Basic Information


GTO ID GTC1809
Trial ID NCT03326336
Disease Retinitis Pigmentosa
Altered gene ChrimsonR-tdTomato
Therapeutic/Target gene Therapeutic gene
TherapyGene transfer
Treatment GS030
PhasePhase1|Phase2
Recruitment statusRecruiting
TitleA Phase 1/2a, Open-Label, Non-Randomized, Dose-Escalation Study to Evaluate the Safety and Tolerability of GS030 in Subjects With Retinitis Pigmentosa
Year2017
CountryFrance|United Kingdom|United States
Company sponsorGenSight Biologics
Other ID(s)GS030_CLIN_001|2017-002204-27
Vector information
Vectoradeno-associated virus
ConstructrAAV2.7m8-CAG-ChrimsonR-tdTomato
Vector typerecombinant adeno-associated virus (rAAV) vector
Transgene/Inserted genethe optimized channelrhodopsin ChrimsonR-tdTomato gene
Regulatory elementthe ubiquitous CAG promoter

Clinical Result

Cohort 1
Administration route intravitreal injection
Age Adult, Older_Adult
References : Optogenetic engineering of retinal ganglion cells with AAV2.7m8-ChrimsonR-tdTomato (GS030) in combination with an event-based camera photostimulation device

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