The application of small nucleic acids is slowly moving from chemistry and molecular biology laboratories into the clinic. However, the development of a new family of therapeutics is always difficult. Research must be performed to produce chemically compatible compounds in vivo and pharmacological investigations are necessary to precisely define the in vivo mechanisms of action of these potentially new drugs. The "2nd International Conference on Antisense Nucleic Acids: Biology, Pharmacology, Therapy" held in Garmisch-Partenkirchen addressed these questions.