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Amicus Therapeutics
Amicus Therapeutics, Inc. is a public American biopharmaceutical company based in Philadelphia, PA. The company went public in 2007 under the NASDAQ trading symbol FOLD. This followed a 2006 planned offering and subsequent withdrawal, which would have established the trading symbol as AMTX Prior to their IPO, Amicus was funded by a variety of venture capital firms including Radius Ventures, Canaan Partners and New Enterprise Associates. History The therapeutic focus of Amicus is on rare and orphan diseases, particularly disorders collectively called lysosomal storage disorders. The company has focused on pharmacological chaperones and enzyme replacement therapy. In 2008, the company expanded from its single site in New Jersey to a second research site in San Diego. In late 2009, the company faced a major financial setback with the termination of a multi-year collaboration agreement with Shire for its lead compound, migalastat, and two other products. Amicus cut 20% of it ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Public Company
A public company is a company whose ownership is organized via shares of share capital, stock which are intended to be freely traded on a stock exchange or in over-the-counter (finance), over-the-counter markets. A public (publicly traded) company can be listed on a stock exchange (listing (finance), 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 so 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 Kin ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Canaan Partners
Canaan Partners, LLC (Canaan) is an American venture capital firm headquartered in Stamford, Connecticut with offices in San Francisco, Menlo Park and New York City. The firm focuses on investing in early stage companies in the technology and healthcare industries. Since 2014, the firm has also been investing in technology companies based in Israel. Background In 1987, Harry Rein, Eric Young and several other employees at the venture capital unit of General Electric performed a management buyout A management buyout (MBO) is a form of acquisition in which a company's existing managers acquire a large part, or all, of the company, whether from a parent company or individual. Management- and/or leveraged buyouts became noted phenomena of 19 ... to spin out the unit as an independent firm. The firm was subsequently named Canaan Partners and would be based in Westport, Connecticut. In 2003, Rein left Canaan for a position with Foundation Medical Partners. Towards the end of 2 ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
GlaxoSmithKline
GSK plc (an acronym from its former name GlaxoSmithKline plc) is a British Multinational corporation, multinational Pharmaceutics, pharmaceutical and biotechnology company with headquarters in London. It was established in 2000 by a Mergers and acquisitions, merger of Glaxo Wellcome and SmithKline Beecham, which was itself a merger of a number of pharmaceutical companies around the Smith, Kline & French firm. GSK is the tenth largest pharmaceutical company and No. 294 on the 2022 Fortune Global 500, ''Fortune'' Global 500, ranked behind other pharmaceutical companies China Resources, Sinopharm (company), Sinopharm, Johnson & Johnson, Pfizer, Roche, AbbVie, Novartis, Bayer, and Merck & Co., Merck Sharp & Dohme. The company has a primary listing on the London Stock Exchange and is a constituent of the FTSE 100 Index. As of February 2024, it had a Market capitalization, market capitalisation of £69 billion, the eighth largest on the London Stock Exchange. The company developed ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Migalastat
Migalastat, sold under the brand name Galafold, is a medication for the treatment of Fabry disease, a rare genetic disorder. It was developed by Amicus Therapeutics. The US Food and Drug Administration (FDA) granted it orphan drug status in 2004, and the European Commission followed in 2006. The European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) granted the drug a marketing approval under the name Galafold in May 2016. The U.S. Food and Drug Administration (FDA) considers it to be a first-in-class medication. Medical uses Migalastat is used for the long-term treatment of Fabry disease in adults and adolescents aged 16 or older with an amenable mutation of the enzyme alpha-galactosidase A (α-GalA). An "amenable" mutation is one that leads to Protein folding, misfolding of the enzyme, but otherwise would not significantly impair its function. Based on an ''in vitro'' test, Amicus Therapeutics has published a list of 269 amenable and nearly 600 non- ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Shire (pharmaceutical Company)
Shire plc was a UK-founded Jersey-registered specialty biopharmaceutical company. Originating in the United Kingdom with an operational base in the United States, its brands and products included Vyvanse, Lialda, and Adderall XR. Shire was acquired by Takeda Pharmaceutical Company on 8 January 2019. Shire was a global biotechnology company focused on serving people with rare diseases and other highly specialized conditions. The company's products were available in more than 100 countries across core therapeutic areas including Hematology, Immunology, Neuroscience, Lysosomal Storage Disorders, Gastrointestinal / Internal Medicine / Endocrine and Hereditary Angioedema; a growing franchise in Oncology; and an emerging, innovative pipeline in Ophthalmics. The original corporate headquarters was located in Basingstoke, Hampshire, England. Main offices are located in Dublin, Ireland, the United States in Cambridge, Massachusetts, and Chicago, Illinois, and in Zug, Switzerl ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Biotech Week
''Biotech Week'' is a weekly biotechnology and pharmaceutical trade magazine. The magazine was established in 1999. It is published by NewsRX. The magazine covers a broad range of medical issues and financial related information about companies in these industries. The magazine is headquartered in Atlanta, Georgia Atlanta ( ) is the List of capitals in the United States, capital and List of municipalities in Georgia (U.S. state), most populous city in the U.S. state of Georgia (U.S. state), Georgia. It is the county seat, seat of Fulton County, Georg .... References External links * * 1999 establishments in Georgia (U.S. state) English-language magazines Magazines established in 1999 Magazines published in Atlanta Medical magazines Professional and trade magazines Science and technology magazines published in the United States Weekly magazines published in the United States {{trade-mag-stub ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
