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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 ...
By Mariam Sunny and Kamal Choudhury (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 ...
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.
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
Ionos SE ([]; proper spelling IONOS) is a German Internet service provider that is known for its web hosting, domain and cloud computing products. The company is part of the United Internet Group and operates in several continental European countries, (excluding Portugal) the UK and North America.
US FDA approves Ionis Pharma's genetic disorder drug (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…
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 ...
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.