Pipeline of RNA-Targeted Therapies

PIPELINE

An Unprecedented Opportunity to Change the Course of Human Health

We are relentlessly pursuing the innovations of tomorrow with our broad and diverse pipeline of potential first or best-in-class investigational medicines designed to treat a range of diseases across our focus areas of neurology, cardiometabolic disease and select areas of high patient needs.

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Last updated: April 29, 2026

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Ionis’ science is inspired by a deep understanding of the genetic foundations of disease and a commitment to advancing next-generation technology. Read more about our science and innovation.