Brensocatib
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Brensocatib
Brensocatib is an investigational new drug that is being evaluated to treat bronchiectasis. It is a dipeptidyl-peptidase I (also known as cathepsin C Cathepsin C (CTSC) also known as dipeptidyl peptidase I (DPP-I) is a lysosomal exo-cysteine protease belonging to the peptidase C1 protein family, a subgroup of the cysteine cathepsins. In humans, it is encoded by the ''CTSC'' gene. Function ...) inhibitor. A phase 3 clinical trial, known as the ASPEN trial, was conducted to evaluate the safety and efficacy of brensocatib in patients with non-cystic fibrosis bronchiectasis. References Anti-inflammatory agents Carboxamides Benzoxazoles Nitriles Oxazepines {{musculoskeletal-drug-stub ...
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Investigational New Drug
The United States Food and Drug Administration's Investigational New Drug (IND) program is the means by which a pharmaceutical industry, pharmaceutical company obtains permission to start human clinical trials and to ship an experimental drug interstate commerce, across state lines (usually to clinical investigators) before a marketing application for the drug has been approved. Regulations are primarily at . Similar procedures are followed in the European Union, Japan, and Canada due to regulatory harmonization efforts by the International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use, International Council for Harmonisation. Types * Research or investigator INDs are non-commercial INDs filed by researchers to study an unapproved drug or to study an approved drug for a new indication or in a new patient population. * Expanded access, Emergency Use INDs, also called compassionate use or single-patient INDs, are filed for emergency use of a ...
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Bronchiectasis
Bronchiectasis is a disease in which there is permanent enlargement of parts of the bronchi, airways of the lung. Symptoms typically include a chronic cough with sputum, mucus production. Other symptoms include shortness of breath, hemoptysis, coughing up blood, and chest pain. Wheezing and nail clubbing may also occur. Those with the disease often get lung infections. Bronchiectasis may result from a number of infection, infectious and acquired causes, including measles, pneumonia, tuberculosis, immune system problems, as well as the genetic disorder cystic fibrosis. Cystic fibrosis eventually results in severe bronchiectasis in nearly all cases. The cause in 10–50% of those without cystic fibrosis is unknown. The mechanism of disease is breakdown of the airways due to an excessive inflammatory response. Involved airways (bronchi) become enlarged and thus less able to clear secretions. These secretions increase the amount of bacteria in the lungs, resulting in airway blockage ...
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Dipeptidyl-peptidase I
Dipeptidyl peptidase I (, ''cathepsin C'', ''dipeptidyl aminopeptidase I'', ''dipeptidyl transferase'', ''dipeptide arylamidase I'', ''DAP I'') is an enzyme. This enzyme catalyses the following chemical reaction : Release of an N-terminal dipeptide, Xaa-Yaa!Zaa-, except when Xaa is Arg or Lys, or Yaa or Zaa is Pro This Cl-dependent, lysosomal cysteine-type peptidase is maximally active at acidic An acid is a molecule or ion capable of either donating a proton (i.e. hydrogen cation, H+), known as a Brønsted–Lowry acid, or forming a covalent bond with an electron pair, known as a Lewis acid. The first category of acids are the ... pH. References External links * EC 3.4.14 {{Enzyme-stub ...
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Cathepsin C
Cathepsin C (CTSC) also known as dipeptidyl peptidase I (DPP-I) is a lysosomal exo-cysteine protease belonging to the peptidase C1 protein family, a subgroup of the cysteine cathepsins. In humans, it is encoded by the ''CTSC'' gene. Function Cathepsin C appears to be a central coordinator for activation of many serine proteases in immune/inflammatory cells. Cathepsin C catalyses excision of dipeptides from the N-terminus of protein and peptide substrates, except if (i) the amino group of the N-terminus is blocked, (ii) the site of cleavage is on either side of a proline residue, (iii) the N-terminal residue is lysine or arginine, or (iv) the structure of the peptide or protein prevents further digestion from the N-terminus. Structure The cDNAs encoding rat, human, murine, bovine, dog and two ''Schistosome'' cathepsin Cs have been cloned and sequenced and show that the enzyme is highly conserved. The human and rat cathepsin C cDNAs encode precursors (prepro-cathepsin C) c ...
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Anti-inflammatory Agents
Anti-inflammatory is the property of a substance or treatment that reduces inflammation, fever or swelling. Anti-inflammatory drugs, also called anti-inflammatories, make up about half of analgesics. These drugs reduce pain by inhibiting mechanisms of inflammation, as opposed to opioids, which affect the central nervous system to block pain. Common anti-inflammatory drugs include nonsteroidal anti-inflammatory drugs (NSAIDs), corticosteroids, antileukotrienes, and monoclonal antibodies. Drugs Clinically approved Nonsteroidal anti-inflammatory drugs NSAIDs alleviate pain by counteracting the cyclooxygenase (COX) enzyme involved in pain mechanisms. Some common examples of NSAIDs are aspirin, ibuprofen, and naproxen. Selective COX-2 inhibitors, such as celecoxib, block the enzymatic conversion of arachidonic acid into prostaglandin, inhibiting inflammation and pain. Analgesics commonly associated with anti-inflammatory drugs, such as acetaminophen (paracetamol), have no periphe ...
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Benzoxazoles
Benzoxazole is an aromatic organic compound with a molecular formula C7H5NO, a benzene-fused oxazole ring structure, and an odor similar to pyridine. Although benzoxazole itself is of little practical value, many derivatives of benzoxazoles are commercially important. Being a heterocyclic compound, benzoxazole finds use in research as a starting material for the synthesis of larger, usually bioactive structures. Its aromaticity makes it relatively stable, although as a heterocycle, it has reactive sites which allow for functionalization. Occurrence and applications It is found within the chemical structures of pharmaceutical drugs such as flunoxaprofen and tafamidis. Benzoxazole derivatives are also of interest for optical brighteners in laundry detergents.E. Smulders, E. Sung "Laundry Detergents, 2. Ingredients and Products" in ''Ullmann's Encyclopedia of Industrial Chemistry'', Wiley-VCH, Weinheim, 2012. Benzoxazoles belong to the group of well-known antifungal agents with an ...
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Nitriles
In organic chemistry, a nitrile is any organic compound that has a functional group. The name of the compound is composed of a base, which includes the carbon of the , suffixed with "nitrile", so for example is called "propionitrile" (or propanenitrile). The prefix ''cyano-'' is used interchangeably with the term ''nitrile'' in industrial literature. Nitriles are found in many useful compounds, including methyl cyanoacrylate, used in super glue, and nitrile rubber, a nitrile-containing polymer used in latex, latex-free laboratory and medical gloves. Nitrile rubber is also widely used as automotive and other seals since it is resistant to fuels and oils. Organic compounds containing multiple nitrile groups are known as cyanocarbons. Inorganic compounds containing the group are not called nitriles, but cyanides instead. Though both nitriles and cyanides can be derived from cyanide salts, most nitriles are not nearly as toxic. Structure and basic properties The N−C−C geome ...
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