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Alnylam
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, 200 ...
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John Maraganore
John Maraganore is an American scientist, entrepreneur, and life sciences industry leader. Education John was born in Chicago, Illinois, U.S. in 1962 to Greek immigrant parents. In 1984, he completed his B.A. from University of Chicago in Division of Biological Sciences. In 1985, he completed his M.S. from the University of Chicago in Department of Biochemistry and Molecular Biology. In 1986, Maraganore received his Ph.D. from the University of Chicago in Department of Biochemistry and Molecular Biology. Career Maraganore started his career as a post-doctoral research scientist at Upjohn in Kalamazoo, Michigan, from 1985-86. From 1986-87, he was a senior scientist at Zymogenetics in Seattle, Washington. From 1987-1997, he was a senior scientist, group leader of thrombosis and hemostasis research, director of biological research, director of market and business development, and program executive at Biogen in Cambridge, Massachusetts. At Biogen, he invented bivalirudin, a di ...
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RNA Interference
RNA interference (RNAi) is a biological process in which RNA molecules are involved in sequence-specific suppression of gene expression by double-stranded RNA, through translational or transcriptional repression. Historically, RNAi was known by other names, including ''co-suppression'', ''post-transcriptional gene silencing'' (PTGS), and ''quelling''. The detailed study of each of these seemingly different processes elucidated that the identity of these phenomena were all actually RNAi. Andrew Fire and Craig C. Mello shared the 2006 Nobel Prize in Physiology or Medicine for their work on RNAi in the nematode worm '' Caenorhabditis elegans'', which they published in 1998. Since the discovery of RNAi and its regulatory potentials, it has become evident that RNAi has immense potential in suppression of desired genes. RNAi is now known as precise, efficient, stable and better than antisense therapy for gene suppression. Antisense RNA produced intracellularly by an expression vector m ...
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Yvonne Greenstreet
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Christoph Westphal
Christoph Westphal, M.D., Ph.D., is a biomedical entrepreneur. Background and training Westphal graduated from Columbia College of Columbia University, summa cum laude, in 1990 and finished the MD–PhD program at Harvard University in six years. Career He worked at McKinsey & Company for two years after getting his degrees. From 2000 to 2005 he was a partner at Polaris Venture Partners, a venture capital firm. VC and Serial Entrepreneur In 2001, he worked with Robert Langer to found Mimeon based on work by Langer on glycoengineering; the company changed its name to Momenta Pharmaceuticals the next year and went public in 2004. Westphal was the founding CEO. Momenta brought to market the first generic, low-molecular-weight heparin. To expand the portfolio of treatments for autoimmune disorders, Johnson & Johnson acquired Momenta Pharmaceuticals for $6.5 billion in an all-cash deal in 2020, after nipocalimab, a treatment developed by Momenta received a rare paediatric ...
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Phillip Sharp
Phillip Allen Sharp (born June 6, 1944) is an American geneticist and molecular biologist who co-discovered RNA splicing. He shared the 1993 Nobel Prize in Physiology or Medicine with Richard J. Roberts for "the discovery that genes in eukaryotes are not contiguous strings but contain introns, and that the splicing of messenger RNA to delete those introns can occur in different ways, yielding different proteins from the same DNA sequence". He has been selected to receive the 2015 Othmer Gold Medal. Research Sharp's current research focuses on small RNAs and other types of non-coding RNAs. His laboratory works to identify the target mRNAs of microRNAs (miRNAs), and has discovered a class of miRNAs that are produced from sequences adjacent to transcription start sites. His laboratory also studies how miRNA gene regulation functions in angiogenesis and cellular stress. Biography Sharp was born in Falmouth, Kentucky, the son of Kathrin (Colvin) and Joseph Walter Sharp. He married ...
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Cambridge, Massachusetts
Cambridge ( ) is a city in Middlesex County, Massachusetts, United States. As part of the Boston metropolitan area, the cities population of the 2020 U.S. census was 118,403, making it the fourth most populous city in the state, behind Boston, Worcester, and Springfield. It is one of two de jure county seats of Middlesex County, although the county's executive government was abolished in 1997. Situated directly north of Boston, across the Charles River, it was named in honor of the University of Cambridge in England, once also an important center of the Puritan theology embraced by the town's founders. Harvard University, the Massachusetts Institute of Technology (MIT), Lesley University, and Hult International Business School are in Cambridge, as was Radcliffe College before it merged with Harvard. Kendall Square in Cambridge has been called "the most innovative square mile on the planet" owing to the high concentration of successful startups that have emerged in the vicinity ...
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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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David Bartel
David P. Bartel is an American molecular biologist best known for his work on microRNAs. Bartel is a Professor of Biology at the Massachusetts Institute of Technology, Member of the Whitehead Institute, and investigator of the Howard Hughes Medical Institute (HHMI). Biography Bartel earned his B.A. degree in Biology from Goshen College in 1982 and his Ph.D. degree in Virology from Harvard University in 1993, under the mentorship of Jack W. Szostak Jack William Szostak (born November 9, 1952) is a Canadian American biologist of Polish British descent, Nobel Prize laureate, university professor at the University of Chicago, former Professor of Genetics at Harvard Medical School, and Alexan .... While in the Szostak lab, Bartel isolated the first ribozymes directly from random sequence, using ''in vitro'' evolution (among these, the Class I ligase). After he became independent at the Whitehead Institute, he further evolved this ribozyme to function as a RNA-dependent RNA poly ...
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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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Russell 1000
The Russell 1000 Index is a stock market index that tracks the highest-ranking 1,000 stocks in the Russell 3000 Index, which represent about 93% of the total market capitalization of that index. , the stocks of the Russell 1000 Index had a weighted average market capitalization of $608.1 billion and a median market capitalization of $15.1 billion. , components ranged in market capitalization from $1.8 billion to $1.4 trillion. The index, which was launched on January 1, 1984, is maintained by FTSE Russell, a subsidiary of the London Stock Exchange Group. The ticker symbol is ^RUI. There are several exchange-traded funds and mutual funds that track the index. Record values Annual returns Top sectors by weight *Technology *Consumer Discretionary * Health Care *Industrials *Financial services Top 10 holdings *Apple () *Microsoft () *Amazon () *Alphabet (Class A) () * Tesla () *Alphabet (Class C) () * Meta () *Nvidia () *Berkshire Hathaway () *UnitedHealth Group () (as of Dece ...
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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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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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