CN117980479A - 用于治疗基于无义介导的rna衰变的病状和疾病的反义寡聚体 - Google Patents

用于治疗基于无义介导的rna衰变的病状和疾病的反义寡聚体 Download PDF

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Publication number
CN117980479A
CN117980479A CN202280057124.6A CN202280057124A CN117980479A CN 117980479 A CN117980479 A CN 117980479A CN 202280057124 A CN202280057124 A CN 202280057124A CN 117980479 A CN117980479 A CN 117980479A
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China
Prior art keywords
nucleotides
fold
mrna
phip
exon
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Pending
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CN202280057124.6A
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Chinese (zh)
Inventor
伊莎贝尔·阿兹纳雷兹
安娜·科里奥内罗·赛斯
雅各布·艾伯特·卡赫
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Stoke Therapeutics Inc
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Stoke Therapeutics Inc
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Publication of CN117980479A publication Critical patent/CN117980479A/zh
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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • 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
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/11—Antisense
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
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    • C12N2310/314—Phosphoramidates
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/32—Chemical structure of the sugar
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/323—Chemical structure of the sugar modified ring structure
    • C12N2310/3231—Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
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    • C12N2310/3233—Morpholino-type ring
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    • C12N2320/00—Applications; Uses
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    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/33—Alteration of splicing

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  • Health & Medical Sciences (AREA)
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  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
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  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
CN202280057124.6A 2021-06-21 2022-06-21 用于治疗基于无义介导的rna衰变的病状和疾病的反义寡聚体 Pending CN117980479A (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202163212981P 2021-06-21 2021-06-21
US63/212,981 2021-06-21
PCT/US2022/034344 WO2022271699A2 (en) 2021-06-21 2022-06-21 Antisense oligomers for treatment of non-sense mediated rna decay based conditions and diseases

Publications (1)

Publication Number Publication Date
CN117980479A true CN117980479A (zh) 2024-05-03

Family

ID=84544915

Family Applications (1)

Application Number Title Priority Date Filing Date
CN202280057124.6A Pending CN117980479A (zh) 2021-06-21 2022-06-21 用于治疗基于无义介导的rna衰变的病状和疾病的反义寡聚体

Country Status (5)

Country Link
US (1) US20240254488A1 (https=)
EP (1) EP4359538A4 (https=)
JP (1) JP2024524974A (https=)
CN (1) CN117980479A (https=)
WO (1) WO2022271699A2 (https=)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN115867657A (zh) 2020-05-11 2023-03-28 斯托克制药公司 用于治疗疾患和疾病的opa1反义寡聚物
TW202504646A (zh) * 2023-06-16 2025-02-01 北京大學 利用環狀ｒｎａ抑制無義介導的降解的系統和方法

Family Cites Families (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA2408632A1 (en) * 2000-05-11 2001-11-15 Maria Rozakis-Adcock Ph domain-interacting protein
US20130288967A1 (en) * 2012-04-25 2013-10-31 Euclid Diagnostics Llc Materials and methods for cancer diagnosis by evaluation of the methylation status of cpg islands on chromosomes 6 and 8
WO2015191780A2 (en) * 2014-06-10 2015-12-17 The General Hospital Corporation Ccctc-binding factor (ctcf) rna interactome
HUE070436T2 (hu) * 2017-10-23 2025-06-28 Stoke Therapeutics Inc Antiszensz oligomerek nonszensz-mediált RNS-lebomláson alapuló állapotok és betegségek kezelésére
EP4017979A4 (en) * 2019-08-19 2024-03-27 Stoke Therapeutics, Inc. COMPOSITIONS AND METHODS FOR MODULATING PROTEIN SPLICING AND EXPRESSION

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Publication number Publication date
WO2022271699A3 (en) 2023-02-09
JP2024524974A (ja) 2024-07-09
WO2022271699A2 (en) 2022-12-29
US20240254488A1 (en) 2024-08-01
EP4359538A4 (en) 2025-09-17
EP4359538A2 (en) 2024-05-01

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