JP2024522783A - 不均衡なヌクレオチドプールによって引き起こされたミトコンドリア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ミトコンドリア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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JP2024522783A
JP2024522783A JP2023577845A JP2023577845A JP2024522783A JP 2024522783 A JP2024522783 A JP 2024522783A JP 2023577845 A JP2023577845 A JP 2023577845A JP 2023577845 A JP2023577845 A JP 2023577845A JP 2024522783 A JP2024522783 A JP 2024522783A
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prodrug
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aryl
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Japanese (ja)
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JP2024522783A5 (https=
JPWO2022266237A5 (https=
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ディピエトロ，ダニエル
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ゾジェニックス，インコーポレイテッド
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Publication of JP2024522783A5 publication Critical patent/JP2024522783A5/ja
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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
    • 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
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • 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
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • 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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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Medicinal Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Molecular Biology (AREA)
  • Organic Chemistry (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Nutrition Science (AREA)
  • Physiology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Saccharide Compounds (AREA)
JP2023577845A 2021-06-18 2022-06-15 不均衡なヌクレオチドプールによって引き起こされたミトコンドリア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 Pending JP2024522783A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202163212468P 2021-06-18 2021-06-18
US63/212,468 2021-06-18
PCT/US2022/033654 WO2022266237A1 (en) 2021-06-18 2022-06-15 Prodrugs of deoxynucleosides for treatment of mitochondrial diseases caused by unbalanced nucleotide pools

Publications (3)

Publication Number Publication Date
JP2024522783A true JP2024522783A (ja) 2024-06-21
JP2024522783A5 JP2024522783A5 (https=) 2025-06-24
JPWO2022266237A5 JPWO2022266237A5 (https=) 2025-06-24

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ID=84526701

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JP2023577845A Pending JP2024522783A (ja) 2021-06-18 2022-06-15 不均衡なヌクレオチドプールによって引き起こされたミトコンドリア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

Country Status (9)

Country Link
US (2) US20230000890A1 (https=)
EP (1) EP4355336A4 (https=)
JP (1) JP2024522783A (https=)
CN (1) CN117500510A (https=)
AU (1) AU2022291783A1 (https=)
CA (1) CA3222484A1 (https=)
IL (1) IL309421A (https=)
MX (1) MX2023014944A (https=)
WO (1) WO2022266237A1 (https=)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP4313068A4 (en) * 2021-03-26 2024-12-25 UCB Biosciences, Inc. Aqueous solutions containing purines and pyrimidines and uses thereof

Family Cites Families (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
JP6599484B2 (ja) * 2015-06-17 2019-10-30 ザ・トラスティーズ・オブ・コロンビア・ユニバーシティ・イン・ザ・シティ・オブ・ニューヨーク ミトコンドリアｄｎａ枯渇症候群を含む不均衡なヌクレオチドプールによって引き起こされる疾患のためのデオキシヌクレオシド療法
US10745435B2 (en) * 2015-11-16 2020-08-18 Cerecor, Inc. Nucleic acid prodrugs
US11628182B2 (en) * 2018-04-12 2023-04-18 Zogenix Mds, Inc. Prodrugs of deoxynucleosides for treatment of diseases cased by unbalanced nucleotide pools

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Publication number Publication date
AU2022291783A1 (en) 2024-02-01
US20230000890A1 (en) 2023-01-05
EP4355336A1 (en) 2024-04-24
EP4355336A4 (en) 2025-05-07
US20250049836A1 (en) 2025-02-13
CA3222484A1 (en) 2022-12-22
IL309421A (en) 2024-02-01
MX2023014944A (es) 2024-04-25
WO2022266237A1 (en) 2022-12-22
CN117500510A (zh) 2024-02-02

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