JP7620781B2 - 不均衡なヌクレオチドプールによって引き起こされた疾患を治療するためのデオキシヌクレオシドのプロドラッグ - Google Patents

不均衡なヌクレオチドプールによって引き起こされた疾患を治療するためのデオキシヌクレオシドのプロドラッグ Download PDF

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JP7620781B2
JP7620781B2 JP2020556284A JP2020556284A JP7620781B2 JP 7620781 B2 JP7620781 B2 JP 7620781B2 JP 2020556284 A JP2020556284 A JP 2020556284A JP 2020556284 A JP2020556284 A JP 2020556284A JP 7620781 B2 JP7620781 B2 JP 7620781B2
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ディピエトロ，ダニエル
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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
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    • A—HUMAN NECESSITIES
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    • 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
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    • A61K31/44—Non condensed pyridines; Hydrogenated derivatives thereof
    • A61K31/4427—Non condensed pyridines; Hydrogenated derivatives thereof containing further heterocyclic ring systems
    • A61K31/4439—Non condensed pyridines; Hydrogenated derivatives thereof containing further heterocyclic ring systems containing a five-membered ring with nitrogen as a ring hetero atom, e.g. omeprazole
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    • A61K31/505—Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim
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    • A61K31/7052—Compounds having saccharide radicals and heterocyclic rings having nitrogen as a ring hetero atom, e.g. nucleosides, nucleotides
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    • A61K31/7068—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
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    • 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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    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/7125—Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
    • A—HUMAN NECESSITIES
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    • C07D401/14—Heterocyclic compounds containing two or more hetero rings, having nitrogen atoms as the only ring hetero atoms, at least one ring being a six-membered ring with only one nitrogen atom containing three or more hetero rings
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D487/00—Heterocyclic compounds containing nitrogen atoms as the only ring hetero atoms in the condensed system, not provided for by groups C07D451/00 - C07D477/00
    • C07D487/02—Heterocyclic compounds containing nitrogen atoms as the only ring hetero atoms in the condensed system, not provided for by groups C07D451/00 - C07D477/00 in which the condensed system contains two hetero rings
    • C07D487/04—Ortho-condensed systems
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    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
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  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Biochemistry (AREA)
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  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
JP2020556284A 2018-04-12 2019-04-12 不均衡なヌクレオチドプールによって引き起こされた疾患を治療するためのデオキシヌクレオシドのプロドラッグ Active JP7620781B2 (ja)

Priority Applications (1)

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JP2023215758A JP7784411B2 (ja) 2018-04-12 2023-12-21 不均衡なヌクレオチドプールによって引き起こされた疾患を治療するためのデオキシヌクレオシドのプロドラッグ

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US201862656861P 2018-04-12 2018-04-12
US62/656,861 2018-04-12
PCT/US2019/027364 WO2019200340A1 (en) 2018-04-12 2019-04-12 Prodrugs of deoxynucleosides for treatment of diseases cased by unbalanced nucleotide pools

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JP2023215758A Division JP7784411B2 (ja) 2018-04-12 2023-12-21 不均衡なヌクレオチドプールによって引き起こされた疾患を治療するためのデオキシヌクレオシドのプロドラッグ

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JP2021521210A JP2021521210A (ja) 2021-08-26
JP2021521210A5 JP2021521210A5 (https=) 2022-04-19
JPWO2019200340A5 JPWO2019200340A5 (https=) 2022-04-19
JP7620781B2 true JP7620781B2 (ja) 2025-01-24

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JP2023215758A Active JP7784411B2 (ja) 2018-04-12 2023-12-21 不均衡なヌクレオチドプールによって引き起こされた疾患を治療するためのデオキシヌクレオシドのプロドラッグ

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US (3) US11628182B2 (https=)
EP (1) EP3773603A4 (https=)
JP (2) JP7620781B2 (https=)
CN (1) CN112384228A (https=)
AU (2) AU2019253023B2 (https=)
BR (1) BR112020020804A2 (https=)
CA (1) CA3096264A1 (https=)
IL (1) IL277725B2 (https=)
MX (1) MX2020010667A (https=)
WO (1) WO2019200340A1 (https=)

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US11628182B2 (en) 2018-04-12 2023-04-18 Zogenix Mds, Inc. Prodrugs of deoxynucleosides for treatment of diseases cased by unbalanced nucleotide pools
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

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20160279159A1 (en) 2015-03-26 2016-09-29 The Trustees Of Columbia University In The City Of New York Deoxyribonucleoside monophospate bypass therapy for mitochondrial dna depletion syndrome
WO2016205671A1 (en) 2015-06-17 2016-12-22 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
WO2017087517A1 (en) 2015-11-16 2017-05-26 Demeter Therapeutics, Llc Nucleic acid prodrugs

Family Cites Families (7)

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Publication number Priority date Publication date Assignee Title
US5432272A (en) 1990-10-09 1995-07-11 Benner; Steven A. Method for incorporating into a DNA or RNA oligonucleotide using nucleotides bearing heterocyclic bases
EP0753525A4 (en) 1994-03-31 1997-09-03 Nippon Shinyaku Co Ltd CYCLIC NUCLEOTIDE DERIVATIVES
US6875751B2 (en) 2000-06-15 2005-04-05 Idenix Pharmaceuticals, Inc. 3′-prodrugs of 2′-deoxy-β-L-nucleosides
MY141594A (en) 2000-06-15 2010-05-14 Novirio Pharmaceuticals Ltd 3'-PRODRUGS OF 2'-DEOXY-ß-L-NUCLEOSIDES
WO2002057425A2 (en) 2001-01-22 2002-07-25 Merck & Co., Inc. Nucleoside derivatives as inhibitors of rna-dependent rna viral polymerase
BR112018076913A2 (pt) 2016-06-24 2019-04-02 Emory University fosforamidatos para o tratamento do vírus da hepatite b
US11628182B2 (en) 2018-04-12 2023-04-18 Zogenix Mds, Inc. Prodrugs of deoxynucleosides for treatment of diseases cased by unbalanced nucleotide pools

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20160279159A1 (en) 2015-03-26 2016-09-29 The Trustees Of Columbia University In The City Of New York Deoxyribonucleoside monophospate bypass therapy for mitochondrial dna depletion syndrome
WO2016205671A1 (en) 2015-06-17 2016-12-22 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
WO2017087517A1 (en) 2015-11-16 2017-05-26 Demeter Therapeutics, Llc Nucleic acid prodrugs

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EP3773603A1 (en) 2021-02-17
IL277725B2 (en) 2024-02-01
IL277725A (en) 2020-11-30
US20230181617A1 (en) 2023-06-15
WO2019200340A1 (en) 2019-10-17
JP7784411B2 (ja) 2025-12-11
US12419901B2 (en) 2025-09-23
US20210077519A1 (en) 2021-03-18
JP2021521210A (ja) 2021-08-26
JP2024038011A (ja) 2024-03-19
US11628182B2 (en) 2023-04-18
CN112384228A (zh) 2021-02-19
AU2019253023A1 (en) 2020-11-12
BR112020020804A2 (pt) 2021-01-12
MX2020010667A (es) 2021-01-08
IL277725B1 (en) 2023-10-01
US20230181616A1 (en) 2023-06-15
EP3773603A4 (en) 2022-03-30
AU2019253023B2 (en) 2025-01-23
AU2025201857A1 (en) 2025-04-03
CA3096264A1 (en) 2019-10-17

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