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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 ...
(Reuters) -The U.S. Food and Drug Administration has approved Ionis Pharmaceuticals' drug to treat a rare genetic disorder, making it the company's first wholly-owned drug, the health regulator's ...
Ionis CEO Brett Monia said the company estimates that only 20% of the patients suffering from ATTR-PN are on treatment. "This is a market where more than one player can have great success," he added.
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]
Clinical trial number NCT04136184 for "NEURO-TTRansform: A Study to Evaluate the Efficacy and Safety of Eplontersen (Formerly Known as ION-682884, IONIS-TTR-LRx and AKCEA-TTR-LRx) in Participants With Hereditary Transthyretin-Mediated Amyloid Polyneuropathy" at ClinicalTrials.gov
The company’s lead program Tividenofusp al ... which might receive accelerated approval and priority review by late 2025. ... (NASDAQ:ALNY), and Ionis Pharmaceuticals, Inc. ...
The organization was founded by Stanley T. Crooke, former CEO of Ionis Pharmaceuticals and head of research at GlaxoSmithKline, in 2020. [3] The n-Lorem Foundation was established in January 2020 with the goal of taking advantage of the technology developed at Ionis to provide experimental treatments to patients suffering from rare genetic disorders.
IONIS-GCCR Rx, also known as ISIS-426115, is an antiglucocorticoid which is under development by Ionis Pharmaceuticals (formerly Isis Pharmaceuticals) for the treatment of diabetes mellitus type 2. [ 1 ] [ 2 ] It has also been under investigation for the treatment of Cushing's syndrome , but no development has been reported. [ 1 ]