PE20211197A1 - Oligonucleotidos para modular la expresion de tmem106b - Google Patents

Oligonucleotidos para modular la expresion de tmem106b

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Publication number
PE20211197A1
PE20211197A1 PE2020001186A PE2020001186A PE20211197A1 PE 20211197 A1 PE20211197 A1 PE 20211197A1 PE 2020001186 A PE2020001186 A PE 2020001186A PE 2020001186 A PE2020001186 A PE 2020001186A PE 20211197 A1 PE20211197 A1 PE 20211197A1
Authority
PE
Peru
Prior art keywords
oligonucleotide
nucleotides
tmem106b
oligonucleotides
modulate
Prior art date
Application number
PE2020001186A
Other languages
English (en)
Inventor
Peter Hagedorn
Marianne L Jensen
Lukasz Kielpinski
Amy Easton
Benny Chih
Original Assignee
Genentech 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 Genentech Inc filed Critical Genentech Inc
Publication of PE20211197A1 publication Critical patent/PE20211197A1/es

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    • C—CHEMISTRY; METALLURGY
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    • 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
    • C12N15/1138—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 receptors or cell surface proteins
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • 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
    • C12N2310/11—Antisense
    • 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
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    • 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
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/323—Chemical structure of the sugar modified ring structure
    • C12N2310/3231—Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
    • 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
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/341—Gapmers, i.e. of the type ===---===
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications
    • C—CHEMISTRY; METALLURGY
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    • 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
    • C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3521—Methyl

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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)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Epidemiology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)

Abstract

SE REFIERE A UN OLIGONUCLEOTIDO ANTISENTIDO MONOCATENARIO DE 10 A 50 NUCLEOTIDOS DE LONGITUD, QUE COMPRENDE UNA SECUENCIA DE NUCLEOTIDOS CONTIGUOS DE 10 A 30 NUCLEOTIDOS DE LONGITUD QUE ES AL MENOS 90 % COMPLEMENTARIO DE UN ACIDO NUCLEICO DIANA DE LA PROTEINA TRANSMEMBRANA 106B DE MAMIFERO (TMEM106B); EN DONDE EL OLIGONUCLEOTIDO ES UN siARN O shARN O FORMA LA CADENA GUIA DE UN COMPLEJO DE siARN O shARN. TAMBIEN SE REFIERE A UNA COMPOSICION FARMACEUTICA QUE COMPRENDE DICHO OLIGONUCLEOTIDO QUE ES UTIL EN EL TRATAMIENTO DE ENFERMEDADES NEURODEGENERATIVAS COMO DEGENERACION LOBULAR FRONTOTEMPORAL (FTLD), ENFERMEDAD DE PARKINSON, LEUCODISTROFIAS HIPOMIELINIZANTES, ENFERMEDAD DE ALZHEIMER.
PE2020001186A 2018-02-09 2019-02-08 Oligonucleotidos para modular la expresion de tmem106b PE20211197A1 (es)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
EP18156142 2018-02-09
US201862669251P 2018-05-09 2018-05-09
PCT/EP2019/053116 WO2019154979A1 (en) 2018-02-09 2019-02-08 Oligonucleotides for modulating tmem106b expression

Publications (1)

Publication Number Publication Date
PE20211197A1 true PE20211197A1 (es) 2021-07-01

Family

ID=65433647

Family Applications (1)

Application Number Title Priority Date Filing Date
PE2020001186A PE20211197A1 (es) 2018-02-09 2019-02-08 Oligonucleotidos para modular la expresion de tmem106b

Country Status (17)

Country Link
US (1) US11485975B2 (es)
EP (1) EP3749766A1 (es)
JP (1) JP7281474B2 (es)
KR (1) KR20200108315A (es)
CN (1) CN111868245A (es)
AU (1) AU2019219194A1 (es)
BR (1) BR112020016170A2 (es)
CA (1) CA3088071A1 (es)
CL (1) CL2020002038A1 (es)
CR (1) CR20200346A (es)
IL (1) IL276357A (es)
MA (1) MA51795A (es)
MX (1) MX2020008290A (es)
PE (1) PE20211197A1 (es)
RU (1) RU2020125769A (es)
SG (1) SG11202007093YA (es)
WO (1) WO2019154979A1 (es)

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EP4058152A4 (en) * 2019-11-14 2024-05-08 The Board of Regents of the University of Oklahoma PROSTATE CANCER TREATMENTS USING OLIGONUCLEOTIDES INTERFERENCE
JP2023527684A (ja) * 2020-05-11 2023-06-30 ジェネンテック， インコーポレイテッド 神経疾患を治療するための補体成分ｃ１ｓ阻害剤、並びにそれを使用する関連組成物、システム及び方法
WO2023141507A1 (en) * 2022-01-20 2023-07-27 Genentech, Inc. Antisense oligonucleotides for modulating tmem106b expression

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Also Published As

Publication number Publication date
JP2021514182A (ja) 2021-06-10
RU2020125769A (ru) 2022-03-10
BR112020016170A2 (pt) 2020-12-15
MX2020008290A (es) 2020-09-25
RU2020125769A3 (es) 2022-03-10
KR20200108315A (ko) 2020-09-17
MA51795A (fr) 2020-12-16
AU2019219194A1 (en) 2020-08-06
US20210054383A1 (en) 2021-02-25
CL2020002038A1 (es) 2020-10-23
IL276357A (en) 2020-09-30
CA3088071A1 (en) 2019-08-15
CN111868245A (zh) 2020-10-30
JP7281474B2 (ja) 2023-05-25
US11485975B2 (en) 2022-11-01
SG11202007093YA (en) 2020-08-28
WO2019154979A1 (en) 2019-08-15
EP3749766A1 (en) 2020-12-16
CR20200346A (es) 2020-10-19

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