Antisense oligonucleotides are essentially short, single-stranded synthetic fragments of DNA or RNA that bind specifically to the target RNA sequence inside cells and modulate protein expression through several different mechanisms, blocking the ability of the RNA to make a protein or function in other ways. In recent years, these drugs have gained popularity among drug developers, as they are a highly specific, targeted treatment that can treat a wide range of diseases. In this article, we take a look at six antisense oligonucleotide companies leading the way in this emerging field. As the first company to ever have an antisense oligonucleotide drug approved by the U.S. Food and Drug Administration (FDA), Ionis Pharmaceuticals is now seen as a pioneer in the field. Its drug, called fomivirsen, was given the green light in 1998 to treat cytomegalovirus (CMV) retinitis – a serious infection of the retina that can lead to blindness – in immunocompromised patients with AIDS.

Full report : Six antisense oligonucleotide companies shaping the future of genetic medicine.