JP2024535888A - インヒビンサブユニットベータｅ（ｉｎｈｂｅ）モジュレーター組成物およびその使用方法 - Google Patents

インヒビンサブユニットベータｅ（ｉｎｈｂｅ）モジュレーター組成物およびその使用方法 Download PDF

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JP2024535888A
JP2024535888A JP2024517496A JP2024517496A JP2024535888A JP 2024535888 A JP2024535888 A JP 2024535888A JP 2024517496 A JP2024517496 A JP 2024517496A JP 2024517496 A JP2024517496 A JP 2024517496A JP 2024535888 A JP2024535888 A JP 2024535888A
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nucleotides
modulator
inhbe
ome
length
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JP2024535888A5 (enExample
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エム ディートン，エイミー
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Alnylam Pharmaceuticals Inc
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Alnylam Pharmaceuticals Inc
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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
    • C12N15/1136—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 growth factors, growth regulators, cytokines, lymphokines or hormones
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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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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
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
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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
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/322—2'-R Modification
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/33—Chemical structure of the base
    • C12N2310/334—Modified C
    • C12N2310/3341—5-Methylcytosine
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/341—Gapmers, i.e. of the type ===---===
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    • 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
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    • 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

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  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
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  • General Engineering & Computer Science (AREA)
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  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Endocrinology (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Cardiology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Heart & Thoracic Surgery (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Epidemiology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicinal Preparation (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
  • Peptides Or Proteins (AREA)
JP2024517496A 2021-09-20 2022-09-19 インヒビンサブユニットベータｅ（ｉｎｈｂｅ）モジュレーター組成物およびその使用方法 Pending JP2024535888A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202163246084P 2021-09-20 2021-09-20
US63/246,084 2021-09-20
PCT/US2022/043948 WO2023044094A1 (en) 2021-09-20 2022-09-19 Inhibin subunit beta e (inhbe) modulator compositions and methods of use thereof

Publications (2)

Publication Number Publication Date
JP2024535888A true JP2024535888A (ja) 2024-10-02
JP2024535888A5 JP2024535888A5 (enExample) 2025-09-29

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JP2024517496A Pending JP2024535888A (ja) 2021-09-20 2022-09-19 インヒビンサブユニットベータｅ（ｉｎｈｂｅ）モジュレーター組成物およびその使用方法

Country Status (12)

Country Link
US (1) US20250051779A1 (enExample)
EP (1) EP4405478A1 (enExample)
JP (1) JP2024535888A (enExample)
KR (1) KR20240083183A (enExample)
CN (1) CN118159654A (enExample)
AU (1) AU2022345881A1 (enExample)
CA (1) CA3232420A1 (enExample)
CL (1) CL2024000821A1 (enExample)
CO (1) CO2024004482A2 (enExample)
IL (1) IL311454A (enExample)
MX (1) MX2024003157A (enExample)
WO (1) WO2023044094A1 (enExample)

Families Citing this family (18)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2024331015A1 (en) 2023-08-30 2026-02-26 Arrowhead Pharmaceuticals, Inc. Rnai agents for inhibiting expression of inhibin subunit beta e (inhbe), pharmaceutical compositions thereof, and methods of use
EP4770650A2 (en) * 2023-09-08 2026-07-08 Wave Life Sciences Ltd. Inhibin subunit beta e-related double stranded oligonucleotide compositions and methods relating thereto
WO2025082057A1 (zh) * 2023-10-16 2025-04-24 润佳(上海)医药技术有限公司 抑制抑制素Beta E基因表达的抑制剂及其用途
IL330083A (en) * 2023-12-21 2026-08-01 Shanghai Argo Biopharmaceutical Co Ltd Compositions and methods for inhibiting inhibin beta subunit E (INHBE) expression
TW202539702A (zh) * 2024-03-08 2025-10-16 大陸商上海拓界生物醫藥科技有限公司 靶向ＩＮＨＢＥ的ＲＮＡｉ劑
WO2025193754A2 (en) * 2024-03-11 2025-09-18 Basecure Therapeutics Llc Compositions and methods for inhibition of expression of inhibin subunit beta e (inhbe) genes
WO2025223537A1 (zh) * 2024-04-26 2025-10-30 信达生物制药(苏州)有限公司 一种dsRNA、其应用及制备方法
WO2025228441A2 (en) * 2024-04-30 2025-11-06 Sanegene Bio Usa Inc. Small interfering rna targeting inhbe and uses thereof
WO2025247212A1 (zh) * 2024-05-27 2025-12-04 大睿生物医药科技(上海)有限公司 调控INHBE表达的dsRNA分子
WO2025256588A1 (zh) * 2024-06-13 2025-12-18 苏州时安生物技术有限公司 一种调节inhbe表达的双链rna组合物、其缀合物和药物组合物及用途
WO2025264948A2 (en) * 2024-06-21 2025-12-26 Eli Lilly And Company Inhbe rna interference agents
WO2026006275A2 (en) * 2024-06-26 2026-01-02 Amgen Inc. Rnai constructs and methods for inhibiting expression of inhbe
WO2026007966A1 (zh) * 2024-07-01 2026-01-08 武汉人福创新药物研发中心有限公司 抑制INHBE基因表达的siRNA、偶联物及其应用
TW202606729A (zh) * 2024-07-03 2026-02-16 美商靖因藥業（上海）有限公司 靶向inhbe之聚核酸分子及其用途
WO2026021537A1 (en) * 2024-07-26 2026-01-29 Qilu Pharmaceutical Co., Ltd. iRNA COMPOSITION AND METHOD OF USING SAME
WO2026026983A1 (zh) * 2024-08-02 2026-02-05 北京安龙生物医药有限公司 靶向inhbe基因的寡核苷酸及其用途
WO2026052140A1 (zh) * 2024-09-06 2026-03-12 北京福元医药股份有限公司 用于抑制inhbe基因表达的双链核糖核酸及其修饰物、缀合物和用途
WO2026132332A2 (en) 2024-12-18 2026-06-25 Silence Therapeutics Gmbh Nucleic acids for inhibiting expression of inhbe in a cell

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