VIRALLY INACTIVATED HUMAN RED CELLS FOR TRANSFUSION

长期目标是通过减少血液传播病毒感染的可能性来提高红细胞输注的安全性。仅在美国，每年就有超过1200万例红细胞输注，而且没有病毒灭活程序可用来清除它们的污染。因此，有效的病毒净化程序将解决一个重大的未得到满足的健康需求。INACTINE TM病毒灭活技术目前处于第一阶段临床试验。这项提案描述了进一步开发这项技术的步骤，以实现用于输血的人红细胞去污染的实用商业程序。这些研究包括：1-支持关键的临床试验和产品前许可证申请的病毒验证研究；2.测试一组潜在细菌污染物对治疗的敏感性；3.灭活器械的一次性部件的设计和制造；4.在动物身上进行的经处理的红细胞的研究，以证实缺乏新的抗原性；5.额外的毒理学研究，以证实经处理的红细胞的安全性。这些研究的结果将为在这项提议的五年时间框架内进行关键的临床试验奠定基础。

The long-term goal is to increase the safety of red blood cell transfusions by reducing the possibility of blood-borne virus infections. In the United States alone, there are over 12,000,000 red blood cell transfusions annually and there is no virus inactivation procedure available to decontaminate them. Thus, an effective virus decontamination procedure would address a significant unmet health need. The INACTINE TM virus inactivation technology is currently in Phase 1 Clinical Trials. This proposal describes steps to develop this technology further toward a practical commercial procedure for decontaminating human red blood cells for transfusion. These studies include: 1 - Virus validation studies to support a pivotal clinical trial and pre- product license application; 2. Testing the sensitivity of a panel of potential bacterial contaminants to the treatment; 3. Design and manufacture of the disposable parts of the inactivation apparatus; 4. Studies of treated red blood cells in animals to confirm lack of neoantigenicity; 5. Additional toxicology studies to confirm the safety of treated red blood cells. The results of these studies will set the stage for pivotal clinical trials to be carried out during the five year time-frame of this proposal.