DEVELOPMENT OF PLX-R18 CELL THERAPY AS A COUNTERMEASURE FOR HEMATOPOIETIC ACUTE RADIATION SYNDROME

这项建议的目标是开发人类胎盘来源的基质细胞，PLX-R18细胞疗法，以缓解H-ARS。Pluri Biotech的主要分包商是武装部队放射生物学研究所，要约人建议广泛进行CRISPR修饰细胞的体外表征和生产，以及辐射啮齿动物和NHP产品功效研究。承包商应进行研究，以支持PLX-R18作为预防、缓解和治疗H-ARS所有方面的新型蜂窝MCM的开发。研究将评估PLX-R18在体外和H-ARS动物模型中的疗效和作用机制，当在辐射暴露后至少24小时给药时。该项目预计将提供所有必要的信息，以证明和设计未来充分和良好控制的研究，以支持PLX-R18的许可。重要的是，这项拟议的研究旨在证明PLX-R18即使在辐射暴露后48小时或更晚的情况下也是有效的。此外，承包商应根据美国FDA动物规则执行必要的监管步骤，以获得FDA的批准。

The goal of this proposal is to develop human placenta-derived stromal cells, PLX-R18 cell therapy, for mitigation of H-ARS. Pluri Biotech’s main subcontractor is the Armed Forces Radiobiology Research Institute, and the Offeror proposes extensive in vitro characterization and production of CRISPR-modified cells, as well as irradiated rodent and NHP product efficacy studies. The Contractor shall perform studies to support the development of PLX-R18 as a novel cellular MCM for the prevention, mitigation, and treatment of all aspects of H-ARS. Studies will evaluate the efficacy and mechanism of action of PLX-R18 in vitro and in animal models of H-ARS, when administered at least 24 hours after radiation exposure. This project is expected to deliver all information required to justify and design future adequate and well-controlled studies to support licensure of PLX-R18. Importantly, the proposed study aims to demonstrate efficacy of PLX-R18 even when administered 48 hours or later after radiation exposure. In addition, the Contractor shall perform the necessary regulatory steps towards FDA approval under the US FDA Animal Rule.