Casey Eye Institute

X-Linked Retinitis Pigmentosa (RPGR) Gene Therapy Trial

An Open-Label Dose Escalation Study to Evaluate the Safety and Efficacy of AGTC-501 (rAAV2tYF-GRK1- RPGR) in Subjects with X-linked Retinitis Pigmentosa caused by RPGR-ORF15 mutations 

The purpose of this study is to learn about a new investigational gene therapy that may help patients with XLRP. The study investigators want to find out whether it is safe and to see if it can improve your vision and other symptoms of XLRP. The gene therapy is given by a surgical injection into the retina (the lining of the back of the eye that detects light) of one eye. The eye with worse vision will receive the gene therapy. 

Eligibility Criteria: 

  • Age: 
    • 18 years or older for Groups 1-3 
    • 6 years and older for Group 4 
  • Male 
  • Must have a clinical diagnosis of XLRP 
  • Must have a documented mutation within the ORF15 exon of the RPGR gene, which is linked to XLRP 
  • Must have visual acuity no better than: 
    • 20/50 in the eye that will receive gene therapy for Groups 1-3
    • 20/32 in the eye that will receive gene therapy for Group 4 
  • Good general health, including no history of diabetes or other ocular diseases like glaucoma 
  • No prior receipt of any similar gene therapy product 

The study will occur at the Casey Eye Institute in Portland, OR. Participation in this study will last for a total of 5 years. There are 12 required study visits in the first year of the study. After the first year, there will be additional visits at Months 18, 24 and 36, then once a year for the next 2 years. Each study visit may span between 1-2 days and may involve functional vision tests, dilated eye exams, and blood draws. 

All study procedures and reasonable travel costs will be covered by the study sponsor. 

If you are interested in learning more about this study or have a patient that you believe would be a good candidate, please call the study coordinator at 503-494-0020 or email 

Principal Investigator: Paul Yang, M.D., Ph.D.
Casey Eye Institute
3375 SW Terwilliger Blvd.
Portland, OR 97239 

eIRB#: 17801
IRB Approved: 11/30/2017 

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