BR112021011061A2 - Construtos de rnai para inibir a expressão de pnpla3 - Google Patents

Construtos de rnai para inibir a expressão de pnpla3 Download PDF

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Publication number
BR112021011061A2
BR112021011061A2 BR112021011061-5A BR112021011061A BR112021011061A2 BR 112021011061 A2 BR112021011061 A2 BR 112021011061A2 BR 112021011061 A BR112021011061 A BR 112021011061A BR 112021011061 A2 BR112021011061 A2 BR 112021011061A2
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BR
Brazil
Prior art keywords
rnai construct
pnpla3
invab
rnai
cugcuucau
Prior art date
Application number
BR112021011061-5A
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English (en)
Portuguese (pt)
Inventor
Ingrid Rulifson
Justin K. Murray
Michael OLLMANN
Oliver HOMANN
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Amgen Inc.
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Publication of BR112021011061A2 publication Critical patent/BR112021011061A2/pt

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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
    • 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
    • A61K31/7105—Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
    • 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
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    • C12N9/10—Transferases (2.)
    • C12N9/1025—Acyltransferases (2.3)
    • C12N9/1029—Acyltransferases (2.3) transferring groups other than amino-acyl groups (2.3.1)
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    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/18—Carboxylic ester hydrolases (3.1.1)
    • C12N9/20—Triglyceride splitting, e.g. by means of lipase
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    • C12Y203/00—Acyltransferases (2.3)
    • C12Y203/01—Acyltransferases (2.3) transferring groups other than amino-acyl groups (2.3.1)
    • C12Y203/01051—1-Acylglycerol-3-phosphate O-acyltransferase (2.3.1.51)
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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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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
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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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    • C12N2310/32—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
    • C12N2310/35—Nature of the modification
    • C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
    • 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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  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
BR112021011061-5A 2018-12-10 2019-12-10 Construtos de rnai para inibir a expressão de pnpla3 BR112021011061A2 (pt)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201862777714P 2018-12-10 2018-12-10
US62/777,714 2018-12-10
PCT/US2019/065481 WO2020123508A2 (en) 2018-12-10 2019-12-10 Rnai constructs for inhibiting pnpla3 expression and methods of use thereof

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BR112021011061A2 true BR112021011061A2 (pt) 2021-08-31

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US (2) US20220017906A1 (https=)
EP (1) EP3894555A2 (https=)
JP (2) JP2022511550A (https=)
KR (1) KR20210102932A (https=)
CN (2) CN121320339A (https=)
AR (1) AR117297A1 (https=)
AU (1) AU2019396419B2 (https=)
BR (1) BR112021011061A2 (https=)
CA (1) CA3121510A1 (https=)
CL (1) CL2021001489A1 (https=)
CO (1) CO2021008997A2 (https=)
CR (1) CR20210371A (https=)
EA (1) EA202191629A1 (https=)
IL (1) IL283473A (https=)
JO (1) JOP20210142A1 (https=)
MX (2) MX2021006784A (https=)
PE (1) PE20211225A1 (https=)
PH (1) PH12021551326A1 (https=)
SG (1) SG11202105711SA (https=)
TW (1) TW202039843A (https=)
UY (1) UY38503A (https=)
WO (1) WO2020123508A2 (https=)

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US20250243491A1 (en) * 2021-09-01 2025-07-31 Aligos Therapeutics, Inc. Pnpla3-targeting short interfering rna (sirna) molecules and uses thereof
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PH12021551326A1 (en) 2022-04-25
WO2020123508A2 (en) 2020-06-18
EA202191629A1 (ru) 2021-09-03
CN121320339A (zh) 2026-01-13
UY38503A (es) 2020-06-30
AR117297A1 (es) 2021-07-28
JP2022511550A (ja) 2022-01-31
US20220017906A1 (en) 2022-01-20
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