AU2018269935B2 - Compositions and methods for the treatment of complications and disorders relating to von Willebrand factor - Google Patents

Compositions and methods for the treatment of complications and disorders relating to von Willebrand factor Download PDF

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AU2018269935B2
AU2018269935B2 AU2018269935A AU2018269935A AU2018269935B2 AU 2018269935 B2 AU2018269935 B2 AU 2018269935B2 AU 2018269935 A AU2018269935 A AU 2018269935A AU 2018269935 A AU2018269935 A AU 2018269935A AU 2018269935 B2 AU2018269935 B2 AU 2018269935B2
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vwf
synthetic polynucleotide
synthetic
disorder
aptamer
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AU2018269935A1 (en
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James C. Gilbert
Shuhao Zhu
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Band Therapeutics LLC
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Band Therapeutics LLC
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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
    • C12N15/115Aptamers, i.e. nucleic acids binding a target molecule specifically and with high affinity without hybridising therewith ; Nucleic acids binding to non-nucleic acids, e.g. aptamers
    • 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
    • A61K31/713Double-stranded nucleic acids or oligonucleotides
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    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00Drugs for disorders of the blood or the extracellular fluid
    • A61P7/02Antithrombotic agents; Anticoagulants; Platelet aggregation inhibitors
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • A61P7/06Antianaemics
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61P9/00Drugs for disorders of the cardiovascular system
    • A61P9/10Drugs for disorders of the cardiovascular system for treating ischaemic or atherosclerotic diseases, e.g. antianginal drugs, coronary vasodilators, drugs for myocardial infarction, retinopathy, cerebrovascula insufficiency, renal arteriosclerosis
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    • C07HSUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • 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
    • C07H21/02Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with ribosyl as saccharide radical
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    • C07HSUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • 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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    • C12N2310/00Structure or type of the nucleic acid
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    • C12N2320/50Methods for regulating/modulating their activity
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    • YGENERAL TAGGING OF NEW TECHNOLOGICAL DEVELOPMENTS; GENERAL TAGGING OF CROSS-SECTIONAL TECHNOLOGIES SPANNING OVER SEVERAL SECTIONS OF THE IPC; TECHNICAL SUBJECTS COVERED BY FORMER USPC CROSS-REFERENCE ART COLLECTIONS [XRACs] AND DIGESTS
    • Y02TECHNOLOGIES OR APPLICATIONS FOR MITIGATION OR ADAPTATION AGAINST CLIMATE CHANGE
    • Y02ATECHNOLOGIES FOR ADAPTATION TO CLIMATE CHANGE
    • Y02A50/00TECHNOLOGIES FOR ADAPTATION TO CLIMATE CHANGE in human health protection, e.g. against extreme weather
    • Y02A50/30Against vector-borne diseases, e.g. mosquito-borne, fly-borne, tick-borne or waterborne diseases whose impact is exacerbated by climate change

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  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
  • Saccharide Compounds (AREA)
AU2018269935A 2017-05-19 2018-05-18 Compositions and methods for the treatment of complications and disorders relating to von Willebrand factor Active AU2018269935B2 (en)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201762508530P 2017-05-19 2017-05-19
US62/508,530 2017-05-19
US201862638579P 2018-03-05 2018-03-05
US62/638,579 2018-03-05
PCT/US2018/033376 WO2018213697A1 (en) 2017-05-19 2018-05-18 Compositions and methods for the treatment of complications and disorders relating to von willebrand factor

Publications (2)

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AU2018269935A1 AU2018269935A1 (en) 2019-11-28
AU2018269935B2 true AU2018269935B2 (en) 2024-05-16

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AU2018269935A Active AU2018269935B2 (en) 2017-05-19 2018-05-18 Compositions and methods for the treatment of complications and disorders relating to von Willebrand factor

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US (3) US11060094B2 (enExample)
EP (1) EP3624801A4 (enExample)
JP (2) JP7317805B2 (enExample)
KR (1) KR102830459B1 (enExample)
CN (2) CN117487811A (enExample)
AU (1) AU2018269935B2 (enExample)
CA (1) CA3058001A1 (enExample)
IL (1) IL269641B2 (enExample)
MX (1) MX390865B (enExample)
TW (1) TWI806868B (enExample)
WO (1) WO2018213697A1 (enExample)

