JP6063380B2 - サンフィリポ症候群b型の処置 - Google Patents

サンフィリポ症候群b型の処置 Download PDF

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Publication number
JP6063380B2
JP6063380B2 JP2013516847A JP2013516847A JP6063380B2 JP 6063380 B2 JP6063380 B2 JP 6063380B2 JP 2013516847 A JP2013516847 A JP 2013516847A JP 2013516847 A JP2013516847 A JP 2013516847A JP 6063380 B2 JP6063380 B2 JP 6063380B2
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Prior art keywords
naglu
brain
protein
nmol
lysosomal
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JP2013542913A5 (OSRAM
JP2013542913A (ja
Inventor
マイケル エフ. コンシーノ,
マイケル エフ. コンシーノ,
ペリクルス カリアス,
ペリクルス カリアス,
ジン パン,
ジン パン,
ケビン ホームズ,
ケビン ホームズ,
パオロ マーティーニ,
パオロ マーティーニ,
アラ ロマシュコ,
アラ ロマシュコ,
ムスラマン メイヤッパン,
ムスラマン メイヤッパン,
ボーホン ジャン,
ボーホン ジャン,
アンドレア イスケンデリアン,
アンドレア イスケンデリアン,
ダイアナ ルンドバーグ,
ダイアナ ルンドバーグ,
アンジェラ ノートン,
アンジェラ ノートン,
ベッティーナ シュトラック−ローグ,
ベッティーナ シュトラック−ローグ,
ヤン ファン,
ヤン ファン,
メアリー アレサンドリーニ,
メアリー アレサンドリーニ,
リチャード ファイファー,
リチャード ファイファー,
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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    • 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
    • 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)
    • A61K38/47Hydrolases (3) acting on glycosyl compounds (3.2), e.g. cellulases, lactases
    • 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
    • A61K47/00Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/02Inorganic compounds
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/06Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
    • A61K47/26Carbohydrates, e.g. sugar alcohols, amino sugars, nucleic acids, mono-, di- or oligo-saccharides; Derivatives thereof, e.g. polysorbates, sorbitan fatty acid esters or glycyrrhizin
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/14Particulate form, e.g. powders, Processes for size reducing of pure drugs or the resulting products, Pure drug nanoparticles
    • A61K9/19Particulate 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/18Antipsychotics, i.e. neuroleptics; Drugs for mania or schizophrenia
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/20Hypnotics; Sedatives
    • 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
    • A61P27/00Drugs for disorders of the senses
    • A61P27/16Otologicals
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00Drugs for disorders of the metabolism
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • 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)
    • C12Y301/06013Iduronate-2-sulfatase (3.1.6.13)
    • 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

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  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Medicinal Chemistry (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Epidemiology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Biomedical Technology (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Biochemistry (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Immunology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • General Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Oil, Petroleum & Natural Gas (AREA)
  • Inorganic Chemistry (AREA)
  • Psychology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Zoology (AREA)
  • Psychiatry (AREA)
  • Genetics & Genomics (AREA)
  • Wood Science & Technology (AREA)
  • Diabetes (AREA)
  • Hematology (AREA)
  • Obesity (AREA)
  • Hospice & Palliative Care (AREA)
  • Anesthesiology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Peptides Or Proteins (AREA)
JP2013516847A 2010-06-25 2011-06-25 サンフィリポ症候群b型の処置 Expired - Fee Related JP6063380B2 (ja)

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/US2011/041928 WO2011163652A2 (en) 2010-06-25 2011-06-25 Treatment of sanfilippo syndrome type b

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2015246589A Division JP2016094451A (ja) 2010-06-25 2015-12-17 サンフィリポ症候群b型の処置

Publications (3)

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JP2013542913A JP2013542913A (ja) 2013-11-28
JP2013542913A5 JP2013542913A5 (OSRAM) 2014-08-14
JP6063380B2 true JP6063380B2 (ja) 2017-01-18

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JP2013516847A Expired - Fee Related JP6063380B2 (ja) 2010-06-25 2011-06-25 サンフィリポ症候群b型の処置

Country Status (11)

Country Link
US (1) US20110318327A1 (OSRAM)
EP (1) EP2585105B1 (OSRAM)
JP (1) JP6063380B2 (OSRAM)
KR (1) KR20130043166A (OSRAM)
CN (1) CN103269709A (OSRAM)
AU (2) AU2011270672B2 (OSRAM)
CA (1) CA2805449A1 (OSRAM)
ES (1) ES2643015T3 (OSRAM)
MX (1) MX2013000324A (OSRAM)
NZ (2) NZ702801A (OSRAM)
WO (1) WO2011163652A2 (OSRAM)

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HRP20170698T1 (hr) * 2008-05-07 2017-09-22 Biomarin Pharmaceutical Inc. Lizosomski usmjereni peptidi i njihove uporabe
JP2013507131A (ja) 2009-10-09 2013-03-04 アーメイゲン・テクノロジーズ・インコーポレイテッド Cnsにおけるイズロン酸2−スルファターゼ活性を増加させるための方法および組成物
SMT201600385T1 (it) 2010-06-25 2017-03-08 Shire Human Genetic Therapies Trasporto di agenti terapeutici all’snc
BR112012033197A2 (pt) 2010-06-25 2017-06-06 Shire Human Genetic Therapies métodos e composições para liberação de cns de n-sulfatase de heparano.
PT2588130T (pt) 2010-06-25 2016-11-25 Shire Human Genetic Therapies Entrega de agentes terapêuticos no cns
KR102720061B1 (ko) 2010-06-25 2024-10-22 다케다 파머수티컬 컴패니 리미티드 아릴설파타제 a의 cns 전달을 위한 방법들 및 조성물들
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KR101380740B1 (ko) 2012-06-29 2014-04-11 쉐어 휴먼 제네텍 세러피스, 인코포레이티드 이듀로네이트-2-설파타제의 정제
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JP6623213B2 (ja) 2014-08-11 2019-12-18 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 リソソーム酵素に融合したマンノース−6−リン酸含有ペプチド
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Also Published As

Publication number Publication date
EP2585105A2 (en) 2013-05-01
EP2585105B1 (en) 2017-08-09
WO2011163652A2 (en) 2011-12-29
MX2013000324A (es) 2013-02-01
WO2011163652A3 (en) 2013-07-11
CA2805449A1 (en) 2011-12-29
AU2017202344A1 (en) 2017-04-27
AU2011270672A1 (en) 2013-02-14
US20110318327A1 (en) 2011-12-29
NZ605871A (en) 2015-02-27
JP2013542913A (ja) 2013-11-28
NZ702801A (en) 2016-08-26
ES2643015T3 (es) 2017-11-21
KR20130043166A (ko) 2013-04-29
EP2585105A4 (en) 2014-07-09
CN103269709A (zh) 2013-08-28
AU2011270672B2 (en) 2017-01-12

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