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Sci Transl Med. 2018 Apr 4;10(435). pii: eaao4097. doi: 10.1126/scitranslmed.aao4097.

A bioengineered retinal pigment epithelial monolayer for advanced, dry age-related macular degeneration.

Author information

1
University of Southern California (USC) Roski Eye Institute, USC Institute for Biomedical Therapeutics, and Department of Ophthalmology, Keck School of Medicine, USC, 1450 San Pablo, Los Angeles, CA 90033, USA. humayun@usc.edu ahkashan@usc.edu.
2
Regenerative Patch Technologies, 150 Gabarda Way, Portola Valley, CA 94028-7445, USA.
3
Retina Vitreous Associates Medical Group, 9001 Wilshire Boulevard, Suite 301, Beverly Hills, CA 90211, USA.
4
California Retina Consultants, 525 East Micheltorena Street, Santa Barbara, CA 93103, USA.
5
Atlantis Eyecare, 7777 Edinger Avenue, Suite 234, Huntington Beach, CA 92647, USA.
6
Center for Biomedicine and Genetics, Beckman Research Institute of City of Hope, 1500 East Duarte Road, Duarte, CA 91010, USA.
7
University of Southern California (USC) Roski Eye Institute, USC Institute for Biomedical Therapeutics, and Department of Ophthalmology, Keck School of Medicine, USC, 1450 San Pablo, Los Angeles, CA 90033, USA.
8
Department of Pathology, Keck School of Medicine, USC, 1441 Eastlake Avenue, NRT 7503, Los Angeles, CA 90033, USA.
9
Center for Stem Cell Biology and Engineering, Neuroscience Research Institute, Mail Code 5060, University of California, Santa Barbara, CA 93016, USA.
10
Department of Biomedical Engineering, 1042 Downey Way, Denney Research Center (DRB) 140, USC, Los Angeles, CA 90089-1111, USA.

Abstract

Retinal pigment epithelium (RPE) dysfunction and loss are a hallmark of non-neovascular age-related macular degeneration (NNAMD). Without the RPE, a majority of overlying photoreceptors ultimately degenerate, leading to severe, progressive vision loss. Clinical and histological studies suggest that RPE replacement strategies may delay disease progression or restore vision. A prospective, interventional, U.S. Food and Drug Administration-cleared, phase 1/2a study is being conducted to assess the safety and efficacy of a composite subretinal implant in subjects with advanced NNAMD. The composite implant, termed the California Project to Cure Blindness-Retinal Pigment Epithelium 1 (CPCB-RPE1), consists of a polarized monolayer of human embryonic stem cell-derived RPE (hESC-RPE) on an ultrathin, synthetic parylene substrate designed to mimic Bruch's membrane. We report an interim analysis of the phase 1 cohort consisting of five subjects. Four of five subjects enrolled in the study successfully received the composite implant. In all implanted subjects, optical coherence tomography imaging showed changes consistent with hESC-RPE and host photoreceptor integration. None of the implanted eyes showed progression of vision loss, one eye improved by 17 letters and two eyes demonstrated improved fixation. The concurrent structural and functional findings suggest that CPCB-RPE1 may improve visual function, at least in the short term, in some patients with severe vision loss from advanced NNAMD.

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