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ALACHUA– Applied Genetic Technologies Corporation, (AGTC), a privately-held, clinical stage biotechnology company developing gene therapy products to treat rare retinal diseases, has secured $37.5 million in a Series B round of venture capital funding.

AGTC is a graduate company of the University of Florida Sid Martin Biotechnology Incubator located in Alachua’s Progress Corporate Park.

Alta Partner and S.R. One, Limited led the financing, with new investor Osage University Partners joining existing investors InterWest, Intersouth, and MedImmune Ventures in the round.

This brings AGTC’s total venture capital funding to almost $88 million. AGTC began life in the Sid Martin Biotechnology Incubator in 2000 and graduated in 2006.

The funding will allow AGTC to continue development of its Phase 2 program in Alpha-1 Antitrypsin Deficiency (Alpha-1) and initiate full development of potential treatments for Achromatopsia (ACHM) and X-Linked Rentinoschisis (XLRS).

ACHM is an inherited genetic condition that presents at birth with impaired visual acuity. Most patients are legally blind, lacking color discrimination and experiencing extreme light sensitivity, resulting in daytime blindness. ACHM is caused by mutations in a group of genes which make the cone cells concentrated in the central retina non-functional. There is no treatment for Achromatopsia, although deep red tinted spectacles or contact lenses can reduce symptoms of light sensitivity. Approximately 22,000 patients in the U.S. and Europe suffer from this disease.

AGTC’s potential treatment uses an adeno-associated virus (AAV), a safe, man-made virus that delivers healthy copies of the ACHM gene to the cells of the retina, replacing the defective copies of the gene. A single treatment is expected to halt the disease for several years, perhaps a lifetime. The AAV delivery system is successfully being used in clinical trials of Leber congenital amaurosis gene therapy that have restored vision in more than 50 adults and children who were virtually blind. Previous research has shown promising signs of efficacy in dog models of ACHM.

XLRS, an inherited genetic condition, is a leading cause of juvenile macular degeneration in males. It is caused by mutations in the RS1 gene, which results in the layers of the central retina splitting. Patients typically begin to experience progressive vision loss between the ages of 5 and 10. Other early symptoms include the inability to focus both eyes and roving, involuntary eye movements. No treatment for XLRS is currently available. Approximately 35,000 patients in the U.S. and Europe suffer from this disease. Previous research has shown promising signs of efficacy in rodent models of XLRS.

AGTC is focused on the research and development of novel therapeutics for patients with unmet medical needs utilizing AGTC’s proprietary, non-pathogenic adeno-associated virus (AAV) delivery system. The company has demonstrated that this system can be used to deliver a normal form of a gene in both animals and humans thus allowing their own body to produce sustained therapeutic levels of important biologics. The company’s most advanced programs in development are treatments for Alpha-1 antitrypsin deficiency (Alpha-1) a disease causing a progressive loss of lung function, and Leber’s Congenital Amaurosis, an inherited condition causing early blindness. Both utilize AGTC’s proprietary AAV system and production methods.

"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.”

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