CO2024001403A2 - Composiciones de arni y métodos para silenciar el angiotensinógeno (agt) - Google Patents

Composiciones de arni y métodos para silenciar el angiotensinógeno (agt)

Info

Publication number
CO2024001403A2
CO2024001403A2 CONC2024/0001403A CO2024001403A CO2024001403A2 CO 2024001403 A2 CO2024001403 A2 CO 2024001403A2 CO 2024001403 A CO2024001403 A CO 2024001403A CO 2024001403 A2 CO2024001403 A2 CO 2024001403A2
Authority
CO
Colombia
Prior art keywords
agt
methods
angiotensinogen
arni
silence
Prior art date
Application number
CONC2024/0001403A
Other languages
English (en)
Spanish (es)
Inventor
Adam Castoreno
James D Mcininch
Mark K Schlegel
Paul Nioi
Joseph Barry
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 CO2024001403A2 publication Critical patent/CO2024001403A2/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
    • 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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    • 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
    • A61K31/33—Heterocyclic compounds
    • A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/41—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having five-membered rings with two or more ring hetero atoms, at least one of which being nitrogen, e.g. tetrazole
    • A61K31/4164—1,3-Diazoles
    • A61K31/4178—1,3-Diazoles not condensed 1,3-diazoles and containing further heterocyclic rings, e.g. pilocarpine, nitrofurantoin
    • 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
    • A61K31/33—Heterocyclic compounds
    • A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/41—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having five-membered rings with two or more ring hetero atoms, at least one of which being nitrogen, e.g. tetrazole
    • A61K31/4245—Oxadiazoles
    • 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
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • A61P9/12—Antihypertensives
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
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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
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/312—Phosphonates
    • C12N2310/3125—Methylphosphonates
    • 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
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    • 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
    • C12N2310/321—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
    • 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
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate

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  • Health & Medical Sciences (AREA)
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  • Molecular Biology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Zoology (AREA)
  • Biochemistry (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Epidemiology (AREA)
  • Cardiology (AREA)
  • Endocrinology (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicinal Preparation (AREA)
CONC2024/0001403A 2021-08-04 2024-02-09 Composiciones de arni y métodos para silenciar el angiotensinógeno (agt) CO2024001403A2 (es)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202163229085P 2021-08-04 2021-08-04
US202163272769P 2021-10-28 2021-10-28
PCT/US2022/039242 WO2023014765A1 (en) 2021-08-04 2022-08-03 iRNA COMPOSITIONS AND METHODS FOR SILENCING ANGIOTENSINOGEN (AGT)

Publications (1)

Publication Number Publication Date
CO2024001403A2 true CO2024001403A2 (es) 2024-02-26

Family

ID=83151961

Family Applications (1)

Application Number Title Priority Date Filing Date
CONC2024/0001403A CO2024001403A2 (es) 2021-08-04 2024-02-09 Composiciones de arni y métodos para silenciar el angiotensinógeno (agt)

Country Status (13)

Country Link
US (1) US20240392294A1 (https=)
EP (1) EP4381071A1 (https=)
JP (1) JP2024531914A (https=)
KR (1) KR20240042016A (https=)
AU (1) AU2022324003A1 (https=)
CA (1) CA3228255A1 (https=)
CL (1) CL2024000341A1 (https=)
CO (1) CO2024001403A2 (https=)
IL (1) IL310295A (https=)
MX (1) MX2024001445A (https=)
PE (1) PE20241132A1 (https=)
TW (1) TW202337474A (https=)
WO (1) WO2023014765A1 (https=)

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* Cited by examiner, † Cited by third party
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TWI851574B (zh) 2018-05-14 2024-08-11 美商阿尼拉製藥公司 血管收縮素原（ＡＧＴ）ｉＲＮＡ組成物及其使用方法
IL312811A (en) * 2021-11-16 2024-07-01 Shanghai Argo Biopharmaceutical Co Ltd Composition and method for inhibiting angiotensinogen (agt) protein expression
CN120882730A (zh) * 2023-04-14 2025-10-31 苏州炫景生物科技有限公司 抑制agt基因表达的化合物、药物组合物及其用途
WO2024234831A1 (zh) * 2023-05-15 2024-11-21 苏州时安生物技术有限公司 一种用于调节血管紧张素原基因表达的双链rna、其缀合物、药物组合物及用途
EP4562158A1 (en) 2023-06-13 2025-06-04 Arnatar Therapeutics, Inc Advanced rna targeting (arnatar) for angiotensinogen
WO2025007961A1 (zh) * 2023-07-06 2025-01-09 大睿生物 调控AGT表达的dsRNA分子
CN119585433A (zh) * 2023-07-06 2025-03-07 石药集团中奇制药技术（石家庄）有限公司 一种抑制AGT基因表达的RNAi剂及其应用
AU2024318590A1 (en) * 2023-07-31 2026-02-26 Basecure Therapeutics Inc. Conjugates of n-acetyl-galactosamine (galnac) and oligonucleotides and uses thereof
CN119585432B (zh) * 2023-08-22 2025-08-12 成都先衍生物技术有限公司 一种靶向AGT基因表达的siRNA及其缀合物和用途
WO2025087237A1 (zh) * 2023-10-23 2025-05-01 广东东阳光药业股份有限公司 一种新型的双链siRNA、其缀合物及其用途
WO2025130862A1 (zh) * 2023-12-18 2025-06-26 广东东阳光药业股份有限公司 一种新型的双链siRNA、其缀合物及其用途

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