C03765: Enhanced and Effective Gene Therapy Delivery MethodNovelty:
This technology is an effective gene delivery system that enhances gene transfer and expression to a solid mass of cells, particularly a solid organ, while resulting in minimal degradation and injury to endothelial cells.
Value Proposition:
Effective delivery of nucleic acids to desired cells within tissue with high levels of expression has been difficult to achieve with current gene transfer technology. Current methods fail to extend nucleic acids across the entire area of interest, preventing sufficient gene expression while possibly causing serious injury to endothelial cells through the means of administration. This system enhances gene transfer efficiency across a solid mass of cells by incorporating a single or multiple vasculature permeability agents alongside a permeability enhancement agent. Additional advantages include:
• Highly efficient and widespread delivery of desired nucleic acids
• Less injurious to endothelial cells
• Provides therapeutics to solid organs in a variety of disease states
• Effectively treats larger areas and more cells than previous gene therapy strategies
Technical Details:
Johns Hopkins researchers have developed a gene transfer method which involves administration of nucleic acids into tissue alongside vascular permeability agents such as PDE-3 and a permeability enhancing agent such as VEGF. The combined effect of the vascular permeability agent and the permeability enhancing agent has shown to significantly enhance nucleic acid delivery to targeted tissue. With better access to the cells, genetic information is more readily accepted by target cells, markedly increasing effectiveness of gene transfer and ultimately, expression of desired gene.
Looking for Partners:
To develop & commercialize the technology as a novel, more efficient method for gene transfer technology.
Stage of Development:
Pre-Clinical
Data Availability:
Under CDA / NDA
Publications/Associated Cases:
Not available at this time
