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 毒性研究支持 IND 申请，以进行支架上诱导多能干细胞衍生的视网膜色素上皮的 I 期研究

• 批准号: 9175829
• 负责人: NANCY BORDELON
• 金额: $ 93.7万
• 依托单位国家: 美国
• 财政年份: 2015
• 资助国家: 美国
• 起止时间: 2015-09-30 至 2016-06-30
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/9175829
• 关键词: Age related macular degeneration; Animals; Area; Autologous; Biological Assay; Cell Line; Cell Therapy; Cells; Clinical Research; Contractor; Contracts; Control Animal; Control Groups; Controlled Study; Data; Data Analyses; Data Collection; Detection; Documentation; Drug usage; Elderly; Equipment; Evaluation; Eye; Feedback; Hematoxylin and Eosin Staining Method; Histopathology; Human; Human Resources; Immunohistochemistry; Injection of therapeutic agent; Life; Longitudinal Studies; Maintenance; Measures; Methods; Operative Surgical Procedures; Outcome; Patients; Performance; Pharmaceutical Preparations; Phase; Phase I Clinical Trials; Pilot Projects; Preparation; Procedures; Protocols documentation; Published Comment; Rattus; Records; Regulation; Reporting; Research Design; Rodent; Sampling; Site; Specimen; Staining method; Stains; Sterility; Structure of retinal pigment epithelium; Surgeon; System; Teratoma; Testing; Tissue Fixation; Tissue Transplantation; Toxic effect; Training; Transplantation; Tumorigenicity; Validation; Work; base; design; experience; follow-up; good laboratory practice; induced pluripotent stem cell; meetings; monolayer; phase 1 study; preclinical study; preclinical toxicity; safety study; sample collection; scaffold; segregation; subretinal injection; tissue preparation

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).

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。研究产品由在可生物降解支架上生长的iPSC分化的RPE单层组成，并且需要在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。