The Foundation Fighting Blindness (FFB) is a private non-profit organization which – by soliciting donations from individual donors, corporations, government agencies and non-profits – funds cutting-edge research aimed at providing preventions, treatments and cures for blinding retinal diseases affecting more than 10 million Americans alone. FFB accomplishes this mission by maintaining a Research Grants Program which, over four decades, has facilitated scores of research advances, including a sight-restoring treatment now in clinical, or human, trials. The Foundation currently funds or had a hand in facilitating 15 clinical trials and intends to eradicate blindness due to retinal diseases by adhering to the following measures: • Research: Since its inception, in 1971, FFB has raised more than $500 million for research that has pinpointed the primary causes of retinal diseases and laid the foundation for treatments now being tested in clinical trials. • Public Education: FFB raises public awareness about retinal diseases through events, seminars and the media and addresses the needs of those living with retinal diseases by providing information on research and low vision aids and services.

Medical research is costly, particularly now, when the Foundation's four decades' worth of investments is paying off, with promising treatments being readied for evaluation in clinical trials, each costing tens of millions of dollars and subject to regulatory oversight by the U. S. Food and Drug Administration. FFB leverages its experience, expertise and partnerships with its members and leading retinal disease researchers to: a) raise funds through events, membership drives, planned giving and one-on-one queries; and b) invest in the most promising research. Our strategies include: Research • Recruiting and training promising medical students, researchers and faculty with career development grants so that they can pursue careers in retinal disease research. • Facilitating collaborations that cross disciplinary, institutional and geographic boundaries through research center grants, which support research likely to accelerate the development of treatments and cures. • Attracting individual investigator grant applicants from a worldwide pool of scientists who potentially bring new ideas, perspectives and expertise to retinal disease research; and providing qualified, independent scientists with investigator research grants. Public Education • Providing comprehensive patient-focused materials, including information on the symptoms, progression and available preventions and/or treatments for various retinal degenerative diseases, free of charge to individuals who request them via phone, mail or email. • Sharing basic information, research news and stories about the impact of retinal diseases and research via print and online newsletters, emails, the Foundation's website and blog and internet social-media sites, such as Facebook and Twitter. • Promoting FFB's impact by supplying print, internet, TV and radio media with stories and public service announcements. • Training and utilizing our nationwide network of 47 chapters to provide those affected by retinal diseases with support, resources and vital information. • Hosting public health education events and meetings during which information about FFB and retinal research is disseminated.

Since its establishment in 1971, the Foundation has raised more than $500 million and become the world's premier non-governmental funder of retinal disease research. FFB's Research Grants Program currently supports 123 research projects at 76 of the world's leading scientific institutions. There are 25 members on FFB's board of directors as well as 115 national trustees, and its Scientific Advisory Board consists of more than 40 renowned retinal experts. FFB has a network of 47 chapters serving the visually impaired in metropolitan areas nationwide, and its work is facilitated by a 90-person staff based in regional offices in California, Colorado, Florida, Illinois, New York, North Carolina, Texas and at its national headquarters in Maryland. They are aided by thousands of volunteers nationwide. FFB also has: • The support of corporate sponsors and partners, the National Institutes of Health and generous individual donors. • A passionate membership network raising awareness and funds locally, regionally and nationally via chapter meetings; more than 50 5K VisionWalks nationwide; 20-plus “Dining in the Dark" events; and traditional and social media. • The Partners for Retinal Health program, through which FFB is promoted and supported by retinal specialists and practices nationwide.

