Transcription
Retinal Therapies to Restore Vision
Slide 1
Disease Overview Macula, part of the retinaresponsible for clearvision begins to thin Affects people over theage of 50 11 Million patients in US 196 Million worldwideDrivers of Disease RPE Injury & Dysfunction Mitochondrial Dysfunction Drusen DevelopmentRPE Retinal Pigment EpitheliumSlide 2
Our Ideal Target Product Profile Novel small molecules Formulated as eye drops that willreach the back of the eye Targets mitochondrial dysfunction Protects RPE from oxidativedamage Easier use and higher compliance Composition of matter patent withbroad utility claims filed in 2018Slide 3
In vitro models of AMD Oxidative stress UV-B light damage Aged Bruch’s membraneSlide 4In vivo models of AMD Rotenone-induced retinaldegeneration Superoxide dismutase 1 -/retinal degeneration
RPE Cell SurvivalOxidative Stress Model50HPly48MTBHP4 TBonon448MM lyly484VBVBPERUun 448MUtedTBtreaHPlyPHTBM484ononlylyonPERTBHP tert-butyl hydroperoxideATP adenosine triphosphate5000.010on0.5 PE1.0****100R**OCR (pmol/min)Protective*Slide 5ATP ProductionProtectivePercent survivalRelative Cell ViabilityUV-Light Damage Model15Non-toxic1.5Mitochondrial Function
Mitochondrial FunctionRPE Cell dative Stress Model12001000***50020OCR (pmol/min)4500Protective1510543Mcm pdpd onl T yBHPno TBHtrea Ptmentcmcm pdpd onl T yBHPno TBHtrea Ptment4 TBonPHTBcmlylyon443MlloceHP0M414EC50Slide 6****0nlyLuminescence Signal Level(Viable Cells)Non-toxicM414225 nMM434112.5 nMM434**p 0.001
MoleculeTypeShort PeptideSmall Subcutaneous InjectionTopical Eye DropPharma Partnerships Solving the Dry AMD DiseaseSlide 7
6-8 months2-3 monthsSAR ActivityMed ChemistrySlide 84-6 monthsPharmacokineticsIn Vivo EfficacyStudiesGenerate Usable LeadCompounds andFormulationAssess Topicaland SystemicDelivery to TargetOcular TissueTesting inAnimal Modelsof RetinalDegeneration 40K- 50K 62K 80KYale Center for MolecularDiscoveryPharmOptima,LLCPharmOptima,LLC
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Retinal Therapies to Restore Vision. Slide 1. Disease Overview Macula, part of the retina responsible for clear vision begins to thin Affects people over the age of 50 11 Million patients in US 196 Million worldwide . Yale Center for Molecular Discovery Assess Topical and Systemic Delivery to Target Ocular Tissue 62K
