CN103096918B - 治疗试剂的cns递送 - Google Patents

治疗试剂的cns递送 Download PDF

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Publication number
CN103096918B
CN103096918B CN201180040978.5A CN201180040978A CN103096918B CN 103096918 B CN103096918 B CN 103096918B CN 201180040978 A CN201180040978 A CN 201180040978A CN 103096918 B CN103096918 B CN 103096918B
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purposes
tissue
brain
enzymatic activity
enzyme
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CN201180040978.5A
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Chinese (zh)
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CN103096918A (zh
Inventor
P·加莱斯
J·潘
J·鲍威尔
L·查纳斯
T·麦考利
T·L·莱特
R·菲佛
Z·夏若克
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Takeda Pharmaceutical Co Ltd
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Shire Human Genetics Therapies Inc
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Application filed by Shire Human Genetics Therapies Inc filed Critical Shire Human Genetics Therapies Inc
Priority to CN201510512218.XA priority Critical patent/CN105233277B/zh
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    • 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
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/66—Microorganisms or materials therefrom
    • A61K35/76—Viruses; Subviral particles; Bacteriophages
    • A—HUMAN NECESSITIES
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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/66—Microorganisms or materials therefrom
    • A61K35/76—Viruses; Subviral particles; Bacteriophages
    • A61K35/761—Adenovirus
    • 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
    • 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
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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • 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
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    • 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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    • A61K9/00—Medicinal preparations characterised by special physical form
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    • 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
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    • 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
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • A61P27/02—Ophthalmic agents
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00—Drugs for disorders of the senses
    • A61P27/16—Otologicals
    • A—HUMAN NECESSITIES
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    • A—HUMAN NECESSITIES
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/24—Hydrolases (3) acting on glycosyl compounds (3.2)
    • C12N9/2402—Hydrolases (3) acting on glycosyl compounds (3.2) hydrolysing O- and S- glycosyl compounds (3.2.1)
    • 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)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y302/00—Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
    • C12Y302/01—Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
    • C12Y302/01045—Glucosylceramidase (3.2.1.45), i.e. beta-glucocerebrosidase
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y302/00—Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
    • C12Y302/01—Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
    • C12Y302/0105—Alpha-N-acetylglucosaminidase (3.2.1.50)
    • 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)

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  • Engineering & Computer Science (AREA)
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  • Pharmacology & Pharmacy (AREA)
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  • Public Health (AREA)
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  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Epidemiology (AREA)
  • Biomedical Technology (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Genetics & Genomics (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Virology (AREA)
  • Microbiology (AREA)
  • Immunology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
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  • Orthopedic Medicine & Surgery (AREA)
  • Mycology (AREA)
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  • Hospice & Palliative Care (AREA)
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  • Urology & Nephrology (AREA)
  • Cardiology (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Pain & Pain Management (AREA)
  • Psychiatry (AREA)
CN201180040978.5A 2010-06-25 2011-06-25 治疗试剂的cns递送 Active CN103096918B (zh)

Priority Applications (1)

Application Number Priority Date Filing Date Title
CN201510512218.XA CN105233277B (zh) 2010-06-25 2011-06-25 治疗试剂的cns递送

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/US2011/041924 WO2011163648A1 (en) 2010-06-25 2011-06-25 Cns delivery of therapeutic agents

Related Child Applications (1)

Application Number Title Priority Date Filing Date
CN201510512218.XA Division CN105233277B (zh) 2010-06-25 2011-06-25 治疗试剂的cns递送

Publications (2)

Publication Number Publication Date
CN103096918A CN103096918A (zh) 2013-05-08
CN103096918B true CN103096918B (zh) 2015-09-23

