CY1122605T1 - Θεραπεια δεοξυνουκλεοσιδιου για νοσους οι οποιες οφειλονται σε μη ισορροπημενες ομαδες νουκλεοτιδιων, οπου συμπεριλαμβανονται συνδρομα εξαντλησης μιτοχονδριακου dna - Google Patents

Θεραπεια δεοξυνουκλεοσιδιου για νοσους οι οποιες οφειλονται σε μη ισορροπημενες ομαδες νουκλεοτιδιων, οπου συμπεριλαμβανονται συνδρομα εξαντλησης μιτοχονδριακου dna

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Publication number
CY1122605T1
CY1122605T1 CY20191101127T CY191101127T CY1122605T1 CY 1122605 T1 CY1122605 T1 CY 1122605T1 CY 20191101127 T CY20191101127 T CY 20191101127T CY 191101127 T CY191101127 T CY 191101127T CY 1122605 T1 CY1122605 T1 CY 1122605T1
Authority
CY
Cyprus
Prior art keywords
mitochondrial dna
dna depletion
deoxynucleoside
unbalanced nucleotide
diseases due
Prior art date
Application number
CY20191101127T
Other languages
Greek (el)
English (en)
Inventor
Michio Hirano
Caterina Garone
Ramon MARTI
Original Assignee
The Trustees Of Columbia University In The City Of New York
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 The Trustees Of Columbia University In The City Of New York filed Critical The Trustees Of Columbia University In The City Of New York
Publication of CY1122605T1 publication Critical patent/CY1122605T1/el

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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
    • A61K31/7042—Compounds having saccharide radicals and heterocyclic rings
    • A61K31/7052—Compounds having saccharide radicals and heterocyclic rings having nitrogen as a ring hetero atom, e.g. nucleosides, nucleotides
    • A61K31/706—Compounds having saccharide radicals and heterocyclic rings having nitrogen as a ring hetero atom, e.g. nucleosides, nucleotides containing six-membered rings with nitrogen as a ring hetero atom
    • A61K31/7064—Compounds having saccharide radicals and heterocyclic rings having nitrogen as a ring hetero atom, e.g. nucleosides, nucleotides containing six-membered rings with nitrogen as a ring hetero atom containing condensed or non-condensed pyrimidines
    • A61K31/7068—Compounds having saccharide radicals and heterocyclic rings having nitrogen as a ring hetero atom, e.g. nucleosides, nucleotides containing six-membered rings with nitrogen as a ring hetero atom containing condensed or non-condensed pyrimidines having oxo groups directly attached to the pyrimidine ring, e.g. cytidine, cytidylic acid
    • 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
    • A61K31/33—Heterocyclic compounds
    • A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/495—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with two or more nitrogen atoms as the only ring heteroatoms, e.g. piperazine or tetrazines
    • A61K31/505—Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim
    • A61K31/513—Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim having oxo groups directly attached to the heterocyclic ring, e.g. cytosine
    • 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
    • A61K31/7042—Compounds having saccharide radicals and heterocyclic rings
    • A61K31/7052—Compounds having saccharide radicals and heterocyclic rings having nitrogen as a ring hetero atom, e.g. nucleosides, nucleotides
    • A61K31/706—Compounds having saccharide radicals and heterocyclic rings having nitrogen as a ring hetero atom, e.g. nucleosides, nucleotides containing six-membered rings with nitrogen as a ring hetero atom
    • A61K31/7064—Compounds having saccharide radicals and heterocyclic rings having nitrogen as a ring hetero atom, e.g. nucleosides, nucleotides containing six-membered rings with nitrogen as a ring hetero atom containing condensed or non-condensed pyrimidines
    • A61K31/7068—Compounds having saccharide radicals and heterocyclic rings having nitrogen as a ring hetero atom, e.g. nucleosides, nucleotides containing six-membered rings with nitrogen as a ring hetero atom containing condensed or non-condensed pyrimidines having oxo groups directly attached to the pyrimidine ring, e.g. cytidine, cytidylic acid
    • A61K31/7072—Compounds having saccharide radicals and heterocyclic rings having nitrogen as a ring hetero atom, e.g. nucleosides, nucleotides containing six-membered rings with nitrogen as a ring hetero atom containing condensed or non-condensed pyrimidines having oxo groups directly attached to the pyrimidine ring, e.g. cytidine, cytidylic acid having two oxo groups directly attached to the pyrimidine ring, e.g. uridine, uridylic acid, thymidine, zidovudine
    • 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
    • A61K31/7042—Compounds having saccharide radicals and heterocyclic rings
    • A61K31/7052—Compounds having saccharide radicals and heterocyclic rings having nitrogen as a ring hetero atom, e.g. nucleosides, nucleotides
    • A61K31/706—Compounds having saccharide radicals and heterocyclic rings having nitrogen as a ring hetero atom, e.g. nucleosides, nucleotides containing six-membered rings with nitrogen as a ring hetero atom
    • A61K31/7064—Compounds having saccharide radicals and heterocyclic rings having nitrogen as a ring hetero atom, e.g. nucleosides, nucleotides containing six-membered rings with nitrogen as a ring hetero atom containing condensed or non-condensed pyrimidines
    • A61K31/7076—Compounds having saccharide radicals and heterocyclic rings having nitrogen as a ring hetero atom, e.g. nucleosides, nucleotides containing six-membered rings with nitrogen as a ring hetero atom containing condensed or non-condensed pyrimidines containing purines, e.g. adenosine, adenylic acid
    • 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
    • A61K31/7042—Compounds having saccharide radicals and heterocyclic rings
    • A61K31/7052—Compounds having saccharide radicals and heterocyclic rings having nitrogen as a ring hetero atom, e.g. nucleosides, nucleotides
    • A61K31/706—Compounds having saccharide radicals and heterocyclic rings having nitrogen as a ring hetero atom, e.g. nucleosides, nucleotides containing six-membered rings with nitrogen as a ring hetero atom
    • A61K31/7064—Compounds having saccharide radicals and heterocyclic rings having nitrogen as a ring hetero atom, e.g. nucleosides, nucleotides containing six-membered rings with nitrogen as a ring hetero atom containing condensed or non-condensed pyrimidines
    • A61K31/7076—Compounds having saccharide radicals and heterocyclic rings having nitrogen as a ring hetero atom, e.g. nucleosides, nucleotides containing six-membered rings with nitrogen as a ring hetero atom containing condensed or non-condensed pyrimidines containing purines, e.g. adenosine, adenylic acid
    • A61K31/708—Compounds having saccharide radicals and heterocyclic rings having nitrogen as a ring hetero atom, e.g. nucleosides, nucleotides containing six-membered rings with nitrogen as a ring hetero atom containing condensed or non-condensed pyrimidines containing purines, e.g. adenosine, adenylic acid having oxo groups directly attached to the purine ring system, e.g. guanosine, guanylic acid
    • 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
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
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    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0053—Mouth and digestive tract, i.e. intraoral and peroral administration
    • 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
    • A61P3/00—Drugs for disorders of the metabolism
    • A61P3/04—Anorexiants; Antiobesity agents
    • 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Q—MEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
    • C12Q1/00—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
    • C12Q1/68—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving nucleic acids
    • C12Q1/6813—Hybridisation assays
    • C12Q1/6827—Hybridisation assays for detection of mutation or polymorphism
    • G—PHYSICS
    • G01—MEASURING; TESTING
    • G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N33/00—Investigating or analysing materials by specific methods not covered by groups G01N1/00 - G01N31/00
    • G01N33/48—Biological material, e.g. blood, urine; Haemocytometers
    • G01N33/50—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K2300/00—Mixtures or combinations of active ingredients, wherein at least one active ingredient is fully defined in groups A61K31/00 - A61K41/00

