CN117500510A - 用于治疗由失衡的核苷酸池引起的线粒体疾病的脱氧核苷的前药 - Google Patents

用于治疗由失衡的核苷酸池引起的线粒体疾病的脱氧核苷的前药 Download PDF

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Publication number
CN117500510A
CN117500510A CN202280043337.3A CN202280043337A CN117500510A CN 117500510 A CN117500510 A CN 117500510A CN 202280043337 A CN202280043337 A CN 202280043337A CN 117500510 A CN117500510 A CN 117500510A
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China
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optionally substituted
alkyl
prodrug
group
independently selected
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CN202280043337.3A
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English (en)
Chinese (zh)
Inventor
D·迪皮特罗
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Ushibi Bioscience Co
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Zhouge Knicks Co
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Publication of CN117500510A publication Critical patent/CN117500510A/zh
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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
    • A61K31/7076—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
    • 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
    • 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
    • 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

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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)
CN202280043337.3A 2021-06-18 2022-06-15 用于治疗由失衡的核苷酸池引起的线粒体疾病的脱氧核苷的前药 Pending CN117500510A (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202163212468P 2021-06-18 2021-06-18
US63/212468 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 (1)

Publication Number Publication Date
CN117500510A true CN117500510A (zh) 2024-02-02

Family

ID=84526701

Family Applications (1)

Application Number Title Priority Date Filing Date
CN202280043337.3A Pending CN117500510A (zh) 2021-06-18 2022-06-15 用于治疗由失衡的核苷酸池引起的线粒体疾病的脱氧核苷的前药

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

Also Published As

Publication number Publication date
AU2022291783A1 (en) 2024-02-01
JP2024522783A (ja) 2024-06-21
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

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Legal Events

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PB01 Publication
PB01 Publication
SE01 Entry into force of request for substantive examination
SE01 Entry into force of request for substantive examination
TA01 Transfer of patent application right

Effective date of registration: 20241118

Address after: North Carolina, USA

Applicant after: Ushibi Bioscience Co.

Country or region after: U.S.A.

Address before: California, USA

Applicant before: Zhouge Knicks Co.

Country or region before: U.S.A.

TA01 Transfer of patent application right