CN103260637B - 乙酰肝素n-硫酸酯酶cns递送的方法和组合物 - Google Patents

乙酰肝素n-硫酸酯酶cns递送的方法和组合物 Download PDF

Info

Publication number
CN103260637B
CN103260637B CN201180040902.2A CN201180040902A CN103260637B CN 103260637 B CN103260637 B CN 103260637B CN 201180040902 A CN201180040902 A CN 201180040902A CN 103260637 B CN103260637 B CN 103260637B
Authority
CN
China
Prior art keywords
concentration
purposes
hns
protein
stabilization formulations
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Expired - Fee Related
Application number
CN201180040902.2A
Other languages
English (en)
Chinese (zh)
Other versions
CN103260637A (zh
Inventor
F·纳托里
G·朱
J·特略
Y·江
J·宗
Z·夏若克
B·维纳格利亚
J·潘
R·菲佛
P·加莱斯
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Shire Human Genetics Therapies Inc
Original Assignee
Shire Human Genetics Therapies Inc
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Shire Human Genetics Therapies Inc filed Critical Shire Human Genetics Therapies Inc
Publication of CN103260637A publication Critical patent/CN103260637A/zh
Application granted granted Critical
Publication of CN103260637B publication Critical patent/CN103260637B/zh
Expired - Fee Related legal-status Critical Current
Anticipated expiration legal-status Critical

Links

Classifications

    • 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
    • A61K47/30Macromolecular organic or inorganic compounds, e.g. inorganic polyphosphates
    • 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)
    • 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
    • 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
    • A61K47/30Macromolecular organic or inorganic compounds, e.g. inorganic polyphosphates
    • A61K47/36Polysaccharides; Derivatives thereof, e.g. gums, starch, alginate, dextrin, hyaluronic acid, chitosan, inulin, agar or pectin
    • 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
    • A61P25/02Drugs for disorders of the nervous system for peripheral neuropathies
    • 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
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14Hydrolases (3)
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14Hydrolases (3)
    • C12N9/24Hydrolases (3) acting on glycosyl compounds (3.2)
    • C12N9/2402Hydrolases (3) acting on glycosyl compounds (3.2) hydrolysing O- and S- glycosyl compounds (3.2.1)
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y302/00Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
    • C12Y302/01Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
    • C12Y302/0105Alpha-N-acetylglucosaminidase (3.2.1.50)
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y310/00Hydrolases acting on sulfur-nitrogen bonds (3.10)
    • C12Y310/01Hydrolases acting on sulfur-nitrogen bonds (3.10) acting on sulfur-nitrogen bonds (3.10.1)
    • C12Y310/01001N-Sulfoglucosamine sulfohydrolase (3.10.1.1)
    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Medicinal Chemistry (AREA)
  • Organic Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Epidemiology (AREA)
  • Zoology (AREA)
  • Genetics & Genomics (AREA)
  • Wood Science & Technology (AREA)
  • Biomedical Technology (AREA)
  • Biochemistry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • General Engineering & Computer Science (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Molecular Biology (AREA)
  • Microbiology (AREA)
  • Biotechnology (AREA)
  • Inorganic Chemistry (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Immunology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Psychology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Oil, Petroleum & Natural Gas (AREA)
  • Psychiatry (AREA)
  • Anesthesiology (AREA)
  • Hospice & Palliative Care (AREA)
  • Diabetes (AREA)
  • Hematology (AREA)
  • Obesity (AREA)
CN201180040902.2A 2010-06-25 2011-06-25 乙酰肝素n-硫酸酯酶cns递送的方法和组合物 Expired - Fee Related CN103260637B (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/US2011/041922 WO2011163647A2 (en) 2010-06-25 2011-06-25 Methods and compositions for cns delivery of heparan n-sulfatase

Related Child Applications (1)

Application Number Title Priority Date Filing Date
CN201610086722.2A Division CN105664142A (zh) 2010-06-25 2011-06-25 乙酰肝素n-硫酸酯酶cns递送的方法和组合物

Publications (2)

Publication Number Publication Date
CN103260637A CN103260637A (zh) 2013-08-21
CN103260637B true CN103260637B (zh) 2016-04-06

Family

ID=46889509

Family Applications (1)

Application Number Title Priority Date Filing Date
CN201180040902.2A Expired - Fee Related CN103260637B (zh) 2010-06-25 2011-06-25 乙酰肝素n-硫酸酯酶cns递送的方法和组合物

Country Status (13)

Country Link
US (1) US9320711B2 (https=)
EP (1) EP2588131B1 (https=)
JP (1) JP6073783B2 (https=)
KR (1) KR20140005842A (https=)
CN (1) CN103260637B (https=)
AU (2) AU2011270667B2 (https=)
BR (1) BR112012033197A2 (https=)
CA (1) CA2805413A1 (https=)
MX (1) MX2013000320A (https=)
NZ (1) NZ605874A (https=)
RU (1) RU2012154576A (https=)
TW (1) TWI546078B (https=)
WO (1) WO2011163647A2 (https=)

