SI2640734T1 - Aminoglycosides and their uses in the treatment of genetic disorders - Google Patents
Aminoglycosides and their uses in the treatment of genetic disorders Download PDFInfo
- Publication number
- SI2640734T1 SI2640734T1 SI201131387T SI201131387T SI2640734T1 SI 2640734 T1 SI2640734 T1 SI 2640734T1 SI 201131387 T SI201131387 T SI 201131387T SI 201131387 T SI201131387 T SI 201131387T SI 2640734 T1 SI2640734 T1 SI 2640734T1
- Authority
- SI
- Slovenia
- Prior art keywords
- compound
- hydrogen
- protecting group
- alkyl
- compound according
- Prior art date
Links
- 208000026350 Inborn Genetic disease Diseases 0.000 title claims abstract 7
- 208000016361 genetic disease Diseases 0.000 title claims abstract 7
- 229940126575 aminoglycoside Drugs 0.000 title claims abstract 5
- 125000000217 alkyl group Chemical group 0.000 claims abstract 9
- 239000008194 pharmaceutical composition Substances 0.000 claims abstract 3
- 238000000034 method Methods 0.000 claims abstract 2
- 230000035772 mutation Effects 0.000 claims abstract 2
- 150000003839 salts Chemical class 0.000 claims abstract 2
- 150000001875 compounds Chemical class 0.000 claims 18
- 229910052739 hydrogen Inorganic materials 0.000 claims 9
- 239000001257 hydrogen Substances 0.000 claims 9
- 125000004435 hydrogen atom Chemical group [H]* 0.000 claims 5
- UFHFLCQGNIYNRP-UHFFFAOYSA-N Hydrogen Chemical compound [H][H] UFHFLCQGNIYNRP-UHFFFAOYSA-N 0.000 claims 4
- 125000004432 carbon atom Chemical group C* 0.000 claims 4
- 241000894006 Bacteria Species 0.000 claims 2
- 201000003883 Cystic fibrosis Diseases 0.000 claims 2
- 206010013801 Duchenne Muscular Dystrophy Diseases 0.000 claims 2
- 239000000463 material Substances 0.000 claims 2
- 239000000203 mixture Substances 0.000 claims 2
- 125000002924 primary amino group Chemical group [H]N([H])* 0.000 claims 2
- 125000006239 protecting group Chemical group 0.000 claims 2
- 230000009752 translational inhibition Effects 0.000 claims 2
- 206010003594 Ataxia telangiectasia Diseases 0.000 claims 1
- 241000206602 Eukaryota Species 0.000 claims 1
- 201000003542 Factor VIII deficiency Diseases 0.000 claims 1
- 241000192125 Firmicutes Species 0.000 claims 1
- 208000015178 Hurler syndrome Diseases 0.000 claims 1
- 206010056886 Mucopolysaccharidosis I Diseases 0.000 claims 1
- 108020004485 Nonsense Codon Proteins 0.000 claims 1
- 208000014769 Usher Syndromes Diseases 0.000 claims 1
- 230000008878 coupling Effects 0.000 claims 1
- 238000010168 coupling process Methods 0.000 claims 1
- 238000005859 coupling reaction Methods 0.000 claims 1
- 239000003937 drug carrier Substances 0.000 claims 1
- 208000009429 hemophilia B Diseases 0.000 claims 1
- 125000002887 hydroxy group Chemical group [H]O* 0.000 claims 1
- 238000004519 manufacturing process Methods 0.000 claims 1
- 238000012856 packing Methods 0.000 claims 1
- 108090000623 proteins and genes Proteins 0.000 claims 1
- 102000004169 proteins and genes Human genes 0.000 claims 1
- 208000011580 syndromic disease Diseases 0.000 claims 1
- 108020004705 Codon Proteins 0.000 abstract 1
- 125000003118 aryl group Chemical group 0.000 abstract 1
- 125000000753 cycloalkyl group Chemical group 0.000 abstract 1
- 230000003013 cytotoxicity Effects 0.000 abstract 1
- 231100000135 cytotoxicity Toxicity 0.000 abstract 1
- 230000000694 effects Effects 0.000 abstract 1
- 230000001747 exhibiting effect Effects 0.000 abstract 1
