ES2833938T3 - Aminoglucósidos y usos de los mismos en el tratamiento de trastornos genéticos - Google Patents
Aminoglucósidos y usos de los mismos en el tratamiento de trastornos genéticos Download PDFInfo
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- ES2833938T3 ES2833938T3 ES17192028T ES17192028T ES2833938T3 ES 2833938 T3 ES2833938 T3 ES 2833938T3 ES 17192028 T ES17192028 T ES 17192028T ES 17192028 T ES17192028 T ES 17192028T ES 2833938 T3 ES2833938 T3 ES 2833938T3
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Classifications
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- C07H1/00—Processes for the preparation of sugar derivatives
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- A—HUMAN NECESSITIES
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- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/70—Carbohydrates; Sugars; Derivatives thereof
- A61K31/7028—Compounds having saccharide radicals attached to non-saccharide compounds by glycosidic linkages
- A61K31/7034—Compounds having saccharide radicals attached to non-saccharide compounds by glycosidic linkages attached to a carbocyclic compound, e.g. phloridzin
- A61K31/7036—Compounds having saccharide radicals attached to non-saccharide compounds by glycosidic linkages attached to a carbocyclic compound, e.g. phloridzin having at least one amino group directly attached to the carbocyclic ring, e.g. streptomycin, gentamycin, amikacin, validamycin, fortimicins
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
- A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
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- A—HUMAN NECESSITIES
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- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P13/00—Drugs for disorders of the urinary system
- A61P13/12—Drugs for disorders of the urinary system of the kidneys
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P21/00—Drugs for disorders of the muscular or neuromuscular system
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- A—HUMAN NECESSITIES
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- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P21/00—Drugs for disorders of the muscular or neuromuscular system
- A61P21/04—Drugs for disorders of the muscular or neuromuscular system for myasthenia gravis
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- A—HUMAN NECESSITIES
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P7/00—Drugs for disorders of the blood or the extracellular fluid
- A61P7/04—Antihaemorrhagics; Procoagulants; Haemostatic agents; Antifibrinolytic agents
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- B—PERFORMING OPERATIONS; TRANSPORTING
- B65—CONVEYING; PACKING; STORING; HANDLING THIN OR FILAMENTARY MATERIAL
- B65D—CONTAINERS FOR STORAGE OR TRANSPORT OF ARTICLES OR MATERIALS, e.g. BAGS, BARRELS, BOTTLES, BOXES, CANS, CARTONS, CRATES, DRUMS, JARS, TANKS, HOPPERS, FORWARDING CONTAINERS; ACCESSORIES, CLOSURES, OR FITTINGS THEREFOR; PACKAGING ELEMENTS; PACKAGES
- B65D79/00—Kinds or details of packages, not otherwise provided for
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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
- C07H13/00—Compounds containing saccharide radicals esterified by carbonic acid or derivatives thereof, or by organic acids, e.g. phosphonic acids
- C07H13/02—Compounds containing saccharide radicals esterified by carbonic acid or derivatives thereof, or by organic acids, e.g. phosphonic acids by carboxylic acids
- C07H13/04—Compounds containing saccharide radicals esterified by carbonic acid or derivatives thereof, or by organic acids, e.g. phosphonic acids by carboxylic acids having the esterifying carboxyl radicals attached to acyclic carbon atoms
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
- C07H15/00—Compounds containing hydrocarbon or substituted hydrocarbon radicals directly attached to hetero atoms of saccharide radicals
- C07H15/20—Carbocyclic rings
- C07H15/22—Cyclohexane rings, substituted by nitrogen atoms
- C07H15/222—Cyclohexane rings substituted by at least two nitrogen atoms
- C07H15/226—Cyclohexane rings substituted by at least two nitrogen atoms with at least two saccharide radicals directly attached to the cyclohexane rings
