BR122020024446B8 - Compostos para modulação da expressão do receptor do hormônio de crescimento - Google Patents

Compostos para modulação da expressão do receptor do hormônio de crescimento

Info

Publication number
BR122020024446B8
BR122020024446B8 BR122020024446A BR122020024446A BR122020024446B8 BR 122020024446 B8 BR122020024446 B8 BR 122020024446B8 BR 122020024446 A BR122020024446 A BR 122020024446A BR 122020024446 A BR122020024446 A BR 122020024446A BR 122020024446 B8 BR122020024446 B8 BR 122020024446B8
Authority
BR
Brazil
Prior art keywords
growth hormone
compounds
hormone receptor
modulation
receptor expression
Prior art date
Application number
BR122020024446A
Other languages
English (en)
Portuguese (pt)
Other versions
BR122020024446B1 (pt
Inventor
P Prakash Thazha
P Seth Punit
E Swayze Eric
Bhanot Sanjay
M Freier Susan
Bui Huynh-Hoa
Original Assignee
Ionis 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 Ionis Pharmaceuticals Inc filed Critical Ionis Pharmaceuticals Inc
Publication of BR122020024446B1 publication Critical patent/BR122020024446B1/pt
Publication of BR122020024446B8 publication Critical patent/BR122020024446B8/pt

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    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • C12N15/1138Non-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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50Medicinal 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
    • A61K47/51Medicinal 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
    • A61K47/54Medicinal 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 the modifying agent being an organic compound
    • A61K47/549Sugars, nucleosides, nucleotides or nucleic acids
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50Medicinal 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
    • A61K47/51Medicinal 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
    • A61K47/54Medicinal 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 the modifying agent being an organic compound
    • A61K47/555Medicinal 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 the modifying agent being an organic compound pre-targeting systems involving an organic compound, other than a peptide, protein or antibody, for targeting specific cells
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50Medicinal 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
    • A61K47/51Medicinal 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
    • A61K47/56Medicinal 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 the modifying agent being an organic macromolecular compound, e.g. an oligomeric, polymeric or dendrimeric molecule
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P19/00Drugs for skeletal disorders
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    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P5/00Drugs for disorders of the endocrine system
    • A61P5/06Drugs for disorders of the endocrine system of the anterior pituitary hormones, e.g. TSH, ACTH, FSH, LH, PRL, GH
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    • C07H21/00Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • C07H21/04Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with deoxyribosyl as saccharide radical
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    • C07K1/00General methods for the preparation of peptides, i.e. processes for the organic chemical preparation of peptides or proteins of any length
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    • C12N2310/00Structure or type of the nucleic acid
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    • C12N2310/3212'-O-R Modification
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    • C12N2310/32Chemical structure of the sugar
    • C12N2310/323Chemical structure of the sugar modified ring structure
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    • C12N2310/33415-Methylcytosine
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    • C12N2310/34Spatial arrangement of the modifications
    • C12N2310/341Gapmers, i.e. of the type ===---===
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    • C12N2310/352Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3525MOE, methoxyethoxy

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  • Cell Biology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Saccharide Compounds (AREA)
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BR122020024446A 2014-05-01 2015-05-01 Compostos para modulação da expressão do receptor do hormônio de crescimento BR122020024446B8 (pt)

Applications Claiming Priority (8)

Application Number Priority Date Filing Date Title
US201461987470P 2014-05-01 2014-05-01
US61/987,470 2014-05-01
US201462061071P 2014-10-07 2014-10-07
US62/061,071 2014-10-07
US201462082511P 2014-11-20 2014-11-20
US62/082,511 2014-11-20
PCT/US2015/028887 WO2015168618A2 (en) 2014-05-01 2015-05-01 Compositions and methods for modulating growth hormone receptor expression
BR112016022742-5A BR112016022742B1 (pt) 2014-05-01 2015-05-01 Composto químico, composição compreendendo o composto e uso dos mesmos

Publications (2)

Publication Number Publication Date
BR122020024446B1 BR122020024446B1 (pt) 2022-04-26
BR122020024446B8 true BR122020024446B8 (pt) 2022-06-28

Family

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Application Number Title Priority Date Filing Date
BR112016022742-5A BR112016022742B1 (pt) 2014-05-01 2015-05-01 Composto químico, composição compreendendo o composto e uso dos mesmos
BR122020024446A BR122020024446B8 (pt) 2014-05-01 2015-05-01 Compostos para modulação da expressão do receptor do hormônio de crescimento

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BR112016022742-5A BR112016022742B1 (pt) 2014-05-01 2015-05-01 Composto químico, composição compreendendo o composto e uso dos mesmos

Country Status (14)

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US (5) US9994855B2 (https=)
EP (3) EP3757215A3 (https=)
JP (2) JP6667453B2 (https=)
KR (2) KR102149571B1 (https=)
CN (2) CN110903337A (https=)
AU (2) AU2015252841B2 (https=)
BR (2) BR112016022742B1 (https=)
CA (1) CA2942570A1 (https=)
DK (1) DK3137604T3 (https=)
ES (1) ES2812099T3 (https=)
IL (2) IL247716B (https=)
MX (2) MX373283B (https=)
RU (1) RU2724527C2 (https=)
WO (1) WO2015168618A2 (https=)

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US11459564B2 (en) 2017-12-21 2022-10-04 Ionis Pharmaceuticals, Inc. Modulation of frataxin expression
JP7672394B2 (ja) * 2019-09-03 2025-05-07 アークトゥラス・セラピューティクス・インコーポレイテッド 治療的に活性な複合体のアシアロ糖タンパク質受容体媒介送達
CA3158917A1 (en) * 2019-10-24 2021-04-29 Genevant Sciences Gmbh Conjugates and methods for treating acromegaly
GB2613225B (en) 2020-03-04 2024-01-03 Verve Therapeutics Inc Compositions and methods for targeted RNA delivery
US20240335551A1 (en) * 2021-05-20 2024-10-10 Olix Us, Inc. Functional moieties and their uses and synthetic preparation
WO2023015223A2 (en) * 2021-08-03 2023-02-09 Verve Therapeutics, Inc. Compositions and methods for targeted rna delivery
EP4396193A4 (en) * 2021-08-30 2025-07-02 Hongene Biotech Corp FUNCTIONALIZED N-ACETYLGALACTOSAMINE ANALOGUES
JP2024546585A (ja) 2021-12-15 2024-12-26 ホンジーン バイオテック コーポレイション 官能基化n-アセチルガラクトサミンアナログ
CN119654412A (zh) * 2022-06-27 2025-03-18 中美瑞康核酸技术(南通)研究院有限公司 激活补体因子h表达的寡核苷酸调节剂
WO2024118503A1 (en) 2022-11-28 2024-06-06 Hongene Biotech Corporation Functionalized n-acetylgalactosamine analogs
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