MX2022011907A - Agentes de arni para inhibir la expresión de pnpla3, composiciones farmacéuticas de estos, y métodos de uso. - Google Patents
Agentes de arni para inhibir la expresión de pnpla3, composiciones farmacéuticas de estos, y métodos de uso.Info
- Publication number
- MX2022011907A MX2022011907A MX2022011907A MX2022011907A MX2022011907A MX 2022011907 A MX2022011907 A MX 2022011907A MX 2022011907 A MX2022011907 A MX 2022011907A MX 2022011907 A MX2022011907 A MX 2022011907A MX 2022011907 A MX2022011907 A MX 2022011907A
- Authority
- MX
- Mexico
- Prior art keywords
- pnpla3
- rnai agents
- methods
- alcoholic
- pharmaceutical compositions
- Prior art date
Links
- 102100031251 1-acylglycerol-3-phosphate O-acyltransferase PNPLA3 Human genes 0.000 title abstract 7
- 108091030071 RNAI Proteins 0.000 title abstract 7
- 230000009368 gene silencing by RNA Effects 0.000 title abstract 7
- 230000014509 gene expression Effects 0.000 title abstract 3
- 238000000034 method Methods 0.000 title abstract 3
- 239000008194 pharmaceutical composition Substances 0.000 title abstract 2
- 101001129184 Homo sapiens 1-acylglycerol-3-phosphate O-acyltransferase PNPLA3 Proteins 0.000 title 1
- 230000002401 inhibitory effect Effects 0.000 title 1
- 108050003337 1-acylglycerol-3-phosphate O-acyltransferase PNPLA3 Proteins 0.000 abstract 6
- 239000003795 chemical substances by application Substances 0.000 abstract 6
- 208000008338 non-alcoholic fatty liver disease Diseases 0.000 abstract 4
- 206010053219 non-alcoholic steatohepatitis Diseases 0.000 abstract 2
- 208000022309 Alcoholic Liver disease Diseases 0.000 abstract 1
- 206010016654 Fibrosis Diseases 0.000 abstract 1
- 206010019668 Hepatic fibrosis Diseases 0.000 abstract 1
- 101100244213 Homo sapiens PNPLA3 gene Proteins 0.000 abstract 1
- 230000001476 alcoholic effect Effects 0.000 abstract 1
- 210000004027 cell Anatomy 0.000 abstract 1
- 230000007882 cirrhosis Effects 0.000 abstract 1
- 208000019425 cirrhosis of liver Diseases 0.000 abstract 1
- 208000037765 diseases and disorders Diseases 0.000 abstract 1
- 210000003494 hepatocyte Anatomy 0.000 abstract 1
- 238000001727 in vivo Methods 0.000 abstract 1
- 230000005764 inhibitory process Effects 0.000 abstract 1
- 239000003446 ligand Substances 0.000 abstract 1
- 230000008685 targeting Effects 0.000 abstract 1
Classifications
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- 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
- C12N15/1137—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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- 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
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- 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
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- 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
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- 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
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- 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
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- C12N2310/14—Type of nucleic acid interfering N.A.
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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
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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
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- 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
- C12N2310/331—Universal or degenerate base
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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
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- C12N2310/35—Nature of the modification
- C12N2310/351—Conjugate
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- C12N2310/30—Chemical structure
- C12N2310/35—Nature of the modification
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- C12N2310/3521—Methyl
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- 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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- 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)
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- Health & Medical Sciences (AREA)
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- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- General Health & Medical Sciences (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Organic Chemistry (AREA)
- Molecular Biology (AREA)
- Biomedical Technology (AREA)
- Veterinary Medicine (AREA)
- Medicinal Chemistry (AREA)
- Biochemistry (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- General Engineering & Computer Science (AREA)
- Biotechnology (AREA)
- Epidemiology (AREA)
- Biophysics (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Virology (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Gastroenterology & Hepatology (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, incluida la cirrosis.