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US (1) US9283181B2 (https=)
EP (3) EP3875107A1 (https=)
JP (1) JP6045491B2 (https=)
KR (6) KR20240068752A (https=)
CN (1) CN103096918B (https=)
AU (4) AU2011270668B8 (https=)
CA (1) CA2803166C (https=)
CY (2) CY1118265T1 (https=)
DK (1) DK2588130T3 (https=)
ES (1) ES2650689T3 (https=)
HR (1) HRP20161505T1 (https=)
IL (3) IL291554B2 (https=)
LT (1) LT2588130T (https=)
MX (1) MX389903B (https=)
NZ (1) NZ605865A (https=)
PE (1) PE20230169A1 (https=)
PL (1) PL3103469T3 (https=)
RU (1) RU2626514C2 (https=)
SM (1) SMT201600385T1 (https=)
UA (1) UA115648C2 (https=)
WO (1) WO2011163648A1 (https=)
ZA (1) ZA201300671B (https=)

Families Citing this family (50)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2003222568B2 (en) 2002-01-11 2009-05-07 Bioasis Technologies, Inc. Use of P97 as an enzyme delivery system for the delivery of therapeutic lysosomal enzymes
NZ605874A (en) 2010-06-25 2015-02-27 Shire Human Genetic Therapies Methods and compositions for cns delivery of heparan n-sulfatase
RS62620B1 (sr) 2010-06-25 2021-12-31 Shire Human Genetic Therapies Metode i kompozicije za isporuku cns akrilsulfataze a
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
NZ605873A (en) 2010-06-25 2015-02-27 Shire Human Genetic Therapies Methods and compositions for cns delivery of arylsulfatase a
SMT201600385T1 (it) 2010-06-25 2017-03-08 Shire Human Genetic Therapies Trasporto di agenti terapeutici all’snc
WO2012101671A1 (en) * 2011-01-25 2012-08-02 Jcr Pharmaceuticals Co., Ltd. Method for production of human recombinant iduronate 2-sulfatase
ES2582869T3 (es) 2011-07-05 2016-09-15 Bioasis Technologies Inc Conjugados de P97-anticuerpo
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
US20140377244A1 (en) 2011-12-23 2014-12-25 Shire Human Genetic Therapies, Inc. Stable formulations for cns delivery of arylsulfatase a
EP2793933B1 (en) * 2011-12-23 2019-04-17 Shire Human Genetic Therapies, Inc. Treatment of cognitive impairment of hunter syndrome by intrathecal delivery of iduronate-2-sulfatase
ES2647082T3 (es) * 2012-07-31 2017-12-19 Bioasis Technologies Inc Proteínas desfosforiladas de la enfermedad de depósito lisosómico y métodos de uso de las mismas
AU2014205441B2 (en) 2013-01-09 2019-12-12 Takeda Pharmaceutical Company Limited Methods for purification of arylsulfatase A
EP2981551B1 (en) 2013-02-20 2020-06-03 Valerion Therapeutics, LLC Methods and compositions for treatment of pompe disease
JP6586412B2 (ja) 2013-03-13 2019-10-02 バイオアシス テクノロジーズ インコーポレイテッド ｐ９７のフラグメントおよびその使用
CN105377039A (zh) * 2013-05-15 2016-03-02 明尼苏达大学董事会 腺相关病毒介导的基因向中枢神经系统转移
JP6603227B2 (ja) 2014-02-03 2019-11-06 バイオアシス テクノロジーズ インコーポレイテッド Ｐ９７融合タンパク質
DK3107562T3 (da) 2014-02-19 2019-12-16 Bioasis Technologies Inc P97-ids-fusionsproteiner
US10058619B2 (en) 2014-05-01 2018-08-28 Bioasis Technologies, Inc. P97-polynucleotide conjugates
US10722559B2 (en) * 2014-08-11 2020-07-28 Shire Human Genetic Therapies, Inc. Mannose-6-phosphate bearing peptides fused to lysosomal enzymes
US10413597B2 (en) 2014-09-29 2019-09-17 Alexion Pharmaceuticals, Inc. Methods of treating Mucopolysaccharidosis IIIB (MPSIIIB)
WO2016100591A1 (en) * 2014-12-19 2016-06-23 Biogen Ma Inc. Apparatus for compound dispersion
