Safety and Tolerability of MA09-hRPE Cells in Patients With Stargardt's Macular Dystrophy(SMD)

A Phase I, Open-Label, Prospective Study to Determine the Safety and Tolerability of Sub-retinal Transplantation of Human Embryonic Stem Cell Derived Retinal Pigmented Epithelial(MA09-hRPE) Cells in Patients With Stargardt's Macular Dystrophy(SMD)

The purpose of this study is to evaluate the safety and tolerability of RPE cellular therapy in patients with SMD

• to evaluate the safety and tolerability of RPE cellular therapy in patients with SMD
• to evaluate the preliminary efficacy of MA09-hPRE in patients with SMD

Inclusion Criteria:

• Adult male or female over 20 years of age.
• Clinical diagnosis of advanced SMD.
• The visual acuity of the eye to receive the transplant will be no better than hand movement.
• The visual acuity of the eye that is not to receive the transplant will be no better than 24 (20/320) Early Treatment of Diabetic Retinopathy Study (ETDRS) letters.

Exclusion Criteria:

• History of malignancy.
• History of myocardial infarction in previous 12 months.
• History of diabetes mellitus.
• Any immunodeficiency.
• Any current immunosuppressive therapy other than intermittent or low dose cortico steroids.
• Serologic evidence of infection with Hepatitis B, Hepatitis C, or HIV.
• Current participation in any other clinical trial.
• Participation within previous 6 months in any clinical trial of a drug by ocular or systemic administration.
• Any other sight-threatening ocular disease.
• Any chronic ocular medications. Any history of retinal vascular disease (compromised blood-retinal barrier). Glaucoma. Uveitis or other intraocular inflammatory disease. Significant lens opacities or other media opacity. Ocular lens removal within previous 3 months