CL2024000130A1 - Composiciones de arni de gen diana asociado con trastorno metabólico y sus métodos de uso - Google Patents

Composiciones de arni de gen diana asociado con trastorno metabólico y sus métodos de uso

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Publication number
CL2024000130A1
CL2024000130A1 CL2024000130A CL2024000130A CL2024000130A1 CL 2024000130 A1 CL2024000130 A1 CL 2024000130A1 CL 2024000130 A CL2024000130 A CL 2024000130A CL 2024000130 A CL2024000130 A CL 2024000130A CL 2024000130 A1 CL2024000130 A1 CL 2024000130A1
Authority
CL
Chile
Prior art keywords
metabolic disorder
methods
target gene
associated target
gene rna
Prior art date
Application number
CL2024000130A
Other languages
English (en)
Spanish (es)
Inventor
M Deaton Aimee
Zuber Jeffrey
Original Assignee
Alnylam Pharmaceuticals Inc
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 Alnylam Pharmaceuticals Inc filed Critical Alnylam Pharmaceuticals Inc
Publication of CL2024000130A1 publication Critical patent/CL2024000130A1/es

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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
    • A61P3/00—Drugs for disorders of the metabolism
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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
    • C—CHEMISTRY; METALLURGY
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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
    • C12N15/1138—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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • 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]
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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
    • 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
    • C12N2310/32—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
    • 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
    • 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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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Virology (AREA)
  • Veterinary Medicine (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Diabetes (AREA)
  • Epidemiology (AREA)
  • Hematology (AREA)
  • Obesity (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • 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)
CL2024000130A 2021-07-21 2024-01-16 Composiciones de arni de gen diana asociado con trastorno metabólico y sus métodos de uso CL2024000130A1 (es)

Applications Claiming Priority (6)

Application Number Priority Date Filing Date Title
US202163223995P 2021-07-21 2021-07-21
US202163278126P 2021-11-11 2021-11-11
US202163285143P 2021-12-02 2021-12-02
US202163287578P 2021-12-09 2021-12-09
US202263321799P 2022-03-21 2022-03-21
US202263323543P 2022-03-25 2022-03-25

Publications (1)

Publication Number Publication Date
CL2024000130A1 true CL2024000130A1 (es) 2024-08-16

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Family Applications (1)

Application Number Title Priority Date Filing Date
CL2024000130A CL2024000130A1 (es) 2021-07-21 2024-01-16 Composiciones de arni de gen diana asociado con trastorno metabólico y sus métodos de uso

Country Status (13)

Country Link
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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CN121752722A (zh) * 2023-08-21 2026-03-27 苏州炫景生物科技有限公司 靶向inhbe基因的双链寡核苷酸、缀合物、组合物及其用途
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
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