KR102537084B1 - 아릴설파타제 a의 cns 전달을 위한 방법들 및 조성물들 - Google Patents

아릴설파타제 a의 cns 전달을 위한 방법들 및 조성물들 Download PDF

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KR102537084B1
KR102537084B1 KR1020217034302A KR20217034302A KR102537084B1 KR 102537084 B1 KR102537084 B1 KR 102537084B1 KR 1020217034302 A KR1020217034302 A KR 1020217034302A KR 20217034302 A KR20217034302 A KR 20217034302A KR 102537084 B1 KR102537084 B1 KR 102537084B1
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formulation
rhasa
brain
protein
administration
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KR20210131449A (ko
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나질라 살라맛-밀러
케써린 타일러
폴 캄폴리에토
자흐라 샤로크
징 판
로렌스 찰나스
테레사 레아 라이트
페리클레스 칼리아스
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샤이어 휴먼 지네틱 테라피즈 인크.
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    • 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
    • A61K9/08—Solutions
    • 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)
    • A61K38/47—Hydrolases (3) acting on glycosyl compounds (3.2), e.g. cellulases, lactases
    • 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
    • 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)
    • 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
    • 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
    • A61K38/54—Mixtures of enzymes or proenzymes covered by more than a single one of groups A61K38/44 - A61K38/46 or A61K38/51 - A61K38/53
    • 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
    • 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
    • A61K47/02—Inorganic compounds
    • 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
    • A61K47/06—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
    • A61K47/26—Carbohydrates, e.g. sugar alcohols, amino sugars, nucleic acids, mono-, di- or oligo-saccharides; Derivatives thereof, e.g. polysorbates, sorbitan fatty acid esters or glycyrrhizin
    • 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
    • A61K9/14—Particulate form, e.g. powders, Processes for size reducing of pure drugs or the resulting products, Pure drug nanoparticles
    • A61K9/19—Particulate form, e.g. powders, Processes for size reducing of pure drugs or the resulting products, Pure drug nanoparticles lyophilised, i.e. freeze-dried, solutions or dispersions
    • 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
    • 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
    • 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
    • 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
    • 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

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  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Veterinary Medicine (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Epidemiology (AREA)
  • Organic Chemistry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Biomedical Technology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Immunology (AREA)
  • Biochemistry (AREA)
  • Psychology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Inorganic Chemistry (AREA)
  • Molecular Biology (AREA)
  • Oil, Petroleum & Natural Gas (AREA)
  • General Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Hospice & Palliative Care (AREA)
  • Psychiatry (AREA)
  • Diabetes (AREA)
  • Hematology (AREA)
  • Obesity (AREA)
  • Dermatology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
KR1020217034302A 2010-06-25 2011-06-25 아릴설파타제 a의 cns 전달을 위한 방법들 및 조성물들 Active KR102537084B1 (ko)

Priority Applications (1)

Application Number Priority Date Filing Date Title
KR1020237017347A KR102720061B1 (ko) 2010-06-25 2011-06-25 아릴설파타제 a의 cns 전달을 위한 방법들 및 조성물들

Applications Claiming Priority (16)

Application Number Priority Date Filing Date Title
US35885710P 2010-06-25 2010-06-25
US61/358,857 2010-06-25
US36078610P 2010-07-01 2010-07-01
US61/360,786 2010-07-01
US38786210P 2010-09-29 2010-09-29
US61/387,862 2010-09-29
US201161435710P 2011-01-24 2011-01-24
US61/435,710 2011-01-24
US201161442115P 2011-02-11 2011-02-11
US61/442,115 2011-02-11
US201161476210P 2011-04-15 2011-04-15
US61/476,210 2011-04-15
US201161495268P 2011-06-09 2011-06-09
US61/495,268 2011-06-09
KR1020207008105A KR102318997B1 (ko) 2010-06-25 2011-06-25 아릴설파타제 a의 cns 전달을 위한 방법들 및 조성물들
PCT/US2011/041926 WO2011163650A2 (en) 2010-06-25 2011-06-25 Methods and compositions for cns delivery of arylsulfatase a

Related Parent Applications (1)

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KR1020207008105A Division KR102318997B1 (ko) 2010-06-25 2011-06-25 아릴설파타제 a의 cns 전달을 위한 방법들 및 조성물들

Related Child Applications (1)

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KR1020237017347A Division KR102720061B1 (ko) 2010-06-25 2011-06-25 아릴설파타제 a의 cns 전달을 위한 방법들 및 조성물들

Publications (2)

Publication Number Publication Date
KR20210131449A KR20210131449A (ko) 2021-11-02
KR102537084B1 true KR102537084B1 (ko) 2023-05-26

