A Clinical Trial Evaluating the Safety and Efficacy of a Single Subretinal Injection of AGTC-501 in Participants With XLRP

Eligibility Criteria

• 1. Provide written informed consent or assent (per local regulation), prior to the conduct of any study-related procedure. Participants who provide assent must have a parent, guardian, or legal representative provide written informed consent.
• 2. Be between 12 and 50 years of age (inclusive) at the time of informed consent and assent (as applicable).
• 3. Be male (XY chromosome) and have at least one documented pathogenic or likely pathogenic variant in the RPGR gene.
• 4. Have a clinical diagnosis of XLRP.
• 5. Be able and willing, as assessed by the Investigator, to follow study instructions, complete study assessments, comply with the protocol, and attend study visits for the duration of the study.
• 6. Have a BCVA ≤ 78 letters (approximately Snellen, 20/32) and ≥ 34 letters (approximately Snellen, 20/200)
• 7. Have a LLVA ≤64 letters (approximately Snellen 20/50) in the study eye
• 8. Be able to perform all tests of visual and retinal function and structure in both eyes based on the participant's reliability, and fixation, in the study eye per the Investigator's discretion.
• 9. Have detectable baseline mean macular sensitivity .
• 10. Have a detectable sub-foveal ellipsoid zone (EZ) line as assessed by SD-OCT in the study eye and confirmed by the CRC.
• 11. If study eye will be at the discretion of the Investigator and/or Surgeon.
• 1. Have other known disease-causing mutations documented in the participant's medical history or identified through a retinal dystrophy gene panel, that in the opinion of the Investigator would interfere with the potential therapeutic effect of the study agent or the quality of the assessments.
• 2. For participants with herpes simplex virus (HSV):
• 1. Have history of oral or genital herpes and unable and/or unwilling to utilize prophylactic antiviral medication.
• 2. Have a history of ocular herpes.
• 3. Have active oral or genital herpes or are currently receiving treatment for HSV infection.
• 3. Have known sensitivity or allergy to systemic corticosteroids or other immunosuppressive medications.
• 4. Have used anti-coagulant agents that may alter coagulation (e.g., warfarin, heparin, apixaban, or high dose docosahexaenoic acid \[DHA; fish oil\]) within 7 days prior to study treatment administration (ibuprofen, aspirin, or similar are acceptable).
• 5. Have used systemic corticosteroids or other immunosuppressive medications within 3 months prior to screening and/or intend to use during screening. Corticosteroids used on an as-needed basis administered by insufflation, inhalation or local administration to the skin and mucosa such as Symbicort (budesonide/formoterol), Flonase (fluticasone propionate), and skin creams and ointments containing corticosteroids shall not be exclusionary.
• 6. If sexually active or planning to become sexually active, are unwilling to use barrier contraception for 3 months following treatment administration.
• 7. Are currently participating or recently participated in any other research
• 8. Have previously received any AAV gene therapy product, stem cell therapy, cell-based therapy, or similar biologics.
• 9. Have significant media opacity impacting evaluation of the retina or vitreous. administration.
• 10. Had intraocular surgery within 90 days of study treatment administration.
• 11. Have any active ocular/intraocular infection or inflammation (e.g., severe blepharitis, infectious conjunctivitis, keratitis, scleritis, endophthalmitis, idiopathic or autoimmune associated uveitis, or herpetic lesions).
• 12. Have a history of corticosteroid-induced raised IOP of \>25 mmHg following corticosteroid exposure, despite topical IOP-lowering pharmacologic therapy.
• 13. Have any artificial retinal implant or prosthesis.
• 14. Have absence of clear ocular media and/or inadequate pupil dilation to facilitate good quality SD-OCT images.
• 15. Have any history of rhegmatogenous retinal detachment.
• 16. Have myopia (spherical equivalent) exceeding -10 diopters (or axial length of \>30 mm if the Principal Investigator \[PI\] deems it appropriate to measure) or presence of pathologic myopia in the study eye.
• 17. Have passed the Low Contrast Ora-VNC mobility course at ≤0.35 lux light level in either eye or binocularly at any screening visit.