CMC SUPPORT FOR NCATS RARE DISEASE PLATFORM GENE THERAPY TRIAL PROJECTS

该 SOW 的目标是让 Leidos-BDP 进行化学、制造和控制 (CMC) 开发和制造任务，以便为两种治疗甲基丙二酸血症 (MMA) 的基因治疗产品实现监管备案和临床研究。 该目标有两个子目标： 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 AAV9 基因载体制造工艺，并具有知识产权操作自由。 这两个子目标可以单独或并行地实现，例如。 g。初始临床批次可能不一定使用无 IP 或优化技术制造，技术开发仍在继续，而初始批次则使用当前可用的方法制造。

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.