CY1120082T1 - Αμινογλυκοσιδες και χρησεις αυτων στη θεραπευτικη αγωγη γενετικων διαταραχων - Google Patents

Αμινογλυκοσιδες και χρησεις αυτων στη θεραπευτικη αγωγη γενετικων διαταραχων

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Publication number
CY1120082T1
CY1120082T1 CY20181100039T CY181100039T CY1120082T1 CY 1120082 T1 CY1120082 T1 CY 1120082T1 CY 20181100039 T CY20181100039 T CY 20181100039T CY 181100039 T CY181100039 T CY 181100039T CY 1120082 T1 CY1120082 T1 CY 1120082T1
Authority
CY
Cyprus
Prior art keywords
aminoglycosides
genetic disorders
therapeutic treatment
alkyl
group
Prior art date
Application number
CY20181100039T
Other languages
English (en)
Inventor
Timor Baasov
Dana Atia-Glikin
Jeyakumar Kandasamy
Valery Belakhov
Original Assignee
Technion Research & Development Foundation Ltd.
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Technion Research & Development Foundation Ltd. filed Critical Technion Research & Development Foundation Ltd.
Publication of CY1120082T1 publication Critical patent/CY1120082T1/el

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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)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Molecular Biology (AREA)
  • Genetics & Genomics (AREA)
  • Biotechnology (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)
  • Neurology (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)

Abstract

Παρέχεται μια νέα κατηγορία αμινογλυκοσιδών ψευδο-τρισακχαριτών που έχουν μια αλκυλ ομάδα στη θέση 5", οι οποίες επιδεικνύουν αποτελεσματική δραστικότητα ανάγνωσης μετάλλαξης κωδικονίου τερματισμού, χαμηλή κυτταροτοξικότητα και υψηλή εκλεκτικότητα προς ευκαρυωτικά συστήματα μετάφρασης. Επίσης παρέχονται φαρμακευτικές συνθέσεις που τις περιέχουν, και χρήσεις αυτών στη θεραπευτική αγωγή γενετικών διαταραχών, καθώς επίσης διαδικασίες παρασκευής αυτών των αμινογλυκοσιδών. Οι αποκαλυπτόμενες αμινογλυκοσίδες μπορούν να αντιπροσωπευθούν από τον γενικό τύπο I: τύπος I ή ένα φαρμακευτικώς αποδεκτό άλας αυτού, όπου το R1 επιλέγεται από την ομάδα που αποτελείται από αλκυλ, κυκλοαλκυλ και αρυλ, και όλες οι άλλες μεταβλητές και χαρακτηριστικά είναι όπως περιγράφονται στην περιγραφή.
CY20181100039T 2010-11-18 2018-01-15 Αμινογλυκοσιδες και χρησεις αυτων στη θεραπευτικη αγωγη γενετικων διαταραχων CY1120082T1 (el)

Applications Claiming Priority (2)

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

Publications (1)

Publication Number Publication Date
CY1120082T1 true CY1120082T1 (el) 2018-12-12

Family

ID=45390144

Family Applications (1)

Application Number Title Priority Date Filing Date
CY20181100039T CY1120082T1 (el) 2010-11-18 2018-01-15 Αμινογλυκοσιδες και χρησεις αυτων στη θεραπευτικη αγωγη γενετικων διαταραχων

Country Status (18)

Country Link
US (7) US8895519B2 (el)
EP (2) EP3296311B1 (el)
JP (3) JP5960712B2 (el)
CA (1) CA2816789C (el)
CY (1) CY1120082T1 (el)
DK (1) DK2640734T3 (el)
ES (2) ES2833938T3 (el)
HK (1) HK1248709A1 (el)
HR (1) HRP20180056T1 (el)
HU (1) HUE036248T2 (el)
IL (3) IL266495B (el)
LT (1) LT2640734T (el)
NO (1) NO2640734T3 (el)
PL (1) PL2640734T3 (el)
PT (1) PT2640734T (el)
RS (1) RS56739B1 (el)
SI (1) SI2640734T1 (el)
WO (1) WO2012066546A1 (el)