EP3292206B8 (en) 2015-05-07 2022-02-09 Takeda Pharmaceutical Company Limited Glucocerebrosidase gene therapy for parkinson's disease
GB201508025D0 (en) 2015-05-11 2015-06-24 Ucl Business Plc Fabry disease gene therapy
KR20170004814A (ko) * 2015-07-02 2017-01-11 주식회사 녹십자 헌터증후군 치료제
WO2017062666A2 (en) * 2015-10-06 2017-04-13 Patricia Dickson Enzyme replacement therapy for mucopolysaccharidosis iiid
US10123969B2 (en) 2015-10-15 2018-11-13 Wisconsin Alumni Research Foundation Osmotic enhancement of drug/therapeutic delivery to the brain following infusion or injection into the cerebrospinal fluid
IL259964B (en) * 2015-12-15 2022-09-01 Genzyme Corp Adenovirus-associated vectors for the treatment of mucolipodosis type ii
WO2017116066A1 (en) 2015-12-30 2017-07-06 Green Cross Corporation Methods and compositions for treating hunter syndrome
EP3416678A1 (en) * 2016-02-17 2018-12-26 Shire Human Genetic Therapies, Inc. Methods and compositions for cns delivery of arylsulfatase a
IL305449B2 (en) 2016-04-15 2026-01-01 Univ Pennsylvania Gene therapy for the treatment of mucocutaneous type ii diabetes
AU2017342555A1 (en) 2016-10-14 2019-05-30 Children's Medical Center Corporation Compositions and methods for treating diseases and disorders of the central nervous system
WO2018136434A1 (en) 2017-01-17 2018-07-26 Children's Medical Center Corporation Compositions and methods for diagnosing and treating peroxisomal diseases
GB201709459D0 (en) 2017-06-14 2017-07-26 Intrabio Ltd Treatment for migraine
KR102655498B1 (ko) 2017-09-07 2024-04-11 제이씨알 파마 가부시키가이샤 수성 의약 조성물
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
AU2018345303B2 (en) 2017-10-02 2025-09-18 Denali Therapeutics Inc. Fusion proteins comprising enzyme replacement therapy enzymes
LT3697399T (lt) 2017-10-18 2026-01-12 Intrabio Ltd Terapiniai agentai, skirti panaudoti gydant neramių kojų sindromą
US12458614B2 (en) 2017-10-18 2025-11-04 Intrabio Ltd Therapeutic agents for improved mobility and cognitive function and for treating neurodegenerative diseases and lysosomal storage disorders
WO2019126376A1 (en) 2017-12-19 2019-06-27 Shire Human Genetic Therapies, Inc. Purified arylsulfatase a and compositons thereof
JP7337081B2 (ja) 2018-02-15 2023-09-01 イントラバイオ リミティド レストレスレッグズ症候群を治療するための治療薬
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
WO2020178721A1 (en) 2019-03-02 2020-09-10 Intrabio Ltd. Leucine, acetyl leucine, and related analogs for treating disease
SI3989962T1 (sl) 2019-06-28 2025-11-28 Intrabio Ltd Kombinirano zdravljenje z acetil-levcinom in miglustatom za zdravljenje lizosomske bolezni kopičenja
AU2020336984B2 (en) * 2019-08-29 2026-04-23 Biomarin Pharmaceutical Inc. Methods for treating CLN2 disease in pediatric subjects
US20230051742A1 (en) 2020-01-13 2023-02-16 Intrabio Ltd. Treatment of late-onset neurodegenerative diseases in heterozygous npc1 gene mutation carriers
JP2023526517A (ja) 2020-05-22 2023-06-21 イントラビオ エルティーディー 運動失調症を治療するためのアセチルロイシンと４－アミノピリジン又はアセタゾラミドとの組合せ
JP2025538189A (ja) * 2022-11-09 2025-11-26 ジーエムピー・バイオテクノロジー・リミテッド Ｃｎｓ疾患のための組成物および方法
WO2024176112A1 (en) 2023-02-21 2024-08-29 Takeda Pharmaceutical Company Limited Large scale production of recombinant arylsulfatase a and compositions thereof
WO2025264957A2 (en) 2024-06-20 2025-12-26 Intrabio Inc. Acetyl leucine for treating neurodevelopmental disorders

