A Phase 1/2a, Open-Label, Non-Randomized, Dose-Escalation Study to Evaluate the Safety and Tolerability of GS030 in Subjects With Retinitis Pigmentosa
The objective of this study is to evaluate the safety and tolerability of escalating doses of a gene therapy called GS030-DP (injected study treatment) administered via a single intravitreal injection and repeated light stimulation using a medical device called GS030-MD (stimulating glasses) in subjects with documented diagnosis of non-syndromic Retinitis Pigmentosa
• Age ≥18 years to ≤75 years at the time of ICF signature.
• Diagnosis of non-syndromic RP defined as:
‣ Clinical diagnosis of non-syndromic RP based on history, mid-peripheral visual dysfunction, and fundoscopic appearance.
⁃ Diagnosis of non-syndromic RP is confirmed on full-field ERG
• Visual acuity:
‣ Visual acuity in the dose-escalation cohorts of no better LP.
⁃ Visual acuity in the extension cohort of no better than CF pending review of dose-escalation cohort data by the DSMB.
• Relatively preserved ganglion cell layer volume and retinal nerve fiber layer thickness, as measured with spectral domain optical coherence tomography (SD-OCT).
• Interpupillary distance of ≥51 mm and ≤72 mm.
• Refractive error of the study eye between -6 diopters and +6 diopters.