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Bluebird Bio
bluebird bio, Inc., based in Somerville, Massachusetts, is a biotechnology company that develops gene therapies for severe genetic disorders. The company is owned by investment funds managed by The Carlyle Group and SK Capital Partners. One of the company's approved drugs is betibeglogene autotemcel (Zynteglo), which treats transfusion-dependent beta thalassemia (TDT), a rare genetic blood disorder, and has been approved for use by the European Medicines Agency. The company has been criticized for the $1.8 million cost of the drug, which is the second most expensive drug in the world. The company is developing LentiGlobin gene therapy for the treatment of sickle cell disease and cerebral adrenoleukodystrophy. It is also developing T cell product candidates to treat acute myeloid leukemia, Merkel-cell carcinoma, diffuse large B-cell lymphoma, and MAGEA4 solid tumors. History The company was founded as Genetix Pharmaceuticals in April 1992 by MIT faculty members Philippe Lebou ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Privately-held Company
A privately held company (or simply a private company) is a company whose Stock, shares and related rights or obligations are not offered for public subscription or publicly negotiated in their respective listed markets. Instead, the Private equity, company's stock is offered, owned, traded or exchanged privately, also known as "over-the-counter (finance), over-the-counter". Related terms are unlisted organisation, unquoted company and private equity. Private companies are often less well-known than their public company, publicly traded counterparts but still have major importance in the world's economy. For example, in 2008, the 441 list of largest private non-governmental companies by revenue, largest private companies in the United States accounted for $1.8 trillion in revenues and employed 6.2 million people, according to ''Forbes''. In general, all companies that are not owned by the government are classified as private enterprises. This definition encompasses both publ ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Forbes
''Forbes'' () is an American business magazine founded by B. C. Forbes in 1917. It has been owned by the Hong Kong–based investment group Integrated Whale Media Investments since 2014. Its chairman and editor-in-chief is Steve Forbes. The company is headquartered in Jersey City, New Jersey. Sherry Phillips is the current CEO of Forbes as of January 1, 2025. Published eight times per year, ''Forbes'' feature articles on finance, industry, investing, and marketing topics. It also reports on related subjects such as technology, communications, science, politics, and law. It has an international edition in Asia as well as editions produced under license in 27 countries and regions worldwide. The magazine is known for its lists and rankings, including its lists of the richest Americans (the Forbes 400, ''Forbes'' 400), of 30 notable people under the age of 30 (the Forbes 30 Under 30, ''Forbes'' 30 under 30), of America's wealthiest celebrities, of the world's top companies (the Fo ... [...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] |
Marina Cavazzana
Marina Cavazzana is a professor of Paediatric Immunology at the Necker-Enfants Malades Hospital and the Imagine Institute, as well as an academic at Paris Descartes University. She was awarded the Irène Joliot-Curie Prize in 2012 and elected to the National Academy of Medicine in 2019. Early life and education Cavazzana was born in Venice. Her father worked on the railway and her mother was a teacher. She studied medicine in Padua, where she completed her residency at the University of Padua. She moved to Paris, where she earned her doctorate at the Paris Diderot University studying bone marrow transplants with Élaine Gluckman at the Hôpital Saint-Louis. Research and career Cavazzana joined the Necker-Enfants Malades Hospital in 1987, where she worked with Alain Fischer and Claude Gricelli. She was appointed to Professor of Immunology in 1994. She served as Director of the Institut national de la santé et de la recherche médicale (INSERM) in 1994. In 2003 she was made ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Nature (journal)
''Nature'' is a British weekly scientific journal founded and based in London, England. As a multidisciplinary publication, ''Nature'' features Peer review, peer-reviewed research from a variety of academic disciplines, mainly in science and technology. It has core editorial offices across the United States, continental Europe, and Asia under the international scientific publishing company Springer Nature. ''Nature'' was one of the world's most cited scientific journals by the Science Edition of the 2022 ''Journal Citation Reports'' (with an ascribed impact factor of 50.5), making it one of the world's most-read and most prestigious academic journals. , it claimed an online readership of about three million unique readers per month. Founded in the autumn of 1869, ''Nature'' was first circulated by Norman Lockyer and Alexander MacMillan (publisher), Alexander MacMillan as a public forum for scientific innovations. The mid-20th century facilitated an editorial expansion for the j ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Necker–Enfants Malades Hospital
The Necker–Enfants Malades Hospital ( ) is a French teaching hospital in the 15th arrondissement of Paris. It is a hospital of the Assistance Publique – Hôpitaux de Paris group and is affiliated to the Université Paris Cité. Necker–Enfants Malades Hospital was created in 1920 by the merger of Necker Hospital (), which was founded in 1778 by Suzanne Necker, with the physically contiguous Sick Children's Hospital (), the oldest children's hospital in the Western world, founded in 1801. History The ''Hôpital Necker'' was founded in 1778 by Madame Necker, born Suzanne Curchod, mother of Madame de Staël and wife of Jacques Necker, Louis XVI's finance minister. Jacques Necker was a leader in the movement to reform crowded hospitals by building smaller treatment centers closer to the patients' neighborhoods. Madame Necker subsequently remodeled an old monastery into the hospital, which prior to the French Revolution was known as the Hospice de Charité. It was a Catholic in ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
