Feasibility Analysis of a Novel System for Gene Delivery

描述（申请人提供）：基于质粒的基因治疗策略 已经提出DNA（pDNA）递送用于多种应用，包括 疫苗和治疗性蛋白质的内源性生产。不幸的是， 这些方法的临床实施受到缺乏 临床上可行的基因递送系统。最近的研究表明， 电穿孔可显著改善pDNA的递送。但 目前的方法和装置不适合广泛的临床应用 应用程序.由于lchor已经开发了临床电穿孔技术 技术的药物输送，该公司处于有利地位， 开发pDNA递送技术。关键的一步是发展 安全、可靠、有效的给药手段。它也必须简单 以使患者的不适最小化。鉴于这些 标准，Ichor建议开发一种综合管理设备， 使得能够临床实施电穿孔介导的pDNA递送。 在第一阶段，Ichor将展示其集成的基本可行性， pDNA施用系统。研究范围包括设计和 开发原型装置并进行测试，以验证安全性和有效性， 小动物。第一阶段的成功完成将导致 适用于大型动物并最终适用于人类的器械。 拟议商业应用：不可用

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 etectroporation can dramatically improve the delivery of pDNA. However, the current methods and devices are inappropriate for widespread clinical application. Since lchor has already developed clinical electroporation technology for pharmaceutical delivery, the company is well positioned to develop the technique for pDNA delivery. 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 patients. In light of these criteria, Ichor proposes to develop an integrated administration device to enable clinical implementation of electroporation mediated pDNA delivery. During Phase I, Ichor will demonstrate basic feasibility for its integrated pDNA administration system. The scope of the research includes design and development of a prototype unit and testing to verify safety and efficacy in small animals. Successful completion of Phase I will lead to development of devices appropriate for application in larger animals and eventually, humans. PROPOSED COMMERCIAL APPLICATION: NOT AVAILABLE