- C07H15/228—Cyclohexane rings substituted by at least two nitrogen atoms with at least two saccharide radicals directly attached to the cyclohexane rings attached to adjacent ring-carbon atoms of the cyclohexane rings
- C07H15/23—Cyclohexane rings substituted by at least two nitrogen atoms with at least two saccharide radicals directly attached to the cyclohexane rings attached to adjacent ring-carbon atoms of the cyclohexane rings with only two saccharide radicals in the molecule, e.g. ambutyrosin, butyrosin, xylostatin, ribostamycin
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
- C07H5/00—Compounds containing saccharide radicals in which the hetero bonds to oxygen have been replaced by the same number of hetero bonds to halogen, nitrogen, sulfur, selenium, or tellurium
- C07H5/04—Compounds containing saccharide radicals in which the hetero bonds to oxygen have been replaced by the same number of hetero bonds to halogen, nitrogen, sulfur, selenium, or tellurium to nitrogen
- C07H5/06—Aminosugars
Landscapes
- Health & Medical Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Organic Chemistry (AREA)
- Life Sciences & Earth Sciences (AREA)
- General Health & Medical Sciences (AREA)
- Engineering & Computer Science (AREA)
- Animal Behavior & Ethology (AREA)
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- Biochemistry (AREA)
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- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Epidemiology (AREA)
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- Orthopedic Medicine & Surgery (AREA)
- Physical Education & Sports Medicine (AREA)
- Mechanical Engineering (AREA)
- Urology & Nephrology (AREA)
- Diabetes (AREA)
- Hematology (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Saccharide Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
Applications Claiming Priority (1)
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| SI2640734T1 (en) * | 2010-11-18 | 2018-02-28 | Technion Research & Development Foundation Ltd. | Aminoglycosides and their uses in the treatment of genetic disorders |
| KR101703137B1 (ko) | 2014-05-30 | 2017-02-07 | 선문대학교 산학협력단 | 당 부가 아미노글리코사이드 화합물, 이의 제조방법 및 이를 유효성분으로 함유하는 약학적 조성물 |
| JP2017516835A (ja) * | 2014-06-05 | 2017-06-22 | エロックス ファーマシューティカルズ リミテッド | レット症候群治療におけるアミノグリコシド類縁体の使用 |
| EP3344641A4 (en) * | 2015-09-02 | 2019-09-18 | Eloxx Pharmaceuticals Ltd. | AMINO-LIKES DERIVATIVES AND USES THEREOF FOR THE TREATMENT OF GENETIC DISEASES |
| US11306115B2 (en) | 2015-09-02 | 2022-04-19 | Eloxx Pharmaceuticals Ltd. | Aminoglycoside derivatives and uses thereof in treating genetic disorders |
| EP3350194A4 (en) | 2015-09-02 | 2019-04-24 | Eloxx Pharmaceuticals Ltd. | AMINO-LIKES DERIVATIVES AND USES THEREOF FOR THE TREATMENT OF GENETIC DISEASES |
| WO2017118968A1 (en) * | 2016-01-05 | 2017-07-13 | Eloxx Pharmaceuticals Ltd. | Aminoglycoside derivatives and uses thereof in treating genetic disorders |
| CN117466958A (zh) * | 2017-03-15 | 2024-01-30 | Eloxx制药有限公司 | 假三糖氨基糖苷及其中间体的大规模制备 |
| WO2019237076A1 (en) * | 2018-06-07 | 2019-12-12 | Eloxx Pharmaceuticals, Inc. | Methods, compositions, and kits for inducing readthrough |
| KR102067477B1 (ko) * | 2018-06-20 | 2020-01-17 | 고려대학교 산학협력단 | 스킬로이노사민을 포함하는 아미노사이클리톨 화합물, 이의 제조방법 및 이를 함유하는 약학 조성물 |
| AU2019387385A1 (en) * | 2018-11-29 | 2021-07-08 | Eloxx Pharmaceuticals | Methods, compositions, and kits for treating ocular diseases |
| WO2021061873A1 (en) * | 2019-09-23 | 2021-04-01 | Eloxx Pharmaceuticals, Inc. | Methods, compositions, and kits for treating polycystic kidney disease |
| EP4230196A1 (en) | 2022-02-21 | 2023-08-23 | Som Innovation Biotech, S.A. | Compounds for use in the treatment of dystrophinopathies |
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| WO2005078099A1 (en) | 2004-02-17 | 2005-08-25 | The Hospital For Sick Children | Mecp2e1 gene |
| EP2390255B1 (en) * | 2006-04-03 | 2016-07-13 | Technion Research & Development Foundation Ltd. | Novel aminoglycosides and uses thereof in the treatment of genetic disorders |
| SI2640734T1 (en) * | 2010-11-18 | 2018-02-28 | Technion Research & Development Foundation Ltd. | Aminoglycosides and their uses in the treatment of genetic disorders |
| JP2017516835A (ja) * | 2014-06-05 | 2017-06-22 | エロックス ファーマシューティカルズ リミテッド | レット症候群治療におけるアミノグリコシド類縁体の使用 |
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