virus (AAV). This non- replicable virus is used as a delivery mechanism to supply 34 florida.HIGH.TECH 2012 AGTC is working on many other projects. The Foundation Fighting Blindness awarded AGTC a $1.5 million grant to conduct a pre-clinical study evaluating gene therapy treatment for another genetic eye disease, X-linked Retinoschisis (XLRS), the leading cause of juvenile macular degeneration in young males. The company's most advanced product is currently in phase two clinical trials to help treat Alpha-1 Antitrypsin Defi ciency, a form of inherited emphysema. A longstanding supporter of the Corridor's life sciences industry, Washer is the current chair of BioFlorida and serves on the Florida High Tech Corridor Council. "The Matching Grants Research Program is an excellent initiative that allows companies to leverage their research dollars and get more information for the same amount of money, which is especially critical for early stage companies" said Washer. "It can make the difference in getting that initial bit of data needed to then receive larger grants and investment funding."