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Ionis Innovation

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.

The Ionis antisense advantage

Optimizing antisense through advancements in medicinal chemistry and pioneering new ways to deliver antisense has been a central focus of our research efforts, allowing us to bring options to those who have had none.

A perpetual and efficient innovation machine

Our platform has served as a springboard for drug discovery and realized hope for patients with unmet needs. Our broad, diverse pipeline has more than 40 first-in-class and/or best-in-class medicines designed to treat a broad range of diseases.