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BR112022011412A2 - Composições de irna de domínio de fosfolipase semelhante à patatina contendo proteína 3 (pnpla3) e métodos de uso das mesmas - Google Patents

Composições de irna de domínio de fosfolipase semelhante à patatina contendo proteína 3 (pnpla3) e métodos de uso das mesmas

Info

Publication number
BR112022011412A2
BR112022011412A2 BR112022011412A BR112022011412A BR112022011412A2 BR 112022011412 A2 BR112022011412 A2 BR 112022011412A2 BR 112022011412 A BR112022011412 A BR 112022011412A BR 112022011412 A BR112022011412 A BR 112022011412A BR 112022011412 A2 BR112022011412 A2 BR 112022011412A2
Authority
BR
Brazil
Prior art keywords
pnpla3
methods
patatin
compositions containing
phospholipase domain
Prior art date
Application number
BR112022011412A
Other languages
English (en)
Inventor
Tremblay Frederic
D Mcininch James
Castoreno Adam
K Schlegel Mark
Kaittanis Charalambos
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
Priority claimed from PCT/US2020/064776 external-priority patent/WO2021126734A1/en
Publication of BR112022011412A2 publication Critical patent/BR112022011412A2/pt

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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
    • 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
    • A61K47/02—Inorganic compounds
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0075—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the delivery route, e.g. oral, subcutaneous
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
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    • A—HUMAN NECESSITIES
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    • 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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    • 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
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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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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
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  • Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biotechnology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
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  • Pharmacology & Pharmacy (AREA)
  • Biochemistry (AREA)
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  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Virology (AREA)
  • Inorganic Chemistry (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)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Enzymes And Modification Thereof (AREA)

Abstract

COMPOSIÇÕES DE IRNA DE DOMÍNIO DE FOSFOLIPASE SEMELHANTE À PATATINA CONTENDO PROTEÍNA 3 (PNPLA3) E MÉTODOS DE USO DAS MESMAS. A presente invenção refere-se aos agentes de RNAi, por exemplo, agentes de RNA de fita dupla (dsRNA), que têm como alvo o gene de Domínio de Fosfolipase Semelhante à patatina contendo proteína 3 (PNPLA3). A invenção também se refere aos métodos de uso de tais agentes de RNAi para inibir a expressão de um gene PNPLA3 e aos métodos de prevenção e tratamento de um distúrbio associado ao PNPLA3, por exemplo, doença hepática gordurosa não alcoólica (NA-FLD).
BR112022011412A 2019-12-16 2020-12-14 Composições de irna de domínio de fosfolipase semelhante à patatina contendo proteína 3 (pnpla3) e métodos de uso das mesmas BR112022011412A2 (pt)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201962948445P 2019-12-16 2019-12-16
US202063040602P 2020-06-18 2020-06-18
PCT/US2020/064776 WO2021126734A1 (en) 2019-12-16 2020-12-14 Patatin-like phospholipase domain containing 3 (pnpla3) irna compositions and methods of use thereof

Publications (1)

Publication Number Publication Date
BR112022011412A2 true BR112022011412A2 (pt) 2022-08-30

Family

ID=76437193

Family Applications (1)

Application Number Title Priority Date Filing Date
BR112022011412A BR112022011412A2 (pt) 2019-12-16 2020-12-14 Composições de irna de domínio de fosfolipase semelhante à patatina contendo proteína 3 (pnpla3) e métodos de uso das mesmas

Country Status (12)

Country Link
US (2) US11149276B2 (pt)
EP (1) EP4077666A1 (pt)
JP (1) JP2023506258A (pt)
KR (1) KR20220116520A (pt)
CN (1) CN115176007A (pt)
AU (1) AU2020404905A1 (pt)
BR (1) BR112022011412A2 (pt)
CA (1) CA3164628A1 (pt)
CO (1) CO2022008105A2 (pt)
IL (1) IL293860A (pt)
MX (1) MX2022007144A (pt)
PH (1) PH12022551417A1 (pt)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2021195467A2 (en) * 2020-03-26 2021-09-30 Arrowhead Pharmaceuticals, Inc. RNAi AGENTS FOR INHIBITING EXPRESSION OF PNPLA3, PHARMACEUTICAL COMPOSITIONS THEREOF, AND METHODS OF USE
WO2024120412A1 (en) * 2022-12-07 2024-06-13 Shanghai Argo Biopharmaceutical Co., Ltd. Compositions and methods for inhibiting expression of patatin-like phospholipase domain-containing 3 (pnpla3)

Family Cites Families (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US7790867B2 (en) 2002-12-05 2010-09-07 Rosetta Genomics Inc. Vaccinia virus-related nucleic acids and microRNA
MX2018012038A (es) 2011-11-18 2021-09-06 Alnylam Pharmaceuticals Inc Agentes de iarn modificados.
WO2013075035A1 (en) 2011-11-18 2013-05-23 Alnylam Pharmaceuticals Rnai agents, compositions and methods of use thereof for treating transthyretin (ttr) associated diseases
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
EP3350328A1 (en) 2015-09-14 2018-07-25 Alnylam Pharmaceuticals, Inc. Polynucleotide agents targeting patatin-like phospholipase domain containing 3 (pnpla3) and methods of use thereof

Also Published As

Publication number Publication date
US20210189400A1 (en) 2021-06-24
IL293860A (en) 2022-08-01
AU2020404905A1 (en) 2022-06-23
US20220348926A1 (en) 2022-11-03
CA3164628A1 (en) 2021-06-24
MX2022007144A (es) 2022-07-11
JP2023506258A (ja) 2023-02-15
PH12022551417A1 (en) 2023-11-20
CO2022008105A2 (es) 2022-07-08
EP4077666A1 (en) 2022-10-26
US11149276B2 (en) 2021-10-19
KR20220116520A (ko) 2022-08-23
CN115176007A (zh) 2022-10-11

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