Development of a Novel Gene Delivery System

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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.