CA3057292A1 - Modified oligonucleotides and therapeutic uses thereof - Google Patents

Modified oligonucleotides and therapeutic uses thereof Download PDF

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Publication number
CA3057292A1
CA3057292A1 CA3057292A CA3057292A CA3057292A1 CA 3057292 A1 CA3057292 A1 CA 3057292A1 CA 3057292 A CA3057292 A CA 3057292A CA 3057292 A CA3057292 A CA 3057292A CA 3057292 A1 CA3057292 A1 CA 3057292A1
Authority
CA
Canada
Prior art keywords
compound
moiety
pharmaceutical composition
protein
weight
Prior art date
Legal status (The legal status 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 status listed.)
Abandoned
Application number
CA3057292A
Other languages
English (en)
French (fr)
Inventor
Alexander Roloff
Nathan C. Gianneschi
Cassandra E. CALLMANN
Matthew P. THOMPSON
Paul A. Bertin
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Vybyl Holdings Inc
University of California San Diego UCSD
Original Assignee
Vybyl Holdings Inc
University of California San Diego UCSD
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 Vybyl Holdings Inc, University of California San Diego UCSD filed Critical Vybyl Holdings Inc
Publication of CA3057292A1 publication Critical patent/CA3057292A1/en
Abandoned legal-status Critical Current

Links

Classifications

    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • General Health & Medical Sciences (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Epidemiology (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Molecular Biology (AREA)
  • Organic Chemistry (AREA)
  • Inorganic Chemistry (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Dermatology (AREA)
  • Immunology (AREA)
  • Biochemistry (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)
CA3057292A 2017-03-22 2018-03-21 Modified oligonucleotides and therapeutic uses thereof Abandoned CA3057292A1 (en)

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
CA3057292A1 true CA3057292A1 (en) 2018-09-27

Family

ID=63586170

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3057292A Abandoned CA3057292A1 (en) 2017-03-22 2018-03-21 Modified oligonucleotides and therapeutic uses thereof

Country Status (9)

Country Link
US (1) US20200046846A1 (enExample)
EP (1) EP3600439A4 (enExample)
JP (1) JP2020514383A (enExample)
KR (1) KR20190123351A (enExample)
CN (1) CN110636865A (enExample)
AU (1) AU2018237139A1 (enExample)
CA (1) CA3057292A1 (enExample)
SG (1) SG11201908771YA (enExample)
WO (1) WO2018175592A1 (enExample)

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20250270248A1 (en) * 2020-09-16 2025-08-28 Astrazeneca Ab Oligonucleotides conjugated to fatty acids
CN117980003A (zh) * 2021-09-10 2024-05-03 嘉德治疗有限责任公司 核酸的脂肪酸缀合物
WO2025028656A1 (ja) * 2023-08-02 2025-02-06 日東電工株式会社 オリゴヌクレオチドを製造する方法

Family Cites Families (14)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP0821001A4 (en) * 1995-03-31 2002-07-17 Drug Delivery System Inst Ltd AMIDITY 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
WO2007112414A2 (en) * 2006-03-27 2007-10-04 Isis Pharmaceuticals, Inc. 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
CN103649311A (zh) * 2011-06-03 2014-03-19 国立大学法人北海道大学 寡核苷酸衍生物、包含寡核苷酸衍生物的治疗用医药组合物及诊断用医药组合物、以及miRNA机能抑制用寡核苷酸衍生物
RU2599449C1 (ru) * 2011-12-15 2016-10-10 Байонир Корпорейшн Новые конъюгаты олигонуклеотидов и их применение
DK3019200T3 (da) * 2013-07-11 2022-06-20 Alnylam Pharmaceuticals Inc Oligonukleotid-ligand-konjugater og fremgangsmåde til fremstiling deraf
CN105792851B (zh) * 2013-09-13 2023-10-10 斯克利普斯研究所 修饰的治疗剂及其组合物
AU2014364589B2 (en) * 2013-12-18 2020-02-27 The California Institute For Biomedical Research Modified therapeutic agents, stapled peptide lipid conjugates, and compositions thereof
JP6773677B2 (ja) * 2015-03-17 2020-10-21 アローヘッド ファーマシューティカルズ インコーポレイテッド 改善されたジスルフィド含有アルキン連結剤
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
AU2016326392B2 (en) * 2015-09-22 2021-02-11 The Regents Of The University Of California Modified cytotoxins and their therapeutic use

Also Published As

Publication number Publication date
AU2018237139A1 (en) 2019-10-17
EP3600439A1 (en) 2020-02-05
KR20190123351A (ko) 2019-10-31
CN110636865A (zh) 2019-12-31
JP2020514383A (ja) 2020-05-21
SG11201908771YA (en) 2019-10-30
WO2018175592A1 (en) 2018-09-27
US20200046846A1 (en) 2020-02-13
EP3600439A4 (en) 2021-01-13

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Legal Events

Date Code Title Description
FZDE Discontinued

Effective date: 20230921