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The University of Florida and AGTC Secure $8.4M Grant to Study Rare Eye Disease

Funds will be used to develop a gene therapy treatment for Achromatopsia in both animal models and human clinical trials

July 23, 2013 - The University of Florida Department of Ophthalmology and AGTC, a privately-held, clinical stage biotechnology company, announced today that they have secured $8.4 million in funding from the National Eye Institute (NEI), a division of the National Institutes of Health. This is only the third grant awarded by the NEI this year. The grant will support the partners’ development of a pre-clinical program to treat the eye disease Achromatopsia.

“Thanks to this funding, we will be able to work with our partners to move quickly toward the goal of developing a treatment for patients with this debilitating condition,” says Sue Washer, President and CEO of AGTC.

Achromatopsia (ACHM) is an inherited genetic condition that affects about 22,000 patients in the U.S. and Europe. Patients demonstrate impaired visual acuity at birth. ACHM is caused by genetic mutations in certain photoreceptor cells in patients’ eyes; when these cells do not function properly patients lose their fine visual acuity. Most are legally blind, lack color discrimination and experience extreme light sensitivity, resulting in daytime blindness. There is no treatment for Achromatopsia, although deep red tinted glasses or contact lenses can reduce symptoms of light sensitivity.

The potential treatment being tested uses an engineered 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 stop the disease for at least 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 and mouse models of ACHM.

"We already have very encouraging results in several animal models that this treatment has the potential to restore vision function,” says William Hauswirth, Ph.D., a Research Professor of ophthalmology in the UF College of Medicine, a part of UF Health. “Our world-class clinical team stands ready to test this gene-based therapy in Achromatopsia patients and begin developing treatments.”

UF and AGTC will be working with several academic and commercial partners including the Bascom Palmer Eye Institute at the University of Miami, Oregon Health Science Center, The Chicago Lighthouse, the Medical College of Wisconsin and Covance Laboratories. 

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