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Moderna, Inc. (/ m ə ˈ d ɜːr n ə / mə-DUR-nə) [4] is an American pharmaceutical and biotechnology company based in Cambridge, Massachusetts, that focuses on RNA therapeutics, primarily mRNA vaccines. These vaccines use a copy of a molecule called messenger RNA (mRNA) to carry instructions for proteins to produce an immune response.
RNA therapeutics are a new class of medications based on ribonucleic acid (RNA). Research has been working on clinical use since the 1990s, with significant success in cancer therapy in the early 2010s. [1] In 2020 and 2021, mRNA vaccines have been developed globally for use in combating the coronavirus disease (COVID-19 pandemic). [2]
The company develops RNA therapeutics for the treatment of rare diseases such as ornithine transcarbamylase deficiency, and respiratory diseases such as cystic fibrosis. As of 2021 [update] , vaccine medicines include a vaccine candidate for COVID-19 and an influenza vaccine in the preclinical development phase.
Arrowhead Pharmaceuticals is a publicly traded biopharmaceutical company based in Pasadena, California. Arrowhead’s products in development act through RNA interference (RNAi) mechanisms of action. [2] [3] The company focuses on treatments for hepatitis B, liver disease associated with alpha 1-antitrypsin deficiency and cardiovascular disease ...
Ultragenyx Pharmaceutical Inc. is an American biopharmaceutical company involved in the research and development of novel products for treatment of rare and ultra-rare genetic diseases for which there are typically no approved treatments and high unmet medical need. The company works with multiple drug modalities including biologics, small ...
Acuitas Therapeutics Inc. is a Canadian biotechnology company based in Vancouver, British Columbia. The company was established in February 2009 to specialize in the development of delivery systems for nucleic acid therapeutics based on lipid nanoparticle (LNP) technology, a key component of the mRNA vaccines deployed for COVID-19 .
This listing is limited to those independent companies and subsidiaries notable enough to have their own articles in Wikipedia. Both going concerns and defunct firms are included, as well as firms that were part of the pharmaceutical industry at some time in their existence, provided they were engaged in the production of human (as opposed to veterinary) therapeutics.
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 ...