JP2019509270A - アリールスルファターゼaのcns送達のための方法および組成物 - Google Patents

アリールスルファターゼaのcns送達のための方法および組成物 Download PDF

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Publication number
JP2019509270A
JP2019509270A JP2018543324A JP2018543324A JP2019509270A JP 2019509270 A JP2019509270 A JP 2019509270A JP 2018543324 A JP2018543324 A JP 2018543324A JP 2018543324 A JP2018543324 A JP 2018543324A JP 2019509270 A JP2019509270 A JP 2019509270A
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Prior art keywords
enzyme
subject
recombinant
arylsulfatase
asa
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Japanese (ja)
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JP2019509270A5 (https=
Inventor
マーガレット ワシレウスキ,
マーガレット ワシレウスキ,
アンナ ワイジャタイク,
アンナ ワイジャタイク,
Original Assignee
シャイアー ヒューマン ジェネティック セラピーズ インコーポレイテッド
シャイアー ヒューマン ジェネティック セラピーズ インコーポレイテッド
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Publication of JP2019509270A publication Critical patent/JP2019509270A/ja
Publication of JP2019509270A5 publication Critical patent/JP2019509270A5/ja
Priority to JP2021124414A priority Critical patent/JP2021169528A/ja
Priority to JP2023138821A priority patent/JP2023159405A/ja
Priority to JP2024082556A priority patent/JP2024102370A/ja
Pending legal-status Critical Current

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    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46Hydrolases (3)
    • A61K38/465Hydrolases (3) acting on ester bonds (3.1), e.g. lipases, ribonucleases
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/0012Galenical forms characterised by the site of application
    • A61K9/0019Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/0012Galenical forms characterised by the site of application
    • A61K9/0085Brain, e.g. brain implants; Spinal cord
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/28Drugs 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00Drugs for disorders of the metabolism
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y301/00Hydrolases acting on ester bonds (3.1)
    • C12Y301/06Sulfuric ester hydrolases (3.1.6)
    • C12Y301/06001Arylsulfatase (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)
JP2018543324A 2016-02-17 2017-02-17 アリールスルファターゼaのcns送達のための方法および組成物 Pending JP2019509270A (ja)

Priority Applications (3)

Application Number Priority Date Filing Date Title
JP2021124414A JP2021169528A (ja) 2016-02-17 2021-07-29 アリールスルファターゼaのcns送達のための方法および組成物
JP2023138821A JP2023159405A (ja) 2016-02-17 2023-08-29 アリールスルファターゼaのcns送達のための方法および組成物
JP2024082556A JP2024102370A (ja) 2016-02-17 2024-05-21 アリールスルファターゼaのcns送達のための方法および組成物

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
JP2021124414A Division JP2021169528A (ja) 2016-02-17 2021-07-29 アリールスルファターゼaのcns送達のための方法および組成物

Publications (2)

Publication Number Publication Date
JP2019509270A true JP2019509270A (ja) 2019-04-04
JP2019509270A5 JP2019509270A5 (https=) 2020-03-26

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Family Applications (4)

Application Number Title Priority Date Filing Date
JP2018543324A Pending JP2019509270A (ja) 2016-02-17 2017-02-17 アリールスルファターゼaのcns送達のための方法および組成物
JP2021124414A Withdrawn JP2021169528A (ja) 2016-02-17 2021-07-29 アリールスルファターゼaのcns送達のための方法および組成物
JP2023138821A Pending JP2023159405A (ja) 2016-02-17 2023-08-29 アリールスルファターゼaのcns送達のための方法および組成物
JP2024082556A Pending JP2024102370A (ja) 2016-02-17 2024-05-21 アリールスルファターゼaのcns送達のための方法および組成物

Family Applications After (3)

Application Number Title Priority Date Filing Date
JP2021124414A Withdrawn JP2021169528A (ja) 2016-02-17 2021-07-29 アリールスルファターゼaのcns送達のための方法および組成物
JP2023138821A Pending JP2023159405A (ja) 2016-02-17 2023-08-29 アリールスルファターゼaのcns送達のための方法および組成物
JP2024082556A Pending JP2024102370A (ja) 2016-02-17 2024-05-21 アリールスルファターゼaのcns送達のための方法および組成物

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=)

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2022531861A (ja) * 2019-05-03 2022-07-12 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア 異染性白質ジストロフィーの治療に有用な組成物

Families Citing this family (1)

* 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

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2013538788A (ja) * 2010-06-25 2013-10-17 シャイアー ヒューマン ジェネティック セラピーズ, インコーポレイテッド アリールスルファターゼaのcns送達の方法および組成物

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
JP2013538788A (ja) * 2010-06-25 2013-10-17 シャイアー ヒューマン ジェネティック セラピーズ, インコーポレイテッド アリールスルファターゼaのcns送達の方法および組成物

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
CLINICAL TRIAL PROTOCOL: HGT-MLD-070, (2015.08.26), <URL:HTTPS://CLINICALTRIALS.GOV/PROVIDEDDOCS/28/, JPN6021002883, ISSN: 0004829169 *
MOL. GENET. METAB., (2016.02.13), 117, [2], P.S38(ARTICLE NO.73), JPN6021002879, ISSN: 0004829168 *

Cited By (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2022531861A (ja) * 2019-05-03 2022-07-12 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア 異染性白質ジストロフィーの治療に有用な組成物
JP7715640B2 (ja) 2019-05-03 2025-07-30 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア 異染性白質ジストロフィーの治療に有用な組成物

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
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
US20200179492A1 (en) 2020-06-11

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