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CV of Invited Speaker Daniel C. CHUNG Position: Clinical Ophthalmic Lead Organization: Spark Therapeutics, Inc. Major Field: Ophthalmology, Gene Education: 1981-85 B.S. Eastern Nazarene College, Quincy, MA (Biology) 1988-88 M.A. Eastern Nazarene College, Quincy, MA (Family Counseling) 1990-94 D.O. New York College of Osteopathic Medicine, Westbury, NY (Medicine) Postgraduate Training and Fellowship Appointments: 1985-88 Research Assistant, Brigham and Women’s Hospital/Harvard Medical School, Boston, MA. 1988-90 Research Biologist, U.S. Dept. of Defense, Bethesda, MD. 1994-97 Fellow, Ocular Gene Therapy Section, Laboratory of Immunology, National Eye Institute/NIH, Bethesda, MD. 1997-98 Transitional Intern, Summa Health Systems, Akron, OH (ACGME accredited) 1998-01 Ophthalmology Resident, Summa Health Systems, Akron, OH (ACGME accredited) 2000-01 Profession Experiences: Chief Resident, Dept. of Ophthalmology, Summa Health Systems, Akron, OH. 2001-03 Fellow, Pediatric Ophthalmology, Cole Eye Institute, Cleveland Clinic Foundation, Cleveland, OH 2001-03 Research Fellow, Ophthalmic Genetics, Cole Eye Institute, Cleveland Clinic Foundation, Cleveland, OH Appointments: 2006-2010Instructor of Ophthalmology, Scheie Eye Institute, Department of Ophthalmology University of Pennsylvania School of Medicine 2008-2014Scientific Advisor, Leber Congenital Amaurosis Gene Therapy Clinical Trial Team Phase 1/3, The Children’s Hospital of Philadelphia. 2010-2014Senior Investigator, FM Kirby Center for Molecular Ophthalmology, Scheie Eye Institute, Department of Ophthalmology University of Pennsylvania School of Medicine 2014-2016Medical Affairs Ophthalmic Lead, Spark Therapeutics, Inc, Philadelphia, PA Global ophthalmic disease lead 2016-Clinical Ophthalmic Lead, Spark Therapeutics, Inc, Philadelphia, PA Global ophthalmic disease lead Honors & Awards: Dr. Chung is the Ophthalmic Lead for Spark Therapeutics. Prior to joining Spark Therapeutics, he was a senior investigator at the FM Kirby Center for Molecular Ophthalmology at the Scheie Eye Institute at the Perelman School of Medicine of the University of Pennsylvania, working in retinal gene therapy and transfer. Concurrently, he served as the scientific advisor on the RPE65 gene therapy study team at the Children’s Hospital of Philadelphia (CHOP). Dr. Chung earned his medical degree from the New Short Bio: York Institute of Technology College of Osteopathic Medicine and completed his residency in Akron, Ohio. He then completed fellowships in pediatric ophthalmology and ocular genetics research at the Cole Eye Institute at the Cleveland Clinic, with additional training in retinal gene therapy at the National Eye Institute in Bethesda, MD. In his current duties, he continues to be connected to the RPE65 gene therapy trial, as Spark Therapeutics is now the sponsor. As the global clinical ophthalmic lead, he works in the areas of medical affairs, clinical development and operations, and research and development. Abstract of Invited Speaker Title: Developing Patient Relevant Outcomes in Gene Therapy Trials Currently accepted endpoints for ophthalmic interventions are visual acuity loss and visual fields; however these endpoints of visual function may not accurately assess the functional vision of patients with inherited retinal dystrophies (IRD) or the benefits received from potential pharmacologic interventions. Therefore, endpoints that focus more on functional vision outcomes are being developed. Functional vision is defined as the vision that is required for the ability to conduct activities of daily living, and encompass aspects of light sensitivity, visual acuity, visual field and contrast sensitivity. Regulatory groups are also advocating endpoints that reflect improvements in the quality of life. Recent gene Abstract: therapy clinical trials using gene therapy for individuals with inherited retinal disease due to autosomal-recessive RPE65 gene mutations have developed a novel multi-luminance mobility course aimed at demonstrating changes in functional vision. This mobility course represents every day challenges faced by these and other IRD patients by requiring them to use their functional vision to follow a path and avoid obstacles. This population also suffers from nyctalopia, night blindness, and by administering the test at a number of different light levels, any changes in low light vision can be evaluated. The test was refined and standardized for use in the pivotal Phase 3 trial and Multi-luminance Mobility Testing Study was performed to confirm test construct and testing validity.