GLOSSARY 26 AAA Adeno Associated Virus IBC Institutional Biosafety Committee AMD Age-Related Macular Degeneration ICH International Conference on Harmonization ANSM Agence nationale de securité du médicament et des produits IDE Investigational Device Exemption de santé IND Investigational New Drug ATIS Asynchronous Time-Based Image Sensor IOP Intraocular pressure ATMP Advanced Therapeutic Medicinal Product IRB Institutional Review Board ATP Adenosine TriPhosphate IVT Intravitreal BCVA Best corrected visual acuity LCA Leber congenital amaurosis BLA Biological License Application LHON Leber hereditary optic neuropathy BPCIA Biologics Price Competition and Innovation Act MAA Marketing Access Authorization CBER Center for Biological Evaluation & Research MIT Massachusetts Institute of Technology CDER Center for Drug Evaluation & Research MTS Mitochondrial Targeting Sequence CAT Committee for Advanced Therapies mtDNA Mitochondrial ribonucleic acid CE European Conformity mRNA Messenger RNA cGMP Certified Good Manufacturing Practices MTS Mitochondrial Targeting Sequence CHMP Committee on Human Medicinal Products NDA New Drug Application CMC Chemistry, Manufacturing and Controls ND4 NADH dehydrogenase 4 CMO Contract Manufacturing Organization NIH National Institutes of Health CMS Center for Medicare & Medicated Services NHP Non-human primate Cox10 Cytochrome c oxidase assembly homolog 10 OCT Optical coherence tomography CRO Contract Research Organization PDCO Paediatric Committee CTA Clinical Trial Application PDUFA Prescription Drug User Fee Act DNA Deoxyribonucleic acid PHS Public Health Service DSMB Data Safety Monitoring Board PMA PreMarket Approval EEA European Economic Area PPACA Patient Protection and Affordable Care Act EMA European Medicines Agency RAC Recombinant DNA Advisory Committee ETDRS Early Treatment Diabetic Retinopathy Study rAAV Recombinant adeno-associated Virus FDA Food and Drugs Administration REMS Risk Evaluation and Mitigation Strategy FD&C Federal Food, Drug, and Cosmetic Act RGC Retinal Ganglion Cells GA Geographic Atrophy RNA Ribo Nucleic Acid GCP Good Clinical Practices RP Retinitis Pigmentosa GLP Good Laboratory Practices SOP Standard operating procedure GMP Good Manufacturing Practices SPC Supplementary Protection Certificate GTP Good Tissue Practices USPTO United States Patent & Trademark Office HCT/Ps Human Cells, Tissues, and Cellular and Tissue-Based UTR UnTranslated Region Products VEP Visual evoked potential HITECH Health Information Technology for Economic and Clinical Health Act Wt Wild type HIPAA Health Insurance Portability and Accountability Act 242– GENSIGHT BIOLOGICS – 2017 Registration Document