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Application Number Title Priority Date Filing Date
KR1020217034302A Active KR102537084B1 (ko) 2010-06-25 2011-06-25 아릴설파타제 a의 cns 전달을 위한 방법들 및 조성물들
KR1020207008105A Active KR102318997B1 (ko) 2010-06-25 2011-06-25 아릴설파타제 a의 cns 전달을 위한 방법들 및 조성물들
KR1020137001752A Active KR101997935B1 (ko) 2010-06-25 2011-06-25 아릴설파타제 a의 cns 전달을 위한 방법들 및 조성물들
KR1020237017347A Active KR102720061B1 (ko) 2010-06-25 2011-06-25 아릴설파타제 a의 cns 전달을 위한 방법들 및 조성물들
KR1020187014958A Active KR102094094B1 (ko) 2010-06-25 2011-06-25 아릴설파타제 a의 cns 전달을 위한 방법들 및 조성물들

Family Applications After (4)

Application Number Title Priority Date Filing Date
KR1020207008105A Active KR102318997B1 (ko) 2010-06-25 2011-06-25 아릴설파타제 a의 cns 전달을 위한 방법들 및 조성물들
KR1020137001752A Active KR101997935B1 (ko) 2010-06-25 2011-06-25 아릴설파타제 a의 cns 전달을 위한 방법들 및 조성물들
KR1020237017347A Active KR102720061B1 (ko) 2010-06-25 2011-06-25 아릴설파타제 a의 cns 전달을 위한 방법들 및 조성물들
KR1020187014958A Active KR102094094B1 (ko) 2010-06-25 2011-06-25 아릴설파타제 a의 cns 전달을 위한 방법들 및 조성물들

Country Status (26)

Country Link
US (1) US9770410B2 (https=)
EP (3) EP3964230A1 (https=)
JP (1) JP6045493B2 (https=)
KR (5) KR102537084B1 (https=)
CN (1) CN103282046B (https=)
AU (5) AU2011270670B2 (https=)
BR (1) BR112012033216B1 (https=)
CA (1) CA2803003C (https=)
CY (2) CY1122282T1 (https=)
DK (1) DK2585104T3 (https=)
ES (1) ES2754776T3 (https=)
HR (1) HRP20191923T1 (https=)
IL (3) IL291556B2 (https=)
LT (1) LT2585104T (https=)
ME (1) ME03647B (https=)
MX (2) MX344795B (https=)
NZ (2) NZ605873A (https=)
PL (2) PL3626257T3 (https=)
RS (1) RS59468B1 (https=)
RU (1) RU2671503C2 (https=)
SI (1) SI2585104T1 (https=)
SM (1) SMT201900589T1 (https=)
TW (1) TWI667037B (https=)
UA (1) UA115650C2 (https=)
WO (1) WO2011163650A2 (https=)
ZA (1) ZA201300675B (https=)

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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
RS62620B1 (sr) 2010-06-25 2021-12-31 Shire Human Genetic Therapies Metode i kompozicije za isporuku cns akrilsulfataze a
SMT201600385T1 (it) 2010-06-25 2017-03-08 Shire Human Genetic Therapies Trasporto di agenti terapeutici all’snc
NZ605873A (en) 2010-06-25 2015-02-27 Shire Human Genetic Therapies Methods and compositions for cns delivery of arylsulfatase a
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US20140377244A1 (en) * 2011-12-23 2014-12-25 Shire Human Genetic Therapies, Inc. Stable formulations for cns delivery of arylsulfatase a
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EP3416678A1 (en) * 2016-02-17 2018-12-26 Shire Human Genetic Therapies, Inc. Methods and compositions for cns delivery of arylsulfatase a
KR102783452B1 (ko) * 2016-06-01 2025-03-19 세르비에 아이피 유케이 리미티드 폴리알킬렌 옥시드-아스파라기나제의 제형, 및 그의 제조 및 사용 방법
US10940185B2 (en) * 2016-12-28 2021-03-09 Jcr Pharmaceuticals Co., Ltd. Lyophilized preparation
TWI835747B (zh) 2017-09-22 2024-03-21 賓州大學委員會 用於治療黏多醣病 ii 型之基因治療
WO2019126376A1 (en) 2017-12-19 2019-06-27 Shire Human Genetic Therapies, Inc. Purified arylsulfatase a and compositons thereof
WO2020004368A1 (ja) * 2018-06-25 2020-01-02 Ｊｃｒファーマ株式会社 蛋白質含有水性液剤
BR112021021908A2 (pt) * 2019-05-03 2022-02-01 Univ Pennsylvania Composições úteis no tratamento da leucodistrofia metacromática
WO2021061517A1 (en) * 2019-09-23 2021-04-01 University Of Washington Assay of arylsulfatase a enzymatic activity in dried blood spots by mass spectrometry and fluorometry
WO2024176112A1 (en) 2023-02-21 2024-08-29 Takeda Pharmaceutical Company Limited Large scale production of recombinant arylsulfatase a and compositions thereof

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