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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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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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
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    • 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
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    • 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
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    • A61K9/0053—Mouth and digestive tract, i.e. intraoral and peroral administration
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    • C12Q1/6813—Hybridisation assays
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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 (https=)
EP (3) EP3505174B1 (https=)
JP (3) JP6599484B2 (https=)
KR (3) KR20250048602A (https=)
CN (2) CN107847512A (https=)
AU (3) AU2016280293B2 (https=)
BR (2) BR112017027079B1 (https=)
CA (1) CA2989653A1 (https=)
CY (2) CY1122605T1 (https=)
DK (2) DK3310362T3 (https=)
ES (2) ES2748556T3 (https=)
HK (1) HK1252133A1 (https=)
HR (2) HRP20191794T1 (https=)
HU (2) HUE046399T2 (https=)
IL (3) IL275256B2 (https=)
LT (2) LT3505174T (https=)
MX (2) MX2017016425A (https=)
PL (2) PL3505174T3 (https=)
PT (2) PT3505174T (https=)
RS (2) RS60572B1 (https=)
RU (1) RU2721492C2 (https=)
SI (2) SI3310362T1 (https=)
SM (2) SMT202000399T1 (https=)
WO (1) WO2016205671A1 (https=)

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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
WO2017112701A1 (en) 2015-12-22 2017-06-29 Zogenix International Limited Metabolism resistant fenfluramine analogs and methods of using the same
ES3024232T3 (en) 2015-12-22 2025-06-04 Zogenix International Ltd Fenfluramine compositions and methods of preparing the same
SG11201900975XA (en) 2016-08-24 2019-03-28 Zogenix International Ltd 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
CA3096264A1 (en) * 2018-04-12 2019-10-17 Modis Therapeutics Inc. Prodrugs of deoxynucleosides for treatment of diseases cased by unbalanced nucleotide pools
CN112312929A (zh) 2018-04-18 2021-02-02 纽约市哥伦比亚大学理事会 用于包括线粒体dna耗竭综合征在内的由不平衡的核苷酸库引起的疾病的基因疗法
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
CN114929240B (zh) * 2019-08-19 2024-10-29 周格尼克斯公司 脱氧胞苷的多晶型形式、包含其的组合物和用途
IL322998A (en) * 2019-09-05 2025-10-01 Mitorainbow Therapeutics Inc Treatment of mitochondrial DNA depletion disorders
JP2023518395A (ja) 2020-03-19 2023-05-01 インテリア セラピューティクス，インコーポレイテッド 指向性ゲノム編集のための方法及び組成物
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
US20240041910A1 (en) * 2020-12-28 2024-02-08 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
US20230000890A1 (en) * 2021-06-18 2023-01-05 Zogenix Mds, Inc. Prodrugs of Deoxynucleosides for Treatment of Mitochondrial Diseases Caused by Unbalanced Nucleotide Pools
CA3261416A1 (en) * 2022-07-12 2024-01-18 Ucb Biosciences, Inc. METHODS TO INCREASE THE BIOAVAILABILITY OF NUCLEOSID 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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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
EP1808177B1 (en) * 1999-02-23 2018-08-08 The Regents of The University of California Methods of treatment of mitochondrial disorders
EA005890B1 (ru) * 2000-05-26 2005-06-30 Айденикс (Кайман) Лимитед СПОСОБЫ ЛЕЧЕНИЯ ИНФЕКЦИЙ, ВЫЗЫВАЕМЫХ ВИРУСОМ ГЕПАТИТА ДЕЛЬТА, С ИСПОЛЬЗОВАНИЕМ β-L-2'-ДЕЗОКСИНУКЛЕОЗИДОВ
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
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
BR112017027079B1 (pt) 2021-04-27
AU2020204042B2 (en) 2021-07-15
LT3505174T (lt) 2020-09-10
LT3310362T (lt) 2019-11-11
RU2020112901A (ru) 2020-06-05
RS59724B1 (sr) 2020-01-31
WO2016205671A1 (en) 2016-12-22
CN107847512A (zh) 2018-03-27
ES2748556T3 (es) 2020-03-17
HUE046399T2 (hu) 2020-03-30
HRP20191794T1 (hr) 2020-02-07
SMT202000399T1 (it) 2020-09-10
KR20220018623A (ko) 2022-02-15
JP2018521993A (ja) 2018-08-09
HRP20200949T1 (hr) 2020-10-02
US11666592B2 (en) 2023-06-06
KR20180039624A (ko) 2018-04-18
US20250360154A1 (en) 2025-11-27
EP3505174B1 (en) 2020-04-29
KR20250048602A (ko) 2025-04-09
MX2017016425A (es) 2018-12-11
JP7036782B2 (ja) 2022-03-15
SI3310362T1 (sl) 2020-02-28
JP6599484B2 (ja) 2019-10-30
US12251392B2 (en) 2025-03-18
IL275255A (en) 2020-07-30
AU2016280293A1 (en) 2018-02-08
CA2989653A1 (en) 2016-12-22
EP3505174A1 (en) 2019-07-03
JP2020019791A (ja) 2020-02-06
EP3310362B1 (en) 2019-08-28
IL275256A (en) 2020-07-30
CY1122605T1 (el) 2021-03-12
SMT201900571T1 (it) 2019-11-13
PT3505174T (pt) 2020-07-06
US20210244754A1 (en) 2021-08-12
RU2018101305A (ru) 2019-07-17
IL256331B (en) 2020-08-31
CN116726035A (zh) 2023-09-12
WO2016205671A9 (en) 2018-01-04
IL275256B1 (en) 2023-05-01
BR122020021913B1 (pt) 2021-07-06
HUE050678T2 (hu) 2020-12-28
IL256331A (en) 2018-02-28
EP3310362A1 (en) 2018-04-25
EP3310362A4 (en) 2018-12-19
MX389016B (es) 2025-03-20
US20180133241A1 (en) 2018-05-17
AU2016280293B2 (en) 2020-07-02
JP6675037B2 (ja) 2020-04-01
DK3505174T3 (da) 2020-07-13
EP3569236A1 (en) 2019-11-20
MX2020000269A (es) 2022-01-03
HK1252133A1 (zh) 2019-05-17
PL3310362T3 (pl) 2020-04-30
US10471087B2 (en) 2019-11-12
RU2721492C2 (ru) 2020-05-19
PL3505174T3 (pl) 2020-08-24
IL275256B2 (en) 2023-09-01
SI3505174T1 (sl) 2021-02-26
AU2021250841A1 (en) 2021-11-04
US20200038424A1 (en) 2020-02-06
IL275255B (en) 2021-04-29
US11110111B2 (en) 2021-09-07
CY1123107T1 (el) 2021-10-29
ES2808148T3 (es) 2021-02-25
AU2021250841B2 (en) 2023-02-09
AU2020204042A1 (en) 2020-07-09
RS60572B1 (sr) 2020-08-31
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