CN103269709A - Sanfilippo综合症类型B的治疗 - Google Patents

Sanfilippo综合症类型B的治疗 Download PDF

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Publication number
CN103269709A
CN103269709A CN2011800407953A CN201180040795A CN103269709A CN 103269709 A CN103269709 A CN 103269709A CN 2011800407953 A CN2011800407953 A CN 2011800407953A CN 201180040795 A CN201180040795 A CN 201180040795A CN 103269709 A CN103269709 A CN 103269709A
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China
Prior art keywords
naglu
protein
described method
brain
sheath
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Pending
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CN2011800407953A
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English (en)
Chinese (zh)
Inventor
M·F·康西诺
P·加莱斯
J·潘
K·霍姆斯
P·马尔蒂尼
A·罗曼什柯
M·梅亚潘
B·张
A·伊斯肯德瑞安
D·伦德伯格
A·诺顿
B·斯特拉克-罗格
Y·黄
M·亚里山德里尼
R·菲佛
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Shire Human Genetics Therapies Inc
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Transkaryotic Therapies Inc
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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)
  • General Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Medicinal Chemistry (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Epidemiology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (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)
  • Molecular Biology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Psychology (AREA)
  • Inorganic Chemistry (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Psychiatry (AREA)
  • Genetics & Genomics (AREA)
  • Oil, Petroleum & Natural Gas (AREA)
  • General Engineering & Computer Science (AREA)
  • Hospice & Palliative Care (AREA)
  • Diabetes (AREA)
  • Hematology (AREA)
  • Obesity (AREA)
  • Anesthesiology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Peptides Or Proteins (AREA)
CN2011800407953A 2010-06-25 2011-06-25 Sanfilippo综合症类型B的治疗 Pending CN103269709A (zh)

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

Publications (1)

Publication Number Publication Date
CN103269709A true CN103269709A (zh) 2013-08-28

Family

ID=46888842

Family Applications (1)

Application Number Title Priority Date Filing Date
CN2011800407953A Pending CN103269709A (zh) 2010-06-25 2011-06-25 Sanfilippo综合症类型B的治疗

Country Status (11)

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

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WO2008022349A2 (en) 2006-08-18 2008-02-21 Armagen Technologies, Inc. Agents for blood-brain barrier delivery
AU2008282496B2 (en) 2007-07-27 2013-04-04 Armagen Technologies, Inc. Methods and compositions for increasing alpha-iduronidase activity in the CNS
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
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
NZ605874A (en) 2010-06-25 2015-02-27 Shire Human Genetic Therapies Methods and compositions for cns delivery of heparan n-sulfatase
EP2596123B1 (en) 2010-07-22 2015-11-25 Shire Human Genetic Therapies, Inc. Crystal structure of human alpha-n-acetylglucosaminidase
US8580922B2 (en) 2011-03-04 2013-11-12 Shire Human Genetic Therapies, Inc. Peptide linkers for polypeptide compositions and methods for using same
BR112013022557A2 (pt) * 2011-03-04 2017-08-01 Shire Human Genetic Therapies ligantes de peptídeo composições de polipeptídeo e métodos para uso dos mesmos
JP6329483B2 (ja) * 2011-10-12 2018-05-23 シナジェバ・バイオファーマ・コーポレイションSynageva Biopharma Corp. 組換えヒトnagluタンパク質およびその利用
AU2012346448B2 (en) 2011-12-02 2017-09-14 Armagen, Inc. Methods and compositions for increasing arylsulfatase A activity in the CNS
US20140377244A1 (en) 2011-12-23 2014-12-25 Shire Human Genetic Therapies, Inc. Stable formulations for cns delivery of arylsulfatase a
KR101380740B1 (ko) * 2012-06-29 2014-04-11 쉐어 휴먼 제네텍 세러피스, 인코포레이티드 이듀로네이트-2-설파타제의 정제
HUE039334T2 (hu) 2012-11-27 2018-12-28 Biomarin Pharm Inc Célzott terápiás lizoszómális enzim fúziós fehérjék és azok alkalmazásai
FR3011468B1 (fr) * 2013-10-04 2015-12-04 Inventiva Utilisation de l'odiparcil dans le traitement d'une mucopolysaccharidose
US20170267986A1 (en) * 2014-06-25 2017-09-21 Shire Human Genetic Therapies, Inc. Generation of mannose-6-phosphate containing recombinant alpha-n-acetyl glucosaminidase
US10722559B2 (en) 2014-08-11 2020-07-28 Shire Human Genetic Therapies, Inc. Mannose-6-phosphate bearing peptides fused to lysosomal enzymes
WO2016025523A1 (en) 2014-08-11 2016-02-18 Shire Human Genetic Therapies, Inc. Lysosomal targeting and uses thereof
US10413597B2 (en) 2014-09-29 2019-09-17 Alexion Pharmaceuticals, Inc. Methods of treating Mucopolysaccharidosis IIIB (MPSIIIB)
WO2016065319A1 (en) * 2014-10-24 2016-04-28 Shire Human Genetic Therapies, Inc. Lysosomal targeting of enzymes, and uses thereof
MA41022A (fr) 2014-11-24 2017-10-03 Shire Human Genetic Therapies Ciblage lysosomial et utilisations correspondantes
US10538589B2 (en) * 2015-01-14 2020-01-21 Armagen Inc. Methods and compositions for increasing N-acetylglucosaminidase (NAGLU) activity in the CNS using a fusion antibody comprising an anti-human insulin receptor antibody and NAGLU
EP4275751A3 (en) 2016-02-24 2023-12-27 BioMarin Pharmaceutical Inc. Improved naglu fusion protein formulation
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
AU2021333365B2 (en) * 2020-08-28 2026-04-02 Jcr Pharmaceuticals Co., Ltd. Mutant of alpha-N-acetylglucosaminidase

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Publication number Publication date
WO2011163652A3 (en) 2013-07-11
ES2643015T3 (es) 2017-11-21
NZ702801A (en) 2016-08-26
KR20130043166A (ko) 2013-04-29
US20110318327A1 (en) 2011-12-29
NZ605871A (en) 2015-02-27
JP6063380B2 (ja) 2017-01-18
CA2805449A1 (en) 2011-12-29
WO2011163652A2 (en) 2011-12-29
AU2011270672A1 (en) 2013-02-14
MX2013000324A (es) 2013-02-01
JP2013542913A (ja) 2013-11-28
EP2585105A4 (en) 2014-07-09
EP2585105B1 (en) 2017-08-09
AU2017202344A1 (en) 2017-04-27
AU2011270672B2 (en) 2017-01-12
EP2585105A2 (en) 2013-05-01

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Application publication date: 20130828