RISK FACTORS 4 • restrict the marketing or manufacturing of the product; We face significant competition in an environment of rapid • seize or detain the product or otherwise require the withdrawal technological change, and our competitors, many of which have of the product from the market; more resources and experience than we have, may achieve regulatory approval before us or develop therapies that are more • refuse to permit the import or export of products; or advanced or effective than ours. • refuse to al low us to enter into supply contracts, including The biotechnology and pharmaceutical industries, including government contracts. the gene therapy field, are characterized by rapidly changing Any government investigation could require us to expend technologies, significant competition and a strong emphasis on significant time and resources in response and could generate intellectual property. We face substantial competition from many negative publicity. The occurrence of any event or penalty different sources, including large and specialty pharmaceutical described above may inhibit our ability to commercialize our and biotechnology companies, academic research institutions, product candidates and adversely affect our business, financial government agencies and public and private research institutions. condition, results of operations and prospects. We are aware of several companies focused on developing gene In addition, the FDA or EMA’s policies, and those of equivalent therapies for various indications, including Adverum, Dimension other regulatory agencies, may change and additional government Therapeutics, Inc., Applied Genetic Technologies Corporation, regulations may be enacted that could prevent, limit or delay Asklepios BioPharmaceutical, Inc., Audentes Therapeutics, Inc., regulatory approval of our product candidates. We cannot predict bluebird bio, Inc., GlaxoSmithKline, Nightstar Therapeutics Ltd., the likelihood, nature or extent of government regulation that Spark Therapeutics, Inc., uniQure and Voyager Therapeutics, may arise from future legislation or administrative action. If we Inc., as well as several companies addressing other methods for are slow or unable to adapt to changes in existing requirements or modifying genes and regulating gene expression. Advances in the adoption of new requirements or policies, or if we are not able gene therapy technology made by a competitor may be used to to maintain regulatory compliance, we may lose any marketing develop therapies that could compete against any of our product approval that we may have obtained and we may not achieve or candidates. In addition, our individual product candidates face sustain profitability. competition from companies outside the gene therapy approach who are using other treatment methods to address the same If we fail to develop and commercialize other product candidates, target indications, such as companies developing retinal implants we may be unable to grow our business. or other stem cell approaches. See Section 6.11, “Competition” of Although the development and commercialization of GS010 this Registration Document. for the treatment of LHON and GS030 for the treatment of Many of our potential competitors have substantial ly greater Retinitis Pigmentosa, or RP, is our primary focus, as part of our financial, technical and other resources, such as larger research growth strategy, we believe that GS030, if successful in the and development, clinical, manufacturing and marketing treatment of RP, may be transferable to the treatment of other departments. Established competitors may invest heavily to diseases of photoreceptor degeneration, including dry AMD quickly discover and develop novel compounds that could make and GA. In addition, we believe our technology platforms have our product candidates obsolete or uneconomical. Any new broad applicability both within and outside ophthalmology as product that competes with an approved product may need to well as in certain CNS disorders. These other indications as well demonstrate compel ling advantages in efficacy, convenience, as additional product candidates wil l require additional, time- tolerability and safety to be commercially successful. Any of our consuming and costly development efforts prior to commercial product candidates that are approved in the future will also face sale, including preclinical studies, clinical trials and approval other competitive factors, including generic competition, which by the FDA, the EMA and/or applicable foreign regulatory could force us to lower prices or could result in reduced sales. authorities, which may also differ appreciably from those of our In addition, new products developed by others could emerge as initial product candidates focused on retinal neurodegenerative competitors to our product candidates. diseases. All product candidates are prone to the risks of failure that are inherent in pharmaceutical product development, In addition, mergers and acquisitions in the biotechnology and including the possibility that the product candidate wil l not pharmaceutical industries may result in even more resources be shown to be sufficiently safe and effective for approval by being concentrated among a smal ler number of competitors. regulatory authorities. In addition, we cannot assure you that Our commercial opportunities could be reduced or eliminated if any such products that are approved wil l be manufactured or competitors develop and commercialize products that are safer, produced economically, successfully commercialized or widely more effective, have fewer or less severe side effects, are more prescribed. convenient or are less expensive than any products that we may 28 – GENSIGHT BIOLOGICS – 2017 Registration Document