Therapeutic oligonucleotides exert their effect through suppression of, or interference with mRNA translation, immune stimulation, protein binding, or through induction of exon skipping. These oligonucleotides can target a broad range of mRNAs (encode all cellular proteins) including those protein targets considered “undruggable” by small molecule or protein therapeutics. Oligonucleotides are a class of new therapeutics that offer promising treatment solutions to a broad range of diseases. They also present unique scientific and regulatory challenges
Omgene is planning to develop oligonucleotides like Nusinersen (used in treating spinal muscular atrophy (SMA), a rare neuromuscular disorder), Patisiran etc.