Page 8 - 19-3599 Sarepta Report

7 CONFIDENTIAL - FOR INTERNAL USE ONLY: DO NOT DISTRIBUTE The Gene Therapy Trajectory – Lessons For 2019 And Beyond With the promise of unprecedented, transformative medical treatment for patients coping with serious diseases, gene therapy is now poised to define the next era in global healthcare – as well as in market access. A brief chronology of major milestones in gene therapy is summarized below. As many readers of this report are aware, the first use of a gene therapy took place in West Germany in 1970 in an effort to treat two young sisters diagnosed with a rare genetic disorder that blocks the production of arginase, an enzyme necessary for avoiding the accumulation of arginine in the body. While both patients unfortunately failed to respond to the intervention, the science of gene therapy captured the interest of researchers and clinicians across the globe. The first approved clinical research in the U.S. involving gene therapy occurred two decades later on September 14, 1990 at the National Institutes of Health, when a four-year-old child was treated for a genetic defect that had resulted in a severe immune system deficiency. In 2003, China’s Food and Drug Administration approved Gendicine® (recombinant human p53 adenovirus), making it the world’s first gene therapy approved for commercial production. In 2012, the European Medicines Agency (EMA) granted a 5-year approval to Glybera® (alipogene tiparvovec) as the first gene therapy in the Western world. Glybera was designed to reverse lipoprotein lipase deficiency, a genetic disorder that can lead to severe pancreatitis. In late 2017, the Food and Drug Administration in the U.S. gave approval to the country’s first gene therapy, Luxturna® (voretigene neparvovec-rzyl), a one-time treatment for Leber’s congenital amaurosis (LCA), a rare, inherited retinal disease in adults and children that results in vision loss. Rose ME, Huerbin MB, Melick J, Marion DW, Palmer AM, Schiding JK, et al. Regulation of interstitial excitatory amino acid concentrations after cortical contusion injury. Brain Res. 2002;935(1-2):40-6. This area will be used for addtional callouts and points of interests. Heading Here Adipiscing elit, sed do eiusmod tempor incididunt ut labore et dolore magna aliqua. Ut enim ad minim veniam. quis nostrud exercitation ullamco laboris nisi ut aliquip ex ea commodo consequat. Duis aute irure dolor in reprehenderit in voluptate velit esse cillum dolore eu fugiat nulla pariatur.