Regulation FD Presentation (Form 8-K)

RDH12KO mice lack retinal phenotype observed in human RDH12 IRD However, mice are susceptible to bright light induced retinal degeneration vs. WT mice1 AAV8 encoding for hRDH12 in RDH12-/- mouse models of light induced retinal degeneration restored structure and function, supporting potential clinical applications1 OPGx-RDH12 Gene Therapy Restored Structure and Function Preventative treatment of RDH12 with AAV8 restores structure and function (water maze test)2 AAV8, adeno-Associated Virus Serotype 8; hRDH12, human retinol dehydrogenase 12; IRD, inherited retinal disease; RDH12, retinol dehydrogenase 12; WT, wild type. 1. Bian J, et al. Drug Des Devel Ther. 2021;15:3531-3591.

46 Signs and Symptoms Directly Inform Selection of Key Endpoints Signs Vessel Narrowing, Pigment Retinopathy, Optic Disc Pallor, RPE Changes ONL Thinning, Ellipsoid Zone Disruption, IRF/SRF Fluid, Macular Atrophy Symptoms Decreased central vision Night blindness Visual field loss / tunnel vision Nystagmus Photophobia Key Structural Endpoints Fundus Photography, Fundus Autofluorescence (FAF) Spectral Domain Optical Coherence Tomography (SD-OCT) Key Functional Endpoints BCVA LLVA FST Microperimetry Contrast sensitivity MLoMT Static perimetry Kinetic visual field testing Pupillometry QoL parameters BCVA, best corrected visual acuity; FST, full-field stimulus test, MLoMT, multi-luminance orientation and mobility test; QoL, quality of life.