DK3505174T3 - Deoxynukleosidterapi mod sygdomme forårsaget af ubalancerede nukleotid-pools, herunder mitokondrielt dna-depletionssyndromer - Google Patents

Deoxynukleosidterapi mod sygdomme forårsaget af ubalancerede nukleotid-pools, herunder mitokondrielt dna-depletionssyndromer Download PDF

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Publication number
DK3505174T3
DK3505174T3 DK19156021.8T DK19156021T DK3505174T3 DK 3505174 T3 DK3505174 T3 DK 3505174T3 DK 19156021 T DK19156021 T DK 19156021T DK 3505174 T3 DK3505174 T3 DK 3505174T3
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Denmark
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mitocondrial
nucleotid
pools
unbalanced
diseases caused
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DK19156021.8T
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English (en)
Inventor
Michio Hirano
Caterina Garone
Ramon Marti
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Univ Columbia
Fundacio Hospital Univ Vall D Hebron Institut De Recerca
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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
    • 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
    • A61K31/7068—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
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • 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
    • A61K31/7076—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
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    • 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
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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
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    • 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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    • 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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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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
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    • 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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DK19156021.8T 2015-06-17 2016-06-17 Deoxynukleosidterapi mod sygdomme forårsaget af ubalancerede nukleotid-pools, herunder mitokondrielt dna-depletionssyndromer DK3505174T3 (da)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201562180914P 2015-06-17 2015-06-17
EP16812537.5A EP3310362B1 (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
DK3505174T3 true DK3505174T3 (da) 2020-07-13

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DK19156021.8T DK3505174T3 (da) 2015-06-17 2016-06-17 Deoxynukleosidterapi mod sygdomme forårsaget af ubalancerede nukleotid-pools, herunder mitokondrielt dna-depletionssyndromer
DK16812537.5T DK3310362T3 (da) 2015-06-17 2016-06-17 Deoxynukleosidterapi mod sygdomme forårsaget af ubalancerede nukleotid-pools, herunder mitokondrielt dna-depletionssyndromer

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DK16812537.5T DK3310362T3 (da) 2015-06-17 2016-06-17 Deoxynukleosidterapi mod sygdomme forårsaget af ubalancerede nukleotid-pools, herunder mitokondrielt dna-depletionssyndromer

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US (4) US10471087B2 (da)
EP (3) EP3569236A1 (da)
JP (3) JP6599484B2 (da)
KR (2) KR20180039624A (da)
CN (2) CN116726035A (da)
AU (3) AU2016280293B2 (da)
BR (2) BR122020021913B1 (da)
CA (1) CA2989653A1 (da)
CY (2) CY1122605T1 (da)
DK (2) DK3505174T3 (da)
ES (2) ES2808148T3 (da)
HK (1) HK1252133A1 (da)
HR (2) HRP20191794T1 (da)
HU (2) HUE046399T2 (da)
IL (3) IL275256B2 (da)
LT (2) LT3310362T (da)
MX (2) MX2017016425A (da)
PL (2) PL3310362T3 (da)
PT (2) PT3505174T (da)
RS (2) RS59724B1 (da)
RU (2) RU2721492C2 (da)
SI (2) SI3310362T1 (da)
WO (1) WO2016205671A1 (da)

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US10292996B2 (en) 2015-03-26 2019-05-21 The Trustees Of Columbia University In The City Of New York Deoxyribonucleoside monophospate bypass therapy for mitochondrial DNA depletion syndrome
RU2721492C2 (ru) * 2015-06-17 2020-05-19 Зе Трастис Оф Коламбия Юниверсити Ин Зе Сити Оф Нью-Йорк Дезоксинуклеозидная терапия заболеваний, вызванных несбалансированными пулами нуклеотидов, в том числе синдромов истощения митохондриальной днк
JP6893213B2 (ja) 2015-12-22 2021-06-23 ゾゲニクス インターナショナル リミテッド フェンフルラミン組成物およびその調製法
JP2019507111A (ja) 2015-12-22 2019-03-14 ゾゲニクス インターナショナル リミテッド 代謝抵抗性フェンフルラミン類縁体およびその使用法
CA3032996A1 (en) 2016-08-24 2018-03-01 Zogenix International Limited Formulation for inhibiting formation of 5-ht 2b agonists and methods of using same
US11649442B2 (en) 2017-09-08 2023-05-16 The Regents Of The University Of California RNA-guided endonuclease fusion polypeptides and methods of use thereof
US10682317B2 (en) 2017-09-26 2020-06-16 Zogenix International Limited Ketogenic diet compatible fenfluramine formulation
BR112020020804A2 (pt) * 2018-04-12 2021-01-12 Modis Therapeutics Inc. Pró-fármacos de desoxinucleosídeos para tratamento de doenças causadas por agrupamentos de nucleotídeos não balanceados
EP3790537A1 (en) 2018-05-11 2021-03-17 Zogenix International Limited Compositions and methods for treating seizure-induced sudden death
AU2020334060A1 (en) * 2019-08-19 2022-03-24 Zogenix, Inc. Polymorphic forms of deoxycytidine, compositions comprising the same and uses
CA3146835A1 (en) * 2019-09-05 2021-03-11 Mitorainbow Therapeutics, Inc. Treating mitochondrial dna depletion disorders
WO2021188840A1 (en) 2020-03-19 2021-09-23 Rewrite Therapeutics, Inc. Methods and compositions for directed genome editing
US11612574B2 (en) 2020-07-17 2023-03-28 Zogenix International Limited Method of treating patients infected with severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2)
US20220096514A1 (en) * 2020-09-29 2022-03-31 Zogenix International Limited Methods of treating thymidine kinase 2 deficiency by administering deoxycytidine and deoxythymidine
EP4268829A1 (en) * 2020-12-28 2023-11-01 Yamasa Corporation Muscular atrophy inhibitor and method for inhibiting muscular atrophy
WO2022204555A1 (en) * 2021-03-26 2022-09-29 Zogenix Mds, Inc. Aqueous solutions containing purines and pyrimidines and uses thereof
WO2022266237A1 (en) * 2021-06-18 2022-12-22 Zogenix Mds, Inc. Prodrugs of deoxynucleosides for treatment of mitochondrial diseases caused by unbalanced nucleotide pools
US20240016938A1 (en) * 2022-07-12 2024-01-18 Zogenix Mds, Inc. Methods for increasing the bioavailability of nucleoside medicinal agents

