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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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Denmark
Prior art keywords
mitocondrial
diseases caused
dna depletion
nucleotide pools
unbalanced nucleotide
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Application number
DK16812537.5T
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English (en)
Inventor
Michio Hirano
Caterina Garone
Ramon Marti
Original Assignee
Univ Columbia
Fundacio Hospital Univ Vall D Hebron Institut De Recerca
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Publication of DK3310362T3 publication Critical patent/DK3310362T3/da

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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
    • A61K31/33—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
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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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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    • A61K9/0053—Mouth and digestive tract, i.e. intraoral and peroral administration
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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
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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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Family Applications (2)

Application Number Title Priority Date Filing Date
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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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 (da)
EP (3) EP3505174B1 (da)
JP (3) JP6599484B2 (da)
KR (3) KR20220018623A (da)
CN (2) CN116726035A (da)
AU (3) AU2016280293B2 (da)
BR (2) BR112017027079B1 (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) HUE050678T2 (da)
IL (3) IL275256B2 (da)
LT (2) LT3505174T (da)
MX (2) MX389016B (da)
PL (2) PL3310362T3 (da)
PT (2) PT3505174T (da)
RS (2) RS60572B1 (da)
RU (1) RU2721492C2 (da)
SI (2) SI3505174T1 (da)
SM (2) SMT202000399T1 (da)
WO (1) WO2016205671A1 (da)

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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
LT3505174T (lt) * 2015-06-17 2020-09-10 The Trustees Of Columbia University In The City Of New York Deoksinukleozidų terapija, skirta gydyti ligas, kurias sukelia nesubalansuoti nukleotidų kiekiai, įskaitant mitochondrinės dnr išsekimo sindromus
US20170174614A1 (en) 2015-12-22 2017-06-22 Zogenix International Limited Metabolism resistant fenfluramine analogs and methods of using the same
CN108883399B (zh) 2015-12-22 2021-06-22 周格尼克斯国际有限公司 芬氟拉明组合物及其制备方法
RU2021106383A (ru) 2016-08-24 2021-04-05 Зодженикс Интернэшнл Лимитед Состав для ингибирования образования агонистов 5-ht2b и способы его применения
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
US11628182B2 (en) * 2018-04-12 2023-04-18 Zogenix Mds, Inc. Prodrugs of deoxynucleosides for treatment of diseases cased by unbalanced nucleotide pools
BR112020021324A2 (pt) 2018-04-18 2021-01-19 The Trustees Of Columbia University In The City Of New York Terapia gênica para doenças causadas por pools de nucleotídeos em desequilíbrio incluindo síndromes de depleção do dna mitocondrial
WO2019216919A1 (en) 2018-05-11 2019-11-14 Zogenix International Limited Compositions and methods for treating seizure-induced sudden death
US12144787B2 (en) 2018-11-19 2024-11-19 Zogenix International Limited Method of treating patients with a mutation in cyclin-dependent kinase-like 5 (CDKL5)
JP7769603B2 (ja) * 2019-08-19 2025-11-13 ユーシービー バイオサイエンシズ，インコーポレイテッド デオキシシチジンの多形形態、それを含む組成物および使用
JP2022546611A (ja) * 2019-09-05 2022-11-04 ミトレインボー セラピューティクス，インコーポレーテッド ミトコンドリアｄｎａ枯渇障害の処置
CN115667505A (zh) 2020-03-19 2023-01-31 因特利亚治疗公司 用于定向基因组编辑的方法和组合物
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
JP2024515025A (ja) 2021-03-26 2024-04-04 ゾジェニックス，インコーポレイテッド プリン及びピリミジンを含有する水性溶液及びその使用
AU2022291783A1 (en) * 2021-06-18 2024-02-01 Ucb Biosciences, Inc. Prodrugs of deoxynucleosides for treatment of mitochondrial diseases caused by unbalanced nucleotide pools
JP2025522100A (ja) * 2022-07-12 2025-07-10 ユーシービー バイオサイエンシス、インコーポレイテッド ヌクレオシド薬剤のバイオアベイラビリティを増加させる方法
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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CN101120947B (zh) * 1998-08-10 2010-11-24 艾丹尼克斯(开曼)有限公司 用于治疗乙型肝炎的β-L-2′-脱氧-核苷
ES2298130T3 (es) * 1999-02-23 2008-05-16 The Regents Of The University Of California Uso de triacetiluridina para el tratamiento de trastornos mitocondriales.
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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
IL289747B2 (en) * 2015-06-05 2025-10-01 Fundacio Hospital Univ Vall Dhebron Institut De Recerca Combination of deoxyribonucleosides for the treatment of mitochondrial DNA depletion syndrome and/or deletion syndrome
LT3505174T (lt) * 2015-06-17 2020-09-10 The Trustees Of Columbia University In The City Of New York Deoksinukleozidų terapija, skirta gydyti ligas, kurias sukelia nesubalansuoti nukleotidų kiekiai, įskaitant mitochondrinės dnr išsekimo sindromus

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

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