A Prospective, Open-label, Phase 1b, Single-arm, Safety Study of an Intravitreal Application of a Recombinant Adeno-associated Virus Vector Expressing CNGA1 (AAV2.NN-CNGA1) in Patients With Retinitis Pigmentosa Due to CNGA1 Mutations
The goal of this phase 1 clinical trial is to learn about the safety and efficacy of a gene therapy, VG901, in patients with a rare disorder of the eye called Retinitis Pigmentosa. The main questions the study aims to answer are: * What is the best tolerated dose and are there any side effects, in particular any inflammatory reactions post drug administration? * Are there any early signs of efficacy on visual function? Participants will be administered a single intravitreal dose of VG901 into the most affected eye through a syringe and followed up for a year to monitor safety and efficacy. There will be two cohorts of participants in this study. Study Cohort 1 will receive the low dose and Study Cohort 2 will receive the high dose as specified in the Protocol.
⁃ To be eligible for study entry, subjects must satisfy all the following criteria:
• Able to understand and willing to consent to study participation by a written informed consent
• Male or female ≥ 18 years of age
• Clinical diagnosis of RP
• Confirmed pathogenic, biallelic variants in the CNGA1 gene
• Ellipsoid zone (EZ) length of the fovea of ≥ 3000 μm in the study eye