AR129311A1 - Composiciones y métodos para inhibir la expresión de mapt - Google Patents

Composiciones y métodos para inhibir la expresión de mapt

Info

Publication number
AR129311A1
AR129311A1 ARP230101180A ARP230101180A AR129311A1 AR 129311 A1 AR129311 A1 AR 129311A1 AR P230101180 A ARP230101180 A AR P230101180A AR P230101180 A ARP230101180 A AR P230101180A AR 129311 A1 AR129311 A1 AR 129311A1
Authority
AR
Argentina
Prior art keywords
gene expression
mapt
pharmaceutical composition
reducing
rnai oligonucleotide
Prior art date
Application number
ARP230101180A
Other languages
English (en)
Inventor
Bob Dale Brown
Henryk T Dudek
Seongmoon Cheong
Shiyu Wang
Travis Grim
Matthew Guese Costales
Maire Jung
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 AR129311A1 publication Critical patent/AR129311A1/es

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    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering N.A.
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/322—2'-R Modification
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/344—Position-specific modifications, e.g. on every purine, at the 3'-end
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C12N2310/3515—Lipophilic moiety, e.g. cholesterol
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3521—Methyl
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/353—Nature of the modification linked to the nucleic acid via an atom other than carbon
    • C12N2310/3533—Halogen

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Chemical & Material Sciences (AREA)
  • Molecular Biology (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Biophysics (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)

Abstract

En el presente documento se proporcionan oligonucleótidos que inhiben la expresión génica MAPT, incluidos los oligonucleótidos conjugados con un ligando de direccionamiento (por ejemplo, un resto lipídico). 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 génica MAPT. Reivindicación 1: Un oligonucleótido de ARNi para reducir la expresión génica de MAPT, comprendiendo el oligonucleótido una cadena de sentido y una cadena antisentido, en donde la cadena de sentido y la cadena antisentido forman una región dúplex, en donde la cadena antisentido comprende una región de complementariedad con una secuencia diana de ARNm de MAPT de cualquiera de SEQ ID Nº 912 - 1295, y en donde la región de complementariedad tiene una longitud de al menos 15 nucleótidos contiguos. Reivindicación 101: Una composición farmacéutica que comprende el oligonucleótido de ARNi de cualquiera de las reivindicaciones 1 a 100, y un portador, agente de administración o excipiente farmacéuticamente aceptable. Reivindicación 104: Un método para reducir la expresión génica de MAPT en una célula, una población de células o un sujeto, comprendiendo el método el paso de: i. poner en contacto la célula o la población de células con el oligonucleótido de ARNi de cualquiera de las reivindicaciones 1 a 100, o la composición farmacéutica de la reivindicación 101; o ii. administrarle al sujeto el oligonucleótido de ARNi de cualquiera de las reivindicaciones 1 a 100, o la composición farmacéutica de la reivindicación 101. Reivindicación 105: El método de la reivindicación 104, en donde reducir la expresión génica de MAPT comprende reducir una cantidad o nivel de ARNm de MAPT, una cantidad o nivel de proteína Tau, o ambos. Reivindicación 113: El método de cualquiera de las reivindicaciones 102 - 112, en donde el oligonucleótido de ARNi o la composición farmacéutica se administra en combinación con una segunda composición o agente terapéutico.
ARP230101180A 2022-05-12 2023-05-12 Composiciones y métodos para inhibir la expresión de mapt AR129311A1 (es)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US202263364609P 2022-05-12 2022-05-12

Publications (1)

Publication Number Publication Date
AR129311A1 true AR129311A1 (es) 2024-08-14

Family

ID=86710745

Family Applications (1)

Application Number Title Priority Date Filing Date
ARP230101180A AR129311A1 (es) 2022-05-12 2023-05-12 Composiciones y métodos para inhibir la expresión de mapt

Country Status (5)

Country Link
US (1) US20230416742A1 (es)
AR (1) AR129311A1 (es)
AU (1) AU2023269281A1 (es)
TW (1) TW202400792A (es)
WO (1) WO2023220349A1 (es)

Family Cites Families (26)

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CZ308053B6 (cs) 2000-12-01 2019-11-27 Max Planck Gesellschaft Izolovaná molekula dvouřetězcové RNA, způsob její výroby a její použití
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GB0605337D0 (en) * 2006-03-17 2006-04-26 Genomica Sau Treatment of CNS conditions
JP5721426B2 (ja) * 2007-04-05 2015-05-20 ザ ジェイ．ディヴィッド グラッドストン インスティテューツ 神経の過度な興奮を緩和する薬剤を識別する方法
ES2708944T3 (es) 2008-09-22 2019-04-12 Dicerna Pharmaceuticals Inc Composiciones y métodos para la inhibición específica de la expresión de genes por DSRNA que tenga modificaciones
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Publication number Publication date
US20230416742A1 (en) 2023-12-28
WO2023220349A1 (en) 2023-11-16
TW202400792A (zh) 2024-01-01
AU2023269281A1 (en) 2024-10-10

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