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See 1 citation in 2018 by Coffey Pj and Da Cruz L:

Nat Biotechnol. 2018 Apr;36(4):328-337. doi: 10.1038/nbt.4114. Epub 2018 Mar 19.

Phase 1 clinical study of an embryonic stem cell-derived retinal pigment epithelium patch in age-related macular degeneration.

Author information

1
The London Project to Cure Blindness, ORBIT, Institute of Ophthalmology, University College London (UCL), London, UK.
2
NIHR Biomedical Research Centre at Moorfields Eye Hospital NHS Foundation Trust, UCL Institute of Ophthalmology, London, UK.
3
Moorfields Eye Hospital NHS Foundation Trust, London, UK.
4
Wellcome/EPSRC Centre for Interventional & Surgical Sciences (WEISS), Charles Bell House, London, UK.
5
Pfizer, Granta Park, Cambridge, UK.
6
Cell and Gene Therapy Catapult, London, UK.
7
Cells for Sight, Transplantation & Research Program, UCL Institute of Ophthalmology, London, UK.
8
UCL Institute of Neurology, Queen Square, London, UK.
9
Center for Stem Cell Biology and Engineering, NRI, UC, Santa Barbara, California, USA.

Abstract

Age-related macular degeneration (AMD) remains a major cause of blindness, with dysfunction and loss of retinal pigment epithelium (RPE) central to disease progression. We engineered an RPE patch comprising a fully differentiated, human embryonic stem cell (hESC)-derived RPE monolayer on a coated, synthetic basement membrane. We delivered the patch, using a purpose-designed microsurgical tool, into the subretinal space of one eye in each of two patients with severe exudative AMD. Primary endpoints were incidence and severity of adverse events and proportion of subjects with improved best-corrected visual acuity of 15 letters or more. We report successful delivery and survival of the RPE patch by biomicroscopy and optical coherence tomography, and a visual acuity gain of 29 and 21 letters in the two patients, respectively, over 12 months. Only local immunosuppression was used long-term. We also present the preclinical surgical, cell safety and tumorigenicity studies leading to trial approval. This work supports the feasibility and safety of hESC-RPE patch transplantation as a regenerative strategy for AMD.

PMID:
29553577
DOI:
10.1038/nbt.4114
[Indexed for MEDLINE]

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