SI2640734T1 - Aminoglikozidi in njihove uporabe v zdravljenju genetskih motenj - Google Patents
Aminoglikozidi in njihove uporabe v zdravljenju genetskih motenj Download PDFInfo
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- 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
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- 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
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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
- 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
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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
- 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
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- 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
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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
- C07H1/00—Processes for the preparation of sugar derivatives
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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
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- 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
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- 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
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- 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)
- Veterinary Medicine (AREA)
- Medicinal Chemistry (AREA)
- Public Health (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Molecular Biology (AREA)
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- Genetics & Genomics (AREA)
- Biotechnology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Epidemiology (AREA)
- Neurology (AREA)
- Physical Education & Sports Medicine (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Mechanical Engineering (AREA)
- Urology & Nephrology (AREA)
- Hematology (AREA)
- Diabetes (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Saccharide Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
Claims (15)
- EP 2 640 734 BI Aminoglikozidi in njihove uporabe v zdravljenju genetskih motenj Patentni zahtevki1. Spojina, ki ima splošno formulo I:ali njena farmacevtsko sprejemljiva sol, kjer: Ri je alkil, ki ima 1-4 ogljikove atome; R-2 je vodik ali (5)-4-amino-2-hidroksibutiril (AHB); R.3 je izbran iz skupine, ki jo sestavljata vodik in alkil, ki ima 1-4 ogljikove atome; in stereo-konfiguracija vsakega izmed položaja 6’ in položaja 5" je neodvisno R konfiguracija ali S konfiguracija.
- 2. Spojina po zahtevku 1, kjer je Ri navedeni alkil.
- 3. Spojina po katerem koli od zahtevkov 1 in 2, kjer sta R2 in R3 vsak vodik.
- 4. Spojina po katerem koli od zahtevkov 1 in 2, kjer je R2 AHB in R3 je vodik.
- 5. Spojina po katerem koli od zahtevkov 1 in 2, kjer je R2 vodik in je R3 navedeni alkil.
- 6. Spojina po katerem koli od zahtevkov 1 in 2, kjer je R2 AHB in je R3 navedeni alkil.
- 7. Spojina po zahtevku 1, ki je izbrana iz skupine, ki jo sestavljajo:
- 8. Spojina po katerem koli od zahtevkov 1-7, značilna po razmerju IC50 translacijske inhibicije v evkariontih proti IC50 translacijski inhibiciji v prokariontih nižjem od 15.
- 9. Spojina po katerem koli od zahtevkov 1-7, značilna po MIC pri gramnegativnih bakterijah višji od 200 μΜ in MIC pri grampozitivnih bakterijah višjih od 20 μΜ.
- 10. Farmacevtski sestavek, ki obsega spojino po katerem koli od zahtevkov 1-9 in farmacevtsko sprejemljiv nosilec.
- 11. Farmacevtski sestavek po zahtevku 10, ki je pakiran v pakimi material in je značilne po tisku, v ali na navedenem pakirnem materialu, za uporabo v zdravljenju genetskih motenj.
- 12. Spojina po katerem koli od zahtevkov 1-9 za uporabo v zdravljenju genetske motnje.
- 13. Sestavek ali spojina za uporabo po zahtevku 11 ali 12, kjer je navedena genetska motnja povezana z mutacijo prezgodnjega stop kodona in/ali s fenotipom s skrajšanjem proteina ("protein truncation phenotype").
- 14. Sestavek ali spojina za uporabo po katerem koli od zahtevkov 11-13, kjer je navedena genetska motnja izbrana iz skupine, ki jo sestavljajo cistična fibroza (CF), Duchennejeva mišična distrofija (DMD), ataksija-teleangiektazija, Hurlerjev sindrom, hemofilija A, hemofilija B, Usherjev sindrom in Tay-Sachsov sindrom.
- 15. Postopek priprave spojine po zahtevku 1, pri čemer postopek obsega: (a) zagotovitev donorske spojine, ki ima formulo II:kjer: R) je alkil, ki ima 1-4 ogljikove atome; R4 je vodik ali donorska amino-zaščitna skupina; R5 je donorska amino-zaščitna skupina, če je R4 je vodik, ali vodik, če je R4 donorska amino-zaščitna skupina; vsak od HPd je donorska hidroksil-zaščitna skupina; in L je odhodna skupina; (b) sklopitev navedene donorske spojine z akceptorsko spojino, ki ima splošno formulo III: kjer:črtkana črta označuje R konfiguracijo ali S konfiguracijo; R3 je izbran iz skupine, ki jo sestavljajo vodik in alkil, ki ima 1-4 ogljikove atome; R^ je akceptorska amino-zaščitna skupina ali (»S)-4-azido-2-0-acetil-1 -butiril; HPa je akceptorska hidroksil- zaščitna skupina; in APa je akceptorska amino-zaščitna skupina; in (c) odstranitev vsake od navedenih amino-zaščitne skupine in hidroksil-zaščitne skupine, s čimer dobimo spojino.
