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

Investigators

Abstract

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).