Applying Antisense Technology

A bstract : Progress in the development of antisense drugs over the last decade has led to the approval of the first such drug—Vitravene® for AIDS‐related CMV retinitis—and the development of a large number of antisense drugs in clinical trials. Antisense drugs are now being studied in Phase 3 trials for patients with cancer and inflammatory bowel disease. Other antisense drugs are in development for rheumatoid arthritis, other inflammatory conditions, and hepatitis C. Still other antisense drugs are entering clinical trials for treatment of metabolic conditions such as diabetes and hyperlipidemia. These latter applications provide the potential for target effects to be more directly measured in the clinic. Improved antisense chemistry, which will enhance the feasibility of subcutaneous and oral administration of antisense drugs and offer the potential of less frequent dosing, is expected to further expand the opportunities for antisense drug development.