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Alnylam Pharmaceuticals
Alnylam Pharmaceuticals, Inc. is an American biopharmaceutical company focused on the discovery, development and commercialization of RNA interference (RNAi) therapeutics for genetically defined diseases. The company was founded in 2002 and is headquartered in Cambridge, Massachusetts. In 2016, ''Forbes'' included the company on its "100 Most Innovative Growth Companies" list. History The company is a spin-off from the Max Planck Institute for Biophysical Chemistry. In 2002, Alnylam was founded by scientists Phillip Sharp, Paul Schimmel, David Bartel, Thomas Tuschl, and Phillip Zamore, and by investors Christoph Westphal and John Kennedy Clarke; John Maraganore was the founding CEO. The company was named after Alnilam, a star in Orion’s belt. The spelling was modified to make it unique. In 2003, the firm merged with the German pharmaceutical company, Ribopharma AG. The newly formed company also received $24.6 million in funding from private-equity firms. On February 27, 2004 ...
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Public Company
A public company is a company whose ownership is organized via shares of stock which are intended to be freely traded on a stock exchange or in over-the-counter markets. A public (publicly traded) company can be listed on a stock exchange (listed company), which facilitates the trade of shares, or not (unlisted public company). In some jurisdictions, public companies over a certain size must be listed on an exchange. In most cases, public companies are ''private'' enterprises in the ''private'' sector, and "public" emphasizes their reporting and trading on the public markets. Public companies are formed within the legal systems of particular states, and therefore have associations and formal designations which are distinct and separate in the polity in which they reside. In the United States, for example, a public company is usually a type of corporation (though a corporation need not be a public company), in the United Kingdom it is usually a public limited company (plc), i ...
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Orion (constellation)
Orion is a prominent constellation located on the celestial equator and visible throughout the world. It is one of the most conspicuous and recognizable constellations in the night sky. It is named after Orion (mythology), Orion, a hunter in Greek mythology. Its brightest stars are the blue-white Rigel (Beta Orionis) and the red Betelgeuse (Alpha Orionis). Characteristics Orion is bordered by Taurus to the northwest, Eridanus to the southwest, Lepus to the south, Monoceros (constellation), Monoceros to the east, and Gemini to the northeast. Covering 594 square degrees, Orion ranks twenty-sixth of the 88 constellations in size. The constellation boundaries, as set by Belgian astronomer Eugène Joseph Delporte, Eugène Delporte in 1930, are defined by a polygon of 26 sides. In the equatorial coordinate system, the right ascension coordinates of these borders lie between and , while the declination coordinates are between and . The constellation's three-letter abbreviation, as ...
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Human Respiratory Syncytial Virus
Respiratory syncytial virus (RSV), also called human respiratory syncytial virus (hRSV) and human orthopneumovirus, is a common, contagious virus that causes infections of the respiratory tract. It is a negative-sense, single-stranded RNA virus. Its name is derived from the large cells known as ''syncytia'' that form when infected cells fuse. RSV is the single most common cause of respiratory hospitalization in infants, and reinfection remains common in later life: it is a notable pathogen in all age groups. Infection rates are typically higher during the cold winter months, causing bronchiolitis in infants, common colds in adults, and more serious respiratory illnesses such as pneumonia in the elderly and immunocompromised. RSV can cause outbreaks both in the community and in hospital settings. Following initial infection via the eyes or nose, the virus infects the epithelial cells of the upper and lower airway, causing inflammation, cell damage, and airway obstruction. A varie ...
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Kyowa Hakko Kirin
is a Japanese pharmaceutical and biotechnology company under the Kirin Holdings, and is among the 40 largest in the world by revenue. The company is headquartered in Chiyoda-ku, Tokyo and is a member of the Nikkei 225 stock index. History On July 1, 1949 the forerunner of the present company, Kyowa Hakko Kogyo Co., Ltd. is established. Following the merger with Kirin Pharma Co., Ltd., on October 1, 2008 the company changed its name to its present. On July 11, 2014, the KHK subsidiary, ProStrakan Group (based in Scotland), acquired Archimedes Pharma from the Novo Nordisk Foundation for $394 million In 2019, the entity was renamed "Kyowa Kirin Co., Ltd.", replacing its prior name of Kyowa Hakko Kirin Co., Ltd. In November 2022, Kyowa Kirin announced plans to spin its international established medicines portfolio into a new joint venture with German company Grünenthal. Business segments and products * Pharmaceuticals business The company developed a method to make afucosyla ...