The Foundation's final goal is to eradicate blindness caused by retinal diseases, a goal that has yet to be met. Progress, however, has been substantial, especially in the past 10 years, as the pace of research advances has quickened. FFB's efforts to maintain this momentum by increasing fundraising levels across the board can be measured against the following benchmarks: Research • When FFB was established in 1971, not one gene had been identified as the cause of a retinal disease; to date, more than 200 have been identified, with each year bringing a new crop of discoveries. • There were no trials for treatments or cures 41 year ago; whereas, currently, there are 15 clinical trials FFB is either funding or had a hand in facilitating. • An ongoing Foundation-funded clinical trial for a gene therapy treatment of a retinal disease that causes legal blindness at birth has, thus far, partly restored vision in more than 40 participants, including children. • The Foundation's Scientific Advisory Board recently awarded $2. 4 million in grants to eight pre-clinical projects as initial steps toward getting treatments to the marketplace. Public Education • Steady year-to-year growth has resulted in a network of 47 chapters of the Foundation serving metropolitan areas in 30 states. • The print version of FFB's newsletter, InFocus, is published three times a year and mailed to approximately 70,000 Foundation members; whereas the electronic version, InFocus Online, is published six times a year and is sent to more than 128,000 online registrants. • In 2012, as part of our public service announcement campaign, our radio messages were broadcast more than 55,000 times, reaching an estimated 2. 6 million listeners and resulting in nearly 900 phone calls requesting information. • The Foundation's low-vision-friendly website, providing visitors with updated information on research, resources and events, had 431,746 unique visitors, resulting in 1. 3 million page views, in 2012. • FFB also launched a lay-audience-targeting blog, “Eye on the Cure," in early 2012, and aggressively stepped up its use of social media to reach hundreds of thousands worldwide and create an online community for people living with vision loss from retinal diseases. • Nearly 600 people from 42 states and seven countries attended the Foundation's annual conference, VISIONS 2012, in Minneapolis, Minnesota, where attendees browsed the displays of 23 vendors, attended research, coping and assistive technology workshops and listened to expert speakers. • Our Vision Seminar Series, a popular annual program of free half-day events held in seven cities, brought in 3,105 people.

Foundation's early investment in LUXTURNA™ boosts vision-restoring treatment for people with RPE65 mutations and will help advance other gene therapies currently in development. (Columbia, MD) — Today's U. S. Food and Drug Administration (FDA) approval of voretigene neparvovec, to be marketed as LUXTURNA, will be life-changing for patients with vision loss due to mutations in the RPE65 gene and a watershed moment for the inherited retinal disease field, says the Foundation Fighting Blindness. The Foundation was an important early investor in LUXTURNA, providing $10 million in critical seed funding for the therapy. The groundbreaking treatment is the first gene therapy for the eye and for any inherited disease to be approved by the FDA. The treatment restores vision by delivering working copies of the RPE65 gene directly into the retina, thereby compensating for the nonfunctional, mutated genes. “We are thrilled for the patients whose lives will change dramatically because of this treatment," says David Brint, Foundation Fighting Blindness chairman. “We are also pleased to have this concrete example of the strength of the Foundation's strategy of identifying and investing early in promising treatments. Doing so helps attract industry investment that can usher promising treatments through clinical trials and ultimately FDA approval. " LUXTURNA is the result of more than two decades of research and development at the University of Florida, the University of Pennsylvania, Children's Hospital of Philadelphia, and Spark Therapeutics. The Foundation Fighting Blindness' seed investment allowed researchers to take the therapy through the early investigational stages critical to any treatment development. “LUXTURNA will be life-changing for people with an inherited retinal disease caused by RPE65 mutations. For them, the treatment means a life of independence. Also important is the momentum this approval provides to other gene-based therapies — for the eye and other diseases — now in the clinic," says Benjamin Yerxa, PhD, Foundation CEO. Twenty-four-year-old Katelyn Corey participated in the clinical trial that led to LUXTURNA's FDA approval. Before the trial, failing vision was causing her to consider giving up her lifelong dream of completing college and working in science. But, in December 2013, she received the RPE65 gene therapy in Spark's Phase 3 clinical trial, and her education and science career got quickly back on track. “Within days, I could see vibrant colors. I could even see the Philadelphia City Hall clock tower at night," she says. “Also, now, I can go to a restaurant and see everything by candlelight, and I can see stars in the night sky. " Corey recently earned a master's degree in epidemiology and now works as a research analyst for the U. S. Department of Veterans Affairs. An additional noteworthy milestone is the demonstrated value of a new clinical endpoint devised by the Spark Therapeutics team to measure LUXTURNA's impact.