A survey of the recent patent literature on the delivery of genes and oligonucleotides

Twenty years ago a biological revolution occurred which was ushered in by the newly found possibilities of recombinant technology. The ability to isolate a particular gene and insert it into an expression system such a bacterium or a mammalian cell started the field of biopharmaceuticals where proteins, and not just small molecules, could be prepared in large quantities with high purity. Some of these same technologies have now been applied toward the next step in reaching the full therapeutic potential of this biological revolution manipulation of gene function in vivo. The possibilities of this manipulation include gene addition, gene augmentation, gene inactivation, etc. and early work has often focused on ex vivo studies which might include suicide genes to prevent run-away events. The next step would be to manipulate gene activity in vivo. This manipulation could be performed by the delivery of genes or polynucleic acid molecules (such as antisense oligonucleotides) designed to either add a new gene activity or regulate the activity of an endogenous gene. Such in vivo manipulation of gene function has raised concerns about the potential for the successful delivery of these agents. In this editorial I review some of the recent patent literature related to targeting aspects of such deliveries.