Ionis’ antisense technology

With RNA as the target that forms the basis of our novel drug discovery platform, we are tackling the most difficult to treat diseases with a focus on neurology and cardiometabolic diseases. Our antisense therapies are disrupting diseases and, we hope, changing their course—from the rarest of conditions, to those that impact millions of people.

And as we continue to advance and expand our technology, the therapies that we discover and develop will become more precise and more potent. Our aim is to provide patients who have few to no effective treatment options, a medicine that specifically targets the root cause of their disease and potentially change its course.

Antisense: Changing the course of disease

Antisense therapies are designed to seek out, bind to and destroy a mRNA in a highly specific manner, so that the amount of disease-causing protein is dramatically decreased. Antisense therapies can also treat diseases caused by too little protein by increasing the production of the protein, thereby restoring the protein to normal levels.