OPKO - Model AntagoNAT - Natural Antisense Transcripts Antagonist Technology

In our Natural Antisense Transcripts Antagonist (AntagoNAT) technology, an oligonucleotide molecule is designed to interfere with regulatory gene expression in order to enhance production of endogenous functional proteins. This strategy is unique and contrasts with established approaches which down-regulate protein production. This platform can lead to the discovery of new drugs to target relevant genes that regulate the optimal production of endogenous functional proteins. One of the leading applications of our AntagoNAT technology is OPK88001 which is designed for the treatment of Dravet syndrome by targeting the mutated SCN1A gene.