Families Citing this family (13)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
ES2833938T3 (es) * 2010-11-18 2021-06-16 Technion Res & Dev Foundation Aminoglucósidos y usos de los mismos en el tratamiento de trastornos genéticos
KR101703137B1 (ko) 2014-05-30 2017-02-07 선문대학교 산학협력단 당 부가 아미노글리코사이드 화합물, 이의 제조방법 및 이를 유효성분으로 함유하는 약학적 조성물
US20170360817A1 (en) * 2014-06-05 2017-12-21 Eloxx Pharmaceuticals Ltd. Use of aminoglycoside analogs in the treatment of rett syndrome
JP6862428B6 (ja) * 2015-09-02 2021-06-02 エロックス ファーマシューティカルズ リミテッド アミノグリコシド誘導体および遺伝性疾患の治療におけるその使用
EP3344640A4 (en) 2015-09-02 2019-05-01 Eloxx Pharmaceuticals Ltd. AMINO-LIKES DERIVATIVES AND USES THEREOF FOR THE TREATMENT OF GENETIC DISEASES
US10786520B2 (en) 2015-09-02 2020-09-29 Eloxx Pharmaceuticals Ltd. Aminoglycoside derivatives and uses thereof in treating genetic disorders
EP3400232A4 (en) * 2016-01-05 2019-10-16 Eloxx Pharmaceuticals Ltd. AMINO-L-KOSIDE DERIVATIVES AND USES THEREOF FOR THE TREATMENT OF GENETIC DISEASES
US11136345B2 (en) 2017-03-15 2021-10-05 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
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

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
WO2005078099A1 (en) 2004-02-17 2005-08-25 The Hospital For Sick Children Mecp2e1 gene
LT2390255T (lt) 2006-04-03 2016-10-25 Technion Research & Development Foundation Ltd. Nauji aminoglikozidai ir jų panaudojimas genetinių sutrikimų gydyme
ES2833938T3 (es) 2010-11-18 2021-06-16 Technion Res & Dev Foundation Aminoglucósidos y usos de los mismos en el tratamiento de trastornos genéticos
US20170360817A1 (en) 2014-06-05 2017-12-21 Eloxx Pharmaceuticals Ltd. Use of aminoglycoside analogs in the treatment of rett syndrome

Also Published As

Publication number Publication date
US20180200276A1 (en) 2018-07-19
US10398718B2 (en) 2019-09-03
US20210228608A1 (en) 2021-07-29
JP2016121168A (ja) 2016-07-07
US9943533B2 (en) 2018-04-17
EP2640734B1 (en) 2017-10-18
US20200078386A1 (en) 2020-03-12
SI2640734T1 (en) 2018-02-28
US20170182078A1 (en) 2017-06-29
US10973839B2 (en) 2021-04-13
DK2640734T3 (en) 2018-01-22
JP2013542981A (ja) 2013-11-28
JP5960712B2 (ja) 2016-08-02
PT2640734T (pt) 2018-01-22
CA2816789A1 (en) 2012-05-24
IL266495B (en) 2020-09-30
CA2816789C (en) 2018-12-11
IL284067B (en) 2022-03-01
HK1248709A1 (zh) 2018-10-19
PL2640734T3 (pl) 2018-04-30
IL277063A (en) 2020-10-29
IL266495A (en) 2019-07-31
JP6758338B2 (ja) 2020-09-23
US20130237489A1 (en) 2013-09-12
US20140357590A1 (en) 2014-12-04
US20160074425A1 (en) 2016-03-17
ES2833938T3 (es) 2021-06-16
JP2018123135A (ja) 2018-08-09
RS56739B1 (sr) 2018-03-30
LT2640734T (lt) 2018-02-12
WO2012066546A1 (en) 2012-05-24
US11865128B2 (en) 2024-01-09
US20190224226A9 (en) 2019-07-25
US9616079B2 (en) 2017-04-11
EP3296311B1 (en) 2020-09-30
NO2640734T3 (el) 2018-03-17
EP2640734A1 (en) 2013-09-25
US9175029B2 (en) 2015-11-03
ES2655916T3 (es) 2018-02-22
HUE036248T2 (hu) 2018-06-28
HRP20180056T1 (hr) 2018-02-09
IL277063B (en) 2021-07-29
IL284067A (en) 2021-07-29
EP3296311A1 (en) 2018-03-21
US8895519B2 (en) 2014-11-25

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