AR124713A1 - Composiciones y métodos para inhibir la expresión de genes en el sistema nervioso central - Google Patents

Composiciones y métodos para inhibir la expresión de genes en el sistema nervioso central

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Publication number
AR124713A1
AR124713A1 ARP220100166A ARP220100166A AR124713A1 AR 124713 A1 AR124713 A1 AR 124713A1 AR P220100166 A ARP220100166 A AR P220100166A AR P220100166 A ARP220100166 A AR P220100166A AR 124713 A1 AR124713 A1 AR 124713A1
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AR
Argentina
Prior art keywords
expression
cns
oligonucleotide
inhibit
antisense
Prior art date
Application number
ARP220100166A
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English (en)
Inventor
Maire Jung
Travis Grim
Weimin Wang
Bob Dale Brown
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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.)
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Publication date
Application filed by Dicerna Pharmaceuticals Inc filed Critical Dicerna Pharmaceuticals Inc
Publication of AR124713A1 publication Critical patent/AR124713A1/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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    • A61K47/51—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
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    • A61K47/51—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
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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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    • C12Y102/00—Oxidoreductases acting on the aldehyde or oxo group of donors (1.2)
    • C12Y102/01—Oxidoreductases acting on the aldehyde or oxo group of donors (1.2) with NAD+ or NADP+ as acceptor (1.2.1)
    • C12Y102/01003—Aldehyde dehydrogenase (NAD+) (1.2.1.3)

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Abstract

En el presente documento se proporcionan conjugados de oligonucleótidos que inhiben o reducen la expresión de genes diana en el SNC. También se proporcionan composiciones que incluyen los mismos y usos de los mismos, particularmente usos relacionados con el tratamiento de enfermedades, trastornos y/o condiciones asociadas con la expresión de genes diana en el SNC. Reivindicación 1: Un conjugado de oligonucleótido-ligando para reducir la expresión de un ARNm diana en el sistema nervioso central (SNC), que comprende (i) un oligonculeótido bicatenario que comprende una cadena antisentido de 15 a 30 nucleótidos de longitud y una cadena sentido de 15 a 40 nucleótidos de longitud, en donde cada una la cadena antisentido y la sentido comprende un extremo 5’ y un extremo 3’, en donde la cadena antisentido y la sentido forman una región dúplex, en donde la cadena antisentido tiene una región de complementariedad con una secuencia diana en el ARNm diana en el SNC, en donde la región de complementariedad es de, al menos, 15 nucleótidos contiguos de longitud, en donde el oligonucleótido comprende un bucle de tallo; y (ii) uno o más restos lipídicos conjugados con el bucle de tallo.
ARP220100166A 2021-01-28 2022-01-27 Composiciones y métodos para inhibir la expresión de genes en el sistema nervioso central AR124713A1 (es)

Applications Claiming Priority (1)

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US202163142877P 2021-01-28 2021-01-28

Publications (1)

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AR124713A1 true AR124713A1 (es) 2023-04-26

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ID=82652781

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ARP220100166A AR124713A1 (es) 2021-01-28 2022-01-27 Composiciones y métodos para inhibir la expresión de genes en el sistema nervioso central

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US (1) US20240117351A1 (es)
EP (1) EP4284933A1 (es)
JP (1) JP2024505035A (es)
CN (1) CN116802295A (es)
AR (1) AR124713A1 (es)
TW (1) TW202246502A (es)
WO (1) WO2022165201A1 (es)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2024097821A1 (en) * 2022-11-02 2024-05-10 Sanegene Bio Usa Inc. Nucleotide-based enhancement agent for rna delivery and therapy

Family Cites Families (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20110118340A1 (en) * 2008-02-08 2011-05-19 Muthiah Manoharan Delivery of rnai constructs to oligodendrocytes
ES2747842T3 (es) * 2014-12-15 2020-03-11 Dicerna Pharmaceuticals Inc Acidos nucleicos de doble hebra modificados por ligando

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TW202246502A (zh) 2022-12-01
WO2022165201A1 (en) 2022-08-04
JP2024505035A (ja) 2024-02-02
EP4284933A1 (en) 2023-12-06
US20240117351A1 (en) 2024-04-11
CN116802295A (zh) 2023-09-22

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