Applications Claiming Priority (2)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US202063000137P | 2020-03-26 | 2020-03-26 | |
PCT/US2021/024299 WO2021195467A2 (en) | 2020-03-26 | 2021-03-26 | RNAi AGENTS FOR INHIBITING EXPRESSION OF PNPLA3, PHARMACEUTICAL COMPOSITIONS THEREOF, AND METHODS OF USE |
Publications (1)
Publication Number | Publication Date |
---|---|
MX2022011907A true MX2022011907A (es) | 2023-01-05 |
Family
ID=75540055
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
MX2022011907A MX2022011907A (es) | 2020-03-26 | 2021-03-26 | Agentes de arni para inhibir la expresión de pnpla3, composiciones farmacéuticas de estos, 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)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
KR20230115344A (ko) * | 2016-09-02 | 2023-08-02 | 애로우헤드 파마슈티컬스 인코포레이티드 | 표적화 리간드 |
JP2020524485A (ja) | 2017-06-02 | 2020-08-20 | 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 |
US11603532B2 (en) | 2017-06-02 | 2023-03-14 | Wave Life Sciences Ltd. | Oligonucleotide compositions and methods of use thereof |
WO2023196941A1 (en) * | 2022-04-08 | 2023-10-12 | Arrowhead Pharmaceuticals, Inc. | Treatment of a non-alcoholic fatty liver disease |
Family Cites Families (15)
Publication number | Priority date | Publication date | Assignee | Title |
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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. |
US5998203A (en) | 1996-04-16 | 1999-12-07 | Ribozyme Pharmaceuticals, Inc. | Enzymatic nucleic acids containing 5'-and/or 3'-cap structures |
MXPA01009073A (es) | 1999-03-10 | 2002-05-06 | Phogen Ltd | Suministro de acidos nucleicos y proteinas a las celulas. |
WO2008022309A2 (en) | 2006-08-18 | 2008-02-21 | F. Hoffmann-La Roche Ag | Polyconjugates for in vivo delivery of polynucleotides |
ES2562817T3 (es) | 2010-02-24 | 2016-03-08 | Arrowhead Research Corporation | Composiciones para el suministro dirigido de ARNip |
US8501930B2 (en) | 2010-12-17 | 2013-08-06 | Arrowhead Madison Inc. | Peptide-based in vivo siRNA delivery system |
US8932572B2 (en) | 2011-08-26 | 2015-01-13 | Arrowhead Madison Inc. | Poly(vinyl ester) polymers for in vivo nucleic acid delivery |
MX2014001965A (es) | 2012-04-18 | 2014-03-31 | Arrowhead Res Corp | Polimeros de poli(acrilato) para suministro de acido nucleico in vivo. |
JP2018510621A (ja) * | 2015-02-13 | 2018-04-19 | アルナイラム ファーマシューティカルズ, インコーポレイテッドAlnylam Pharmaceuticals, Inc. | パタチン様ホスホリパーゼドメイン含有3(PNPLA3)iRNA組成物およびその使用方法 |
KR20230048446A (ko) | 2016-03-07 | 2023-04-11 | 애로우헤드 파마슈티컬스 인코포레이티드 | 치료용 화합물을 위한 표적화 리간드 |
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 |
CN117701562A (zh) * | 2017-09-14 | 2024-03-15 | 箭头药业股份有限公司 | 用于抑制血管生成素-样3(ANGPTL3)的表达的RNAi剂和组合物以及使用方法 |
UY38003A (es) * | 2017-12-12 | 2019-06-28 | Amgen Inc | Construcciones de arni para inhibir la expresión de pnpla3 y métodos de uso de las mismas |
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2021
- 2021-03-26 TW TW110111184A patent/TW202202153A/zh unknown
- 2021-03-26 KR KR1020227036413A patent/KR20220158011A/ko active Search and Examination
- 2021-03-26 UY UY0001039138A patent/UY39138A/es unknown
- 2021-03-26 CN CN202180024737.5A patent/CN115397436A/zh active Pending
- 2021-03-26 IL IL296718A patent/IL296718A/en unknown
- 2021-03-26 CA CA3176418A patent/CA3176418A1/en active Pending
- 2021-03-26 AR ARP210100762A patent/AR121672A1/es unknown
- 2021-03-26 MX MX2022011907A patent/MX2022011907A/es unknown
- 2021-03-26 PE PE2022002098A patent/PE20230345A1/es unknown
- 2021-03-26 JP JP2022557767A patent/JP2023519246A/ja active Pending
- 2021-03-26 WO PCT/US2021/024299 patent/WO2021195467A2/en active Application Filing
- 2021-03-26 AU AU2021242321A patent/AU2021242321A1/en active Pending
- 2021-03-26 CR CR20220532A patent/CR20220532A/es unknown
- 2021-03-26 US US17/213,787 patent/US20210301294A1/en active Pending
- 2021-03-26 BR BR112022019241A patent/BR112022019241A2/pt unknown
- 2021-03-26 EP EP21719475.2A patent/EP4125932A2/en active Pending
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2022
- 2022-09-23 DO DO2022000204A patent/DOP2022000204A/es unknown
- 2022-09-23 CL CL2022002580A patent/CL2022002580A1/es unknown
- 2022-09-26 CO CONC2022/0013746A patent/CO2022013746A2/es unknown
- 2022-09-26 EC ECSENADI202275322A patent/ECSP22075322A/es unknown
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2023
- 2023-08-31 CL CL2023002586A patent/CL2023002586A1/es unknown
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DOP2022000204A (es) | 2023-04-30 |
CL2022002580A1 (es) | 2023-03-24 |
CR20220532A (es) | 2022-11-28 |
TW202202153A (zh) | 2022-01-16 |
UY39138A (es) | 2021-09-30 |
IL296718A (en) | 2022-11-01 |
AR121672A1 (es) | 2022-06-29 |
CN115397436A (zh) | 2022-11-25 |
CL2023002586A1 (es) | 2024-01-26 |
CO2022013746A2 (es) | 2022-10-31 |
ECSP22075322A (es) | 2022-10-31 |
JP2023519246A (ja) | 2023-05-10 |
PE20230345A1 (es) | 2023-03-01 |
EP4125932A2 (en) | 2023-02-08 |
US20210301294A1 (en) | 2021-09-30 |
BR112022019241A2 (pt) | 2022-11-16 |
WO2021195467A2 (en) | 2021-09-30 |
WO2021195467A3 (en) | 2021-11-11 |
KR20220158011A (ko) | 2022-11-29 |
CA3176418A1 (en) | 2021-09-30 |
AU2021242321A1 (en) | 2022-10-06 |
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