NEI Toxicity Studies to Support an IND Filing for Conducting Phase I Study of induced Pluripotent Stem Cell-Derived Retinal Pigment Epithelium on Scaffold

NEI正在开发一种I期研究性新药（IND），使用自体诱导多能干细胞（iPSC）衍生的视网膜色素上皮（RPE）片作为晚期年龄相关性黄斑变性（AMD）患者的细胞疗法。该项目的这一部分需要设计和进行临床前毒性研究，以支持人类1期临床研究的IND申请。研究产品由生长在可生物降解支架上的iPSC分化的RPE单层组成，需要在RNU大鼠眼睛的视网膜下腔中施用。 此次收购的目的是开发和实施IND，以在RNU大鼠中进行临床前研究，评估试验用组合产品（可降解支架上的RPE片）的全身毒性和致瘤性。

The NEI is developing a phase I Investigative New Drug (IND) using autologous induced pluripotent stem cells (iPSC) derived retinal pigment epithelium (RPE) sheets as cell therapy for advanced age related macular degeneration (AMD) patients. This part of the project requires design and conduct preclinical toxicity studies to support an IND filing for a phase 1 clinical study in humans. The investigational product consists of an iPSC differentiated RPE monolayer grown on a biodegradable scaffold and needs to be administered in the subretinal space of the eye of RNU rats. The objective of this acquisition is to develop and conduct an IND enabling preclinical study in RNU rats to assess the systemic toxicity and tumorigenicity of the investigational combination product in (RPE sheet on degradable scaffold).