DK3310362T3 - 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
DK3310362T3
DK3310362T3 DK16812537.5T DK16812537T DK3310362T3 DK 3310362 T3 DK3310362 T3 DK 3310362T3 DK 16812537 T DK16812537 T DK 16812537T DK 3310362 T3 DK3310362 T3 DK 3310362T3
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Prior art keywords
mitocondrial
diseases caused
dna depletion
nucleotide pools
unbalanced nucleotide
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DK16812537.5T
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Danish (da)
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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    • 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
    • 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
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    • 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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    • 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
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DK16812537.5T 2015-06-17 2016-06-17 Deoxynukleosidterapi mod sygdomme forårsaget af ubalancerede nukleotid-pools, herunder mitokondrielt dna-depletionssyndromer DK3310362T3 (da)

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
DK3310362T3 true DK3310362T3 (da) 2019-10-07

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

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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

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US (5) US10471087B2 (enExample)
EP (3) EP3505174B1 (enExample)
JP (3) JP6599484B2 (enExample)
KR (3) KR20180039624A (enExample)
CN (2) CN116726035A (enExample)
AU (3) AU2016280293B2 (enExample)
BR (2) BR122020021913B1 (enExample)
CA (1) CA2989653A1 (enExample)
CY (2) CY1122605T1 (enExample)
DK (2) DK3310362T3 (enExample)
ES (2) ES2748556T3 (enExample)
HK (1) HK1252133A1 (enExample)
HR (2) HRP20191794T1 (enExample)
HU (2) HUE046399T2 (enExample)
IL (3) IL275256B2 (enExample)
LT (2) LT3505174T (enExample)
MX (2) MX2017016425A (enExample)
PL (2) PL3310362T3 (enExample)
PT (2) PT3505174T (enExample)
RS (2) RS60572B1 (enExample)
RU (1) RU2721492C2 (enExample)
SI (2) SI3310362T1 (enExample)
SM (2) SMT202000399T1 (enExample)
WO (1) WO2016205671A1 (enExample)

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US9549909B2 (en) 2013-05-03 2017-01-24 The Katholieke Universiteit Leuven Method for the treatment of dravet syndrome
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
US10471087B2 (en) * 2015-06-17 2019-11-12 The Trustees Of Columbia University In The City Of New York Deoxynucleoside therapy for diseases caused by unbalanced nucleotide pools including mitochondrial DNA depletion syndromes
EP3393470B1 (en) 2015-12-22 2021-01-20 Zogenix International Limited Metabolism resistant fenfluramine analogs and methods of using the same
CA3007286A1 (en) 2015-12-22 2017-06-29 Zogenix International Limited Fenfluramine compositions and methods of preparing the same
MX2019001799A (es) 2016-08-24 2019-06-13 Zogenix International Ltd Formulacion para inhibir la formacion de agonistas de 5-ht2b y metodos de utilizacion de la misma.
WO2019051097A1 (en) 2017-09-08 2019-03-14 The Regents Of The University Of California RNA-GUIDED ENDONUCLEASE FUSION POLYPEPTIDES AND METHODS OF USING SAME
US10682317B2 (en) 2017-09-26 2020-06-16 Zogenix International Limited Ketogenic diet compatible fenfluramine formulation
WO2019200340A1 (en) 2018-04-12 2019-10-17 Modis Therapeutics Inc. Prodrugs of deoxynucleosides for treatment of diseases cased by unbalanced nucleotide pools
MX2020010994A (es) 2018-04-18 2021-01-08 Univ Columbia Terapia de gen para enfermedades causadas por combinaciones desequilibradas de nucleótidos que incluyen síndromes de agotamiento de adn mitocondrial.
WO2019216919A1 (en) 2018-05-11 2019-11-14 Zogenix International Limited Compositions and methods for treating seizure-induced sudden death
EA202191079A1 (ru) 2018-11-19 2021-09-20 Зодженикс Интернэшнл Лимитед Способы лечения синдрома ретта с применением фенфлурамина
EP4017502A4 (en) * 2019-08-19 2023-11-01 Modis Therapeutics Inc. POLYMORPH FORMS OF DEOXYCYTIDINE, COMPOSITIONS COMPRISING THEM AND USES
CN114340641B (zh) * 2019-09-05 2025-04-04 米托瑞因博治疗股份有限公司 治疗线粒体dna缺失障碍
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
WO2022145439A1 (ja) * 2020-12-28 2022-07-07 ヤマサ醤油株式会社 筋萎縮抑制剤及び筋萎縮を抑制させる方法
CN117120060A (zh) * 2021-03-26 2023-11-24 周格尼克斯-莫迪斯公司 含有嘌呤和嘧啶的水溶液及其用途
EP4355336A4 (en) * 2021-06-18 2025-05-07 UCB Biosciences, Inc. DEOXYNUCLEOSIDE PRODRUGS FOR THE TREATMENT OF MITOCHONDRIAL DISEASES CAUSED BY UNBALANCED NUCLEOTIDE GROUPS
EP4554596A1 (en) * 2022-07-12 2025-05-21 UCB Biosciences, Inc. Methods for increasing the bioavailability of nucleoside medicinal agents
WO2024206595A2 (en) * 2023-03-30 2024-10-03 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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KR100879559B1 (ko) * 1999-02-23 2009-01-22 더 리젠츠 오브 더 유니버시티 오브 캘리포니아 미토콘드리아 장애의 치료 또는 예방용 제약조성물
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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
PL3302499T3 (pl) * 2015-06-05 2023-07-24 Fundació Hospital Universitari Vall D'hebron - Institut De Recerca Leczenie chorób mitochondrialnych
US10471087B2 (en) * 2015-06-17 2019-11-12 The Trustees Of Columbia University In The City Of New York Deoxynucleoside therapy for diseases caused by unbalanced nucleotide pools including mitochondrial DNA depletion syndromes

