AR121672A1 - AGENTES DE ARNi PARA INHIBIR LA EXPRESIÓN DE PNPLA3, COMPOSICIONES FARMACÉUTICAS DE ESTA, Y MÉTODOS DE USO - Google Patents

AGENTES DE ARNi PARA INHIBIR LA EXPRESIÓN DE PNPLA3, COMPOSICIONES FARMACÉUTICAS DE ESTA, Y MÉTODOS DE USO

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Publication number
AR121672A1
AR121672A1 ARP210100762A ARP210100762A AR121672A1 AR 121672 A1 AR121672 A1 AR 121672A1 AR P210100762 A ARP210100762 A AR P210100762A AR P210100762 A ARP210100762 A AR P210100762A AR 121672 A1 AR121672 A1 AR 121672A1
Authority
AR
Argentina
Prior art keywords
pnpla3
rnai agents
methods
alcoholic
pharmaceutical compositions
Prior art date
Application number
ARP210100762A
Other languages
English (en)
Inventor
Anthony Nicholas
Tao Pei
Zhao Xu
Casi Schienebeck
Zhi-Ming Ding
Original Assignee
Arrowhead 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 Arrowhead Pharmaceuticals Inc filed Critical Arrowhead Pharmaceuticals Inc
Publication of AR121672A1 publication Critical patent/AR121672A1/es

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    • C—CHEMISTRY; METALLURGY
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    • 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
    • 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
    • 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
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/54—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic compound
    • A61K47/549—Sugars, nucleosides, nucleotides or nucleic acids
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/16—Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
    • 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)
    • C12Y301/01—Carboxylic ester hydrolases (3.1.1)
    • C12Y301/01003—Triacylglycerol lipase (3.1.1.3)
    • 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)
    • C12Y301/01—Carboxylic ester hydrolases (3.1.1)
    • C12Y301/01004—Phospholipase A2 (3.1.1.4)
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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 N.A.
    • 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
    • C12N2310/315—Phosphorothioates
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/317—Chemical structure of the backbone with an inverted bond, e.g. a cap structure
    • 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
    • C12N2310/32—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
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/33—Chemical structure of the base
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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
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    • C12N2310/33—Chemical structure of the base
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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
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    • C12N2310/3521—Methyl
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/353—Nature of the modification linked to the nucleic acid via an atom other than carbon
    • C12N2310/3533—Halogen
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/353—Nature of the modification linked to the nucleic acid via an atom other than carbon
    • C12N2310/3535—Nitrogen
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    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/35—Special therapeutic applications based on a specific dosage / administration regimen

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Molecular Biology (AREA)
  • Biomedical Technology (AREA)
  • Biochemistry (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • Epidemiology (AREA)
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  • Physics & Mathematics (AREA)
  • Gastroenterology & Hepatology (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)
  • Medicinal Preparation (AREA)

Abstract

La presente descripción se relaciona con agentes de ARNi, p. ej., agentes de ARNi bicatenarios, capaces de inhibir la expresión génica de la proteína 3 que contiene el dominio fosfolipasa tipo patatina (PNPLA3). También se describen composiciones farmacéuticas que incluyen agentes de ARNi del PNPLA3 y métodos de uso de estos. Los agentes de ARNi del PNPLA3 descritos en la presente descripción pueden conjugarse con ligandos de direccionamiento para facilitar el suministro a las células, incluidos los hepatocitos. El suministro de los agentes de ARNi del PNPLA3 in vivo proporciona la inhibición de la expresión génica de PNPLA3. Los agentes de ARNi pueden usarse en métodos de tratamiento de enfermedades y trastornos relacionados con PNPLA3, que incluyen hepatopatía grasa no alcohólica (NAFLD), esteatohepatitis no alcohólica (NASH), fibrosis hepática y enfermedades hepáticas no alcohólicas o alcohólicas, que incluyen cirrosis.
ARP210100762A 2020-03-26 2021-03-26 AGENTES DE ARNi PARA INHIBIR LA EXPRESIÓN DE PNPLA3, COMPOSICIONES FARMACÉUTICAS DE ESTA, Y MÉTODOS DE USO AR121672A1 (es)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US202063000137P 2020-03-26 2020-03-26

Publications (1)

Publication Number Publication Date
AR121672A1 true AR121672A1 (es) 2022-06-29

Family

ID=75540055

Family Applications (1)

Application Number Title Priority Date Filing Date
ARP210100762A AR121672A1 (es) 2020-03-26 2021-03-26 AGENTES DE ARNi PARA INHIBIR LA EXPRESIÓN DE PNPLA3, COMPOSICIONES FARMACÉUTICAS DE ESTA, Y MÉTODOS DE USO

Country Status (20)

