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AR125351A1 - Composiciones y métodos para modular la expresión de pnpla3 - Google Patents

Composiciones y métodos para modular la expresión de pnpla3

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Publication number
AR125351A1
AR125351A1 ARP220100950A ARP220100950A AR125351A1 AR 125351 A1 AR125351 A1 AR 125351A1 AR P220100950 A ARP220100950 A AR P220100950A AR P220100950 A ARP220100950 A AR P220100950A AR 125351 A1 AR125351 A1 AR 125351A1
Authority
AR
Argentina
Prior art keywords
pnpla3
expression
rnai oligonucleotide
subject
pharmaceutical composition
Prior art date
Application number
ARP220100950A
Other languages
English (en)
Inventor
Utsav Saxena
Henryk T Dudek
Marc Abrams
Anton Turanov
Bob Dale Brown
Original Assignee
Dicerna 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 Dicerna Pharmaceuticals Inc filed Critical Dicerna Pharmaceuticals Inc
Publication of AR125351A1 publication Critical patent/AR125351A1/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
    • C12N15/1137—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
    • A61K31/7125—Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
    • 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
    • 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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    • C07—ORGANIC CHEMISTRY
    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • C07H21/02—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with ribosyl as saccharide radical
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12P—FERMENTATION OR ENZYME-USING PROCESSES TO SYNTHESISE A DESIRED CHEMICAL COMPOUND OR COMPOSITION OR TO SEPARATE OPTICAL ISOMERS FROM A RACEMIC MIXTURE
    • C12P19/00—Preparation of compounds containing saccharide radicals
    • C12P19/26—Preparation of nitrogen-containing carbohydrates
    • C12P19/28—N-glycosides
    • C12P19/30—Nucleotides
    • C12P19/34—Polynucleotides, e.g. nucleic acids, oligoribonucleotides
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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
    • C12N2310/315—Phosphorothioates
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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
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    • C12N2310/321—2'-O-R Modification
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
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    • C12N2310/351—Conjugate
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    • C12N2310/3515—Lipophilic moiety, e.g. cholesterol
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/50—Physical structure
    • C12N2310/53—Physical structure partially self-complementary or closed
    • C12N2310/531—Stem-loop; Hairpin

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
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  • Biotechnology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Microbiology (AREA)
  • Epidemiology (AREA)
  • Physics & Mathematics (AREA)
  • Virology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Gastroenterology & Hepatology (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)
  • Liquid Crystal Substances (AREA)

Abstract

Se describen oligonucleótidos y composiciones que los incluyen para inhibir o reducir la expresión del gen de la proteína 3 que contiene el dominio de fosfolipasa similar a la patatina (PNPLA3). También se describen métodos para preparar y usar los oligonucleótidos, particularmente usos relacionados con el tratamiento de enfermedades, trastornos y/o afecciones asociados a la expresión de PNPLA3. Reivindicación 69: Una composición farmacéutica que comprende el oligonucleótido de ARNi de cualquiera de las reivindicaciones 1 a 67, y un portador, un agente de administración o un excipiente farmacéuticamente aceptable. Reivindicación 71: Un método para reducir la expresión de PNPLA3 en una célula, una población de células o un sujeto, el que comprende la etapa de: i. poner en contacto la célula o la población de células con el oligonucleótido de ARNi de cualquiera de las reivindicaciones 1 a 67, o la composición farmacéutica de la reivindicación 69; o ii. administrar al sujeto el oligonucleótido de ARNi de cualquiera de las reivindicaciones 1 a 67, o la composición farmacéutica de la reivindicación 69. Reivindicación 92: Un kit que comprende el oligonucleótido de ARNi de cualquiera de las reivindicaciones 1 a 67, un portador farmacéuticamente aceptable opcional y un prospecto que comprende instrucciones para la administración a un sujeto que tiene una enfermedad, un trastorno o una afección asociados a la expresión de PNPLA3.
ARP220100950A 2021-04-14 2022-04-13 Composiciones y métodos para modular la expresión de pnpla3 AR125351A1 (es)

Applications Claiming Priority (1)

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US202163174932P 2021-04-14 2021-04-14

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Country Status (13)

Country Link
US (2) US12084662B2 (es)
EP (1) EP4323519A1 (es)
JP (1) JP2024514880A (es)
KR (1) KR20230171431A (es)
CN (1) CN117120613A (es)
AR (1) AR125351A1 (es)
AU (1) AU2022258459A1 (es)
BR (1) BR112023021109A2 (es)
CA (1) CA3209418A1 (es)
IL (1) IL307625A (es)
MX (1) MX2023012216A (es)
TW (2) TWI857290B (es)
WO (1) WO2022221430A1 (es)

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TWI857290B (zh) 2021-04-14 2024-10-01 美商戴瑟納製藥股份有限公司 用於調節ｐｎｐｌａ３表現之組合物及方法
EP4622983A1 (en) 2022-11-23 2025-10-01 Eli Lilly and Company Method of synthesizing 4'-phosphate analog nucleotide phosphoramidite
AU2023384015A1 (en) 2022-11-23 2025-05-22 Eli Lilly And Company Methods of synthesizing a targeting ligand-conjugated nucleotide phosphoramidite
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MX2023012216A (es) 2023-10-26
TW202300645A (zh) 2023-01-01
CN117120613A (zh) 2023-11-24
TW202500748A (zh) 2025-01-01
US20220364098A1 (en) 2022-11-17
TWI857290B (zh) 2024-10-01
US12084662B2 (en) 2024-09-10
EP4323519A1 (en) 2024-02-21
BR112023021109A2 (pt) 2023-12-12
WO2022221430A1 (en) 2022-10-20
JP2024514880A (ja) 2024-04-03
IL307625A (en) 2023-12-01
US20240401059A1 (en) 2024-12-05
KR20230171431A (ko) 2023-12-20
AU2022258459A1 (en) 2023-09-14
CA3209418A1 (en) 2022-10-20

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