A Clinical Trial of Retinal Gene Therapy for X-linked Retinitis Pigmentosa

This is a Phase 1/2/3, first-in-human, multi-centre, dose-escalation interventional study of
AAV8-RPGR in male subjects with genetically confirmed XLRP. Part I is a dose-selection study;
Part II is a dose-expansion study, comparing 2 doses, and a third untreated group to allow
for a controlled comparison of efficacy and safety.

Key inclusion Criteria:

- Male

- Part I: >= 18 years

- Part II: >= 10 years

- Documentation of a mutation in the RPGR gene

Key exclusion Criteria:

- participated in a gene therapy trial previously or a clinical trial with an
investigational drug in the past 12 weeks