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Company Profile
We are the leading company in RNA-targeted drug discovery and development with a pipeline of first-in-class or best-in-class medicines with the potential to provide high value for patients with significant unmet medical needs. We have created an efficient and broadly applicable drug discovery platform. Using this platform, we believe that we are revolutionizing medicine with the goal to improve the quality of, and save, lives.
Recent Press ReleasesMore >>
06/22/17Enrollment in Phase 1/2a Study of IONIS-HTT Rx in Patients with Huntington's Disease Completed and Open-Label Extension Study to Open in 2H 2017
CARLSBAD, Calif., June 22, 2017 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (NASDAQ: IONS) today announced the completion of enrollment in the Phase 1/2a randomized, placebo-controlled, dose escalation study of IONIS-HTTRx in patients with Huntington's disease (HD). Dosing in the final patient cohort continues, and Ionis plans to report top-line results from this study around year-end 2017. The safety and tolerability profile of IONIS-HTTRx in the completed cohorts of the Phase 1/2a study suppor... 
06/20/17Akcea Therapeutics Launches Proposed Initial Public Offering
CAMBRIDGE, Mass., June 20, 2017 /PRNewswire/ -- Akcea Therapeutics, Inc. today announced that it has launched the proposed initial public offering of its common stock. Akcea is offering 9,620,000 shares of common stock and the underwriters will have a 30-day option to purchase up to an additional 1,443,000 shares of common stock. The initial public offering price is currently expected to be between $12.00 and $14.00 per share.  Akcea has applied to list the s... 
06/01/17SPINRAZA® (nusinersen) Approved in the European Union
First Approved Treatment in EU for SMA Approved to Treat Broad Range of People with SMA Ionis Earns $50 Million Milestone CARLSBAD, Calif., June 1, 2017 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (NASDAQ: IONS) announced today that the European Commission (EC) has granted marketing authorization for SPINRAZA® (nusinersen) for the treatment of 5q spinal muscular atrophy (SMA). 5q SMA is the most common form of the disease and represents approximately 95% of all SMA cases. SPINRAZA is th... 
05/25/17Akcea Announces Publication in The New England Journal of Medicine of Data with AKCEA-ANGPTL3-L Rx Showing Favorable Cardiometabolic Effects
CAMBRIDGE, Mass., May 25, 2017 /PRNewswire/ -- Akcea Therapeutics, a wholly owned subsidiary of Ionis Pharmaceuticals, Inc. (NASDAQ: IONS), today announced the publication in The New England Journal of Medicine of key preclinical findings with angiopoietin-like 3 (ANGPTL3)-targeting drugs and Phase 1/2 clinical study results with AKCEA-ANGPTL3-LRx (formerly IONIS-ANGPTL3-LRx). In the Phase 1/2 clinical study, treatment with AKCEA-ANGPTL3-LRx resulted in substantial, dose-dependent reductions in ... 
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