Classifications
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- 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
-
- 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
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- 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
-
- 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
-
- 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
- A61P21/04—Drugs for disorders of the muscular or neuromuscular system for myasthenia gravis
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
-
- 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
-
- 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
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
- C07H1/00—Processes for the preparation of sugar derivatives
-
- 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)
- Medicinal Chemistry (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Molecular Biology (AREA)
- Biotechnology (AREA)
- Genetics & Genomics (AREA)
- Biochemistry (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Epidemiology (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Physical Education & Sports Medicine (AREA)
- Neurology (AREA)
- Mechanical Engineering (AREA)
- Diabetes (AREA)
- Hematology (AREA)
- Urology & Nephrology (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 (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US41495610P | 2010-11-18 | 2010-11-18 | |
| PCT/IL2011/000889 WO2012066546A1 (en) | 2010-11-18 | 2011-11-17 | Aminoglycosides and uses thereof in treating genetic disorders |
| EP11799501.9A EP2640734B1 (en) | 2010-11-18 | 2011-11-17 | Aminoglycosides and uses thereof in treating genetic disorders |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| SI2640734T1 true SI2640734T1 (en) | 2018-02-28 |
Family
ID=45390144
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| SI201131387T SI2640734T1 (en) | 2010-11-18 | 2011-11-17 | Aminoglycosides and their uses in the treatment of genetic disorders |
Country Status (18)
| Country | Link |
|---|---|
| US (7) | US8895519B2 (enExample) |
| EP (2) | EP2640734B1 (enExample) |
| JP (3) | JP5960712B2 (enExample) |
| CA (1) | CA2816789C (enExample) |
| CY (1) | CY1120082T1 (enExample) |
| DK (1) | DK2640734T3 (enExample) |
| ES (2) | ES2833938T3 (enExample) |
| HR (1) | HRP20180056T1 (enExample) |
| HU (1) | HUE036248T2 (enExample) |
| IL (3) | IL266495B (enExample) |
| LT (1) | LT2640734T (enExample) |
| NO (1) | NO2640734T3 (enExample) |
| PL (1) | PL2640734T3 (enExample) |
| PT (1) | PT2640734T (enExample) |
| RS (1) | RS56739B1 (enExample) |
| SI (1) | SI2640734T1 (enExample) |
| SM (1) | SMT201800019T1 (enExample) |
| WO (1) | WO2012066546A1 (enExample) |
Families Citing this family (13)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| RS56739B1 (sr) | 2010-11-18 | 2018-03-30 | Technion Res & Dev Foundation | Aminoglikozidi i njihova upotreba u tretiranju genetskih poremećaja |
| KR101703137B1 (ko) | 2014-05-30 | 2017-02-07 | 선문대학교 산학협력단 | 당 부가 아미노글리코사이드 화합물, 이의 제조방법 및 이를 유효성분으로 함유하는 약학적 조성물 |
| EP3151840A1 (en) | 2014-06-05 | 2017-04-12 | Eloxx Pharmaceuticals Ltd. | Use of aminoglycoside analogs in the treatment of rett syndrome |
| US10786520B2 (en) | 2015-09-02 | 2020-09-29 | Eloxx Pharmaceuticals Ltd. | Aminoglycoside derivatives and uses thereof in treating genetic disorders |
