FFB\u2019s Investments Are Filling the Pipeline for Vision-Saving Therapies\r\nWith five gene-therapy clinical trials underway or soon to begin, Applied Genetic Technologies Corporation (AGTC) is generating tremendous excitement for the potential to overcome vision loss from several inherited retinal diseases.\r\nAt the Foundation\u2019s Investing in Cures Summit on September 16 in Chicago, Sue Washer, AGTC\u2019s chief executive officer, emphasized FFB\u2019s crucial role in moving the company and its projects forward. \u201cWe as an organization would not be here today without FFB,\u201d she said. \u201cAnd that all started with the work that was funded by the Foundation in Bill Hauswirth\u2019s lab at the University of Florida.\u201d Bill Hauswirth, PhD, is one of AGTC\u2019s scientific co-founders, and his groundbreaking gene-therapy research has been funded by FFB for 20 years.\r\nAGTC is now conducting early human studies for gene therapies for achromatopsia (day blindness) and X-linked retinoschisis (XLRS). The trials are progressing well with ongoing patient treatment and recruitment.\r\nIn addition, the company has submitted an investigational new drug application (IND) to launch a gene-therapy clinical trial for people with X-linked retinitis pigmentosa.\r\nNext year, the company is planning to submit an IND for an optogenetic treatment \u2014 a therapy designed to restore vision in people who are completely blind from a wide range of IRDs.\r\nMs. Washer explained that the Foundation also provided funding at a critical juncture in the company\u2019s growth and development. \u201cWhen we were still privately backed, our funds were short. We needed key funding to push XLRS gene therapy forward,\u201d she said. \u201cThat $1.5 million funding came from FFB. Without that grant and our ability to move that product forward, the company would not have been able to attract venture capital, go public, and be in the position where we will soon have five ongoing clinical trials.\u201d\r\nIn addition to funding promising biotech start-ups, FFB has played a critical role in developing research talent. Shannon Boye, PhD, an up-and-coming gene-therapy developer at the University of Florida who was mentored by Dr. Hauswirth, told the summit audience how important Foundation funding was in getting her career off the ground.\r\n\u201cI received my first individual investigator award from FFB in 2012. I was so excited, I did the happy dance with my kids in my kitchen,\u201d she said. \u201cThe data I obtained from the FFB grant enabled me to get a five-year award from the National Eye Institute in 2014. I got the NEI grant on my first shot, which was awesome, and I very much have the FFB to thank for that outcome.\u201d\r\nDr. Boye is currently partnering with the biotech Sanofi Genzyme to advance a gene therapy for Leber congenital amaurosis (GUCY2D mutations) into a clinical trial. She is also developing a dual-vector gene therapy for Usher syndrome type 1B, which is caused by mutations in the gene MYO7A. Most current vectors don\u2019t have the capacity to deliver large genes like MYO7A.\r\nOther presenters at the Investing in Cures Summit included:\r\nJohn Ash, PhD, an FFB-funded researcher at the University of Florida, reviewed lab research for his emerging neuroprotective treatments \u2014 including a drug and a gene therapy \u2014 which are designed to slow vision loss for a broad range of IRDs. Dr. Ash is also working to repurpose FDA-approved therapies as vision-saving treatments for IRDs.\r\nDaniel Chung, DO, the ophthalmic lead for clinical development at Spark Therapeutics, discussed how Spark\u2019s vision-restoring RPE65 gene therapy for Leber congenital amaurosis and retinitis pigmentosa (RP) is poised to become the first FDA-approved gene therapy for the eye or an inherited condition. The Foundation provided $10 million in research funding that helped make Spark\u2019s clinical trial possible.\r\nHalden Conner, co-founder and president of the start-up Nacuity, presented his company\u2019s emerging drug called N-Acetylcysteine Amide (NACA), an antioxidant that shows promise for slowing vision loss for people with RP and other IRDs. In addition to funding NACA-related lab studies at Johns Hopkins University, the Foundation is investing up to $7.5 million in Nacuity to move the drug into a Phase II clinical trial in 2018.