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  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
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  • Wood Science & Technology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
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  • Urology & Nephrology (AREA)
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CY20191101127T 2015-06-17 2019-10-30 Θεραπεια δεοξυνουκλεοσιδιου για νοσους οι οποιες οφειλονται σε μη ισορροπημενες ομαδες νουκλεοτιδιων, οπου συμπεριλαμβανονται συνδρομα εξαντλησης μιτοχονδριακου dna CY1122605T1 (el)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201562180914P 2015-06-17 2015-06-17
PCT/US2016/038110 WO2016205671A1 (en) 2015-06-17 2016-06-17 Deoxynucleoside therapy for diseases caused by unbalanced nucleotide pools including mitochondrial dna depletion syndromes

Publications (1)

Publication Number Publication Date
CY1122605T1 true CY1122605T1 (el) 2021-03-12

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Application Number Title Priority Date Filing Date
CY20191101127T CY1122605T1 (el) 2015-06-17 2019-10-30 Θεραπεια δεοξυνουκλεοσιδιου για νοσους οι οποιες οφειλονται σε μη ισορροπημενες ομαδες νουκλεοτιδιων, οπου συμπεριλαμβανονται συνδρομα εξαντλησης μιτοχονδριακου dna
CY20201100578T CY1123107T1 (el) 2015-06-17 2020-06-25 Θεραπεια δεοξυνουκλεοσιδιου για νοσους οι οποιες οφειλονται σε μη ισορροπημενες ομαδες νουκλεοτιδιων, οπου συμπεριλαμβανονται συνδρομα εξαντλησης μιτοχονδριακου dna

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CY20201100578T CY1123107T1 (el) 2015-06-17 2020-06-25 Θεραπεια δεοξυνουκλεοσιδιου για νοσους οι οποιες οφειλονται σε μη ισορροπημενες ομαδες νουκλεοτιδιων, οπου συμπεριλαμβανονται συνδρομα εξαντλησης μιτοχονδριακου dna

