Directory of Optogenetic Trials

Here we aim to proviede an up-to-date directory of current and past optogenetic trials. Please us if you find a trial or results missing:

IDPhaseActuatorVectorTarget CellsSponsorConditionResults
NCT02556736I/IIaChR2AAV2 2.7m8RGCAbbVie/RetroSenseRPConference report (Bunnell, Yuan, Lopez, 2025) and Trial results data reported on clincialtrials.gov
NCT03326336 (PIONEER)I/IIaChrimsonRAAV2 7m8RGCGenSight BiologicRPCase report: Object localization in near spase, EEG ( Sahel et al., 2021)
ChiCTR2200062174 (UGX-201)ImGluR4 -MW-Opsin chimera under synuclein gamma promoterAAV2 7m8RGCUgenex TherapeuticsRPConference report: VA improvement (Luo et al., 2025);


Phase 1 Results: VA improvement observed only in treated eye and at biologically plausible time scale. FST improvement similar in treated and untreated eye (Han X et al. AJO, 2026)
NCT04278131I/IIChronosFPAAV2RGCBionic SightRPPress release: Increase in light sensitivity and VA ( Bionic Sight, 2025 )
NCT04919473, NCT05921162I/II + Follow-UpMCO-1 / vMCO-010AAV2BCNanoscope TherapeuticsRP, ABCA4-associatedCase Series: Improvement in VA, visual field and Quality of Life, no controls included (Mohanty et al., 2025 )
NCT04945772 (RESTORE), NCT06162585 (REMAIN)I/II +  Follow-UpMCO-010AAV2BCNanoscope TherapeuticsRPPress release: Significant improvement in VA ( FirstWord, 2025; Nanoscope Therapeutics, 2024 )

Conference report: No SAE ( Boyer et al., 2023) and functional gain prevailing after 3 years ( Mahajan V et al., 2026)
NCT05417126 (STARLIGHT), NCT06048185II +  Follow-UpvMCO-010AAV2BCNanoscope TherapeuticsM. StargardtCase Series: Improvement in VA, visual field and Quality of Life, no controls included (Lam et al., 2025)
NCT06292650, NCT07282457IPsCatCh2.0AAV?RGCZhongmou TherapeuticsRPConference report: VA improvement (Zhou W et al., 2025) and clinically meaningful VA improvement in 83.3% (5/6), equalling mean -0.61 logMAR from 2.42logMAR at baseline. Accliamed improvement in hue discrimination. No improvement in two sham injected patients (Zhou W et al., 2026))
NCT06460844 (ENVISION)IChRownARMMsRGCRay TherapeuticsRP, Choroideremia
NCT07311863IUgenex TherapeuticsRP