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The Foundation Fighting Blindness Partners with AGTC to Advance Gene Therapies into the Clinic
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The Foundation Fighting Blindness (FFB) has announced that it is collaborating with Applied Genetic Technologies Corporation (AGTC), a leading gene therapy development company, to advance gene therapies for retinal degenerative diseases into clinical trials. Specifically, the Foundation’s clinical trial support organization, the National Neurovision Research Institute (NNRI), is funding preclinical investigations of gene therapy to treat X-linked retinoschisis (XLRS), an inherited vision-robbing condition that affects young boys, at the University of Florida, Oregon Health and Sciences University (OHSU), and the University of British Columbia, as well as an achromatopsia (day blindness) study at the University of Pennsylvania. Success in these pivotal preclinical studies will enable AGTC to seek FDA approval to launch clinical trials. “This collaboration is a tremendous boost for the development of gene therapy products for retinal degenerative diseases,” said Stephen Rose, Ph.D., chief research officer, Foundation Fighting Blindness. “It affirms the great potential for science-guided foundations and academic researchers to work in partnership with commercial firms like AGTC that have the commitment and experience to bring these promising treatments directly to patients. AGTC will soon be launching Phase I/II clinical trials of their gene therapy for Leber congenital amaurosis (LCA) at the University of Massachusetts and OHSU, where they will be treating participants with higher doses than those used previously in three other landmark LCA gene therapy studies that restored some vision in 20 young adults and children. In AGTC’s forthcoming clinical trial, both the concentration and volume of the injected solution will be increased as they attempt to treat a larger area of the retina with a more powerful therapy. The hope is that they can restore even more vision. AGTC’s gene therapy technology was derived from preclinical studies that FFB funded almost a decade ago — studies that gave vision to Lancelot and other Briard dogs born blind from Leber congenital amaurosis. Jeff Chulay, M.D., chief medical officer at AGTC, says that AGTC’s adeno-associated virus (AAV) technology, used to deliver the healthy gene to the retina, has a number of advantages, including an excellent safety track record and the ability to readily transfect cells in the retina. Furthermore, once injected into the cells, the AAV treatments have demonstrated effectiveness over long periods of time. “Our AAV technology has performed very well. We are excited for its potential to save and restore vision in people with a variety of retinal conditions,” he adds. “This partnership is all about quickly moving vision-saving treatments into the clinic and out to the people who need them,” say William T. Schmidt, chief executive officer, Foundation Fighting Blindness. “We are excited about the potential for AGTC’s treatments to help so many of our constituents.” |
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