Oligonucleotide Therapeutics--IBC Sixth International Conference. 3-5 May 1999, La Jolla, CA, USA.

ABSTRACT

A wide array of strategies was presented for exploiting antisense oligonucleotides (AS ONs). Vitravene (ISIS Pharmaceuticals Inc), a first-generation phosphorothioate (PS) oligodeoxynucelotide (ODN) has been approved for use in the US and European markets for the treatment of CMV retinitis. A number of pharmaceutical companies introduced numerous compounds in both phase I and pivotal phase II clinical trials, for treatment of a wide range of diseases, including cancer, inflammation and viral agents. Advances in AS ON delivery were also described, including topical and oral routes of administration. New chemical modifications incorporated into second-generation oligonucleotides demonstrated superior potency and duration of action in a number of preclinical models. Finally, in response to the explosion in new genomic sequence information generated by the Human Genome Project, a number of companies are combining bioinformatics with high-throughput screening (HTS) to rapidly discover new drug targets. As a result, there was much excitement exhibited by researchers attending this meeting and a strong feeling that this new drug paradigm is delivering on its initial promise.