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A Novel Method for Importing Peptides with Functional Cargo Into the Cells
This technology enables the deliveryof biological molecules into the interior of a cell. Such a delivery mechanism could be utilized in a variety of therapies including peptide, gene transfer and/or antisense therapy.
Many peptides have been developed to treat a variety of conditions including heart, cancer, respiratory and neurological
conditions. The major obstacle of using such peptides as therapies is their actual delivery. Not only has it been a major challenge tospecifically target therapies to the particular cell type to be treated, but the cell membrane also becomes a major obstacle. Delivery of such biological molecules remains problematic since they are unable to readily cross cell membranes. Although many companies are currently working on delivery methods, current methods include microinjection and permeabilization of the cell membrane, both of which have limitations and adverse side affects. Cell permeabilization can cause severe damage to the cell and often results in cell death. Microinjections require a high skill set and often results in cell damage. Further, these methods have limited applications.
By utilizing a complex comprised of an importation competent signal peptide, Vanderbilt researchers have deviseda mechanism for delivering peptides, polypeptides, proteins, nucleic acids, carbohydrates, lipids and other therapeutic agents across the cell membrane and into the cell. This technology relieves the existing problems by providing a method of importing a biologically active molecule into a cell using mechanisms that normally occur in the cells. Thus, this invention avoids the damage that can occur to the target cells often seen in current delivery mechanisms.
Improved delivery of drugs would not only improve safety and efficacy, butit would also lead to increased patient compliance. Many companies are adopting drug delivery as a strategy to repel genetic competition, which threatens their blockbuster drugs scheduled to lose their patents.
Potential Market Size
According to Frost and Sullivan, the drug delivery market was valuedover $55 billion dollars in 2006 and is predicted to continue to grow. Not only is there a need for new drug delivery systems, but drug delivery systems may also allow pharmaceutical companies to revive late stage products and maintain patent protection for such products.
Currently peptides cross cell membranes either through active transport, cell permeabilization or microinjection. Active transport has been anarea of promise for drug delivery into cells; however, this requires that one knows a great deal about the specific carrier proteins responsible for such transport, such as their distribution on various organs and cell types and their specificity. Both cell permeabilization and microinjection can lead to damage of the cell membrane.
Several patents have been issued and are pending, comprising both composition and method claims. Three United States patents have been issued (5,807,746; 6,043,339;6,495,518) in addition to patents in Great Britian, Ireland, Australia, France and Germany. Patents are still pending in both the United States and Canada.
Inventors:Jack HawigerYao-Zhong Lin