CA3226887A1 - Metabolic disorder-associated target gene irna compositions and methods of use thereof - Google Patents

Metabolic disorder-associated target gene irna compositions and methods of use thereof Download PDF

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Publication number
CA3226887A1
CA3226887A1 CA3226887A CA3226887A CA3226887A1 CA 3226887 A1 CA3226887 A1 CA 3226887A1 CA 3226887 A CA3226887 A CA 3226887A CA 3226887 A CA3226887 A CA 3226887A CA 3226887 A1 CA3226887 A1 CA 3226887A1
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Prior art keywords
dsrna agent
nucleotide
nucleotides
dsrna
strand
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CA3226887A
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English (en)
French (fr)
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Aimee M. DEATON
Jeffrey ZUBER
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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
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • 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
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    • 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
    • C12N15/1138—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 receptors or cell surface proteins
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    • C12Y—ENZYMES
    • C12Y301/00—Hydrolases acting on ester bonds (3.1)
    • C12Y301/04—Phosphoric diester hydrolases (3.1.4)
    • C12Y301/04017—3',5'-Cyclic-nucleotide phosphodiesterase (3.1.4.17)
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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]
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/312—Phosphonates
    • C12N2310/3125—Methylphosphonates
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
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    • C12N2310/322—2'-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
    • C12N2310/323—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
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/344—Position-specific modifications, e.g. on every purine, at the 3'-end
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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
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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
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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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  • Health & Medical Sciences (AREA)
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  • Pharmacology & Pharmacy (AREA)
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  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicinal Preparation (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
CA3226887A 2021-07-21 2022-07-20 Metabolic disorder-associated target gene irna compositions and methods of use thereof Pending CA3226887A1 (en)

Applications Claiming Priority (13)

Application Number Priority Date Filing Date Title
US202163223995P 2021-07-21 2021-07-21
US63/223,995 2021-07-21
US202163278126P 2021-11-11 2021-11-11
US63/278,126 2021-11-11
US202163285143P 2021-12-02 2021-12-02
US63/285,143 2021-12-02
US202163287578P 2021-12-09 2021-12-09
US63/287,578 2021-12-09
US202263321799P 2022-03-21 2022-03-21
US63/321,799 2022-03-21
US202263323543P 2022-03-25 2022-03-25
US63/323,543 2022-03-25
PCT/US2022/037658 WO2023003922A1 (en) 2021-07-21 2022-07-20 Metabolic disorder-associated target gene irna compositions and methods of use thereof

Publications (1)

Publication Number Publication Date
CA3226887A1 true CA3226887A1 (en) 2023-01-26

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CA3226887A Pending CA3226887A1 (en) 2021-07-21 2022-07-20 Metabolic disorder-associated target gene irna compositions and methods of use thereof

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US (1) US20240309370A1 (https=)
EP (1) EP4373937A1 (https=)
JP (1) JP2024526890A (https=)
KR (1) KR20240036041A (https=)
CN (1) CN117716032A (https=)
AU (1) AU2022314619A1 (https=)
CA (1) CA3226887A1 (https=)
CL (1) CL2024000130A1 (https=)
CO (1) CO2024000239A2 (https=)
IL (1) IL309897A (https=)
MX (1) MX2024000981A (https=)
TW (1) TW202421169A (https=)
WO (1) WO2023003922A1 (https=)

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IL326882A (en) 2023-08-30 2026-04-01 Arrowhead Pharmaceuticals Inc RNAi agents for inhibiting expression of inhibitory subunit beta E (INHBE), their pharmaceutical compositions and methods of use
WO2025064660A2 (en) * 2023-09-21 2025-03-27 Alnylam Pharmaceuticals, Inc. Activin a receptor type 1c (acvr1c) irna compositions and methods of use thereof
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
WO2025137390A1 (en) * 2023-12-20 2025-06-26 Arrowhead Pharmaceuticals, Inc. Rnai agents for inhibiting expression of activin receptor-like kinase 7 (alk7), compositions thereof, and methods of use
TW202525307A (zh) * 2023-12-21 2025-07-01 大陸商上海舶望製藥有限公司 抑制抑制素亞基βE（INHBE）的表達的組合物和方法
WO2025157269A1 (zh) * 2024-01-26 2025-07-31 康哲研发有限公司 双链siRNA及其缀合物的应用
WO2025185730A1 (zh) * 2024-03-08 2025-09-12 上海拓界生物医药科技有限公司 靶向INHBE的RNAi剂
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
WO2025195381A1 (zh) * 2024-03-21 2025-09-25 成都先衍生物技术有限公司 一种靶向INHBE基因表达的siRNA及其缀合物和用途
CN120210203A (zh) * 2024-03-27 2025-06-27 施能康医药科技(苏州)有限公司 靶向抑制素亚单位βE的核酸及其用途
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
WO2025228327A1 (zh) * 2024-04-28 2025-11-06 上海拓界生物医药科技有限公司 靶向ACVR2A的RNAi剂及其医药用途
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
WO2025264948A2 (en) 2024-06-21 2025-12-26 Eli Lilly And Company Inhbe rna interference agents
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WO2026021537A1 (en) * 2024-07-26 2026-01-29 Qilu Pharmaceutical Co., Ltd. iRNA COMPOSITION AND METHOD OF USING SAME
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