AU2017220100B2 - Methods and compositions for CNS delivery of arylsulfatase A - Google Patents

Methods and compositions for CNS delivery of arylsulfatase A Download PDF

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Publication number
AU2017220100B2
AU2017220100B2 AU2017220100A AU2017220100A AU2017220100B2 AU 2017220100 B2 AU2017220100 B2 AU 2017220100B2 AU 2017220100 A AU2017220100 A AU 2017220100A AU 2017220100 A AU2017220100 A AU 2017220100A AU 2017220100 B2 AU2017220100 B2 AU 2017220100B2
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AU
Australia
Prior art keywords
enzyme
baseline
brain
subject
leu
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Active
Application number
AU2017220100A
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English (en)
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AU2017220100A1 (en
Inventor
Margaret WASILEWSKI
Anna WIJATYK
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Takeda Pharmaceutical Co Ltd
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Takeda Pharmaceutical Co Ltd
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.)
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Publication of AU2017220100A1 publication Critical patent/AU2017220100A1/en
Assigned to TAKEDA PHARMACEUTICAL COMPANY LIMITED reassignment TAKEDA PHARMACEUTICAL COMPANY LIMITED Request for Assignment Assignors: SHIRE HUMAN GENETIC THERAPIES, INC.
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
    • A61K38/465—Hydrolases (3) acting on ester bonds (3.1), e.g. lipases, ribonucleases
    • 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
    • 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
    • A61K9/0085—Brain, e.g. brain implants; Spinal cord
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
    • 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
    • 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)
    • C12Y301/06001—Arylsulfatase (3.1.6.1)

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • Epidemiology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Hospice & Palliative Care (AREA)
  • Psychiatry (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Immunology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Dermatology (AREA)
  • Psychology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Diabetes (AREA)
  • Genetics & Genomics (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Hematology (AREA)
  • Obesity (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
AU2017220100A 2016-02-17 2017-02-17 Methods and compositions for CNS delivery of arylsulfatase A Active AU2017220100B2 (en)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201662296563P 2016-02-17 2016-02-17
US62/296,563 2016-02-17
US201762453864P 2017-02-02 2017-02-02
US62/453,864 2017-02-02
PCT/US2017/018440 WO2017143233A1 (en) 2016-02-17 2017-02-17 Methods and compositions for cns delivery of arylsulfatase a

Publications (2)

Publication Number Publication Date
AU2017220100A1 AU2017220100A1 (en) 2018-08-30
AU2017220100B2 true AU2017220100B2 (en) 2024-04-04

Family

ID=58192404

Family Applications (1)

Application Number Title Priority Date Filing Date
AU2017220100A Active AU2017220100B2 (en) 2016-02-17 2017-02-17 Methods and compositions for CNS delivery of arylsulfatase A

Country Status (10)

Country Link
US (3) US11020461B2 (https=)
EP (1) EP3416678A1 (https=)
JP (4) JP2019509270A (https=)
CN (2) CN108883162A (https=)
AU (1) AU2017220100B2 (https=)
BR (1) BR112018016874A2 (https=)
CA (1) CA3014909A1 (https=)
MA (1) MA44237A (https=)
MX (2) MX2018009937A (https=)
WO (1) WO2017143233A1 (https=)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2019126376A1 (en) * 2017-12-19 2019-06-27 Shire Human Genetic Therapies, Inc. Purified arylsulfatase a and compositons thereof
BR112021021908A2 (pt) * 2019-05-03 2022-02-01 Univ Pennsylvania Composições úteis no tratamento da leucodistrofia metacromática

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2011163650A2 (en) * 2010-06-25 2011-12-29 Shire Human Genetic Therapies, Inc. Methods and compositions for cns delivery of arylsulfatase a

Family Cites Families (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6217552B1 (en) 1999-03-01 2001-04-17 Coaxia, Inc. Medical device for selective intrathecal spinal cooling in aortic surgery and spinal trauma
US6537785B1 (en) 1999-09-14 2003-03-25 Genzyme Glycobiology Research Institute, Inc. Methods of treating lysosomal storage diseases
NZ605874A (en) * 2010-06-25 2015-02-27 Shire Human Genetic Therapies Methods and compositions for cns delivery of heparan n-sulfatase
SMT201600385T1 (it) * 2010-06-25 2017-03-08 Shire Human Genetic Therapies Trasporto di agenti terapeutici all’snc
CN103179980B (zh) * 2010-06-25 2016-09-28 夏尔人类遗传性治疗公司 艾杜糖醛酸-2-硫酸酯酶的cns递送的方法和组合物

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2011163650A2 (en) * 2010-06-25 2011-12-29 Shire Human Genetic Therapies, Inc. Methods and compositions for cns delivery of arylsulfatase a

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
DALI CHRISTINE ET AL: "Intrathecal delivery of recombinant human arylsulfatase A in children with late-infantile metachromatic leukodystrophy", MOLECULAR GENETICS AND METABOLISM, vol. 117, no. 2, 11 February 2015 (2015-02-11) *

Also Published As

Publication number Publication date
EP3416678A1 (en) 2018-12-26
WO2017143233A1 (en) 2017-08-24
US20210315981A1 (en) 2021-10-14
CN108883162A (zh) 2018-11-23
JP2023159405A (ja) 2023-10-31
MX2024004034A (es) 2024-04-24
MX2018009937A (es) 2018-11-29
AU2017220100A1 (en) 2018-08-30
CN117224659A (zh) 2023-12-15
US20250099555A1 (en) 2025-03-27
US11020461B2 (en) 2021-06-01
JP2024102370A (ja) 2024-07-30
CA3014909A1 (en) 2017-08-24
BR112018016874A2 (pt) 2019-02-05
JP2021169528A (ja) 2021-10-28
MA44237A (fr) 2021-06-02
JP2019509270A (ja) 2019-04-04
US20200179492A1 (en) 2020-06-11

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Legal Events

Date Code Title Description
PC1 Assignment before grant (sect. 113)

Owner name: TAKEDA PHARMACEUTICAL COMPANY LIMITED

Free format text: FORMER APPLICANT(S): SHIRE HUMAN GENETIC THERAPIES, INC.

FGA Letters patent sealed or granted (standard patent)