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AR125992A1 - Composiciones y métodos para inhibir la expresión del componente 1 de reducción de amidoxima mitocondrial (marc1) - Google Patents

Composiciones y métodos para inhibir la expresión del componente 1 de reducción de amidoxima mitocondrial (marc1)

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Publication number
AR125992A1
AR125992A1 ARP220101411A ARP220101411A AR125992A1 AR 125992 A1 AR125992 A1 AR 125992A1 AR P220101411 A ARP220101411 A AR P220101411A AR P220101411 A ARP220101411 A AR P220101411A AR 125992 A1 AR125992 A1 AR 125992A1
Authority
AR
Argentina
Prior art keywords
marc1
expression
adema
galnac
inhibiting
Prior art date
Application number
ARP220101411A
Other languages
English (en)
Inventor
Henryk Han Wen Pursell Natalie Wayne Lai Chengjung Haynes William Geoffrey Ding Zhihao Dudek
Original Assignee
Novo Nordisk As
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 Novo Nordisk As, Dicerna Pharmaceuticals Inc filed Critical Novo Nordisk As
Publication of AR125992A1 publication Critical patent/AR125992A1/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
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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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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Abstract

En la presente descripción se proporcionan oligonucleótidos que inhiben la expresión de MARC1. Además, se proporcionan composiciones que incluyen los mismos y usos de estos, particularmente usos relacionados con el tratamiento de enfermedades, trastornos y/o afecciones asociados con la expresión de MARC1. Reivindicación 1: Un oligonucleótido de ARNi bicatenario (ARNbci) para inhibir la expresión de MARC1, en donde dicho ARNbc comprende una hebra sentido y una hebra antisentido, la hebra antisentido comprende una región de complementariedad con un transcrito de ARN de MARC1, en donde la hebra sentido comprende la secuencia y todas las modificaciones de 5’-mGs-mG-mC-mU-mA-mG-mA-fG-fA-fA-fG-mA-mA-mA-mG-mU-mU-mA-mA-mA-mG-mC-mA-mG-mC-mC-mG-[ademA-GalNAc]-[ademA-GalNAc]-[ademA-GalNAc]-mG-mG-mC-mU-mG-mC-3’ (SEQ ID Nº 1615), y en donde la hebra antisentido comprende la secuencia y todas las modificaciones de 5’-MeFosfonato-4O-mUs-fUs-fUs-fA-fA-mC-fU-mU-mU-fC-mU-mU-mC-fU-mC-mU-mA-mG-mC-mCs-mGs-mG-3’ (SEQ ID Nº 1651), en donde mC, mA, mG, mU = 2’-OMe ribonucleósidos; fA, fC, fG, fU = 2’F ribonucleósidos; s = fosforotioato, y en donde ademA-GalNAc = fórmula (1). Reivindicación 13: Una composición farmacéutica que comprende el oligonucleótido de ARNi de acuerdo con cualquiera de las reivindicaciones 1 - 12, y un portador, agente de suministro o excipiente farmacéuticamente aceptables.
ARP220101411A 2021-05-28 2022-05-27 Composiciones y métodos para inhibir la expresión del componente 1 de reducción de amidoxima mitocondrial (marc1) AR125992A1 (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US202163194395P 2021-05-28 2021-05-28
EP21183860 2021-07-06

Publications (1)

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AR125992A1 true AR125992A1 (es) 2023-08-30

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US (2) US11655473B2 (es)
EP (1) EP4347820A1 (es)
JP (2) JP7463621B2 (es)
KR (1) KR102662071B1 (es)
AR (1) AR125992A1 (es)
AU (1) AU2022282579A1 (es)
BR (1) BR112023023807A2 (es)
CA (1) CA3215836A1 (es)
CL (1) CL2023003363A1 (es)
CO (1) CO2023016136A2 (es)
IL (1) IL308418A (es)
MX (1) MX2023013504A (es)
PE (1) PE20250074A1 (es)
TW (1) TWI831225B (es)
WO (1) WO2022248665A1 (es)

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Publication number Priority date Publication date Assignee Title
US11655473B2 (en) * 2021-05-28 2023-05-23 Novo Nordisk A/S Compositions and methods for inhibiting mitochondria amidoxime reducing component 1 (MARC1) expression
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US20250297260A1 (en) 2024-03-22 2025-09-25 Takeda Pharmaceutical Company Limited Compositions and methods for inhibiting cytochrome p450 family 7 subfamily a member 1 (cyp7a1) expression

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TWI831225B (zh) 2024-02-01
WO2022248665A1 (en) 2022-12-01
JP7463621B2 (ja) 2024-04-08
US20230042451A1 (en) 2023-02-09
KR102662071B1 (ko) 2024-05-03
KR20230130609A (ko) 2023-09-12
PE20250074A1 (es) 2025-01-13
EP4347820A1 (en) 2024-04-10
US11655473B2 (en) 2023-05-23
US20230340490A1 (en) 2023-10-26
MX2023013504A (es) 2024-02-23
CA3215836A1 (en) 2022-12-01
JP2024508069A (ja) 2024-02-22
BR112023023807A2 (pt) 2024-02-20
TW202307206A (zh) 2023-02-16
IL308418A (en) 2024-01-01
CO2023016136A2 (es) 2024-02-15
JP2024071571A (ja) 2024-05-24
AU2022282579A9 (en) 2023-11-16
AU2022282579A1 (en) 2023-11-02
CL2023003363A1 (es) 2024-06-21

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