Family Cites Families (48)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4743265A (en) * 1986-04-23 1988-05-10 Dij Catheter Corp Articulated catheter placement device
US5863782A (en) 1995-04-19 1999-01-26 Women's And Children's Hospital Synthetic mammalian sulphamidase and genetic sequences encoding same
US6118045A (en) 1995-08-02 2000-09-12 Pharming B.V. Lysosomal proteins produced in the milk of transgenic animals
ES2185117T3 (es) 1997-08-22 2003-04-16 Seikagaku Kogyo Co Ltd Agente terapeutico para disco intervertebral herniado.
DK1137762T3 (da) 1998-12-07 2009-02-02 Genzyme Corp Behandling af Pompes sygdom
US6217552B1 (en) 1999-03-01 2001-04-17 Coaxia, Inc. Medical device for selective intrathecal spinal cooling in aortic surgery and spinal trauma
US20020052311A1 (en) 1999-09-03 2002-05-02 Beka Solomon Methods and compostions for the treatment and/or diagnosis of neurological diseases and disorders
US6537785B1 (en) 1999-09-14 2003-03-25 Genzyme Glycobiology Research Institute, Inc. Methods of treating lysosomal storage diseases
US20020099025A1 (en) * 1999-12-30 2002-07-25 Heywood James A. Treatment of neurological disorders
US7560424B2 (en) 2001-04-30 2009-07-14 Zystor Therapeutics, Inc. Targeted therapeutic proteins
US20040005309A1 (en) 2002-05-29 2004-01-08 Symbiontics, Inc. Targeted therapeutic proteins
ATE384736T1 (de) 2001-04-30 2008-02-15 Zystor Therapeutics Inc Subzelluläres targeting von therapeutischen proteinen
US7629309B2 (en) 2002-05-29 2009-12-08 Zystor Therapeutics, Inc. Targeted therapeutic proteins
WO2003032913A2 (en) 2001-10-16 2003-04-24 Symbiontics Inc. Methods and compositions for targeting proteins across the blood brain barrier
US20030072761A1 (en) 2001-10-16 2003-04-17 Lebowitz Jonathan Methods and compositions for targeting proteins across the blood brain barrier
EP1503788B1 (en) * 2002-04-25 2011-06-29 Shire Human Genetic Therapies, Inc. Treatment of alpha-galactosidase a deficiency
CA2487815A1 (en) 2002-05-29 2003-12-11 Biomarin Pharmaceutical Inc. Targeted therapeutic proteins
ATE521701T1 (de) 2003-01-22 2011-09-15 Univ Duke Verbesserte konstrukte zur expression lysosomaler polypeptide
US20050026823A1 (en) * 2003-06-20 2005-02-03 Biomarin Pharmaceutical Inc. Use of the chaperone receptor-associated protein (RAP) for the delivery of therapeutic compounds to the brain and other tissues
CA2525236C (en) 2003-06-20 2015-03-24 Biomarin Pharmaceutical Inc. Delivery of therapeutic compounds to the brain and other tissues
US7442372B2 (en) 2003-08-29 2008-10-28 Biomarin Pharmaceutical Inc. Delivery of therapeutic compounds to the brain and other tissues
US8536315B2 (en) 2004-01-30 2013-09-17 Shire Pharmaceuticals Ireland Limited Production and purification of recombinant arylsulftase
US20060029656A1 (en) 2004-02-03 2006-02-09 Biodelivery Sciences International, Inc. Replacement enzyme cochleates
CA2556245A1 (en) 2004-02-06 2005-08-25 Biomarin Pharmaceutical Inc. Manufacture of highly phosphorylated lysosomal enzymes and uses thereof
CN1922313B (zh) 2004-02-10 2011-10-26 生物马林医药公司 酸性α-糖苷酶及其片段
