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Company Profile

Ionis is the leading company in RNA-targeted drug discovery and development focused on developing drugs for patients who have the highest unmet medical needs, such as those patients with severe and rare diseases. Using its proprietary antisense technology, Ionis has created a large pipeline of first-in-class or best-in-class drugs, with over three dozen drugs in development. SPINRAZA® (nusinersen) has been approved in the U.S., Europe, Japan, Canada and Brazil for the treatment of spinal muscular atrophy (SMA). Biogen is responsible for commercializing SPINRAZA. Drugs that have successfully completed Phase 3 studies include inotersen , an antisense drug Ionis is developing to treat patients with TTR amyloidosis, and volanesorsen, an antisense drug discovered by Ionis and co-developed by Ionis and Akcea Therapeutics to treat patients with either familial chylomicronemia syndrome or familial partial lipodystrophy. Akcea, an affiliate of Ionis, is a biopharmaceutical company focused on developing and commercializing drugs to treat patients with serious cardiometabolic diseases caused by lipid disorders. If approved, volanesorsen will be commercialized through Ionis' affiliate, Akcea. Volanesorsen filings for marketing approval have been submitted in the U.S., EU and Canada. Inotersen is progressing toward regulatory filings for marketing authorization. Ionis' patents provide strong and extensive protection for its drugs and technology.

Recent Press Releases

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Date Title and Summary
Toggle Summary Biogen and Ionis Expand Strategic Collaboration to Develop Drug Candidates for a Broad Range of Neurological Diseases
Transaction builds upon the productive collaboration that produced SPINRAZA ®  as well as two antisense drug candidates currently in the clinic, with the potential to advance up to seven more drug candidates to the clinic within the next two years The antisense platform developed by Ionis is an
Toggle Summary Presentations at the 2018 American Academy of Neurology Highlight the Broad Potential of Antisense Drugs for Neurological Diseases
-- Special conference session focused on therapeutic potential of antisense drugs to treat neurological diseases -- -- Fourteen presentations on Ionis' antisense drugs to treat neurological diseases, including SMA, amyloidosis, Huntington's disease, Alzheimer's disease, and ALS -- CARLSBAD, Calif.
Toggle Summary Akcea and Ionis Complete Licensing Transaction to Commercialize Inotersen for hATTR
  Akcea shareholders approved the transaction on April 16 at a Special Meeting of Stockholders                              Ionis licenses Akcea worldwide rights to inotersen and AKCEA-TTR-L Rx                CAMBRIDGE, Mass. and CARLSBAD, Calif., April 17, 2018 (GLOBE NEWSWIRE) --  Akcea
Toggle Summary Sarah Boyce Joins Akcea Therapeutics as President and Member of the Board of Directors
CAMBRIDGE, Mass. , April 17, 2018 (GLOBE NEWSWIRE) -- Akcea Therapeutics, Inc. (NASDAQ:AKCA), an affiliate of Ionis Pharmaceuticals, Inc. , today announced that, following today’s close of the transaction licensing Akcea the exclusive, worldwide rights to inotersen and AKCEA-TTR-L Rx , Sarah Boyce

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