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FFB Partnership Accelerates Advancement of Gene Therapies into the Clinic A decade ago, five scientific leaders with expertise in gene therapy formed a company to commercialize their promising innovations for the treatment of incurable genetic diseases. They named the company Applied Genetic Technologies Corporation, with their acronym, AGTC, cleverly representing the four organic compounds — adenine, guanine, thymine, and cytosine — that make up the genetic code found in virtually every cell on the planet.  One of those founders was Bill Hauswirth, Ph.D., an FFB-funded researcher from the University of Florida, who at the time had just developed a gene therapy that would restore sight in more than 60 Briard dogs — including the world-famous Lancelot — born blind from Leber congenital amaurosis (LCA). Today, similar treatments in landmark Phase I clinical trials have restored some vision in 20 young adults and children who were virtually blind from LCA.AGTC will soon be launching Phase I/II clinical trials of their LCA gene therapy at the University of Massachusetts and Oregon Health and Science University, where they will be treating younger participants with higher doses than those used previously. 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. In partnership with the Foundation, AGTC is also developing gene therapies for X-linked retinoschisis (XLRS) and achromatopsia (day blindness), with the goal of bringing those to clinical trials shortly. The Foundation is currently funding preclinical studies for XLRS at the University of Florida, Oregon Health and Science University, and the University of British Columbia, as well as an achromatopsia study at the University of Pennsylvania. “We are excited to see AGTC taking laboratory advancements funded by the Foundation and moving them into the clinic,” says Stephen Rose, Ph.D., chief research officer, Foundation Fighting Blindness. “It’s the quickest and most effective path to moving these treatments out to the people who need them, and it is opening the door to use gene therapies to treat a variety of retinal degenerative diseases.” Jeff Chulay, M.D., chief medical officer at AGTC, says that many inherited retinal degenerative diseases are particularly amenable to gene therapy, because they are caused by a single genetic defect. He says, “We believe we have technology that can benefit patients with a variety of eye diseases. To treat the largest number of patients requires a commercial entity like AGTC, which has the production resources and expertise to obtain approval from the FDA.” Chulay 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 penetrate cells in the retina. Furthermore, once injected into the subretinal space, 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. |
