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Ionis Pharmaceuticals, Inc. is a biotechnology company based in Carlsbad, California, that specializes in discovering and developing RNA-targeted therapeutics.The company has three commercially approved medicines: Spinraza (), Tegsedi (), and Waylivra (Volanesorsen), and has four drugs in pivotal studies: tominersen for Huntington's disease (together with Roche), tofersen for SOD1-ALS, AKCEA ...
In October 2024, the Committee for Medicinal Products for Human Use of the European Medicines Agency adopted a positive opinion, recommending the granting of a marketing authorization for the medicinal product Wainzua, intended for the treatment of adults with hereditary transthyretin-mediated amyloidosis (ATTRv) and stage 1 or 2 polyneuropathy. [11]
The results come as companies race to bring the first approved treatment for the disease and tap into a global market expected to surpass $16 billion by 2030, according to market research firm ...
Tofersen was developed by Ionis Pharmaceuticals and was licensed to, and co-developed by, Biogen. [7] [8]The effectiveness of tofersen was evaluated in a 28-week, randomized, double-blind, placebo-controlled clinical study in 147 participants with weakness attributable to amyotrophic lateral sclerosis and a superoxide dismutase 1 (SOD-1) mutation confirmed by a central laboratory. [2]
The life-threatening genetic disease called hereditary angioedema causes unpredictable and frequent severe swelling of the skin, gastrointestinal tract, upper respiratory system, face and throat.
Ionis Pharmaceuticals, another biotech, is also pursuing an HAE program, and it reported results from a phase 3 clinical trial in late May of this year. Patients treated with its therapy once ...
The drug was discovered and developed by Ionis Pharmaceuticals. Volanesorsen was designated an orphan drug by the European Medicines Agency (EMA) in February 2014, for phosphorothioate oligonucleotide targeted to apolipoprotein C-III for treatment of familial chylomicronaemia syndrome. [8]
C. Frank Bennett is an American pharmacologist.Bennett is currently the Senior Vice President of Research and Neurology Franchise Leader at Ionis Pharmaceuticals. [1] He is a 2019 Breakthrough Prize winner in Life Sciences, which he shared with his collaborator Adrian R. Krainer for the development of an effective antisense oligonucleotide therapy for children with the neurodegenerative ...