UGX202 Injection in Patients With Advanced Retinitis Pigmentosa

Study to Evaluate the Safety and Preliminary Efficacy of UGX202 Injection in Patients With Advanced Retinitis Pigmentosa

The primary objective of this clinical trial is to evaluate the safety and tolerability of a single intravitreal injection of the gene therapy drug UGX202 in patients with advanced RP. The secondary objective is, to assess the preliminary efficacy of a single intravitreal injection of the gene therapy drug UGX202 in treating patients with advanced RP.

This study is a non-randomized, open-label investigator-initiated trial (IIT). It plans to enroll approximately 6 subjects with non-syndromic retinitis pigmentosa (RP) who have extremely low vision (the study eye is the eye with lower vision, and the best corrected visual acuity [BCVA] > logMAR 1.9).

The study drug is divided into two dose groups: low dose and high dose. A modified "3+3" dose escalation approach is adopted. The low-dose group (4.2E+10 vg/eye) is planned to include 3 subjects. First, 1 subject (sentinel) will be enrolled and observed for 28 days. If no dose-limiting toxicity (DLT) occurs, 2 more subjects (non-sentinel) will be enrolled and observed for 28 days. The second and third subjects will be enrolled with a 7-day interval.

The high-dose group (1.2E+11 vg/eye) is planned to include 3 subjects. Subjects in the high-dose group will be enrolled and administered the drug in sequence after passing the screening. There will be at least a 1-week interval between each subject. The timing of enrolling the full 3 subjects or stopping enrollment will be determined by the investigator's assessment of safety.All subjects will receive intravitreal injection of the study drug UGX202 after enrollment and will be followed up for 52 weeks to evaluate the safety, tolerability, and preliminary efficacy of UGX202.

Inclusion Criteria:

• Provide written informed consent form (ICF).
• Age ≥18 years at ICF signing.
• Diagnosed as non-syndromic RP；
• BCVA > logMAR 1.9 (assessed by FrACT) in the study eye.
• Confirmation of preserved memory of visual experience
• Spherical equivalent between -9D and +6D.

Exclusion Criteria:

• Prior gene therapy in either eye.
• Received any interventional investigational drug within 90 days prior to screening.
• Any Study eye disease or systemic disease judged by the investigator to affect visual function assessment.
• Hypersensitivity to corticosteroids, intolerance to corticosteroid regimen, active concurrent infection contraindicating treatment.
• History or tendency of psychiatric disorders impacting safety and/or efficacy assessment.
• Any other factor deemed unsuitable by the investigator.