Study of RS1 Ocular Gene Transfer for X-linked Retinoschisis

Exclusion Criteria

• •Participant is actively receiving another study medication/investigational product (IP).
• •Participant has previously enrolled in another gene therapy trial.
• •Participant is currently taking, or has taken in the last three months, a systemic carbonic anhydrase inhibitor prior to enrollment/baseline 1 testing.
• •Participant has any condition that significantly increases risk of systemic corticosteroids or systemic steroid-sparing immuno-modulatory agents, such as HIV, syphilis, tuberculosis, hepatitis B, hepatitis C, or diabetes mellitus (DM).
• •Participant has an underlying serious illness that impairs regular follow-up during the study.
• •Participant has had diagnosis or treatment of a malignancy (excluding non-melanoma skin cancer) within the previous five years.
• •Participant has pre-existing ocular tumors (excluding non-suspicious nevi).
• •Participant has a known allergy to fluorescein dye or other contraindications to obtaining a fluorescein angiogram.
• •Participant is on a medication that prevents safe administration of study related drugs.
• •Participant has uncontrolled hypertension. (Hypertension judged to be adequately controlled at baseline medical evaluation is not exclusionary.)
• •Participant has compromised renal function such that cyclosporine or Cellcept, which could be used to treat any manifest ocular inflammation, would be contraindicated.
• •Participant has significant liver disease with elevated liver enzymes (greater than or equal to 2.5 times upper limit of normal \[ULN\]).
• •Participant has low absolute neutrophil count (ANC\<1.3 x 10(3)/micro liters).
• •Participant has used any biologic immunosuppressive agents within the last three months (within the last six months for rituximab or cyclophosphamide).
• •STUDY EYE ELIGIBILITY CRITERIA:
• •The study eye has a history of other ocular disease likely to contribute significantly to visual loss or likely to present special risks (e.g., optic neuropathy, advanced glaucoma, uveitis, large bullous schisis cavities or bullous retinal detachment precluding safe intravitreal injection).
• •The study eye has lens, cornea, or other media opacities precluding adequate visualization and testing of the retina.
• •The study eye has undergone intraocular surgery within six months prior to enrollment.
• •The study eye is receiving topical carbonic anhydrase inhibitor, or has received topical carbonic anhydrase inhibitors in the past three months.
• •STUDY EYE SELECTION CRITERIA:
• •If both eyes of a participant meet the study eye eligibility criteria, the choice of study eye will be determined as follows:
• •The eye with the worse visual acuity will be selected as the study eye.
• •If both eyes have visual acuities within five letter differences of one another, the choice of study eye will be determined at the discretion of the Investigator in consultation with the participant.