About Isis

Pioneering antisense drug discovery

We are the leading company in antisense drug discovery and development, exploiting a novel drug discovery platform we created to generate a broad pipeline of first-in-class drugs. Antisense technology provides a direct route from genomics to drugs. With our highly efficient and prolific drug discovery platform we can expand our pipeline and our partners' pipelines with antisense drugs that address significant medical needs. Our strategy is to do what we do best—to discover unique antisense drugs and develop these drugs to key clinical value inflection points.

We discover and conduct early development of new drugs and, at the key clinical value inflection points, outlicense our drugs to partners. We maximize the value of the drugs we discover by putting them in the hands of leading pharmaceutical companies with late-stage development, commercialization and marketing expertise, such as Biogen Idec, Genzyme, a Sanofi company, GlaxoSmithKline, AstraZeneca and Roche. For instance, our partner, Genzyme, is commercializing our lead product, KYNAMRO® in the United States for the treatment of patients with homozygous familial hypercholesterolemia, or HoFH. We also work with a consortium of smaller companies that can exploit our drugs and technologies in areas that are outside of our core focus. As a result of our unique strategy, we can keep our organization small and focused. Our strong financial position is a result of the successful execution of our business strategy as well as our inventive and focused research and development capabilities.

Furthermore, as an innovator in RNA-targeted drug discovery and development, we have designed and executed a patent strategy that has provided us with strong and extensive protection for our drugs as well as many aspects of antisense drug discovery, development and manufacturing. We are continually making improvements to our drug discovery platform and our drugs and expanding our antisense technology to include new opportunities. We expect to remain the leader in all aspects of antisense technology advancement.


Antisense technology is opening new doors in the way we treat disease.

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Our pipeline continues to grow with novel therapies in development to treat patients who have significant unmet medical needs.

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TTR Study

Isis Pharmaceuticals is currently enrolling a Phase 3 study that is designed to evaluate the safety and efficacy of ISIS-TTRRx in patients with Familial Amyloid Polyneuropathy (FAP), the form of TTR amyloidosis that causes nerve damage.

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