MAGEA4
Melanoma-associated antigen 4 is a protein that in humans is encoded by the ''MAGEA4'' gene. This gene is a member of the MAGEA gene family. The members of this family encode proteins with 50 to 80% sequence identity to each other. The promoters and first exons of the MAGEA genes show considerable variability, suggesting that the existence of this gene family enables the same function to be expressed under different transcriptional controls. Clinical importance The MAGEA genes are clustered at chromosomal location Xq28. They have been implicated in some hereditary disorders, such as dyskeratosis congenita. At least four variants encoding the same protein have been found for this gene. In salivary gland carcinomas, MAGE4 expression correlates to lower-grade histology, lower likelihood of metastases and more favourable survival. While MAGEA4 is expressed by many tumours, it is almost universally expressed by synovial sarcomas. A targeted treatment to use genetically modified ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Diffuse Large B-cell Lymphoma
Diffuse large B-cell lymphoma (DLBCL) is a cancer of B cells, a type of lymphocyte that is responsible for producing antibodies. It is the most common form of non-Hodgkin lymphoma among adults, with an annual incidence of 7–8 cases per 100,000 people per year in the US and UK. This cancer occurs primarily in older individuals, with a median age of diagnosis at ~70 years, although it can occur in young adults and, in rare cases, children. DLBCL can arise in virtually any part of the body and, depending on various factors, is often a very aggressive malignancy. The first sign of this illness is typically the observation of a rapidly growing mass or tissue infiltration that is sometimes associated with systemic B symptoms, e.g. fever, weight loss, and night sweats. The causes of diffuse large B-cell lymphoma are not well understood. Usually DLBCL arises from normal B cells, but it can also represent a malignant transformation of other types of lymphoma (particularly marginal zone ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Merkel-cell Carcinoma
Merkel cell carcinoma (MCC) is a rare and aggressive skin cancer occurring in about three people per million members of the population. It is also known as cutaneous APUDoma, primary neuroendocrine carcinoma of the skin, primary small cell carcinoma of the skin, and trabecular carcinoma of the skin. Factors involved in the development of MCC include the Merkel cell polyomavirus (MCPyV or MCV), a weakened immune system, and exposure to ultraviolet radiation. Merkel cell carcinoma usually arises on the head, neck, and extremities, as well as in the perianal region and on the eyelid. It is more common in people over sixty years old, Caucasian people, and males. MCC is less common in children. Signs and symptoms Merkel cell carcinoma (MCC) usually presents as a firm nodule (up to 2 cm diameter) or mass (>2 cm diameter). These flesh-colored, red, or blue tumors typically vary in size from 0.5 cm (less than one-quarter of an inch) to more than 5 cm (2 inches ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Acute Myeloid Leukemia
Acute myeloid leukemia (AML) is a cancer of the myeloid line of blood cells, characterized by the rapid growth of abnormal cells that build up in the bone marrow and blood and interfere with haematopoiesis, normal blood cell production. Symptoms may include Fatigue, feeling tired, shortness of breath, Bruise, easy bruising and bleeding, and increased risk of infection. Occasionally, spread may occur to the brain, skin, or gums. As an acute leukemia, AML progresses rapidly, and is typically fatal within weeks or months if left untreated. Risk factors include getting older, being male, smoking, previous chemotherapy or radiation therapy, myelodysplastic syndrome, and exposure to the chemical benzene. The underlying mechanism involves replacement of normal bone marrow with leukemia cells, which results in a anemia, drop in red blood cells, thrombocytopenia, platelets, and normal leukopenia, white blood cells. Diagnosis is generally based on bone marrow aspiration and specific bl ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
T Cell
T cells (also known as T lymphocytes) are an important part of the immune system and play a central role in the adaptive immune response. T cells can be distinguished from other lymphocytes by the presence of a T-cell receptor (TCR) on their cell surface receptor, cell surface. T cells are born from hematopoietic stem cells, found in the bone marrow. Developing T cells then migrate to the thymus gland to develop (or mature). T cells derive their name from the thymus. After migration to the thymus, the precursor cells mature into several distinct types of T cells. T cell differentiation also continues after they have left the thymus. Groups of specific, differentiated T cell subtypes have a variety of important functions in controlling and shaping the immune response. One of these functions is immune-mediated cell death, and it is carried out by two major subtypes: Cytotoxic T cell, CD8+ "killer" (cytotoxic) and T helper cell, CD4+ "helper" T cells. (These are named for the presen ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Adrenoleukodystrophy
Adrenoleukodystrophy (ALD) is a genetic disorder, disease linked to the X chromosome. It is a result of fatty acid buildup caused by failure of peroxisome#Metabolic functions, peroxisomal fatty acid beta oxidation which results in the accumulation of very long chain fatty acids in tissues throughout the body. The most severely affected tissues are the myelin in the central nervous system, the adrenal cortex, and the Leydig cells in the testes. The long chain fatty acid buildup causes damage to the myelin sheath of the neurons of the brain, resulting in seizures and hyperactivity. Other symptoms include problems in speaking, listening, and understanding verbal instructions. Clinically, ALD presents as a heterogeneous disorder, showing several distinct phenotypes, and no clear pattern of genotype–phenotype correlation. As an X-linked disorder, ALD presents more frequently and severely in males; however, approximately 80% of heterozygote females show some symptoms later in life. ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
