Drug and Gene Targeting                                 Efficient Delivery of Agents to Molecular Targets Over the past 10-15 years, the ability to identify individual molecular mediators of disease has allowed drugs to be designed and developed to be very specific for their molecular targets. This advanced drug discovery for unique molecular targets of disease has not been fully realized, however, due to limits of current technology to deliver the novel drugs selectively to the diseased tissue or organs. Targeting these very specific agents to the site of disease will improve the therapeutic response for the patient by increasing the potency of the drugs to allow efficacy at much lower dosages. Targeting should, therefore, reduce undesired side effects and improve the cost/benefit ratio for the patient. The selection of targeting scheme will certainly be based on the disease but may also be unique to the extent of disease and genetic profile of each patient. Introduction of targeting to the clinic is in congruence with the trend to individualize treatment for each patient made possible by advances in diagnostic technology to generate a molecular disease profile on patients. Depending on the patient's molecular disease profile, one targeting scheme or unique combination of schemes may be the optimal therapeutic strategy. GlycoTech's approach to targeting is to utilize our expertise in carbohydrates to design novel targeting schemes based on lectin-carbohydrate interactions. Targeting based on lectin-carbohydrate interactions is advantageous for several reasons. Lectins and carbohydrates are expressed on cell surfaces and are, therefore, accessible to receive a targeted agent. For some diseases, carbohydrates are uniquely expressed or significantly upregulated on the cell surface allowing a targeted agent to be selectively delivered to the diseased cells. Carbohydrates exhibit fast kinetic on-rates with their lectin receptors so targeting can be achieved in environments of convective mass transport such as in the vasculature. The discovery and use of glycoconjugates and glycomimetics will lead to the development of targeting schemes useful in diagnostic imaging, drug delivery, and gene therapy. Technology GlycoTech's expertise in carbohydrates coupled with our extensive carbohydrate, neoglycoconjugate, and carbohydrate antibody libraries allows us to probe cell surfaces to identify native carbohydrate structures and, more specifically, the carbohydrate binding epitope that can be a candidate for targeting. Most of our assays are