Ionis Pharmaceuticals IONS

Ionis Pharmaceuticals Inc. is an American biotechnology company based in Carlsbad, California, that specializes in the discovery and development of RNA-targeted therapeutics. The firm’s products are aimed at patients suffering from a range of life-altering diseases, for which other therapeutic approaches either have not proven effective, or do not exist, including SMA, hATTR and FCS. The company was founded in 1989.

The firm is primarily focused on two core research areas: neurology and cardiometabolic. Commercial medicines available from the company include SPINRAZA, which is for treatment of patients of all ages with spinal muscular atrophy (SMA), and TEGSEDI, for the treatment of patients with polyneuropathy caused by hereditary transthyretin amyloidosis (hATTR), both of which are debilitating, progressive, and potentially fatal diseases.

The company’s therapeutics are based on its antisense technology, in which drugs attach themselves to strands of RNA to prevent them from producing disease-causing proteins. In this way it is potentially possible to create a therapy that fights disease without harming healthy cells. Ionis has a robust pipeline of drugs in development, aimed at addressing neurodegenerative diseases, cardiometabolic diseases, and cancer. The firm is listed on the NASDAQ under the ticker IONS.

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