CMC SUPPORT FOR NCATS RARE DISEASE PLATFORM GENE THERAPY TRIAL PROJECTS

本工作说明书的目的是让Leidos-BDP执行化学、生产和控制（CMC）开发和生产任务，以实现两种治疗甲基丙二酸血症（MMA）的基因治疗产品的监管备案和临床研究。 该目标有两个次级目标： 交付临床前和临床批次以及相关数据包，以及时进行临床试验;开发稳健且可扩展的AAV 9基因载体制造工艺，并享有知识产权自由操作权。 这两个次级目标可以分开和平行处理，G.初始临床批次可能不一定使用无IP或优化技术生产，在使用当前可用方法生产初始批次的同时，技术开发仍在继续。

The objective of this SOW is for Leidos-BDP to conduct the chemistry, manufacturing, and controls (CMC) development and manufacturing tasks to enable regulatory filing and clinical studies for two gene therapy products to treat methylmalonic acidemia (MMA). The objective has two sub-objectives: Delivery of preclinical and clinical lots and associated data packages to timely enable clinical trials; Development of robust and scalable process for manufacture of AAV9 gene vectors, with IP freedom to operate. The two sub-objectives may be addressed separately and in parallel, e. g. initial clinical lots may not necessarily be manufactured using IP-free or optimized technology, with technology development continuing while initial lots are manufactured using currently available methods.