BR112022019241A2 - Agentes de rnai para inibição da expressão de pnpla3, composições farmacêuticas dos mesmos e métodos de uso - Google Patents

Agentes de rnai para inibição da expressão de pnpla3, composições farmacêuticas dos mesmos e métodos de uso

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Publication number
BR112022019241A2
BR112022019241A2 BR112022019241A BR112022019241A BR112022019241A2 BR 112022019241 A2 BR112022019241 A2 BR 112022019241A2 BR 112022019241 A BR112022019241 A BR 112022019241A BR 112022019241 A BR112022019241 A BR 112022019241A BR 112022019241 A2 BR112022019241 A2 BR 112022019241A2
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Brazil
Prior art keywords
rnai agents
pnpla3
methods
pharmaceutical compositions
alcoholic
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BR112022019241A
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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 of BR112022019241A2 publication Critical patent/BR112022019241A2/pt

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    • 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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Abstract

AGENTES DE RNAI PARA INIBIÇÃO DA EXPRESSÃO DE PNPLA3, COMPOSIÇÕES FARMACÊUTICAS DOS MESMOS E MÉTODOS DE USO. A presente invenção refere-se a agentes de RNAi, por exemplo, agentes de RNAi de fita dupla, capazes de inibir a expressão gênica do domínio de fosfolipase tipo patatina contendo proteína 3 (PNPLA3). Também são descritas composições farmacêuticas que incluem agentes de RNAi de PNPLA3 e métodos de uso dos mesmos. Os agentes de RNAi de PNPLA3 descritos na presente invenção podem ser conjugados com ligantes de direcionamento para facilitar a liberação para as células, incluindo os hepatócitos. A liberação dos agentes de RNAi de PNPLA3 in vivo fornece a inibição da expressão do gene PNPLA3. Os agentes de RNAi podem ser usados em métodos de tratamento de doenças e distúrbios relacionados a PNPLA3, incluindo doença hepática gordurosa não alcoólica (DHGNA), esteato-hepatite não alcoólica (EHNA), fibrose hepática e doenças hepáticas alcoólicas ou não alcoólicas, incluindo cirrose.
BR112022019241A 2020-03-26 2021-03-26 Agentes de rnai para inibição da expressão de pnpla3, composições farmacêuticas dos mesmos e métodos de uso BR112022019241A2 (pt)

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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BR112022019241A2 true BR112022019241A2 (pt) 2022-11-16

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US (1) US20210301294A1 (pt)
EP (1) EP4125932A2 (pt)
JP (1) JP2023519246A (pt)
KR (1) KR20220158011A (pt)
CN (1) CN115397436A (pt)
AR (1) AR121672A1 (pt)
AU (1) AU2021242321A1 (pt)
BR (1) BR112022019241A2 (pt)
CA (1) CA3176418A1 (pt)
CL (2) CL2022002580A1 (pt)
CO (1) CO2022013746A2 (pt)
CR (1) CR20220532A (pt)
DO (1) DOP2022000204A (pt)
EC (1) ECSP22075322A (pt)
IL (1) IL296718A (pt)
MX (1) MX2022011907A (pt)
PE (1) PE20230345A1 (pt)
TW (1) TW202202153A (pt)
UY (1) UY39138A (pt)
WO (1) WO2021195467A2 (pt)

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CN111050806A (zh) 2017-06-02 2020-04-21 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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TW202202153A (zh) 2022-01-16
JP2023519246A (ja) 2023-05-10
CA3176418A1 (en) 2021-09-30
EP4125932A2 (en) 2023-02-08
CR20220532A (es) 2022-11-28
AU2021242321A1 (en) 2022-10-06
UY39138A (es) 2021-09-30
CL2023002586A1 (es) 2024-01-26
CN115397436A (zh) 2022-11-25
DOP2022000204A (es) 2023-04-30
WO2021195467A2 (en) 2021-09-30
CL2022002580A1 (es) 2023-03-24
PE20230345A1 (es) 2023-03-01
IL296718A (en) 2022-11-01
ECSP22075322A (es) 2022-10-31
US20210301294A1 (en) 2021-09-30
CO2022013746A2 (es) 2022-10-31
WO2021195467A3 (en) 2021-11-11
MX2022011907A (es) 2023-01-05
KR20220158011A (ko) 2022-11-29
AR121672A1 (es) 2022-06-29

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