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ProQR
ProQR Therapeutics NV (NASDAQ: PRQR) is a Dutch biotechnology company based in Leiden, the Netherlands. The company was funded in 2012 by chief executive officer (CEO) Daniel de Boer. It specializes in the development of RNA therapeutics for rare genetic diseases with an ophthalmologic application. History ProQR was founded in 2012 by chief executive officer (CEO) Daniel de Boer and co-founders Henri Termeer, Dinko Valerio and Gerard Platenburg. The initial pipeline of the company was to develop small-molecule drugs or gene therapy that would treat cystic fibrosis (CF). Positive proof of concept (PoC) was achieved in 2016 with the clinical trial of the molecule QR-010 targeting the gene coding for cystic fibrosis transmembrane conductance regulator (CFTCR) in patients. Subsequently, the company expanded its pipeline into treating other rare diseases. In 2017, a spin-out of ProQR named Amylon Therapeutics was established in Leiden with the focus on the development of therapi ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Henri Termeer
Henri A. Termeer (February 28, 1946 – May 12, 2017) was a Dutch biotechnology executive and entrepreneur who is considered a pioneer in corporate strategy in the biotechnology industry for his tenure as CEO at Genzyme. Termeer created a business model adopted by many others in the biotech industry by garnering steep prices— mainly from insurers and government payers— for therapies for rare genetic disorders known as orphan diseases that mainly affect children. Genzyme uses biological processes to manufacture drugs that are not easily copied by generic-drug makers. The drugs are also protected by orphan drug acts in various countries which provides extensive protection from competition and ensures coverage by publicly funded insurers. As CEO of Genzyme from 1981 to 2011, he developed corporate strategies for growth including optimizing institutional embeddedness nurturing vast networks of influential groups and clusters: doctors, private equity, patient-groups, insurance, hea ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
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), ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Spin-out
A corporate spin-off, also known as a spin-out, or starburst or hive-off, is a type of corporate action where a company "splits off" a section as a separate business or creates a second incarnation, even if the first is still active. Characteristics Spin-offs are divisions of companies or organizations that then become independent businesses with assets, employees, intellectual property, technology, or existing products that are taken from the parent company. Shareholders of the parent company receive equivalent shares in the new company in order to compensate for the loss of equity in the original stocks. However, shareholders may then buy and sell stocks from either company independently; this potentially makes investment in the companies more attractive, as potential share purchasers can invest narrowly in the portion of the business they think will have the most growth. In contrast, divestment can also sever one business from another, but the assets are sold off rather ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Reuters
Reuters ( ) is a news agency owned by Thomson Reuters Corporation. It employs around 2,500 journalists and 600 photojournalists in about 200 locations worldwide. Reuters is one of the largest news agencies in the world. The agency was established in London in 1851 by the German-born Paul Reuter. It was acquired by the Thomson Corporation of Canada in 2008 and now makes up the media division of Thomson Reuters. History 19th century Paul Reuter worked at a book-publishing firm in Berlin and was involved in distributing radical pamphlets at the beginning of the Revolutions in 1848. These publications brought much attention to Reuter, who in 1850 developed a prototype news service in Aachen using homing pigeons and electric telegraphy from 1851 on, in order to transmit messages between Brussels and Aachen, in what today is Aachen's Reuters House. Reuter moved to London in 1851 and established a news wire agency at the London Royal Exchange. Headquartered in London, R ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Cambridge, Massachusetts
Cambridge ( ) is a city in Middlesex County, Massachusetts, United States. As part of the Greater Boston, Boston metropolitan area, the cities population of the 2020 United States Census, 2020 U.S. census was 118,403, making it the fourth most populous city in the state, behind Boston, Massachusetts, Boston, Worcester, Massachusetts, Worcester, and Springfield, Massachusetts, 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 innovati ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Nucleotide
Nucleotides are organic molecules consisting of a nucleoside and a phosphate. They serve as monomeric units of the nucleic acid polymers – deoxyribonucleic acid (DNA) and ribonucleic acid (RNA), both of which are essential biomolecules within all life-forms on Earth. Nucleotides are obtained in the diet and are also synthesized from common nutrients by the liver. Nucleotides are composed of three subunit molecules: a nucleobase, a five-carbon sugar ( ribose or deoxyribose), and a phosphate group consisting of one to three phosphates. The four nucleobases in DNA are guanine, adenine, cytosine and thymine; in RNA, uracil is used in place of thymine. Nucleotides also play a central role in metabolism at a fundamental, cellular level. They provide chemical energy—in the form of the nucleoside triphosphates, adenosine triphosphate (ATP), guanosine triphosphate (GTP), cytidine triphosphate (CTP) and uridine triphosphate (UTP)—throughout the cell for the many c ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
RNA Editing
