Generic API researchOligonucleotides

Oligonucleotides are Nucleic acid polymer chains that can act in a sequence specific manner to control gene expression.

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.

Nusinersen

Nusinersen

Patisiran

Patisiran