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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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)
  • Wood Science & Technology (AREA)
  • Genetics & Genomics (AREA)
  • Medicinal Chemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Molecular Biology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Biomedical Technology (AREA)
  • Microbiology (AREA)
  • Biotechnology (AREA)
  • Epidemiology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Hematology (AREA)
  • Obesity (AREA)
  • Diabetes (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 (cg-RX-API-DMAC7.html)
EP (1) EP2880156B1 (cg-RX-API-DMAC7.html)
JP (1) JP6433424B2 (cg-RX-API-DMAC7.html)
CN (1) CN104662150B (cg-RX-API-DMAC7.html)
AU (1) AU2013296557B2 (cg-RX-API-DMAC7.html)
CA (2) CA3140358A1 (cg-RX-API-DMAC7.html)
ES (1) ES2647082T3 (cg-RX-API-DMAC7.html)
WO (1) WO2014022515A1 (cg-RX-API-DMAC7.html)

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AU2003222568B2 (en) 2002-01-11 2009-05-07 Bioasis Technologies, Inc. Use of P97 as an enzyme delivery system for the delivery of therapeutic lysosomal enzymes
EP2717917B1 (en) 2011-07-05 2016-04-20 biOasis Technologies Inc P97-antibody conjugates
EP3321281B1 (en) 2011-08-05 2019-11-27 biOasis Technologies Inc P97 fragments with transfer activity
AU2013296557B2 (en) 2012-07-31 2019-04-18 Bioasis Technologies Inc. Dephosphorylated lysosomal storage disease proteins and methods of use thereof
WO2014160438A1 (en) 2013-03-13 2014-10-02 Bioasis Technologies Inc. Fragments of p97 and uses thereof
US20160347821A1 (en) 2014-02-03 2016-12-01 Bioasis Technologies, Inc. P97 fusion proteins
WO2015126729A1 (en) * 2014-02-19 2015-08-27 Bioasis Technologies, Inc. P97-ids fusion proteins
EP3137610B1 (en) 2014-05-01 2019-03-06 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
WO2018089789A1 (en) 2016-11-10 2018-05-17 The Research Foundation For The State University Of New York System, method and biomarkers for airway obstruction
WO2019051297A1 (en) * 2017-09-08 2019-03-14 The Nemours Foundation AGENT, DEVICE AND BLOOD CIRCULATION SYSTEM FOR TREATING LYSOSOMAL DISEASES, AND METHODS OF TREATING LYSOSOMAL DISEASES
EP3692070A1 (en) 2017-10-02 2020-08-12 Denali Therapeutics Inc. Fusion proteins comprising enzyme replacement therapy enzymes
JP7577650B2 (ja) * 2018-10-10 2024-11-05 アミカス セラピューティックス インコーポレイテッド ジスルフィド結合により安定したポリペプチド組成物及びその使用方法
CN113301925A (zh) 2018-12-19 2021-08-24 小利兰·斯坦福大学理事会 用于溶酶体靶向的双官能分子以及相关的组合物和方法
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
KR20230086703A (ko) 2020-10-14 2023-06-15 데날리 테라퓨틱스 인크. 설포글루코사민 설포하이드롤라제 효소를 포함하는 융합 단백질 및 이의 방법
CA3226269A1 (en) 2021-07-14 2023-01-19 Lycia Therapeutics, Inc. Asgpr cell surface receptor binding compounds and conjugates
CA3228179A1 (en) * 2021-08-18 2023-02-23 Joint-Stock Company "Generium" Targeted delivery of therapeutic enzymes
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
WO2023204202A1 (ja) * 2022-04-19 2023-10-26 Ｊｃｒファーマ株式会社 マンノース－６－リン酸の数を減少させた糖蛋白質
EP4397687A4 (en) * 2022-04-28 2025-09-24 Linno Pharmaceuticals Inc RECOMBINANT HARSA TO CROSS THE BLOOD-BRAIN BARRIER

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