KR20190123351A - 개질된 올리고뉴클레오티드 및 이의 치료 용도 - Google Patents

개질된 올리고뉴클레오티드 및 이의 치료 용도 Download PDF

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Publication number
KR20190123351A
KR20190123351A KR1020197030864A KR20197030864A KR20190123351A KR 20190123351 A KR20190123351 A KR 20190123351A KR 1020197030864 A KR1020197030864 A KR 1020197030864A KR 20197030864 A KR20197030864 A KR 20197030864A KR 20190123351 A KR20190123351 A KR 20190123351A
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South Korea
Prior art keywords
compound
protein
pharmaceutical composition
moiety
group
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KR1020197030864A
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Korean (ko)
Inventor
알렉산더 롤로프
나탄 씨. 지안네스키
카산드라 이. 콜먼
매튜 피. 톰프슨
폴 에이. 버틴
Original Assignee
더 리젠츠 오브 더 유니버시티 오브 캘리포니아
바이빌 홀딩스, 인코포레이티드
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Application filed by 더 리젠츠 오브 더 유니버시티 오브 캘리포니아, 바이빌 홀딩스, 인코포레이티드 filed Critical 더 리젠츠 오브 더 유니버시티 오브 캘리포니아
Publication of KR20190123351A publication Critical patent/KR20190123351A/ko
Ceased legal-status Critical Current

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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—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
    • A61K47/50—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
    • 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
    • A61K47/68—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 the modifying agent being an antibody, an immunoglobulin or a fragment thereof, e.g. an Fc-fragment
    • A61K47/6801—Drug-antibody or immunoglobulin conjugates defined by the pharmacologically or therapeutically active agent
    • A61K47/6803—Drugs conjugated to an antibody or immunoglobulin, e.g. cisplatin-antibody conjugates
    • A61K47/6807—Drugs conjugated to an antibody or immunoglobulin, e.g. cisplatin-antibody conjugates the drug or compound being a sugar, nucleoside, nucleotide, nucleic acid, e.g. RNA antisense
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—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
    • A61K47/30—Macromolecular organic or inorganic compounds, e.g. inorganic polyphosphates
    • A61K47/42—Proteins; Polypeptides; Degradation products thereof; Derivatives thereof, e.g. albumin, gelatin or zein
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—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
    • A61K47/50—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
    • 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
    • A61K47/54—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 the modifying agent being an organic compound
    • A61K47/542—Carboxylic acids, e.g. a fatty acid or an amino acid
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—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
    • A61K47/50—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
    • 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
    • A61K47/62—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 the modifying agent being a protein, peptide or polyamino acid
    • A61K47/64—Drug-peptide, drug-protein or drug-polyamino acid conjugates, i.e. the modifying agent being a peptide, protein or polyamino acid which is covalently bonded or complexed to a therapeutically active agent
    • A61K47/643—Albumins, e.g. HSA, BSA, ovalbumin or a Keyhole Limpet Hemocyanin [KHL]
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • General Health & Medical Sciences (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Molecular Biology (AREA)
  • Organic Chemistry (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Dermatology (AREA)
  • Inorganic Chemistry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Biochemistry (AREA)
  • Immunology (AREA)
  • Biotechnology (AREA)
  • Genetics & Genomics (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Saccharide Compounds (AREA)
  • Medicinal Preparation (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
KR1020197030864A 2017-03-22 2018-03-21 개질된 올리고뉴클레오티드 및 이의 치료 용도 Ceased KR20190123351A (ko)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201762475185P 2017-03-22 2017-03-22
US62/475,185 2017-03-22
PCT/US2018/023578 WO2018175592A1 (en) 2017-03-22 2018-03-21 Modified oligonucleotides and therapeutic uses thereof

Publications (1)

Publication Number Publication Date
KR20190123351A true KR20190123351A (ko) 2019-10-31

Family

ID=63586170

Family Applications (1)

Application Number Title Priority Date Filing Date
KR1020197030864A Ceased KR20190123351A (ko) 2017-03-22 2018-03-21 개질된 올리고뉴클레오티드 및 이의 치료 용도