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Publication number Priority date Publication date Assignee Title
RU2300381C2 (ru) * 1998-08-10 2007-06-10 Айденикс (Кайман) Лимитед β-L-2'-ДЕЗОКСИНУКЛЕОЗИДЫ ДЛЯ ЛЕЧЕНИЯ ГЕПАТИТА В
KR100879559B1 (ko) * 1999-02-23 2009-01-22 더 리젠츠 오브 더 유니버시티 오브 캘리포니아 미토콘드리아 장애의 치료 또는 예방용 제약조성물
ES2227203T3 (es) * 2000-05-26 2005-04-01 Idenix (Cayman) Limited Metodos para tratar la infeccion por el virus de la hepatitis delta con beta-1-2'-desoxinucleosidos.
WO2012125848A2 (en) * 2011-03-16 2012-09-20 Baylor College Of Medicine A method for comprehensive sequence analysis using deep sequencing technology
US10292996B2 (en) 2015-03-26 2019-05-21 The Trustees Of Columbia University In The City Of New York Deoxyribonucleoside monophospate bypass therapy for mitochondrial DNA depletion syndrome
US11337980B2 (en) * 2015-06-05 2022-05-24 Fundació Hospital Universitari Vall D'hebron-Institut De Recerca Treatment of mitochondrial diseases
RU2721492C2 (ru) * 2015-06-17 2020-05-19 Зе Трастис Оф Коламбия Юниверсити Ин Зе Сити Оф Нью-Йорк Дезоксинуклеозидная терапия заболеваний, вызванных несбалансированными пулами нуклеотидов, в том числе синдромов истощения митохондриальной днк

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AU2020204042B2 (en) 2021-07-15
US10471087B2 (en) 2019-11-12
CN116726035A (zh) 2023-09-12
US11110111B2 (en) 2021-09-07
EP3310362A1 (en) 2018-04-25
KR20220018623A (ko) 2022-02-15
HRP20191794T1 (hr) 2020-02-07
ES2748556T3 (es) 2020-03-17
MX2020000269A (es) 2022-01-03
JP2018521993A (ja) 2018-08-09
PL3310362T3 (pl) 2020-04-30
RU2018101305A3 (da) 2019-10-31
AU2021250841A1 (en) 2021-11-04
DK3310362T3 (da) 2019-10-07
CY1122605T1 (el) 2021-03-12
WO2016205671A9 (en) 2018-01-04
US20180133241A1 (en) 2018-05-17
EP3310362A4 (en) 2018-12-19
RS59724B1 (sr) 2020-01-31
EP3310362B1 (en) 2019-08-28
HK1252133A1 (zh) 2019-05-17
JP6599484B2 (ja) 2019-10-30
IL275255B (en) 2021-04-29
AU2021250841B2 (en) 2023-02-09
RU2020112901A (ru) 2020-06-05
ES2808148T3 (es) 2021-02-25
HUE050678T2 (hu) 2020-12-28
LT3310362T (lt) 2019-11-11
AU2020204042A1 (en) 2020-07-09
PT3505174T (pt) 2020-07-06
US20210244754A1 (en) 2021-08-12
PT3310362T (pt) 2019-10-29
US20230277577A1 (en) 2023-09-07
IL275255A (en) 2020-07-30
JP2020019792A (ja) 2020-02-06
JP6675037B2 (ja) 2020-04-01
IL275256B2 (en) 2023-09-01
AU2016280293B2 (en) 2020-07-02
CN107847512A (zh) 2018-03-27
CY1123107T1 (el) 2021-10-29
KR20180039624A (ko) 2018-04-18
LT3505174T (lt) 2020-09-10
HUE046399T2 (hu) 2020-03-30
SI3310362T1 (sl) 2020-02-28
IL256331A (en) 2018-02-28
HRP20200949T1 (hr) 2020-10-02
IL275256A (en) 2020-07-30
BR112017027079B1 (pt) 2021-04-27
SI3505174T1 (sl) 2021-02-26
AU2016280293A1 (en) 2018-02-08
EP3505174B1 (en) 2020-04-29
PL3505174T3 (pl) 2020-08-24
JP2020019791A (ja) 2020-02-06
RS60572B1 (sr) 2020-08-31
RU2018101305A (ru) 2019-07-17
US11666592B2 (en) 2023-06-06
MX2017016425A (es) 2018-12-11
RU2721492C2 (ru) 2020-05-19
WO2016205671A1 (en) 2016-12-22
JP7036782B2 (ja) 2022-03-15
US20200038424A1 (en) 2020-02-06
BR122020021913B1 (pt) 2021-07-06
EP3505174A1 (en) 2019-07-03
IL275256B1 (en) 2023-05-01
CA2989653A1 (en) 2016-12-22
IL256331B (en) 2020-08-31
EP3569236A1 (en) 2019-11-20

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