BUSINESS OVERVIEW 6 and significant competition. Any product candidates that we Phase I I I data. GlaxoSmithKline plc’s anti-amyloid beta successful ly develop and commercialize wil l have to compete monoclonal for patients with GA is in Phase I I clinical trials. with therapies that may become available in the future. We face Novartis’ LFG-316 C5 monoclonal antibody for patients with competition from pharmaceutical and biotechnology companies, GA is in Phase I I clinical trials. Apel lis Pharmaceuticals, Inc. as well as from academic institutions, governmental agencies and is currently developing a C3 inhibitor, which has completed public and private research institutions. Phase I I clinical trials. We are aware of a number of companies focused on developing • GS030 for the Treatment of Dry AMD: No approved therapy gene therapies in various indications, including Adverum, currently exists for dry AMD. Ophthotech Corporation’s Dimension Therapeutics, Inc., Applied Genetic Technologies Zimura, a complement C5 inhibitor, is currently in a Phase I Ib Corporation, Asklepios BioPharmaceutical, Inc., Audentes clinical trial. Johnson and Johnson is developing Palucorcel, Therapeutics, Inc.,bluebird bio, Inc., GlaxoSmithKline, Nightstar which utilizes human embryonic stem cel ls that recently Therapeutics Ltd., Spark Therapeutics, Inc., uniQure N.V. completed a Phase I/I I trial. Similarly, Astellas Pharma Inc. is and Voyager Therapeutics, Inc., as wel l as several companies developing a stem cell treatment currently in Phase I I. BioTime, addressing other methods for modifying genes and regulating Inc. is developing OpRegen, a dry AMD-targeted therapy that gene expression. Any advances in gene therapy technology made replaces missing retinal pigment epithelium cells with OpRegen by a competitor may be used to develop therapies that could cel ls and is currently in a Phase I/I Ia dose escalation study. compete against any of our product candidates. Regenerative Patch Technologies requires a subretinal implant of stem cells and is in a Phase I/I I trial. For our specific retinal gene therapy products, the main competitors include: Many of our potential competitors, alone or with their strategic • GS010 for the Treatment of LHON: The Bascom Palmer Eye partners, have substantially greater financial, technical and other Institute of the University of Miami Miller School of Medicine resources than we do, such as larger research and development, in the United States has enrolled the first five participants in its clinical, marketing and manufacturing organizations. Mergers and Phase I trial of virus-based gene transferfor this mitochondrial acquisitions in the biotechnology and pharmaceutical industries disorder. The Huazhong University in China, which completed may result in even more resources being concentrated among a Phase I/I I studies in 2013 using gene therapy, recently smaller number of competitors. Our commercial opportunity could published data with nine months follow-up and the evaluation be reduced or eliminated if competitors develop and commercialize of both safety and some visual acuity outcomes. The purpose products that are safer, more effective, have fewer or less severe of these studies is exclusively academic and, to our knowledge, side effects, are more convenient or are less expensive than any Huazhong University is not currently developing any drug products that we may develop. Competitors also may obtain FDA or candidates. We are aware of other companies and institutions other regulatory approval for their products more rapidly than we focused on developing therapies in the LHON space that do may obtain approval for ours, which could result in our competitors not involve gene therapy, including: Santhera Pharmaceuticals establishing a strong market position before we are able to enter the Holding AG using a chemical entity, Idebenone, approved market. Additionally, technologies developed by our competitors under exceptional circumstances in Europe; and Stealth may render our potential product candidates uneconomical or Biotherapeutics Inc. using an antioxidant agent known as obsolete, and we may not be successful in marketing our product Ocuvia, which is currently in Phase I I clinical trials. candidates against competitors. • GS030 for the Treatment of RP: To our knowledge, we are the only company developing a technology that utilizes light at 6.12 near-red wavelength with goggles. RetroSense Therapeutics, GOVERNMENT REGULATION LLC, or RetroSense, is developing a ChR2-based optogenetic We are subject to a variety of laws and regulations in France, the product that is in Phase I studies that will have to utilize blue light to stimulate the ChR2 without goggles. Retrosense was United States and the European Union. Our product candidates acquired by Allergan plc in September 2016. In addition, the use biological products and medical devices that are subject to only approved non-therapeutic treatment for RP is retinal laws and regulations regarding testing, manufacturing, safety, implants. efficacy, labeling, packaging, storage, record keeping, distribution, export and import, reporting, approval, advertising and other • GS030 for the Treatment of GA: No approved therapy promotional practices. currently exists for GA. Most major clinical-stage therapeutic treatments for GA are in the field of cel l therapy, including Clinical Trials on Human Subjects lampalizumab, an anti-Factor D, the development of which Before testing any biological product candidate, including a gene F. Hoffmann-La Roche Ltd. terminated post unsuccessful therapy product, in humans, the product candidate enters the 84 – GENSIGHT BIOLOGICS – 2017 Registration Document