Scalable production of RPE cells from induced pluripotent stem cell under GMP conditions for cellular replacement therapy of the dry form of AMD

年龄相关性黄斑变性（AMD）是西方世界60岁以上人群失明的主要原因，并且每年该问题都在增加。AMD导致视力的中心部分丢失，使其无法欣赏细节。AMD与视网膜支持细胞-视网膜色素上皮细胞（RPE）的缺陷有关。视网膜中的视杆细胞和视锥细胞（光感受器）是感光细胞，它们的生存依赖于这些细胞的正常功能，因此这些细胞的功能衰竭会导致视力逐渐丧失并最终失明。目前还没有预防AMD发展的治疗方法。然而，对于某些形式的AMD，采用一系列干预措施取得了一些成功，但这些方法仅适用于某些患者，并且通常只是暂时的。我们的目标是从来自患者自身的干细胞中产生细胞替代疗法，其可有效替代AMD中发现的功能失调的RPE，这将导致能够稳定和恢复绝大多数患者视力的手术疗法。我们计划在三年内开发从患者皮肤样本中产生干细胞的必要过程，并将这些干细胞转化为RPE。我们将测试这些细胞的安全性和有效性，准备用于患者。我们的目标是在该项目结束时提交临床试验申请，以启动这些患者来源的眼细胞的I/II期临床试验。

Age related macular degeneration (AMD) is the leading cause of blindness in people over the age of 60 in the western world and each year the problem increases. AMD results in the central portion of vision being lost making it impossible to appreciate fine detail. AMD is associated with defects of the retinal support cells - the retinal pigment epithelial cells (RPE). The rods and cones (the photoreceptors) in the retina, which are the light sensitive cells, depend for their survival on the normal functioning of these cells, and so failure of these cells leads to progressive loss of vision and eventual blindness. There is currently no treatment which prevents the development of AMD. However there has been some success for certain forms of AMD with a range of interventions, but these approaches are only suitable for certain patients and are often only temporary. Our aim is to produce a cell replacement therapy from stem cells derived from patients themselves which are effective in replacing dysfunctional RPE found in AMD which will lead to a surgical therapy capable of stabalising and restoring vision in the vast majority of patients. We plan to develop, over three years, the necessary process of producing stem cells derived from patient skin samples and to turn these stem cells into RPE. We will test the safety and efficacy of these cells ready for use in patients. The goal is to submit a clinical trial application at the end of this project for these patient derived eye cells to initiate a Phase I/II clinical trial.