CN108883162A - 用于芳基硫酸酯酶a的cns递送的方法和组合物 - Google Patents

用于芳基硫酸酯酶a的cns递送的方法和组合物 Download PDF

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Publication number
CN108883162A
CN108883162A CN201780022756.8A CN201780022756A CN108883162A CN 108883162 A CN108883162 A CN 108883162A CN 201780022756 A CN201780022756 A CN 201780022756A CN 108883162 A CN108883162 A CN 108883162A
Authority
CN
China
Prior art keywords
enzyme
recombination
subject
arylsulfatase
treatment
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.)
Pending
Application number
CN201780022756.8A
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English (en)
Chinese (zh)
Inventor
M·瓦西莱夫斯基
A·维伽泰科
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.)
Takeda Pharmaceutical Co Ltd
Original Assignee
Shire Human Genetics Therapies Inc
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 Shire Human Genetics Therapies Inc filed Critical Shire Human Genetics Therapies Inc
Priority to CN202311189440.1A priority Critical patent/CN117224659A/zh
Publication of CN108883162A publication Critical patent/CN108883162A/zh
Pending legal-status Critical Current

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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)

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  • 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)
CN201780022756.8A 2016-02-17 2017-02-17 用于芳基硫酸酯酶a的cns递送的方法和组合物 Pending CN108883162A (zh)

Priority Applications (1)

Application Number Priority Date Filing Date Title
CN202311189440.1A CN117224659A (zh) 2016-02-17 2017-02-17 用于芳基硫酸酯酶a的cns递送的方法和组合物

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

Related Child Applications (1)

Application Number Title Priority Date Filing Date
CN202311189440.1A Division CN117224659A (zh) 2016-02-17 2017-02-17 用于芳基硫酸酯酶a的cns递送的方法和组合物

Publications (1)

Publication Number Publication Date
CN108883162A true CN108883162A (zh) 2018-11-23

Family

ID=58192404

Family Applications (2)

Application Number Title Priority Date Filing Date
CN201780022756.8A Pending CN108883162A (zh) 2016-02-17 2017-02-17 用于芳基硫酸酯酶a的cns递送的方法和组合物
CN202311189440.1A Pending CN117224659A (zh) 2016-02-17 2017-02-17 用于芳基硫酸酯酶a的cns递送的方法和组合物

Family Applications After (1)

Application Number Title Priority Date Filing Date
CN202311189440.1A Pending CN117224659A (zh) 2016-02-17 2017-02-17 用于芳基硫酸酯酶a的cns递送的方法和组合物

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
CN103282046A (zh) * 2010-06-25 2013-09-04 夏尔人类遗传性治疗公司 芳基硫酸酯酶 a cns 递送的方法和组合物

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
CN103282046A (zh) * 2010-06-25 2013-09-04 夏尔人类遗传性治疗公司 芳基硫酸酯酶 a cns 递送的方法和组合物

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
CHRISTINE DALI等: "Development program for an intrathecally (IT) administered recombinant human arylsulfatase A in children with metachromatic leukodystrophy (MLD)", 《MOLECULAR GENETICS AND METABOLISM》 *
CHRISTINE DALI等: "Intrathecal delivery of recombinant human arylsulfatase A in children with late-infantile metachromatic leukodystrophy", 《MOLECULAR GENETICS AND METABOLISM》 *

Also Published As

Publication number Publication date
EP3416678A1 (en) 2018-12-26
WO2017143233A1 (en) 2017-08-24
US20210315981A1 (en) 2021-10-14
AU2017220100B2 (en) 2024-04-04
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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