San Diego
San Diego ( , ) is a city on the Pacific coast of Southern California, adjacent to the Mexico–United States border. With a population of over 1.4 million, it is the List of United States cities by population, eighth-most populous city in the United States. San Diego is the county seat, seat of San Diego County. It is known for its mild Mediterranean climate, extensive List of beaches in San Diego County, beaches and List of parks in San Diego, parks, long association with the United States Navy, and recent emergence as a wireless, electronics, List of hospitals in San Diego, healthcare, and biotechnology development center. Historically home to the Kumeyaay people, San Diego has been referred to as the ''Birthplace of California'', as it was the first site visited and settled by Europeans on what is now the West Coast of the United States. In 1542, Juan Rodríguez Cabrillo claimed the area for Spain, forming the basis for the settlement of Alta California, 200 years later. ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Royal Society Of Chemistry
The Royal Society of Chemistry (RSC) is a learned society and professional association in the United Kingdom with the goal of "advancing the chemistry, chemical sciences". It was formed in 1980 from the amalgamation of the Chemical Society, the Royal Institute of Chemistry, the Faraday Society, and the Society for Analytical Chemistry with a new Royal Charter and the dual role of learned society and professional body. At its inception, the Society had a combined membership of 49,000 in the world. The headquarters of the Society are at Burlington House, Piccadilly, London. It also has offices in Thomas Graham House in Cambridge (named after Thomas Graham (chemist), Thomas Graham, the first president of the Chemical Society) where ''RSC Publishing'' is based. The Society has offices in the United States, on the campuses of The University of Pennsylvania and Drexel University, at the University City Science Center in Philadelphia, Pennsylvania, in both Beijing and Shanghai, People' ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Enzyme Replacement Therapy
Enzyme replacement therapy (ERT) is a medical treatment which replaces an enzyme that is deficient or absent in the body. Usually, this is done by giving the patient an intravenous (IV) infusion of a solution containing the enzyme. ERT is available for some lysosomal storage diseases: Gaucher disease, Fabry disease, MPS I, MPS II (Hunter syndrome), MPS VI and Pompe disease. ERT does not correct the underlying genetic defect, but it increases the concentration of the enzyme that the patient is lacking. ERT has also been used to treat patients with severe combined immunodeficiency (SCID) resulting from an adenosine deaminase deficiency ( ADA-SCID). Other treatment options for patients with enzyme or protein deficiencies include substrate reduction therapy, gene therapy, and bone-marrow derived stem cell transplantation. History ERT was developed in 1964 by Christian de Duve and Roscoe Brady. Leading work was done on this subject at the Department of Physiology at the Un ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Pharmacological Chaperone
A pharmacological chaperone or pharmacoperone is a drug that acts as a chaperone (protein), protein chaperone. That is, it contains small molecules that enter cells and serve as a molecular scaffolding in order to cause otherwise-protein folding, misfolded mutant proteins to fold and route correctly within the cell. Mutation of proteins often causes molecular misfolding, which results in protein misrouting within the cell. Accordingly, mutant molecules may retain proper function but end up in parts of the cell where the function is inappropriate, or even deleterious, to cell function. Misfolded proteins are usually recognized by the quality-control system of the cell and retained (and often destroyed or recycled) in the endoplasmic reticulum. Pharmacoperones correct the folding of misfolded proteins, allowing them to pass through the cell's quality-control system and become correctly routed. Since mutations often cause disease by causing misfolding and misrouting, pharmacoperones ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
USSEC
The United States Securities and Exchange Commission (SEC) is an independent agency of the United States federal government, created in the aftermath of the Wall Street crash of 1929. Its primary purpose is to enforce laws against market manipulation. Created by Section 4 of the Securities Exchange Act of 1934 (now codified as and commonly referred to as the Exchange Act or the 1934 Act), the SEC enforces the Securities Act of 1933, the Trust Indenture Act of 1939, the Investment Company Act of 1940, the Investment Advisers Act of 1940, and the Sarbanes–Oxley Act of 2002, among other statutes. Overview The SEC has a three-part mission: to protect investors; maintain fair, orderly, and efficient markets; and facilitate capital formation. To achieve its mandate, the SEC enforces the statutory requirement that public companies and other regulated entities submit quarterly and annual reports, as well as other periodic disclosures. In addition to annual financial reports, ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Lysosomal Storage Disease
Lysosomal storage diseases (LSDs; ) are a group of over 70 rare inherited metabolic disorders that result from defects in lysosomal function. Lysosomes are sacs of enzymes within cells that digest large molecules and pass the fragments on to other parts of the cell for recycling. This process requires several critical enzymes. If one of these enzymes is defective due to a mutation, the large molecules accumulate within the cell, eventually killing it. Lysosomal storage disorders are caused by lysosomal dysfunction usually as a consequence of deficiency of a single enzyme required for the metabolism of lipids, glycoproteins (sugar-containing proteins), or mucopolysaccharides. Individually, lysosomal storage diseases occur with incidences of less than 1:100,000; however, as a group, the incidence is about 1:5,000 – 1:10,000. Most of these disorders are autosomal recessively inherited such as Niemann–Pick disease, type C, but a few are X-linked recessively inherited, such as Fa ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
