BUSINESS OVERVIEW 6 REFLECT is enrolling patients with an onset of vision loss of less Our Technology Platforms and Other Applications than 12 months. For more information on clinical trials protocols, We believe our integrated technology, which combines gene see Section 6.5 “Our Lead Product Candidate: GS010 for the therapy with our core MTS and optogenetics technology Treatment of LHON”, of this Registration Document. platforms, has the potential to replace or restore the function of GS030 for the Treatment of RP retinal cells, either RGCs or photoreceptors that have degenerated We are developing GS030 for the treatment of diseases of in order to regain vision for patients, thereby improving the photoreceptor degeneration that include RP and dry age-related quality of their lives. Beyond our initial product candidate, GS010 macular degeneration, or AMD, with and without geographic for the treatment of LHON, we believe that our MTS technology atrophy, or GA. We initially focused our studies on the treatment platform can be applied to treatments for LHON caused by other of RP, which is an orphan family of diseases caused by multiple single mutations, including our second LHON product candidate, mutations in over 100 genes involved in the visual cycle. On GS011, to treat LHON due to mutation in the ND1 gene. Similarly, average, RP patients begin experiencing vision loss as young we believe that GS030 using our optogenetics platform can adults, eventually becoming blind around the age of 40 to 45. RP is address any disease of photoreceptor degeneration regardless the most widespread hereditary cause of blindness in developed of the etiology and be entirely transferable to dry AMD or nations, with a prevalence of about 1.5 million people throughout GA, offering a meaningful benefit to these diseases that have the world. In Europe and the United States, the prevalence of RP is significant unmet medical needs. approximately one in 3,500 and one in 4,000 and the incidence of In addition, our MTS and optogenetics technologies have potential new patients each year is 15,000 and 20,000. There is currently applicability outside of our initial focus on severe retinal diseases. no existing treatment for RP. We believe our MTS technology platform, given its unique ability GS030 utilizes our novel optogenetics technology platform. to actively shuttle mitochondrial proteins into the mitochondrion, Optogenetics is a biologic technique that involves the transfer of a enables the development of treatments for the many indications gene that is encoding for a light-sensitive protein to cause neuronal involving defects of the mitochondrion, including such rare cells to respond to light stimulation. Our platform of optogenetics diseases as Kearns-Sayre syndrome and Alpers disease, and targets retinal ganglion cells, or RGCs, and modifies them into possibly more common disorders such as Parkinson’s disease and true photoreceptors. This allows us to confer a photoreceptive amyotrophic lateral sclerosis, or ALS. Similarly, we believe this function to the healthy and preserved RGCs independent of any gene therapy approach of our optogenetics platform that permits specific underlying genetic mutation. Light stimulation, which the introduction of proteins sensitive to light stimulation has activates the protein, is amplified and enhanced by an external broad applicability to indications outside ophthalmology that are wearable device designed as goggles. We developed these receptive to light stimulation, such as congenital deafness, pain goggles to amplify the light stimulation upon the transduced treatment and vagus nerve stimulation. neuronal cel ls and expand vision restoration. We believe our We own or have exclusively in-licensed all intellectual property technology would be immediately transferable to any disease in rights covering our MTS and optogenetics platform technologies which photoreceptors are lost while RGCs remain, such as dry and our current product candidates. In addition, we hold AMD and GA. Approximately 15 million people are affected with worldwide commercialization rights to our technology platforms, AMD in the United States, with a global prevalence of 170 million, product candidates and development programs. Because of and dry AMD accounts for approximately 80% of al l cases of the orphan nature of LHON and RP, we believe a limited and late-stage AMD. Given this, we expect to initiate clinical trials of targeted sales and marketing organization would be able to reach GS030 for the treatment of dry AMD and GA. specialized ophthalmology centers and their patients. GS030 has received orphan drug designation for the treatment of RP in the United States and the European Union and advanced Our Management and Scientific Team therapy medicinal product, or ATMP, classification for the treatment We believe that we have a significant competitive advantage of RP in the European Union. Our preclinical proof-of-concept as a result of the collective experience of our management and studies have demonstrated that GS030 can restore light sensitivity scientific team in the biotechnology industry, specifically in the in the retina of blind mice and non-human primates. In other areas of ophthalmology and gene therapy. Our Chief Executive preclinical studies, we have also restored visual behaviors in vivo in Officer and co-founder, Bernard Gilly Ph.D., has over 20 years of blind rats using GS030 with demonstrable effects upon their visual experience in the pharmaceutical sector and as an entrepreneur. cortex. We received approval in December 2017from the UK’s Other members of our executive management team have Medicines and Healthcare Products Regulatory Agency, or MHRA, significant experience in the discovery and development of gene to conduct a Phase I/II clinical trial in severely affected RP subjects therapy and ophthalmology drug products. Our co-founder, and expect to treat the first subject in the second quarter of 2018. José-Alain Sahel M.D. Ph.D., is the Director of Institut de la Vision GENSIGHT BIOLOGICS – 2017 Registration Document– 67