CN115397988A - 用于治疗疾病的反义寡聚体 - Google Patents

用于治疗疾病的反义寡聚体 Download PDF

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Publication number
CN115397988A
CN115397988A CN202180023548.6A CN202180023548A CN115397988A CN 115397988 A CN115397988 A CN 115397988A CN 202180023548 A CN202180023548 A CN 202180023548A CN 115397988 A CN115397988 A CN 115397988A
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ace2
aon
exon
mrna
expression
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CN202180023548.6A
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English (en)
Chinese (zh)
Inventor
S·威尔顿
M·昂-赫图特
M·C·托马斯
R·J·皮克林
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Murdoch University
Monash University
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Murdoch University
Monash University
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Priority claimed from AU2020900883A external-priority patent/AU2020900883A0/en
Application filed by Murdoch University, Monash University filed Critical Murdoch University
Publication of CN115397988A publication Critical patent/CN115397988A/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
    • C12N15/1137—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 against enzymes
    • 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
    • A61K31/7125—Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61P13/00—Drugs for disorders of the urinary system
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A61P3/08—Drugs for disorders of the metabolism for glucose homeostasis
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12—Antivirals
    • A61P31/14—Antivirals for RNA viruses
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/48—Hydrolases (3) acting on peptide bonds (3.4)
    • C12N9/485—Exopeptidases (3.4.11-3.4.19)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y304/00—Hydrolases acting on peptide bonds, i.e. peptidases (3.4)
    • C12Y304/17—Metallocarboxypeptidases (3.4.17)
    • C12Y304/17023—Angiotensin-converting enzyme 2 (3.4.17.23)
    • 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
    • A61K31/712—Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
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    • C12N2310/00—Structure or type of the nucleic acid
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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
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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
    • C12N2310/3233—Morpholino-type ring
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    • C12N2320/00—Applications; Uses
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    • C12N2320/31—Combination therapy
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    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/33—Alteration of splicing

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  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
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  • Plant Pathology (AREA)
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  • Physics & Mathematics (AREA)
  • Diabetes (AREA)
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  • Heart & Thoracic Surgery (AREA)
  • Emergency Medicine (AREA)
  • Endocrinology (AREA)
  • Hematology (AREA)
  • Obesity (AREA)
  • Urology & Nephrology (AREA)
  • Cardiology (AREA)
  • Vascular Medicine (AREA)
CN202180023548.6A 2020-03-23 2021-03-22 用于治疗疾病的反义寡聚体 Pending CN115397988A (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
AU2020900883A AU2020900883A0 (en) 2020-03-23 Antisense Oligomers for Treatment of Disease
AU2020900883 2020-03-23
PCT/AU2021/050261 WO2021189104A1 (en) 2020-03-23 2021-03-22 Antisense oligomers for treatment of disease

Publications (1)

Publication Number Publication Date
CN115397988A true CN115397988A (zh) 2022-11-25

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CN202180023548.6A Pending CN115397988A (zh) 2020-03-23 2021-03-22 用于治疗疾病的反义寡聚体

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US (1) US20230407309A1 (https=)
EP (1) EP4127174A4 (https=)
JP (1) JP2023519518A (https=)
CN (1) CN115397988A (https=)
AU (1) AU2021244774A1 (https=)
WO (1) WO2021189104A1 (https=)

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN114540384A (zh) * 2020-12-31 2022-05-27 中山大学附属第一医院 用于降低血管紧张素转换酶2（ace2）表达的寡核苷酸及其在治疗病毒感染中的用途

Families Citing this family (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP7722666B2 (ja) * 2020-07-28 2025-08-13 神戸天然物化学株式会社 アンギオテンシン変換酵素２遺伝子のエクソンのスキッピングを誘導するアンチセンス核酸
CA3256035A1 (en) * 2022-04-14 2023-10-19 Invisishield Technologies Ltd. COMPOSITIONS FOR PREVENTING OR TREATMENT OF CORONAVIRUS INFECTIONS
US20250333744A1 (en) * 2022-04-14 2025-10-30 Shift Pharmaceuticals Holding Inc. Polynucleotide Treatments for Charcot-Marie-Tooth Disease
WO2023201306A1 (en) 2022-04-14 2023-10-19 Invisishield Technologies Ltd. Compositions for preventing or treating influenza infections
WO2024233583A2 (en) * 2023-05-08 2024-11-14 Carisma Therapeutics Inc. Compositions and methods relating to intronic shrna chimeric antigen receptor constructs

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20070185044A1 (en) * 2005-03-08 2007-08-09 Dobie Kenneth W Modulation of ace2 expression
CN102676529A (zh) * 2006-05-19 2012-09-19 莱顿教学医院 一种反义寡核苷酸

Family Cites Families (4)

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Publication number Priority date Publication date Assignee Title
US6610497B1 (en) * 1997-12-11 2003-08-26 Millennium Pharmaceuticals, Inc. Angiotensin converting enzyme homolog and therapeutic and diagnostic uses therefor
US6989363B1 (en) * 1997-12-11 2006-01-24 Millennium Pharmaceuticals, Inc. Angiotensin converting enzyme homolog and therapeutic and diagnostic uses therefor
US20050203038A1 (en) * 2004-03-10 2005-09-15 Isis Pharmaceuticals Inc. Modulation of ACE2 expression
CN111979273B (zh) * 2020-08-24 2022-05-27 苏州启辰生物科技有限公司 一种制备人源化ace2小鼠模型的方法

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20070185044A1 (en) * 2005-03-08 2007-08-09 Dobie Kenneth W Modulation of ace2 expression
CN102676529A (zh) * 2006-05-19 2012-09-19 莱顿教学医院 一种反义寡核苷酸

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
AARTSMA-RUS A等: "Antisense-mediated exon skipping: a versatile tool with therapeutic and research applications", RNA, vol. 13, no. 10, 31 October 2007 (2007-10-31), pages 1609 - 1624 *
REHMAN SU等: "Alternative splicing of ACE2 possibly generates variants that may limit the entry of SARS-CoV-2: a potential therapeutic approach using SSOs", CLINICAL SCIENCE, vol. 134, no. 10, 29 May 2020 (2020-05-29), pages 1143 - 1150 *

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN114540384A (zh) * 2020-12-31 2022-05-27 中山大学附属第一医院 用于降低血管紧张素转换酶2（ace2）表达的寡核苷酸及其在治疗病毒感染中的用途

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WO2021189104A1 (en) 2021-09-30
EP4127174A4 (en) 2024-05-01
AU2021244774A1 (en) 2022-09-29
WO2021189104A9 (en) 2023-02-02
EP4127174A1 (en) 2023-02-08
JP2023519518A (ja) 2023-05-11
US20230407309A1 (en) 2023-12-21

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