Country Status (9)

Country Link
US (1) US20200046846A1 (cg-RX-API-DMAC10.html)
EP (1) EP3600439A4 (cg-RX-API-DMAC10.html)
JP (1) JP2020514383A (cg-RX-API-DMAC10.html)
KR (1) KR20190123351A (cg-RX-API-DMAC10.html)
CN (1) CN110636865A (cg-RX-API-DMAC10.html)
AU (1) AU2018237139A1 (cg-RX-API-DMAC10.html)
CA (1) CA3057292A1 (cg-RX-API-DMAC10.html)
SG (1) SG11201908771YA (cg-RX-API-DMAC10.html)
WO (1) WO2018175592A1 (cg-RX-API-DMAC10.html)

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP4213883A1 (en) * 2020-09-16 2023-07-26 Astrazeneca AB Oligonucleotides conjugated to fatty acids
EP4398939A1 (en) * 2021-09-10 2024-07-17 Guardian Therapeutics, LLC Fatty acid conjugates of nucleic acids
EP4755898A1 (en) * 2023-08-02 2026-06-10 Nitto Denko Corporation Method for producing oligonucleotide

Family Cites Families (14)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA2216844A1 (en) * 1995-03-31 1996-10-03 Drug Delivery System Institute, Ltd. Amidite derivatives and oligonucleotide derivatives
US8969543B2 (en) * 2003-04-03 2015-03-03 Bioneer Corporation SiRNA-hydrophilic polymer conjugates for intracellular delivery of siRNA and method thereof
US20090306178A1 (en) * 2006-03-27 2009-12-10 Balkrishen Bhat Conjugated double strand compositions for use in gene modulation
KR101224828B1 (ko) * 2009-05-14 2013-01-22 (주)바이오니아 ｓｉＲＮＡ 접합체 및 그 제조방법
GB0910723D0 (en) * 2009-06-22 2009-08-05 Sylentis Sau Novel drugs for inhibition of gene expression
WO2012165616A1 (ja) * 2011-06-03 2012-12-06 国立大学法人北海道大学 オリゴヌクレオチド誘導体、オリゴヌクレオチド誘導体を含む治療用医薬組成物及び診断用医薬組成物、並びにｍｉＲＮＡ機能抑制用オリゴヌクレオチド誘導体
PL2796150T3 (pl) * 2011-12-15 2021-08-30 Bioneer Corporation Nowe koniugaty oligonukleotydowe i ich zastosowanie
WO2015006740A2 (en) * 2013-07-11 2015-01-15 Alnylam Pharmaceuticals, Inc. Oligonucleotide-ligand conjugates and process for their preparation
WO2015038938A1 (en) * 2013-09-13 2015-03-19 The California Institute For Biomedical Research Modified therapeutic agents and compositions thereof
US10039809B2 (en) * 2013-12-18 2018-08-07 The California Institute For Biomedical Research Modified therapeutic agents, stapled peptide lipid conjugates, and compositions thereof
US9803201B2 (en) * 2015-03-17 2017-10-31 Arrowhead Pharmaceuticals, Inc. Disulfide-containing alkyne linking agents
MA41794A (fr) * 2015-03-18 2018-01-23 The California Institute For Biomedical Res Agents thérapeutiques modifiés et compositions associées
WO2017030973A1 (en) * 2015-08-14 2017-02-23 University Of Massachusetts Bioactive conjugates for oligonucleotide delivery
MY193776A (en) * 2015-09-22 2022-10-27 Univ California Modified cytotoxins and their therapeutics use

Also Published As

Publication number Publication date
EP3600439A1 (en) 2020-02-05
CN110636865A (zh) 2019-12-31
US20200046846A1 (en) 2020-02-13
SG11201908771YA (en) 2019-10-30
JP2020514383A (ja) 2020-05-21
CA3057292A1 (en) 2018-09-27
WO2018175592A1 (en) 2018-09-27
AU2018237139A1 (en) 2019-10-17
EP3600439A4 (en) 2021-01-13

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