JP2023547274A - マイクロｒｎａ－２９の化合物、組成物、および治療法における使用 - Google Patents

マイクロｒｎａ－２９の化合物、組成物、および治療法における使用 Download PDF

Info

Publication number
JP2023547274A
JP2023547274A JP2023549131A JP2023549131A JP2023547274A JP 2023547274 A JP2023547274 A JP 2023547274A JP 2023549131 A JP2023549131 A JP 2023549131A JP 2023549131 A JP2023549131 A JP 2023549131A JP 2023547274 A JP2023547274 A JP 2023547274A
Authority
JP
Japan
Prior art keywords
mir
compound
salt
dose
cwt
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Pending
Application number
JP2023549131A
Other languages
English (en)
Japanese (ja)
Other versions
JP2023547274A5 (enExample
Inventor
スチュワート ギルクライスト，デレク
リンジィ ミラー，ニール
Original Assignee
コーズウェイ セラピューティクス リミテッド
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by コーズウェイ セラピューティクス リミテッド filed Critical コーズウェイ セラピューティクス リミテッド
Publication of JP2023547274A publication Critical patent/JP2023547274A/ja
Publication of JP2023547274A5 publication Critical patent/JP2023547274A5/ja
Pending legal-status Critical Current

Links

Images

Classifications

    • 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
    • C12N2310/141—MicroRNAs, miRNAs
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/322—2'-R Modification
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/344—Position-specific modifications, e.g. on every purine, at the 3'-end
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C12N2310/3515—Lipophilic moiety, e.g. cholesterol
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/35—Special therapeutic applications based on a specific dosage / administration regimen

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biochemistry (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicinal Preparation (AREA)
JP2023549131A 2020-10-23 2021-10-25 マイクロｒｎａ－２９の化合物、組成物、および治療法における使用 Pending JP2023547274A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
GBGB2016863.9A GB202016863D0 (en) 2020-10-23 2020-10-23 Microrna-29 compounds, compositions and uses in therapy
GB2016863.9 2020-10-23
PCT/EP2021/079580 WO2022084561A2 (en) 2020-10-23 2021-10-25 Microrna-29 compounds, compositions and uses in therapy

Publications (2)

Publication Number Publication Date
JP2023547274A true JP2023547274A (ja) 2023-11-09
JP2023547274A5 JP2023547274A5 (enExample) 2024-11-01

Family

ID=73726994

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2023549131A Pending JP2023547274A (ja) 2020-10-23 2021-10-25 マイクロｒｎａ－２９の化合物、組成物、および治療法における使用

Country Status (9)

Country Link
US (1) US20240390410A1 (enExample)
EP (1) EP4232580A2 (enExample)
JP (1) JP2023547274A (enExample)
KR (1) KR20230088810A (enExample)
CN (1) CN116744942A (enExample)
AU (1) AU2021365394A1 (enExample)
CA (1) CA3195977A1 (enExample)
GB (1) GB202016863D0 (enExample)
WO (1) WO2022084561A2 (enExample)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2023445724A1 (en) * 2023-04-28 2025-11-06 Causeway Therapeutics Limited Microrna-29 compounds, compositions and uses in therapy, and methods for evaluating tendinopathic lesions

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2017521374A (ja) * 2014-05-29 2017-08-03 クォーク ファーマシューティカルズ インコーポレーティッドＱｕａｒｋ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ，Ｉｎｃ． 臓器の虚血再灌流傷害を予防するための方法及び組成物
JP2017527294A (ja) * 2014-09-08 2017-09-21 ミラゲン セラピューティクス， インコーポレイテッド Ｍｉｒ−２９模倣物およびその使用
US20200318113A1 (en) * 2019-03-05 2020-10-08 MiRagen Therapeutics, Inc. Polynucleotide conjugates and uses thereof

Family Cites Families (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
GB201400598D0 (en) * 2014-01-14 2014-03-05 Univ Glasgow Materials and methods for modulation of tendon healing
GB201503967D0 (en) * 2015-03-09 2015-04-22 Univ Glasgow Biocompatible implants for use in tendon therapy

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2017521374A (ja) * 2014-05-29 2017-08-03 クォーク ファーマシューティカルズ インコーポレーティッドＱｕａｒｋ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ，Ｉｎｃ． 臓器の虚血再灌流傷害を予防するための方法及び組成物
JP2017527294A (ja) * 2014-09-08 2017-09-21 ミラゲン セラピューティクス， インコーポレイテッド Ｍｉｒ−２９模倣物およびその使用
US20200318113A1 (en) * 2019-03-05 2020-10-08 MiRagen Therapeutics, Inc. Polynucleotide conjugates and uses thereof

Also Published As

Publication number Publication date
EP4232580A2 (en) 2023-08-30
CA3195977A1 (en) 2022-04-28
KR20230088810A (ko) 2023-06-20
US20240390410A1 (en) 2024-11-28
WO2022084561A3 (en) 2022-06-02
AU2021365394A1 (en) 2023-06-08
WO2022084561A2 (en) 2022-04-28
CN116744942A (zh) 2023-09-12
GB202016863D0 (en) 2020-12-09

Similar Documents

Publication Publication Date Title
TWI477273B (zh) 低頻率格拉替雷（ｇｌａｔｉｒａｍｅｒ）醋酸鹽之治療
JP2020073479A (ja) アルポート症候群の処置方法
US20210008054A1 (en) Use of cdk9 inhibitors to reduce cartilage degradation
JP2010510807A (ja) 高コレステロール血症を治療するための方法
TW201244724A (en) Method of treating keloids or hypertrophic scars using antisense compounds targeting connective tissue growth factor (CTGF)
JP2022526763A (ja) カシメルセンによる筋ジストロフィーの治療方法
TW202300647A (zh) 用於治療多囊腎病之組成物
JP2005314381A (ja) 増殖性腎疾患の予防・治療・改善剤
JP2026021335A (ja) ａｐｏ（ａ）を標的化するコンジュゲートアンチセンス化合物を用いて心血管事象のリスクを低減する方法
CN120957731A (zh) 用于治疗多囊肾病的修饰的寡核苷酸
JP2023547274A (ja) マイクロｒｎａ－２９の化合物、組成物、および治療法における使用
JP2024012436A (ja) 食道狭窄抑制剤
CN104083761A (zh) microRNA-101抑制剂在制备预防或治疗骨关节炎药物中的应用
US20240191233A1 (en) Compounds and methods for reducing nlrp3 expression
US20240360446A1 (en) Microrna-29 compounds, compositions and uses in therapy, and methods for evaluating tendinopathic lesions
JP7514563B2 (ja) オルガノイドおよび抗炎症剤を含む炎症性腸疾患の予防または治療用薬学組成物
FR3014693A1 (fr) Utilisation de la 3-(2-chloro-phenyl)-1-[4-(4-fluoro-benzyl)-piperazin-1-yl]-propenone pour la prevention et/ou le traitement des pathologies fonctionnelles pelvi-perineales
CN115381950B (zh) Rab 35或其下游调控分子在治疗骨折后慢性疼痛中的应用
WO2020100089A2 (en) Compositions and methods for the treatment of smooth muscle dysfunction
JP2025538360A (ja) 阻害性核酸およびその使用方法
KR20260061479A (ko) 식도 협착 억제제
WO2005120580A2 (en) Compositions and methods for treatment of fibro-proliferative disorders

Legal Events

Date Code Title Description
A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20230703

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20241024

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20241024

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20250930

A977 Report on retrieval

Free format text: JAPANESE INTERMEDIATE CODE: A971007

Effective date: 20250930

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20251224

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20260224