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Cubist Pharmaceuticals
Cubist Pharmaceuticals was an American biopharmaceutical company that targeted pathogens like MRSA. . The company employed 638 people, mostly in Lexington, MA. On 8 December 2014, Merck & Co. acquired Cubist for $102 per share in cash ($8.4 billion). History Cubist was founded in May 1992 by John K. Clarke, Paul R. Schimmel, Ph.D. and Barry M. Bloom, Ph.D, all of whom were also directors. Cubist appeared on Fortune 2010’s List of fastest growing companies, and was named to the 2010 Deloitte Technology Fast 500. In 2011, the company acquired Adolor, maker of a drug for treatment of constipation. The company expected sales of its drug Cubicin to grow to more than 1 billion dollars per year. In July 2013, Cubist Pharmaceuticals agreed to purchase Trius Therapeutics and Optimer Pharmaceuticals for around $1.6 billion. In 2014, succeeding Michael Bonney as President, Robert J. Perez, was announced to take leadership on January 1, 2015. In January 2015 Cubist Pharmaceutic ...
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MicroRNA
MicroRNA (miRNA) are small, single-stranded, non-coding RNA molecules containing 21 to 23 nucleotides. Found in plants, animals and some viruses, miRNAs are involved in RNA silencing and post-transcriptional regulation of gene expression. miRNAs base-pair to complementary sequences in mRNA molecules, then gene silence said mRNA molecules by one or more of the following processes: (1) cleavage of mRNA strand into two pieces, (2) destabilization of mRNA by shortening its poly(A) tail, or (3) translation of mRNA into proteins. This last method of gene silencing is the least efficient of the three, and requires the aid of ribosomes. miRNAs resemble the small interfering RNAs (siRNAs) of the RNA interference (RNAi) pathway, except miRNAs derive from regions of RNA transcripts that fold back on themselves to form short hairpins, whereas siRNAs derive from longer regions of double-stranded RNA. The human genome may encode over 1900 miRNAs, although more recent analysis suggests that ...
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Regulus Therapeutics
Regulus Therapeutics Inc. or Regulus () is a clinical stage biopharmaceutical company focused on the development of first-in-class drugs that target microRNAs to treat a broad range of diseases. Regulus was established in September 2007 by Alnylam Pharmaceuticals and Isis Pharmaceuticals. microRNA explained MicroRNAs are small naturally occurring RNA molecules, typically 20 to 25 nucleotides in length, that do not encode proteins but instead have evolved to regulate gene expression. anti-miR therapeutics Anti-miR therapeutics inhibit specific microRNA targets. Animal models showed that modulating microRNAs through anti-miRs effectively regulates biological processes and provides therapeutic benefit to cardiac dysfunction, cancer and hepatitis C virus infection. Administration of anti-miR oligonucleotides is possible through local or parenteral injection. The company's lead drug candidate, RG-012, is intended as a treatment for alport syndrome in phase 2 clinical trials. Str ...
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Ionis Pharmaceuticals
Ionis Pharmaceuticals, Inc. is a biotechnology company based in Carlsbad, California, that specializes in discovering and developing RNA-targeted therapeutics. The company has 3 commercially approved medicines: Spinraza (Nusinersen), Tegsedi ( Inotersen), and Waylivra ( Volanesorsen) and has 4 drugs in pivotal studies: tominersen for Huntington’s disease, tofersen for SOD1-ALS, AKCEA-APO(a)-LRx for cardiovascular disease, and AKCEA-TTR-LRx for all forms of TTR amyloidosis. The company was named Isis Pharmaceuticals until December 2015. History The company was founded in 1989 as Isis Pharmaceuticals by Stanley T. Crooke, a former head of research of GlaxoSmithKline, with a goal to commercialize antisense therapy. In 1992, the company received its first approval by the Food and Drug Administration for an investigational new drug application in 1992 for a genital warts drug candidate. The FDA approval marked the first time for the company to conduct any antisense therapy and to ...
