GENSIGHT DDR 2017 VA - 6.7 LEVERAGING OUR PLATFORMS TO ADDRESS CENTRAL NERVOUS SYSTEM DISORDERS - 6.8 PHARMACO-ECONOMICS OF BLINDNESS AND VISUAL IMPAIRMENT (84/264)

GENSIGHT DDR 2017 VAGENSIGHT DDR 2017 VA - 6.7 LEVERAGING OUR PLATFORMS TO ADDRESS CENTRAL NERVOUS SYSTEM DISORDERS - 6.8 PHARMACO-ECONOMICS OF BLINDNESS AND VISUAL IMPAIRMENT BUSINESS OVERVIEW the jurisdiction in charge of evaluation. The regulatory pathway is based on the following classification: an IND will be necessary for clinical trial initiation, and a BLA will be required marketing authorization. In order to validate the adequacy of our non-clinical toxicology and safety program designed to support the first-in-man study, we submitted a request for non-clinical scientific advice from the EMA in April 2016 and an early Type C meeting with the FDA. Both the EMA and FDA have agreed with our strategy in principle to evaluate toxicity in a rodent disease model and in non-human primates, and we completed these toxicology studies in 2017. In order to obtain agreement on the adequacy of the data presented to support initiation of an IND in the United States, a formal Type B Pre-IND meeting was held with the FDA in December 2017. During this meeting, the FDA agreed in principle with our Phase I/I I clinical trial design, as well as device aspects to support an IND. The following image illustrates the deterioration of GS030: Our Planned Strategy for the Treatment of Dry AMD and GA We believe that our optogenetics technology platform could be used for the treatment of dry AMD or GA. Because GS030 uses optogenetics and can address diseases of photoreceptor degeneration regardless of the type of mutation, we believe GS030, if successful in the treatment of RP, would be entirely transferable to the dry form of AMD and would offer enormous benefit to a common disease currently unamenable to therapy. Although RP and GA have very different origins, both diseases are characterized by the degeneration of the photoreceptor cells in the patient’s retina. Upon evidence of clinical proof-of-concept in RP, we may initiate clinical trials of GS030 for the treatment of dry AMD and GA. 6.7 LEVERAGING OUR PLATFORMS TO ADDRESS CENTRAL NERVOUS SYSTEM DISORDERS We have established an integrated development platform to replace or restore the function of retinal cel ls that have degenerated in order to regain quality of sight for patients, thereby transforming their lives. We intend to pursue the application of 82 – GENSIGHT BIOLOGICS – 2017 Registration Document 6 Market Opportunity for GS030 in Dry AMD and GA AMD is a degenerative disorder characterized by loss of the at the time ofphotoreceptors and preservation of the RGCs, and is driven by genetic and environmental factors. Central vision, which is essential to read, perform precise tasks and recognize faces, is lost. Peripheral visual field is usual ly preserved. Macular degeneration typically occurs in patients over the age of 55. The early form of AMD is called dry AMD and can evolve over time to late AMD. Late AMD can take two forms, either wet AMD or GA. Dry AMD is six times more prevalent than wet AMD. Approximately 15 million people are affected with AMD in the United States, with a global prevalence of 170 million, and dry AMD accounts for approximately 80% of all cases of late-stage AMD. The prevalence of AMD increases significantly in those older than 75, reaching 22% in the population over 90. CMC and medical normal vision to blindness in AMD: our integrated development platform to other indications beyond ophthalmology, in particular, for degenerative diseases of the central nervous system. that 6.8 PHARMACO-ECONOMICS OF BLINDNESS AND VISUAL IMPAIRMENT Blindness and visual impairment impact not only the individual but also the family, caregivers, and the community, leading to significant societal costs. The total annual cost of vision disorders and blindness in the United States was estimated to be $139 billion in 2013, and the total cost of blindness alone in the European Union was estimated at €32 billion, or up to €60,000 per patient per year. These figures include direct costs such as medical treatment, medical visual prostheses, adaptations and assistance devices, and special training and assistance programs, as well as indirect costs resulting from impaired vision, the loss of productivity and the need for supportive care, long-term care and the costs of social programs. These conditions also can have significant, multidimensional effects on patients’ quality of life, including their physical and emotional well-being.