| EP3344640A4 (en) * | 2015-09-02 | 2019-05-01 | Eloxx Pharmaceuticals Ltd. | AMINO-LIKES DERIVATIVES AND USES THEREOF FOR THE TREATMENT OF GENETIC DISEASES |
| CN114957357A (zh) | 2015-09-02 | 2022-08-30 | Eloxx制药有限公司 | 氨基糖苷衍生物及其在治疗遗传性病症中的应用 |
| US20190016746A1 (en) * | 2016-01-05 | 2019-01-17 | Eloxx Pharmaceuticals Ltd. | Aminoglycoside derivatives and uses thereof in treating genetic disorders |
| BR112019018834A2 (pt) * | 2017-03-15 | 2020-04-14 | Eloxx Pharmaceuticals Ltd | preparação em grande escala de aminoglicosídeos de pseudotrissacarídeos e de seus intermediários |
| 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 |
| US20220008445A1 (en) * | 2018-11-29 | 2022-01-13 | 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 |
Family Cites Families (5)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JPS5960712A (ja) | 1982-09-30 | 1984-04-06 | Toshiba Corp | デ−タ判別装置 |
| CA2554380C (en) | 2004-02-17 | 2015-05-19 | The Hospital For Sick Children | Mecp2e1 gene |
| HUE031643T2 (en) * | 2006-04-03 | 2017-07-28 | Technion Res & Dev Foundation | New aminoglycosides and their applications in the treatment of genetic disorders |
| RS56739B1 (sr) | 2010-11-18 | 2018-03-30 | Technion Res & Dev Foundation | Aminoglikozidi i njihova upotreba u tretiranju genetskih poremećaja |
| EP3151840A1 (en) * | 2014-06-05 | 2017-04-12 | Eloxx Pharmaceuticals Ltd. | Use of aminoglycoside analogs in the treatment of rett syndrome |
-
2011
- 2011-11-17 RS RS20180029A patent/RS56739B1/sr unknown
- 2011-11-17 NO NO11799501A patent/NO2640734T3/no unknown
- 2011-11-17 HR HRP20180056TT patent/HRP20180056T1/hr unknown
- 2011-11-17 LT LTEP11799501.9T patent/LT2640734T/lt unknown
- 2011-11-17 JP JP2013539397A patent/JP5960712B2/ja active Active
- 2011-11-17 HU HUE11799501A patent/HUE036248T2/hu unknown
- 2011-11-17 WO PCT/IL2011/000889 patent/WO2012066546A1/en not_active Ceased
- 2011-11-17 SM SM20180019T patent/SMT201800019T1/it unknown
- 2011-11-17 EP EP11799501.9A patent/EP2640734B1/en active Active
- 2011-11-17 ES ES17192028T patent/ES2833938T3/es active Active
- 2011-11-17 PT PT117995019T patent/PT2640734T/pt unknown
- 2011-11-17 SI SI201131387T patent/SI2640734T1/en unknown
- 2011-11-17 DK DK11799501.9T patent/DK2640734T3/en active
- 2011-11-17 CA CA2816789A patent/CA2816789C/en active Active
- 2011-11-17 PL PL11799501T patent/PL2640734T3/pl unknown
- 2011-11-17 US US13/885,715 patent/US8895519B2/en active Active
- 2011-11-17 EP EP17192028.3A patent/EP3296311B1/en active Active
- 2011-11-17 ES ES11799501.9T patent/ES2655916T3/es active Active
-
2014
- 2014-08-18 US US14/461,477 patent/US9175029B2/en active Active
-
2015
- 2015-09-27 US US14/866,960 patent/US9616079B2/en active Active
-
2016
- 2016-01-22 JP JP2016011038A patent/JP2016121168A/ja active Pending
-
2017
- 2017-03-09 US US15/453,990 patent/US9943533B2/en active Active
-
2018
- 2018-01-15 CY CY20181100039T patent/CY1120082T1/el unknown
- 2018-03-01 JP JP2018036281A patent/JP6758338B2/ja active Active
- 2018-03-07 US US15/914,045 patent/US10398718B2/en active Active
-
2019
- 2019-05-07 IL IL266495A patent/IL266495B/en active IP Right Grant
- 2019-08-23 US US16/550,121 patent/US10973839B2/en active Active
-
2020
- 2020-09-01 IL IL277063A patent/IL277063B/en unknown
-
2021
- 2021-04-08 US US17/226,050 patent/US11865128B2/en active Active
- 2021-06-16 IL IL284067A patent/IL284067B/en unknown
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