KR102094094B1 - 아릴설파타제 a의 cns 전달을 위한 방법들 및 조성물들 - Google Patents

아릴설파타제 a의 cns 전달을 위한 방법들 및 조성물들 Download PDF

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KR102094094B1
KR102094094B1 KR1020187014958A KR20187014958A KR102094094B1 KR 102094094 B1 KR102094094 B1 KR 102094094B1 KR 1020187014958 A KR1020187014958 A KR 1020187014958A KR 20187014958 A KR20187014958 A KR 20187014958A KR 102094094 B1 KR102094094 B1 KR 102094094B1
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rhasa
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brain
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KR20180059580A (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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  • Pharmacology & Pharmacy (AREA)
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  • Bioinformatics & Cheminformatics (AREA)
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  • Organic Chemistry (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Neurology (AREA)
  • Biomedical Technology (AREA)
  • Neurosurgery (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Immunology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Biochemistry (AREA)
  • Psychology (AREA)
  • Molecular Biology (AREA)
  • Oil, Petroleum & Natural Gas (AREA)
  • Inorganic Chemistry (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Wood Science & Technology (AREA)
  • Obesity (AREA)
  • Diabetes (AREA)
  • Hospice & Palliative Care (AREA)
  • Hematology (AREA)
  • Psychiatry (AREA)
  • Dermatology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
KR1020187014958A 2010-06-25 2011-06-25 아릴설파타제 a의 cns 전달을 위한 방법들 및 조성물들 Active KR102094094B1 (ko)

Priority Applications (1)

Application Number Priority Date Filing Date Title
KR1020207008105A KR102318997B1 (ko) 2010-06-25 2011-06-25 아릴설파타제 a의 cns 전달을 위한 방법들 및 조성물들

Applications Claiming Priority (15)

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
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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KR1020137001752A Division KR101997935B1 (ko) 2010-06-25 2011-06-25 아릴설파타제 a의 cns 전달을 위한 방법들 및 조성물들

Related Child Applications (1)

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

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KR20180059580A KR20180059580A (ko) 2018-06-04
KR102094094B1 true KR102094094B1 (ko) 2020-03-27

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KR1020187014958A Active KR102094094B1 (ko) 2010-06-25 2011-06-25 아릴설파타제 a의 cns 전달을 위한 방법들 및 조성물들
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

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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

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US (1) US9770410B2 (https=)
EP (3) EP3626257B1 (https=)
JP (1) JP6045493B2 (https=)
KR (5) KR102094094B1 (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) MX382443B (https=)
NZ (2) NZ605873A (https=)
PL (2) PL2585104T3 (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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PT3103469T (pt) 2010-06-25 2021-03-04 Shire Human Genetic Therapies Administração de agentes terapêuticos no snc
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CN109640961B (zh) * 2016-06-01 2021-11-02 施维雅知识产权英国有限公司 聚亚烷基氧化物-天冬酰胺酶制剂及其制备和使用方法
AU2017386888B2 (en) * 2016-12-28 2024-08-08 Jcr Pharmaceuticals Co., Ltd. Lyophilized preparation
KR102883778B1 (ko) 2017-09-22 2025-11-12 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 Ii형 점액다당류증의 치료를 위한 유전자 요법
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KR20220020261A (ko) 2019-05-03 2022-02-18 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 이염성 백질이영양증의 치료에 유용한 조성물
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