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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 Ionis Pharmaceuticals Licenses Second Orally Delivered Antisense Drug to Janssen
- IONIS-JBI2-2.5 Rx designed to treat GI autoimmune disease - Ionis Earns $5 Million License Fee from Janssen CARLSBAD, Calif. , Nov. 16, 2017 /PRNewswire/ --  Ionis Pharmaceuticals, Inc. (NASDAQ: IONS) announced today that it has licensed a second orally delivered Generation 2.5 antisense drug to
Toggle Summary Akcea and Ionis Announce Acceptance of Marketing Applications in U.S., EU and Canada for Volanesorsen for the Treatment of FCS
FDA Prescription Drug User Fee Act goal date set for August 30, 2018 CAMBRIDGE, Mass., Nov. 15, 2017 (GLOBE NEWSWIRE) -- Akcea Therapeutics, Inc. (NASDAQ:AKCA), an affiliate of Ionis Pharmaceuticals, Inc. (NASDAQ:IONS), focused on developing and commercializing drugs to treat patients with serious
Toggle Summary Akcea Initiates AKCEA-ANGPTL3-LRx Phase 2 Program in Patients with Rare Hyperlipidemias
CAMBRIDGE, Mass., Nov. 13, 2017 (GLOBE NEWSWIRE) -- Akcea Therapeutics, Inc. (NASDAQ:AKCA), an affiliate of Ionis Pharmaceuticals, Inc. (NASDAQ:IONS), focused on developing and commercializing drugs to treat patients with serious cardiometabolic diseases caused by lipid disorders, today announced
Toggle Summary Ionis Licenses Novel Antisense Drug for the Treatment of Centronuclear Myopathy to Dynacure
Ionis earns $5 million license fee from Dynacure CARLSBAD, Calif. and STRASBOURG, France , Nov. 9, 2017 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (NASDAQ: IONS) and Dynacure announced today that Dynacure has licensed IONIS-DNM2-2.5 Rx (Dyn101), a Generation 2.5 antisense drug targeting dynamin 2

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