Development of a Novel Gene Delivery System

描述(由申请人提供)：基于质粒DNA(PDNA)传递的基因治疗策略已被提出用于各种应用，包括疫苗和内源性治疗性蛋白质的生产。不幸的是，由于缺乏临床可行的基因输送系统，这些方法的临床实施一直受到阻碍。最近的研究表明，体内电穿孔可以显著改善pDNA的输送。然而，目前的方法和装置并不适合广泛的临床应用。关键的一步是发展安全、可靠和有效的管理手段。它还必须使用简单，对患者造成的不适降至最低。在第一阶段，Ichor研究了一种适用于肌肉内基因输送的集成应用设备的基本可行性。这项工作证明了拟议装置的基本可行性，并导致了概念的进一步完善。在第二阶段，Ichor将开发一种适用于人类肌肉内基因传递的应用系统。拟议的工作范围包括研究和开发、制造功能原型，最后是安全和性能验证研究。在SBIR第11阶段成功完成后，建议的系统将用于肌肉内注射新的基于基因的产品的临床研究。

DESCRIPTION (provided by applicant): Gene therapy strategies based on plasmid DNA (pDNA) delivery have been proposed for a variety of applications including vaccines and endogenous production of therapeutic proteins. Unfortunately, clinical implementation of these approaches has been hampered by the lack of clinically viable gene delivery systems. Recent studies suggest that in vivo electroporation can dramatically improve the delivery of pDNA. However, the current methods and devices are inappropriate for widespread clinical application. A critical step is the development of safe, reliable, and effective means of administration. It must also be simple to apply and result in minimal discomfort for the patient. During Phase I, Ichor investigated the basic feasibility of an integrated application device suitable for intra-muscular gene delivery. This work demonstrated the basic feasibility of the proposed device and led to further refinement of the concept. During Phase II, Ichor will develop an application system that is suitable for intra-muscular gene delivery in humans. The proposed scope of work includes research and development, manufacture of functional prototypes, and finally, safety and performance validation studies. Following the successful completion of SBIR Phase 11, the proposed system will be utilized in clinical studies for the intra-muscular delivery of novel gene-based products.