Treatment of Advanced Dry Age Related Macular Degeneration With AAVCAGsCD59

Inclusion Criteria:

• Men or women 50 years of age or older
• Advanced dry AMD with GA in the study eye
• BCVA Snellen equivalent of 20/80 or worse in the study eye using ETDRS charts at a starting distance of 4m after the first 3 patients are enrolled and demonstrate favorable safety data
• Total GA lesion size 5mm2 (2 DA) to 20mm2 (8 DA) in the study eye; if multifocal, then at least one focus of GA needs to measure 1.27 mm2 (0.5 DA)
• Fellow eye BCVA of 20/800 or better and must be the eye with the better visual acuity
• Must be willing to undergo paracentesis of the anterior chamber

Exclusion Criteria:

• GA secondary to non AMD etiologies
• Prior or active choroidal neovascularization (CNV) in the study eye
• History of conditions in the study eye during screening which might alter visual acuity or interfere with study testing
• Active uncontrolled glaucoma
• Intraocular surgery in the study eye within 3 months of enrollment or are known or likely candidate for intraocular surgery (including cataract surgery) in the study eye within 1 year of treatment
• Acute or chronic infection in the study eye
• History of inflammation in the study eye or ongoing inflammation in either eye
• History of uveitis in the study eye
• Ongoing ocular inflammation in either eye
• Any contraindication to intravitreal injection
• Currently using or have used treatment for exudative AMD in the study eye only: laser photocoagulation, photodynamic therapy (PDT), ranibizumab (Lucentis®), pegaptanib sodium (Macugen®), bevacizumab (Avastin®) or aflibercept (Eylea®)
• Currently using any periocular (study eye), intravitreal (study eye) or systemic (oral or intravenous) corticosteroids within 3 months prior to screening.

• Any of the following underlying systemic diseases:

  • Unstable or severe cardiovascular disease, e.g., congestive heart failure (New York Heart Association Functional class III or IV), myocardial infarction within 6 months, ventricular tachyarrhythmias requiring ongoing treatment, unstable angina, or critical limb ischemia;
  • Poorly controlled diabetes;
  • Clinically significant impaired renal or hepatic function, for example:
  • Cerebrovascular disease within 12 months prior to Screening;
  • Dementia or neurodegenerative disease (e.g., Alzheimer's disease, Parkinson's disease);
  • Have a malignancy at Screening or a history of malignancy that precludes completion of this 2-year study, including presence of tumor or disease whose treatment may directly affect the ability to evaluate the safety and efficacy of AAVCAGsCD59, or currently undergoing treatment for a specific cancer;
  • Immunocompromised conditions and/or need for immunosuppressive therapy;
• Any significant poorly controlled illness that would preclude study compliance and follow-up
• Current or prior use of any medication known to be toxic to the retina or optic nerve including, but not limited, to chloroquine/hydrochloroquine, deferoxamine, phenothiazines and ethambutol
• Previous treatment with any ocular or systemic gene transfer product
• Received any investigational product within 120 days prior to screening