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US (5) US10471087B2 (https=)
EP (3) EP3310362B1 (https=)
JP (3) JP6599484B2 (https=)
KR (3) KR20180039624A (https=)
CN (2) CN116726035A (https=)
AU (3) AU2016280293B2 (https=)
BR (2) BR112017027079B1 (https=)
CA (1) CA2989653A1 (https=)
CY (2) CY1122605T1 (https=)
DK (2) DK3505174T3 (https=)
ES (2) ES2748556T3 (https=)
HK (1) HK1252133A1 (https=)
HR (2) HRP20191794T1 (https=)
HU (2) HUE046399T2 (https=)
IL (3) IL275256B2 (https=)
LT (2) LT3310362T (https=)
MX (2) MX2017016425A (https=)
PL (2) PL3505174T3 (https=)
PT (2) PT3505174T (https=)
RS (2) RS59724B1 (https=)
RU (1) RU2721492C2 (https=)
SI (2) SI3505174T1 (https=)
SM (2) SMT202000399T1 (https=)
WO (1) WO2016205671A1 (https=)

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EP3790537A1 (en) 2018-05-11 2021-03-17 Zogenix International Limited Compositions and methods for treating seizure-induced sudden death
AU2019384963B2 (en) 2018-11-19 2022-05-26 Zogenix International Limited Methods of treating Rett syndrome using fenfluramine
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EP4313068A4 (en) 2021-03-26 2024-12-25 UCB Biosciences, Inc. Aqueous solutions containing purines and pyrimidines and uses thereof
US20230000890A1 (en) * 2021-06-18 2023-01-05 Zogenix Mds, Inc. Prodrugs of Deoxynucleosides for Treatment of Mitochondrial Diseases Caused by Unbalanced Nucleotide Pools
WO2024015453A1 (en) * 2022-07-12 2024-01-18 Zogenix Mds, Inc. Methods for increasing the bioavailability of nucleoside medicinal agents
EP4687918A2 (en) * 2023-03-30 2026-02-11 The Trustees Of Columbia University In The City Of New York Pharmacological therapy for mitochondrial dna depletion deletions syndrome involving mutations in the guk1 gene

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AU2021250841B2 (en) 2023-02-09
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EP3310362A4 (en) 2018-12-19
DK3505174T3 (da) 2020-07-13
IL275256A (en) 2020-07-30
RS60572B1 (sr) 2020-08-31
SMT202000399T1 (it) 2020-09-10
RU2020112901A (ru) 2020-06-05
SI3505174T1 (sl) 2021-02-26
HK1252133A1 (zh) 2019-05-17
US20250360154A1 (en) 2025-11-27
AU2016280293B2 (en) 2020-07-02
PL3310362T3 (pl) 2020-04-30
CN116726035A (zh) 2023-09-12
EP3505174B1 (en) 2020-04-29
MX2017016425A (es) 2018-12-11
SMT201900571T1 (it) 2019-11-13
EP3505174A1 (en) 2019-07-03
KR20220018623A (ko) 2022-02-15
IL256331B (en) 2020-08-31
HUE046399T2 (hu) 2020-03-30
EP3569236A1 (en) 2019-11-20
AU2020204042B2 (en) 2021-07-15
US11110111B2 (en) 2021-09-07
US20180133241A1 (en) 2018-05-17
JP2018521993A (ja) 2018-08-09
WO2016205671A1 (en) 2016-12-22
AU2020204042A1 (en) 2020-07-09
SI3310362T1 (sl) 2020-02-28
DK3310362T3 (da) 2019-10-07
US12251392B2 (en) 2025-03-18
ES2748556T3 (es) 2020-03-17
EP3310362B1 (en) 2019-08-28
JP6599484B2 (ja) 2019-10-30
IL275255A (en) 2020-07-30
ES2808148T3 (es) 2021-02-25
MX2020000269A (es) 2022-01-03
PT3310362T (pt) 2019-10-29
RU2018101305A3 (https=) 2019-10-31
AU2016280293A1 (en) 2018-02-08
IL275256B2 (en) 2023-09-01
RS59724B1 (sr) 2020-01-31
RU2721492C2 (ru) 2020-05-19
CY1123107T1 (el) 2021-10-29
IL275256B1 (en) 2023-05-01
JP2020019791A (ja) 2020-02-06
WO2016205671A9 (en) 2018-01-04
KR20250048602A (ko) 2025-04-09
LT3505174T (lt) 2020-09-10
CN107847512A (zh) 2018-03-27
JP7036782B2 (ja) 2022-03-15
KR20180039624A (ko) 2018-04-18
HRP20191794T1 (hr) 2020-02-07
RU2018101305A (ru) 2019-07-17
IL256331A (en) 2018-02-28
HRP20200949T1 (hr) 2020-10-02
AU2021250841A1 (en) 2021-11-04
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US20230277577A1 (en) 2023-09-07
US20200038424A1 (en) 2020-02-06
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HUE050678T2 (hu) 2020-12-28
US11666592B2 (en) 2023-06-06
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IL275255B (en) 2021-04-29
PL3505174T3 (pl) 2020-08-24
PT3505174T (pt) 2020-07-06
US20210244754A1 (en) 2021-08-12
US10471087B2 (en) 2019-11-12

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