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期研究新药(IND)，使用自体诱导多能干细胞(IPSC)来源的视网膜色素上皮(RPE)片作为老年黄斑变性(AMD)患者的细胞治疗。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IND提交人类第一阶段临床研究。研究产品由IPSC分化的RPE单层组成，生长在可生物降解的支架上，需要在RNU大鼠的视网膜下空间给药。 此次收购的目的是在RNU大鼠身上开发和进行一项IND启用的临床前研究，以评估研究用组合产品(可降解支架上的RPE Sheet)的系统毒性和致瘤性。

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).