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One in 10 Americans suffer from one of 7,000 rare diseases, defined in the U.S. as any disease affecting fewer than 200,000 people. With a relatively small community to lean on for support, managing a rare disease diagnosis can be lonely. Patients often receive multiple misdiagnoses before even learning the name of their disease.

In Alachua County, Applied Genetic Technologies Corporation (AGTC) is determined to change the narrative. AGTC is a clinical-stage biotechnology company developing genetic therapies for people with rare and debilitating ophthalmic, otologic and central nervous system diseases. Priding itself on 20 years of gene therapy experience, AGTC has found success in designing and constructing all critical gene therapy elements and bringing them together to develop successful treatments for patients.

AGTC’s long-standing relationship with the University of Florida (UF) has brought to fruition multiple Sponsored Research Agreements to cover the exploration of new treatments and to support clinical trials and research projects that will enhance its technology platform. Its project pipeline centers on the exploration of treatment options for Optogenetics, Age-Related Macular Degeneration and Non-Syndromic Hearing Loss and Deafness.

In May, the former UF Innovate | Sid Martin Biotech client executed an agreement to expand its local footprint with the development of quality control labs and a manufacturing suite. Located in Foundation Park, AGTC’s new facility will join other biotechnology and life science companies that desire “more customized, flexible and affordable R&D and manufacturing facilities,” said the developer in a news release.

Expected to be operational in 2022, AGTC’s new facility will help the company meet anticipated demand for its X-Linked Retinites Pigmentosa therapy for rare diseases causing blindness in boys and young men, as well as other clinical and pre-clinical opportunities, explained President and CEO Sue Washer. She also expects the facility to create new jobs.

AGTC’s news of expansion comes on the heels of legislation passed unanimously this spring to establish a 20-member Rare Disease Advisory Council within the Department of Health, making Florida one of only 17 states to have passed this type of legislation and signed it into law. The Council will not only advocate on behalf of patients with rare diseases, but also advise businesses and academic institutions on potential research.

Now, with increased government support, Florida’s innovation community is better positioned to foster the continued success of companies like AGTC – sparking growth of the life sciences sector and, more importantly, offering hope to patients with rare diseases for improved quality of life and perhaps even a cure.

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