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BR122020021913B1 (pt) 2021-07-06
LT3310362T (lt) 2019-11-11
KR20250048602A (ko) 2025-04-09
PT3310362T (pt) 2019-10-29
CY1123107T1 (el) 2021-10-29
RS59724B1 (sr) 2020-01-31
PT3505174T (pt) 2020-07-06
US20210244754A1 (en) 2021-08-12
JP6599484B2 (ja) 2019-10-30
RU2018101305A3 (enExample) 2019-10-31
IL275256A (en) 2020-07-30
CN107847512A (zh) 2018-03-27
KR20180039624A (ko) 2018-04-18
HK1252133A1 (zh) 2019-05-17
SI3505174T1 (sl) 2021-02-26
KR20220018623A (ko) 2022-02-15
AU2021250841A1 (en) 2021-11-04
US11666592B2 (en) 2023-06-06
AU2020204042B2 (en) 2021-07-15
MX389016B (es) 2025-03-20
CN116726035A (zh) 2023-09-12
CY1122605T1 (el) 2021-03-12
IL256331B (en) 2020-08-31
JP6675037B2 (ja) 2020-04-01
WO2016205671A9 (en) 2018-01-04
EP3310362B1 (en) 2019-08-28
ES2808148T3 (es) 2021-02-25
EP3569236A1 (en) 2019-11-20
AU2016280293B2 (en) 2020-07-02
WO2016205671A1 (en) 2016-12-22
HRP20200949T1 (hr) 2020-10-02
RS60572B1 (sr) 2020-08-31
IL275255A (en) 2020-07-30
AU2021250841B2 (en) 2023-02-09
IL275255B (en) 2021-04-29
AU2016280293A1 (en) 2018-02-08
EP3505174A1 (en) 2019-07-03
DK3505174T3 (da) 2020-07-13
US20230277577A1 (en) 2023-09-07
CA2989653A1 (en) 2016-12-22
SI3310362T1 (sl) 2020-02-28
RU2018101305A (ru) 2019-07-17
US11110111B2 (en) 2021-09-07
IL275256B2 (en) 2023-09-01
EP3505174B1 (en) 2020-04-29
SMT202000399T1 (it) 2020-09-10
US12251392B2 (en) 2025-03-18
JP2020019791A (ja) 2020-02-06
US20250360154A1 (en) 2025-11-27
IL256331A (en) 2018-02-28
PL3310362T3 (pl) 2020-04-30
ES2748556T3 (es) 2020-03-17
HUE050678T2 (hu) 2020-12-28
HUE046399T2 (hu) 2020-03-30
EP3310362A1 (en) 2018-04-25
HRP20191794T1 (hr) 2020-02-07
SMT201900571T1 (it) 2019-11-13
AU2020204042A1 (en) 2020-07-09
PL3505174T3 (pl) 2020-08-24
US20180133241A1 (en) 2018-05-17
JP2020019792A (ja) 2020-02-06
JP2018521993A (ja) 2018-08-09
MX2017016425A (es) 2018-12-11
MX2020000269A (es) 2022-01-03
US20200038424A1 (en) 2020-02-06
US10471087B2 (en) 2019-11-12
RU2721492C2 (ru) 2020-05-19
RU2020112901A (ru) 2020-06-05
LT3505174T (lt) 2020-09-10
EP3310362A4 (en) 2018-12-19
IL275256B1 (en) 2023-05-01
JP7036782B2 (ja) 2022-03-15
BR112017027079B1 (pt) 2021-04-27

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