For medical professionals

At Ionis, we are focused on harnessing the power of RNA-targeted medicines to deliver breakthroughs for patients with serious diseases.

Over the last three decades, we have pioneered the field of RNA-targeted medicines and established the now proven field of antisense oligonucleotide (ASO) therapeutics. Now, we are leveraging our expertise to expand into new approaches within RNA, including next-generation ASOs and small interfering RNA (siRNA) technology as well as DNA editing. We’re also building on our best-in-class science and technology by using innovative approaches like Mesyl-phosphoramidate (MsPA) backbone and Bicycle peptides to enhance the profile of new medicines for our pipeline and develop future breakthroughs for additional serious diseases.

Get to Know Us

Our Groundbreaking Science

The science behind our RNA-targeted medicines is inspired by a deep understanding of genetics.

Our Medicines

We have several approved medicines that treat a range of serious diseases.

Our Pipeline

Our robust late-stage pipeline of investigational medicines is poised to deliver significant impact to people with rare and common diseases.

Clinical Trials

We have a number of ongoing clinical trials across cardiovascular, neurologic and rare diseases.

Grants and Sponsorships

We accept requests for grants and sponsorships that are designed to advance knowledge in our core focus areas.

Explore Our Resources for Medical Professionals

Visit our online resource for familial chylomicronemia syndrome (FCS) and severe hypertriglyceridemia (sHTG)

Female Scientist in Lab