AU2013296557B2 - Dephosphorylated lysosomal storage disease proteins and methods of use thereof - Google Patents

Dephosphorylated lysosomal storage disease proteins and methods of use thereof Download PDF

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Publication number
AU2013296557B2
AU2013296557B2 AU2013296557A AU2013296557A AU2013296557B2 AU 2013296557 B2 AU2013296557 B2 AU 2013296557B2 AU 2013296557 A AU2013296557 A AU 2013296557A AU 2013296557 A AU2013296557 A AU 2013296557A AU 2013296557 B2 AU2013296557 B2 AU 2013296557B2
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polypeptide
ids
disease
protein
human
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AU2013296557A
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AU2013296557A1 (en
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Reinhard Gabathuler
Timothy Z. Vitalis
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Bioasis Technologies Inc
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Bioasis Technologies Inc
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Assigned to BIOASIS TECHNOLOGIES INC. reassignment BIOASIS TECHNOLOGIES INC. Request to Amend Deed and Register Assignors: BIOASIS TECHNOLOGIES, INC.
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    • 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
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • 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
    • A61K47/644—Transferrin, e.g. a lactoferrin or ovotransferrin
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • 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
    • C12N11/00—Carrier-bound or immobilised enzymes; Carrier-bound or immobilised microbial cells; Preparation thereof
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y301/00—Hydrolases acting on ester bonds (3.1)
    • C12Y301/06—Sulfuric ester hydrolases (3.1.6)
    • C12Y301/06013—Iduronate-2-sulfatase (3.1.6.13)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Zoology (AREA)
  • Genetics & Genomics (AREA)
  • Wood Science & Technology (AREA)
  • Medicinal Chemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Molecular Biology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Microbiology (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • Epidemiology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Hematology (AREA)
  • Diabetes (AREA)
  • Obesity (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Peptides Or Proteins (AREA)
  • Medicinal Preparation (AREA)
AU2013296557A 2012-07-31 2013-07-31 Dephosphorylated lysosomal storage disease proteins and methods of use thereof Ceased AU2013296557B2 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201261677959P 2012-07-31 2012-07-31
US61/677,959 2012-07-31
PCT/US2013/052939 WO2014022515A1 (en) 2012-07-31 2013-07-31 Dephosphorylated lysosomal storage disease proteins and methods of use thereof

Publications (2)

Publication Number Publication Date
AU2013296557A1 AU2013296557A1 (en) 2015-02-26
AU2013296557B2 true AU2013296557B2 (en) 2019-04-18

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AU2013296557A Ceased AU2013296557B2 (en) 2012-07-31 2013-07-31 Dephosphorylated lysosomal storage disease proteins and methods of use thereof

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US (3) US9932565B2 (https=)
EP (1) EP2880156B1 (https=)
JP (1) JP6433424B2 (https=)
CN (1) CN104662150B (https=)
AU (1) AU2013296557B2 (https=)
CA (2) CA3140358A1 (https=)
ES (1) ES2647082T3 (https=)
WO (1) WO2014022515A1 (https=)

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CN103747807B (zh) 2011-07-05 2016-12-07 比奥阿赛斯技术有限公司 P97‑抗体缀合物和使用方法
AU2012294673B2 (en) 2011-08-05 2015-11-26 Bioasis Technologies Inc. p97 fragments with transfer activity
CA3140358A1 (en) 2012-07-31 2014-02-06 Bioasis Technologies, Inc. Dephosphorylated lysosomal storage disease proteins and methods of use thereof
AU2014243816B2 (en) 2013-03-13 2019-01-31 Bioasis Technologies Inc. Fragments of p97 and uses thereof
JP6603227B2 (ja) 2014-02-03 2019-11-06 バイオアシス テクノロジーズ インコーポレイテッド Ｐ９７融合タンパク質
DK3107562T3 (da) 2014-02-19 2019-12-16 Bioasis Technologies Inc P97-ids-fusionsproteiner
AU2015252906C1 (en) 2014-05-01 2020-09-17 Bioasis Technologies Inc. p97-polynucleotide conjugates
GB201508025D0 (en) 2015-05-11 2015-06-24 Ucl Business Plc Fabry disease gene therapy
RU2673038C2 (ru) * 2016-01-18 2018-11-21 ООО "Международный Биотехнологический Центр "Генериум" Лекарственное средство на основе бифункционального антитела для лечения мукополисахаридоза II типа
CN106282231B (zh) * 2016-09-06 2020-01-03 陕西慧康生物科技有限责任公司 粘多糖贮积症ii型动物模型的构建方法及应用
EP3537961B1 (en) 2016-11-10 2025-06-25 The Research Foundation for The State University of New York System, method and biomarkers for airway obstruction
JP7252211B2 (ja) * 2017-09-08 2023-04-04 ザ ヌムール ファウンデーション リソソーム蓄積症を治療するための薬剤、デバイス、および血液循環システム、ならびにリソソーム蓄積症を治療するための方法
CR20200129A (es) 2017-10-02 2020-08-22 Denali Therapeutics Inc Proteínas de fusión que comprenden enzimas de terapia de reemplazo de enzimas
CN113631182B (zh) * 2018-10-10 2025-03-21 阿米库斯治疗学公司 二硫键稳定的多肽组合物和使用方法
AU2019404061A1 (en) 2018-12-19 2021-07-22 The Board Of Trustees Of The Leland Stanford Junior University Bifunctional molecules for lysosomal targeting and related compositions and methods
CN110609078B (zh) * 2019-09-20 2022-03-11 南京谱利健生物技术有限公司 一种检测蛋白磷酸化和乙酰氨基葡萄糖化关联作用的方法
EP4229192A1 (en) 2020-10-14 2023-08-23 Denali Therapeutics Inc. Fusion proteins comprising sulfoglucosamine sulfohydrolase enzymes and methods thereof
EP4370157A1 (en) 2021-07-14 2024-05-22 Lycia Therapeutics, Inc. Asgpr cell surface receptor binding compounds and conjugates
EP4389772A4 (en) * 2021-08-18 2025-08-20 Joint Stock Company Generium TARGETED ADMINISTRATION OF THERAPEUTIC FERMENTS
CN114181318B (zh) * 2021-11-08 2023-08-01 四川大学 一种组织特异性表达穿透血脑屏障的idua融合蛋白的重组腺相关病毒及应用
US20250257338A1 (en) * 2022-04-19 2025-08-14 Jcr Pharmaceuticals Co., Ltd. Glycoprotein having reduced number of mannose-6-phosphates
WO2023207574A1 (zh) * 2022-04-28 2023-11-02 领诺(上海)医药科技有限公司 一种穿越血脑屏障的重组hARSA

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