Families Citing this family (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
BR112022015326A2 (pt) * 2020-02-04 2022-10-11 Takeda Pharmaceuticals Co Tratamento de menorragia em pacientes com doença de von willebrand severa por administração de vwf recombinante
US20230038761A1 (en) * 2020-02-04 2023-02-09 Band Therapeutics, Llc Regulation of von willebrand factor (vwf)
WO2022115502A1 (en) * 2020-11-24 2022-06-02 Band Therapeutics, Llc Compositions and methods for treatment of bleeding disorders
CN116723863A (zh) * 2020-11-24 2023-09-08 邦德治疗有限公司 用于治疗出血性病症的组合物和方法
US12090243B2 (en) 2021-03-24 2024-09-17 Aleo Bme, Inc. Compositions comprising fluid gels for tissue separation

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2010091396A2 (en) * 2009-02-09 2010-08-12 Archemix Corp. Aptamers to von willerbrand factor and their use as thrombotic, hematologic and cardiovascular disease therapeutics

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JP2008512098A (ja) * 2004-09-07 2008-04-24 アーケミックス コーポレイション フォン・ビルブラント因子に対するアプタマー、および血栓性疾患の処置剤としてのその使用
US7566701B2 (en) 2004-09-07 2009-07-28 Archemix Corp. Aptamers to von Willebrand Factor and their use as thrombotic disease therapeutics
US7563601B1 (en) * 2005-06-01 2009-07-21 City Of Hope Artificial riboswitch for controlling pre-mRNA splicing
WO2008066621A2 (en) * 2006-10-19 2008-06-05 Duke University Reversible platelet inhibition
WO2008150495A2 (en) 2007-06-01 2008-12-11 Archemix Corp. Vwf aptamer formulations and methods for use
US20170044545A1 (en) * 2014-04-24 2017-02-16 Ribomic Inc. Aptamer for bonding to autotaxin and inhibiting biological activity of autotaxin, and use for same
EP3266871B1 (en) * 2015-03-06 2021-04-28 TAGCyx Biotechnologies Inc. Method for stabilizing dna aptamers
US10760083B2 (en) 2015-10-30 2020-09-01 Tagcyx Biotechnologies Inc. DNA aptamer that binds to vWF
US20230038761A1 (en) * 2020-02-04 2023-02-09 Band Therapeutics, Llc Regulation of von willebrand factor (vwf)
WO2022115502A1 (en) * 2020-11-24 2022-06-02 Band Therapeutics, Llc Compositions and methods for treatment of bleeding disorders
WO2023039522A1 (en) * 2021-09-10 2023-03-16 Guardian Therapeutics, Llc Fatty acid conjugates of nucleic acids

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2010091396A2 (en) * 2009-02-09 2010-08-12 Archemix Corp. Aptamers to von willerbrand factor and their use as thrombotic, hematologic and cardiovascular disease therapeutics

Also Published As

Publication number Publication date
IL269641A (en) 2019-11-28
KR102830459B1 (ko) 2025-07-09
EP3624801A4 (en) 2021-02-24
RU2019136508A (ru) 2021-06-22
WO2018213697A1 (en) 2018-11-22
CN110520128B (zh) 2023-11-14
US20210292763A1 (en) 2021-09-23
CN117487811A (zh) 2024-02-02
TWI806868B (zh) 2023-07-01
JP7781815B2 (ja) 2025-12-08
AU2018269935A1 (en) 2019-11-28
EP3624801A1 (en) 2020-03-25
US20260092283A1 (en) 2026-04-02
US11060094B2 (en) 2021-07-13
JP2023093535A (ja) 2023-07-04
KR20200008122A (ko) 2020-01-23
TW201900877A (zh) 2019-01-01
RU2019136508A3 (enExample) 2022-02-14
NZ758741A (en) 2025-09-26
JP7317805B2 (ja) 2023-07-31
IL269641B2 (en) 2024-12-01
JP2020522566A (ja) 2020-07-30
CN110520128A (zh) 2019-11-29
IL269641B1 (en) 2024-08-01
MX390865B (es) 2025-03-12
US20200165612A1 (en) 2020-05-28
CA3058001A1 (en) 2018-11-22
MX2019013700A (es) 2020-01-15

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