SBIR-STTR Award

Recent activity has indicated the tremendous potential of antisense oligonucleotides in a variety of research environments. Of particular interest has been the demonstration that antisense oligonucleotides with a modified phosphate backbone have significant activity against the AIDS virus. Once a suitably active antisense oligonucleotide is identified, there will likely be significant delays in its commercial development because of technical problems in its production on a large scale. The current research plan is designed to lead to the development of a synthesis system capable of being adapted to a pharmaceutical production scale.

Anticipated Results:
This research is designed to identify a synthesis system capable of producing therapeutic modified oligonucleotides on a pharmaceutical production scale.National Institute of Allergy And Infectious Diseases

The use of modified antisense oligonucleotides as potential chemotherapeutics is currently progressing from in vitro studies in research labs to pharmacology studies using animal models. As yet, there exists no commercial DNA synthesizer capable of producing a variety of modified oligonucleotides on a large scale. The proposed research is designed to develop a commercial DNA synthesizer, optimized for H- phosphonate chemistry, capable of producing multigram quantities of modified oligonucleotides will be prepared. Also, techniques will be designed for the purification of these products on a pharmaceutical scale.

Thesaurus Terms:
antisense nucleic acid, biomedical equipment development, deoxyribonucleic acid, nucleic acid chemical synthesis, oligonucleotide design /development, ion exchange chromatography, monomer, phosphonate gel electrophoresis, high performance liquid chromatography, nucleic acid sequencing