DOP2022000204A - Agentes de rani para inhibir la expresión de pnpla3, composiciones farmacéuticas de estos y métodos de uso - Google Patents

Agentes de rani para inhibir la expresión de pnpla3, composiciones farmacéuticas de estos y métodos de uso

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Publication number
DOP2022000204A
DOP2022000204A DO2022000204A DO2022000204A DOP2022000204A DO P2022000204 A DOP2022000204 A DO P2022000204A DO 2022000204 A DO2022000204 A DO 2022000204A DO 2022000204 A DO2022000204 A DO 2022000204A DO P2022000204 A DOP2022000204 A DO P2022000204A
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Dominican Republic
Prior art keywords
pnpla3
rnai agents
methods
agents
pharmaceutical compositions
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DO2022000204A
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English (en)
Inventor
Nicholas Anthony
Pei Tao
Xu Zhao
Schienebeck Casi
Ding Zhi-Ming
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Arrowhead Pharmaceuticals Inc
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Publication date
Application filed by Arrowhead Pharmaceuticals Inc filed Critical Arrowhead Pharmaceuticals Inc
Publication of DOP2022000204A publication Critical patent/DOP2022000204A/es

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    • 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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    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
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    • 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
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    • 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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    • 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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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.
DO2022000204A 2020-03-26 2022-09-23 Agentes de rani para inhibir la expresión de pnpla3, composiciones farmacéuticas de estos y métodos de uso DOP2022000204A (es)

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

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DOP2022000204A true DOP2022000204A (es) 2023-04-30

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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)

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TW202320855A (zh) * 2016-09-02 2023-06-01 美商愛羅海德製藥公司 標靶性配體
US11603532B2 (en) 2017-06-02 2023-03-14 Wave Life Sciences Ltd. Oligonucleotide compositions and methods of use thereof
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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WO2016130806A2 (en) * 2015-02-13 2016-08-18 Alnylam Pharmaceuticals, Inc. Patatin-like phospholipase domain containing 3 (pnpla3) irna compositions and methods of use thereof
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KR20220158011A (ko) 2022-11-29
BR112022019241A2 (pt) 2022-11-16
CA3176418A1 (en) 2021-09-30
AR121672A1 (es) 2022-06-29
UY39138A (es) 2021-09-30
CN115397436A (zh) 2022-11-25
CL2023002586A1 (es) 2024-01-26
MX2022011907A (es) 2023-01-05
EP4125932A2 (en) 2023-02-08
PE20230345A1 (es) 2023-03-01
AU2021242321A1 (en) 2022-10-06
JP2023519246A (ja) 2023-05-10
WO2021195467A3 (en) 2021-11-11
CR20220532A (es) 2022-11-28
ECSP22075322A (es) 2022-10-31
WO2021195467A2 (en) 2021-09-30
CO2022013746A2 (es) 2022-10-31
CL2022002580A1 (es) 2023-03-24
TW202202153A (zh) 2022-01-16
IL296718A (en) 2022-11-01
US20210301294A1 (en) 2021-09-30

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