Country Link
US (1) US20210301294A1 (es)
EP (1) EP4125932A2 (es)
JP (1) JP2023519246A (es)
KR (1) KR20220158011A (es)
CN (1) CN115397436A (es)
AR (1) AR121672A1 (es)
AU (1) AU2021242321A1 (es)
BR (1) BR112022019241A2 (es)
CA (1) CA3176418A1 (es)
CL (2) CL2022002580A1 (es)
CO (1) CO2022013746A2 (es)
CR (1) CR20220532A (es)
DO (1) DOP2022000204A (es)
EC (1) ECSP22075322A (es)
IL (1) IL296718A (es)
MX (1) MX2022011907A (es)
PE (1) PE20230345A1 (es)
TW (1) TW202202153A (es)
UY (1) UY39138A (es)
WO (1) WO2021195467A2 (es)

Families Citing this family (4)

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Publication number Priority date Publication date Assignee Title
KR20230115344A (ko) * 2016-09-02 2023-08-02 애로우헤드 파마슈티컬스 인코포레이티드 표적화 리간드
JP2020522510A (ja) 2017-06-02 2020-07-30 ウェイブ ライフ サイエンシズ リミテッドＷａｖｅ Ｌｉｆｅ Ｓｃｉｅｎｃｅｓ Ｌｔｄ． オリゴヌクレオチド組成物及びその使用方法
CN110997692A (zh) 2017-06-02 2020-04-10 波涛生命科学有限公司 寡核苷酸组合物及其使用方法
WO2023196941A1 (en) * 2022-04-08 2023-10-12 Arrowhead Pharmaceuticals, Inc. Treatment of a non-alcoholic fatty liver disease

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US4522811A (en) 1982-07-08 1985-06-11 Syntex (U.S.A.) Inc. Serial injection of muramyldipeptides and liposomes enhances the anti-infective activity of muramyldipeptides
NL9201440A (nl) 1992-08-11 1994-03-01 Univ Leiden Triantennaire clusterglycosiden, hun bereiding en toepassing.
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AU2007285782B2 (en) 2006-08-18 2010-06-24 Arrowhead Research Corporation Polyconjugates for in vivo delivery of polynucleotides
KR101956623B1 (ko) 2010-02-24 2019-03-12 애로우헤드 파마슈티컬스 인코포레이티드 ｓｉＲＮＡ의 표적 전달용 조성물
US8501930B2 (en) 2010-12-17 2013-08-06 Arrowhead Madison Inc. Peptide-based in vivo siRNA delivery system
CA2842039A1 (en) 2011-08-26 2013-03-07 Arrowhead Research Corporation Poly(vinyl ester) polymers for in vivo nucleic acid delivery
JP2015515530A (ja) 2012-04-18 2015-05-28 アローヘッド リサーチ コーポレイション インビボ核酸送達のためのポリ（アクリラート）ポリマー
CA2976445A1 (en) * 2015-02-13 2016-08-18 Alnylam Pharmaceuticals, Inc. Patatin-like phospholipase domain containing 3 (pnpla3) irna compositions and methods of use thereof
UY37146A (es) 2016-03-07 2017-09-29 Arrowhead Pharmaceuticals Inc Ligandos de direccionamiento para compuestos terapéuticos
US10036024B2 (en) * 2016-06-03 2018-07-31 Purdue Research Foundation siRNA compositions that specifically downregulate expression of a variant of the PNPLA3 gene and methods of use thereof for treating a chronic liver disease or alcoholic liver disease (ALD)
KR20230115344A (ko) * 2016-09-02 2023-08-02 애로우헤드 파마슈티컬스 인코포레이티드 표적화 리간드
BR112020003126A2 (pt) * 2017-09-14 2020-10-13 Arrowhead Pharmaceuticals, Inc. agentes de rnai e composições para inibição da expressão de angiopoietina tipo 3 (angptl3), e métodos de uso
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Also Published As

Publication number Publication date
CL2023002586A1 (es) 2024-01-26
PE20230345A1 (es) 2023-03-01
ECSP22075322A (es) 2022-10-31
WO2021195467A2 (en) 2021-09-30
JP2023519246A (ja) 2023-05-10
CR20220532A (es) 2022-11-28
BR112022019241A2 (pt) 2022-11-16
CO2022013746A2 (es) 2022-10-31
WO2021195467A3 (en) 2021-11-11
TW202202153A (zh) 2022-01-16
CN115397436A (zh) 2022-11-25
CA3176418A1 (en) 2021-09-30
KR20220158011A (ko) 2022-11-29
MX2022011907A (es) 2023-01-05
IL296718A (en) 2022-11-01
AU2021242321A1 (en) 2022-10-06
US20210301294A1 (en) 2021-09-30
UY39138A (es) 2021-09-30
DOP2022000204A (es) 2023-04-30
EP4125932A2 (en) 2023-02-08
CL2022002580A1 (es) 2023-03-24

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