CA2816789C - Aminoglycosides and uses thereof in treating genetic disorders - Google Patents

Aminoglycosides and uses thereof in treating genetic disorders Download PDF

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Publication number
CA2816789C
CA2816789C CA2816789A CA2816789A CA2816789C CA 2816789 C CA2816789 C CA 2816789C CA 2816789 A CA2816789 A CA 2816789A CA 2816789 A CA2816789 A CA 2816789A CA 2816789 C CA2816789 C CA 2816789C
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CA
Canada
Prior art keywords
compound
genetic disorder
pharmaceutical composition
genetic
compounds
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Active
Application number
CA2816789A
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English (en)
French (fr)
Other versions
CA2816789A1 (en
Inventor
Timor Baasov
Dana Atia-Glikin
Jeyakumar Kandasamy
Valery Belakhov
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Technion Research and Development Foundation Ltd
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Technion Research and Development Foundation Ltd
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Publication of CA2816789A1 publication Critical patent/CA2816789A1/en
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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)
CA2816789A 2010-11-18 2011-11-17 Aminoglycosides and uses thereof in treating genetic disorders Active CA2816789C (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US41495610P 2010-11-18 2010-11-18
US61/414,956 2010-11-18
PCT/IL2011/000889 WO2012066546A1 (en) 2010-11-18 2011-11-17 Aminoglycosides and uses thereof in treating genetic disorders

Publications (2)

Publication Number Publication Date
CA2816789A1 CA2816789A1 (en) 2012-05-24
CA2816789C true CA2816789C (en) 2018-12-11

Family

ID=45390144

Family Applications (1)

Application Number Title Priority Date Filing Date
CA2816789A Active CA2816789C (en) 2010-11-18 2011-11-17 Aminoglycosides and uses thereof in treating 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)

* Cited by examiner, † Cited by third party
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)

* Cited by examiner, † Cited by third party
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

Also Published As

Publication number Publication date
EP3296311A1 (en) 2018-03-21
US20130237489A1 (en) 2013-09-12
IL284067B (en) 2022-03-01
IL266495A (en) 2019-07-31
WO2012066546A1 (en) 2012-05-24
LT2640734T (lt) 2018-02-12
US9616079B2 (en) 2017-04-11
US8895519B2 (en) 2014-11-25
PT2640734T (pt) 2018-01-22
EP2640734A1 (en) 2013-09-25
CY1120082T1 (el) 2018-12-12
US20200078386A1 (en) 2020-03-12
PL2640734T3 (pl) 2018-04-30
US20190224226A9 (en) 2019-07-25
JP5960712B2 (ja) 2016-08-02
RS56739B1 (sr) 2018-03-30
ES2833938T3 (es) 2021-06-16
IL277063A (en) 2020-10-29
NO2640734T3 (enExample) 2018-03-17
IL277063B (en) 2021-07-29
US10398718B2 (en) 2019-09-03
IL284067A (en) 2021-07-29
DK2640734T3 (en) 2018-01-22
HK1248709A1 (en) 2018-10-19
SMT201800019T1 (it) 2018-03-08
US10973839B2 (en) 2021-04-13
EP3296311B1 (en) 2020-09-30
US9943533B2 (en) 2018-04-17
HRP20180056T1 (hr) 2018-02-09
SI2640734T1 (en) 2018-02-28
JP6758338B2 (ja) 2020-09-23
US9175029B2 (en) 2015-11-03
HUE036248T2 (hu) 2018-06-28
CA2816789A1 (en) 2012-05-24
JP2016121168A (ja) 2016-07-07
US11865128B2 (en) 2024-01-09
IL266495B (en) 2020-09-30
JP2013542981A (ja) 2013-11-28
US20170182078A1 (en) 2017-06-29
US20180200276A1 (en) 2018-07-19
JP2018123135A (ja) 2018-08-09
US20210228608A1 (en) 2021-07-29
US20140357590A1 (en) 2014-12-04
US20160074425A1 (en) 2016-03-17
ES2655916T3 (es) 2018-02-22
EP2640734B1 (en) 2017-10-18

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