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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 ...
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 ...
In 2019 he was awarded a Breakthrough Prize in Life Sciences for his contributions to the understanding of the RNA gene-splicing process and, in collaboration with fellow Prize Laureate Dr. Frank Bennet of Ionis Pharmaceuticals, the development of medical interventions that target the RNA-splicing process, including Spinraza, which is the first treatment for the genetic disorder Spinal ...
(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 ...
The U.S. Food and Drug Administration (FDA) on Thursday approved Ionis Pharmaceuticals and partner AstraZeneca's drug to treat nerve damage caused by a life-shortening rare disease. The drug ...
The rare genetic disorder, known as familial chylomicronemia syndrome (FCS), prevents the body from breaking down fats and is characterized by extremely high levels of triglyceride. FCS is caused ...
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.
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