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Hoffmann-La Roche
F. Hoffmann-La Roche AG, commonly known as Roche, is a Swiss multinational healthcare company that operates worldwide under two divisions: Pharmaceuticals and Diagnostics. Its holding company, Roche Holding AG, has shares listed on the SIX Swiss Exchange. The company headquarters are located in Basel. Roche is the fifth largest pharmaceutical company in the world by revenue, and the leading provider of cancer treatments globally. The company controls the American biotechnology company Genentech, which is a wholly owned affiliate, and the Japanese biotechnology company Chugai Pharmaceuticals, as well as the United States-based companies Ventana and Foundation Medicine. Roche's revenues during fiscal year 2020 were 58.32 billion Swiss francs. Descendants of the founding Hoffmann and Oeri families own slightly over half of the bearer shares with voting rights (a pool of family shareholders 45%, and Maja Oeri a further 5% apart), with Swiss pharma firm Novartis owning a furth ...
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Progressive Multifocal Leukoencephalopathy
Progressive multifocal leukoencephalopathy (PML) is a rare and often fatal viral disease characterized by progressive damage (''-pathy'') or inflammation of the white matter (''leuko-'') of the brain (''-encephalo-'') at multiple locations (''multifocal''). It is caused by the JC virus, which is normally present and kept under control by the immune system. The JC virus is harmless except in cases of weakened immune systems. In general, PML has a mortality rate of 30–50% in the first few months, and those who survive can be left with varying degrees of neurological disabilities. PML occurs almost exclusively in patients with severe immune deficiency, most commonly among patients with acquired immune deficiency syndrome (AIDS), but people on chronic immunosuppressive medications including chemotherapy are also at increased risk of PML, such as patients with transplants, Hodgkin's lymphoma, multiple sclerosis, psoriasis, rheumatoid arthritis, and other autoimmune diseases. Sign ...
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Biogen Idec
Biogen Inc. is an American multinational biotechnology company based in Cambridge, Massachusetts, specializing in the discovery, development, and delivery of therapies for the treatment of neurological diseases to patients worldwide. History Biogen was founded in 1978 in Geneva as ''Biotechnology Geneva'' by several prominent biologists, including Kenneth Murray from the University of Edinburgh, Phillip Allen Sharp from the Massachusetts Institute of Technology, Walter Gilbert from Harvard University (Gilbert served as CEO during the start-up phase of Biogen), Heinz Schaller from the University of Heidelberg, and Charles Weissmann from the University of Zurich (Weissmann contributed the first product interferon alpha).Werner Grundlehner''Zürcher Antikörper gegen Alzheimer hat Milliardenpotenzial – und Gegenwind.''Neue Zürcher Zeitung, June 8, 2021. Retrieved June 8, 2021. Gilbert and Sharp were subsequently honored with Nobel Prizes: Gilbert was recognized in 1980 with th ...
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Neurodegeneration
A neurodegenerative disease is caused by the progressive loss of structure or function of neurons, in the process known as neurodegeneration. Such neuronal damage may ultimately involve cell death. Neurodegenerative diseases include amyotrophic lateral sclerosis, multiple sclerosis, Parkinson's disease, Alzheimer's disease, Huntington's disease, multiple system atrophy, and prion diseases. Neurodegeneration can be found in the brain at many different levels of neuronal circuitry, ranging from molecular to systemic. Because there is no known way to reverse the progressive degeneration of neurons, these diseases are considered to be incurable; however research has shown that the two major contributing factors to neurodegeneration are oxidative stress and inflammation. Biomedical research has revealed many similarities between these diseases at the subcellular level, including atypical protein assemblies (like proteinopathy) and induced cell death. These similarities suggest that ...
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