US20050208090A1 (en) * 2004-03-18 2005-09-22 Medtronic, Inc. Methods and systems for treatment of neurological diseases of the central nervous system
WO2006121199A1 (ja) 2005-05-11 2006-11-16 Oxygenix Co., Ltd. 脂質小胞体組成物
CN101541172A (zh) 2005-06-08 2009-09-23 阿米库斯治疗学公司 溶酶体酶编码基因突变相关的cns紊乱的治疗
AR059089A1 (es) 2006-01-20 2008-03-12 Genzyme Corp Administracion intraventricular de una enzima para enfermedades de almacenamiento lisosomal
ES2928096T3 (es) 2006-04-04 2022-11-15 Takeda Pharmaceuticals Co Un proceso para la concentración de un polipéptido
GB0611463D0 (en) 2006-06-09 2006-07-19 Novartis Ag Organic compounds
WO2008070769A1 (en) 2006-12-06 2008-06-12 Medtronic, Inc. Intrathecal catheter
WO2008109677A2 (en) 2007-03-06 2008-09-12 Saint Louis University Modified enzyme and treatment method
EP2174661B1 (en) * 2007-06-29 2012-12-19 National University Corporation Nagoya University Agent for improving neuropathic pain
WO2009017005A1 (ja) 2007-07-27 2009-02-05 Sharp Kabushiki Kaisha 移動局装置、基地局装置、通信システム及びプログラム
US8933197B2 (en) 2007-08-15 2015-01-13 Amunix Operating Inc. Compositions comprising modified biologically active polypeptides
TWI543768B (zh) 2007-11-30 2016-08-01 艾伯維生物技術有限責任公司 蛋白質調配物及製造其之方法
US7722865B2 (en) 2008-01-18 2010-05-25 Biomarin Pharmaceutical Inc. Manufacture of active highly phosphorylated human lysosomal sulfatase enzymes and uses thereof
HRP20170698T1 (hr) 2008-05-07 2017-09-22 Biomarin Pharmaceutical Inc. Lizosomski usmjereni peptidi i njihove uporabe
US8436489B2 (en) 2009-06-29 2013-05-07 Lightsail Energy, Inc. Compressed air energy storage system utilizing two-phase flow to facilitate heat exchange
NZ605874A (en) 2010-06-25 2015-02-27 Shire Human Genetic Therapies Methods and compositions for cns delivery of heparan n-sulfatase
NZ605873A (en) 2010-06-25 2015-02-27 Shire Human Genetic Therapies Methods and compositions for cns delivery of arylsulfatase a
SMT201600385T1 (it) 2010-06-25 2017-03-08 Shire Human Genetic Therapies Trasporto di agenti terapeutici all’snc
EP2588132A4 (en) 2010-06-25 2014-10-15 Shire Human Genetic Therapies METHOD AND COMPOSITIONS FOR DELIVERING BETA GALACTOCEREBROSIDASE INTO THE CNS
RS62620B1 (sr) 2010-06-25 2021-12-31 Shire Human Genetic Therapies Metode i kompozicije za isporuku cns akrilsulfataze a
CA2805449A1 (en) 2010-06-25 2011-12-29 Shire Human Genetic Therapies, Inc. Treatment of sanfilippo syndrome type b
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
JP2012062312A (ja) 2010-08-19 2012-03-29 Yoshikatsu Eto ハンター症候群の治療剤

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
"Japan Elaprase ® Treatment (JET) study: Idursulfase enzyme replacement therapy in adult patients with attenuated Hunter syndrome (Mucopolysaccharidosis II, MPS II);Torayuki Okuyama 等;《Molecular Genetics and Metabolism》;20090824;第99卷;18-25 *
INTRATHECAL DELIVERY OF PROTEIN THERAPEUTICS TO TREAT GENETIC DISEASES INVOLVING THE CNS;Zahra Shahrokh 等;《Injectable Drug Delivery 2010: Formulations Focus》;20100531;16-20 *

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