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 (sl) | 2018-02-28 |
Family
ID=45390144
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| SI201131387T SI2640734T1 (sl) | 2010-11-18 | 2011-11-17 | Aminoglikozidi in njihove uporabe v zdravljenju genetskih motenj |
Country Status (18)
| Country | Link |
|---|---|
| US (7) | US8895519B2 (sl) |
| EP (2) | EP3296311B1 (sl) |
| JP (3) | JP5960712B2 (sl) |
| CA (1) | CA2816789C (sl) |
| CY (1) | CY1120082T1 (sl) |
| DK (1) | DK2640734T3 (sl) |
| ES (2) | ES2655916T3 (sl) |
| HK (1) | HK1248709A1 (sl) |
| HR (1) | HRP20180056T1 (sl) |
| HU (1) | HUE036248T2 (sl) |
| IL (3) | IL266495B (sl) |
| LT (1) | LT2640734T (sl) |
| NO (1) | NO2640734T3 (sl) |
| PL (1) | PL2640734T3 (sl) |
| PT (1) | PT2640734T (sl) |
| RS (1) | RS56739B1 (sl) |
| SI (1) | SI2640734T1 (sl) |
| WO (1) | WO2012066546A1 (sl) |
Families Citing this family (13)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CA2816789C (en) | 2010-11-18 | 2018-12-11 | Technion Research & Development Foundation Ltd. | Aminoglycosides and uses thereof in treating genetic disorders |
| KR101703137B1 (ko) | 2014-05-30 | 2017-02-07 | 선문대학교 산학협력단 | 당 부가 아미노글리코사이드 화합물, 이의 제조방법 및 이를 유효성분으로 함유하는 약학적 조성물 |
| WO2015186134A1 (en) | 2014-06-05 | 2015-12-10 | Eloxx Pharmaceuticals Ltd. | Use of aminoglycoside analogs in the treatment of rett syndrome |
| US11306115B2 (en) | 2015-09-02 | 2022-04-19 | Eloxx Pharmaceuticals Ltd. | Aminoglycoside derivatives and uses thereof in treating genetic disorders |
| AU2016314379B2 (en) | 2015-09-02 | 2021-03-11 | Eloxx Pharmaceuticals Ltd. | Aminoglycoside derivatives and uses thereof in treating genetic disorders |
| US10786520B2 (en) | 2015-09-02 | 2020-09-29 | Eloxx Pharmaceuticals Ltd. | Aminoglycoside derivatives and uses thereof in treating genetic disorders |
| US20190016746A1 (en) * | 2016-01-05 | 2019-01-17 | Eloxx Pharmaceuticals Ltd. | Aminoglycoside derivatives and uses thereof in treating genetic disorders |
| AU2018235441B2 (en) * | 2017-03-15 | 2022-07-21 | Eloxx Pharmaceuticals Ltd. | Large scale preparation of pseudo-trisaccharide aminoglycosides and of intermediates thereof |
| 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 |
| KR20220003502A (ko) * | 2018-11-29 | 2022-01-10 | 엘록스 파마슈티칼스 | 안구 질환을 치료하기 위한 방법, 조성물 및 키트 |
| 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 |
| JP5830213B2 (ja) | 2004-02-17 | 2015-12-09 | ザ ホスピタル フォー シック チルドレン | Ｍｅｃｐ２ｅ１遺伝子 |
| ES2689539T3 (es) * | 2006-04-03 | 2018-11-14 | Technion Research & Development Foundation Ltd. | Aminoglucósidos novedosos y usos de los mismos en el tratamiento de trastornos genéticos |
| CA2816789C (en) * | 2010-11-18 | 2018-12-11 | Technion Research & Development Foundation Ltd. | Aminoglycosides and uses thereof in treating genetic disorders |
| WO2015186134A1 (en) | 2014-06-05 | 2015-12-10 | Eloxx Pharmaceuticals Ltd. | Use of aminoglycoside analogs in the treatment of rett syndrome |
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2011
- 2011-11-17 CA CA2816789A patent/CA2816789C/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
- 2011-11-17 WO PCT/IL2011/000889 patent/WO2012066546A1/en active Application Filing
- 2011-11-17 JP JP2013539397A patent/JP5960712B2/ja active Active
- 2011-11-17 PT PT117995019T patent/PT2640734T/pt unknown
- 2011-11-17 SI SI201131387T patent/SI2640734T1/sl unknown
- 2011-11-17 LT LTEP11799501.9T patent/LT2640734T/lt unknown
- 2011-11-17 PL PL11799501T patent/PL2640734T3/pl unknown
- 2011-11-17 RS RS20180029A patent/RS56739B1/sr unknown
- 2011-11-17 EP EP11799501.9A patent/EP2640734B1/en active Active
- 2011-11-17 NO NO11799501A patent/NO2640734T3/no unknown
- 2011-11-17 HU HUE11799501A patent/HUE036248T2/hu unknown
- 2011-11-17 DK DK11799501.9T patent/DK2640734T3/en active
- 2011-11-17 ES ES17192028T patent/ES2833938T3/es active Active
- 2011-11-17 US US13/885,715 patent/US8895519B2/en active Active
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2014
- 2014-08-18 US US14/461,477 patent/US9175029B2/en active Active
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2015
- 2015-09-27 US US14/866,960 patent/US9616079B2/en active Active
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2016
- 2016-01-22 JP JP2016011038A patent/JP2016121168A/ja active Pending
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2017
- 2017-03-09 US US15/453,990 patent/US9943533B2/en active Active
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2018
- 2018-01-12 HR HRP20180056TT patent/HRP20180056T1/hr unknown
- 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
- 2018-06-25 HK HK18108109.1A patent/HK1248709A1/zh unknown
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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
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2020
- 2020-09-01 IL IL277063A patent/IL277063B/en unknown
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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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