RNA editing (also RNA modification) is a molecular process through which some cells can make discrete changes to specific nucleotide sequences within an RNA molecule after it has been generated by RNA polymerase. It occurs in all living organisms and is one of the most evolutionarily conserved properties of RNAs. RNA editing may include the insertion, deletion, and base substitution of nucleotides within the RNA molecule. RNA editing is relatively rare, with common forms of RNA processing (e.g. splicing, 5'-capping, and 3'-polyadenylation) not usually considered as editing. It can affect the activity, localization as well as stability of RNAs, and has been linked with human diseases. RNA editing has been observed in some tRNA, rRNA, mRNA, or miRNA molecules of eukaryotes and their viruses, archaea, and prokaryotes. RNA editing occurs in the cell nucleus, as well as within mitochondria and plastids. In vertebrates, editing is rare and usually consists of a small number of chang ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Usher Syndrome
Usher syndrome, also known as Hallgren syndrome, Usher–Hallgren syndrome, retinitis pigmentosa–dysacusis syndrome or dystrophia retinae dysacusis syndrome, is a rare genetic disorder caused by a mutation in any one of at least 11 genes resulting in a combination of hearing loss and visual impairment. It is a major cause of deafblindness and is at present incurable. Usher syndrome is classed into three subtypes (I, II and III) according to the genes responsible and the onset of deafness. All three subtypes are caused by mutations in genes involved in the function of the inner ear and retina. These mutations are inherited in an autosomal recessive pattern. The occurrence of Usher syndrome varies across the world and across the different syndrome types, with rates as high as 1 in 12,500 in Germany to as low as 1 in 28,000 in Norway. Type I is most common in Medical genetics of Jews, Ashkenazi Jewish and Acadian populations, and type III is rarely found outside Ashkenazi Jewish ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Retinitis Pigmentosa
Retinitis pigmentosa (RP) is a genetic disorder of the eyes that causes loss of vision. Symptoms include trouble seeing at night and decreasing peripheral vision (side and upper or lower visual field). As peripheral vision worsens, people may experience " tunnel vision". Complete blindness is uncommon. Onset of symptoms is generally gradual and often begins in childhood. Retinitis pigmentosa is generally inherited from one or both parents or rarely it can be caused by a miscoding during DNA division. It is caused by genetic miscoding of proteins in one of more than 300 genes involved. The underlying mechanism involves the progressive loss of rod photoreceptor cells that line the retina of the eyeball. The rod cells secrete a neuroprotective substance (Rod-derived cone viability factor, RdCVF) that protects the cone cells from apoptosis (cell death). However, when the rod cells die, this substance is no longer provided. This is generally followed by the loss of cone photorece ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Dystrophic Epidermolysis Bullosa
Epidermolysis bullosa dystrophica or dystrophic EB (DEB) is an inherited disease affecting the skin and other organs. "Butterfly child" is the colloquial name for a child born with the disease, as their skin is seen to be as delicate and fragile as the wings of a butterfly. Signs and symptoms The deficiency in anchoring fibrils impairs the adherence between the epidermis and the underlying dermis. The skin of DEB patients is thus highly susceptible to severe blistering. Collagen VII is also associated with the epithelium of the esophageal lining, and DEB patients may have chronic scarring, webbing, and obstruction of the esophagus. Affected individuals are often severely malnourished due to trauma to the oral and esophageal mucosa and require feeding tubes for nutrition. They also have iron-deficiency anemia of uncertain origin, which leads to chronic fatigue. Open wounds on the skin heal slowly or not at all, often scarring extensively, and are particularly susceptible to infec ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Leber's Congenital Amaurosis
Leber congenital amaurosis (LCA) is a rare inherited eye disease that appears at birth or in the first few months of life. It affects about 1 in 40,000 newborns. LCA was first described by Theodor Leber in the 19th century. It should not be confused with Leber's hereditary optic neuropathy, which is a different disease also described by Theodor Leber. One form of LCA was successfully treated with gene therapy in 2008. Signs and symptoms The term congenital refers to a condition present from birth (not acquired) and amaurosis refers to a loss of vision not associated with a lesion. However, beyond these general descriptions, the presentation of LCA can vary, because it is associated with multiple genes. LCA is typically characterized by nystagmus, sluggish or absent pupillary responses, and severe vision loss or blindness. Genetics It is usually autosomal recessive; however, importantly for family planning, it is sometimes autosomal dominant. It is a disorder thought to be ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Rare Disease
A rare disease is any disease that affects a small percentage of the population. In some parts of the world, an orphan disease is a rare disease whose rarity means there is a lack of a market large enough to gain support and resources for discovering treatments for it, except by the government granting economically advantageous conditions to creating and selling such treatments. Orphan drugs are ones so created or sold. Most rare diseases are genetic and thus are present throughout the person's entire life, even if symptoms do not immediately appear. Many rare diseases appear early in life, and about 30% of children with rare diseases will die before reaching their fifth birthdays. With only four diagnosed patients in 27 years, ribose-5-phosphate isomerase deficiency is considered the rarest known genetic disease. No single cut-off number has been agreed upon for which a disease is considered rare. A disease may be considered rare in one part of the world, or in a particular gr ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