Families Citing this family (43)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
CA2748889A1 (en) 2009-03-18 2010-09-23 Armagen Technologies, Inc. Compositions and methods for blood-brain barrier delivery of igg-decoy receptor fusion proteins
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
MX354217B (es) 2010-05-14 2018-02-19 Dana Farber Cancer Inst Inc Composiciones y metodos para el tratamiento de leucemia.
PL2902030T3 (pl) 2010-05-14 2017-07-31 Dana-Farber Cancer Institute, Inc. Związki tienotriazolodiazepinowe do leczenia nowotworu
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
CA2805449A1 (en) * 2010-06-25 2011-12-29 Shire Human Genetic Therapies, Inc. Treatment of sanfilippo syndrome type b
NZ605874A (en) 2010-06-25 2015-02-27 Shire Human Genetic Therapies Methods and compositions for cns delivery of heparan n-sulfatase
SMT201600385T1 (it) 2010-06-25 2017-03-08 Shire Human Genetic Therapies Trasporto di agenti terapeutici all’snc
AU2010366066B2 (en) * 2010-12-22 2016-01-14 Fondazione Telethon Therapeutic strategies to treat CNS pathology in mucopolysaccharidoses
TW201307563A (zh) * 2011-05-19 2013-02-16 Shire Human Genetic Therapies 純化乙醯肝素-n-硫酸酯酶之方法
ES2954743T3 (es) * 2011-07-22 2023-11-24 Piramal Critical Care Inc Formulaciones farmacéuticas de baclofeno intratecal y sistema de suministro relacionado
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
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
EA201590582A1 (ru) * 2012-12-07 2016-01-29 Шир Хьюман Дженетик Терапис, Инк. Способы и композиции для лечения мукополисахаридоза типа iiia с использованием интратекального введения
WO2014160456A1 (en) * 2013-03-13 2014-10-02 Shire Human Genetic Therapies, Inc. Method of characterizing lysosomal enzymes
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
JP6692293B2 (ja) * 2013-07-22 2020-05-13 アーマジェン・インコーポレイテッドArmagen, Inc. Cnsにおける酵素活性を増大するための方法および組成物
CA2929652A1 (en) 2013-11-08 2015-05-14 Dana-Farber Cancer Institute, Inc. Combination therapy for cancer using bromodomain and extra-terminal (bet) protein inhibitors
SG11201607108XA (en) 2014-02-28 2016-09-29 Tensha Therapeutics Inc Treatment of conditions associated with hyperinsulinaemia
BR112017008714A2 (pt) 2014-10-27 2017-12-19 Tensha Therapeutics Inc inibidores de bromodomínio
JP6781508B2 (ja) 2014-11-18 2020-11-04 塩野義製薬株式会社 安定化されたペプチド組成物
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
WO2017087685A1 (en) 2015-11-20 2017-05-26 The Regents Of The University Of California Deformable nano-scale vehicles (dnvs) for trans-blood brain barrier, trans-mucosal, and transdermal drug delivery
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
US10940185B2 (en) 2016-12-28 2021-03-09 Jcr Pharmaceuticals Co., Ltd. Lyophilized preparation
WO2018187240A1 (en) 2017-04-03 2018-10-11 The Regents Of The University Of California Deformable nano-scale vehicles (dnvs) for trans-blood brain barrier, trans-mucosal, and transdermal drug delivery
US11584777B2 (en) * 2017-08-31 2023-02-21 Green Cross Corporation Method for purifying a sulfatase protein
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
PY1939780A (es) * 2018-05-25 2019-12-04 Genzyme Corp Composiciones farmacéuticas para el tratamiento de la deficiencia de esfingomielinasa ácida
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
BE1026619B1 (fr) 2018-09-17 2020-04-14 Safran Aero Boosters Sa Systeme de mesure pour turbomachine
US12493019B2 (en) 2019-02-20 2025-12-09 Amgen Inc. Methods of determining protein stability
WO2021207273A1 (en) * 2020-04-07 2021-10-14 The Regents Of The University Of California Manufacturing of synthetic exosomes for cns and non-cns delivery of therapeutics
PE20231931A1 (es) 2020-10-14 2023-12-01 Denali Therapeutics Inc Proteinas de fusion que comprenden enzimas sulfoglucosamina sulfohidrolasa y metodos de estas
CN120379648A (zh) * 2022-12-23 2025-07-25 株式会社绿十字 具有改善的稳定性的包含乙酰肝素n-硫酸酯酶的药物组合物
TW202508617A (zh) * 2023-08-28 2025-03-01 南韓商綠十字股份有限公司 包括乙醯肝素n-硫酸酯酶(hns)的用於治療聖菲利波症候群a型(mps iiia)的組合物

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20090017005A1 (en) * 2003-08-29 2009-01-15 Biomarion Pharmaceutical Inc. Delivery of Therapeutic Compounds to the Brain and Other Tissues
CN101410408A (zh) * 2006-04-04 2009-04-15 希尔制药爱尔兰有限责任公司 用于浓缩多肽的方法
CN101431960A (zh) * 2006-01-20 2009-05-13 建新公司 用于溶酶体贮积症的脑室内酶输注系统

Family Cites Families (45)

* 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
ATE384736T1 (de) 2001-04-30 2008-02-15 Zystor Therapeutics Inc Subzelluläres targeting von therapeutischen proteinen
US20040005309A1 (en) 2002-05-29 2004-01-08 Symbiontics, Inc. Targeted therapeutic proteins
US7629309B2 (en) 2002-05-29 2009-12-08 Zystor Therapeutics, Inc. Targeted therapeutic proteins
US7560424B2 (en) 2001-04-30 2009-07-14 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
CA2525236C (en) 2003-06-20 2015-03-24 Biomarin Pharmaceutical Inc. Delivery of therapeutic compounds to the brain and other tissues
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
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
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
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
SMT201600385T1 (it) 2010-06-25 2017-03-08 Shire Human Genetic Therapies Trasporto di agenti terapeutici all’snc
RS62620B1 (sr) 2010-06-25 2021-12-31 Shire Human Genetic Therapies Metode i kompozicije za isporuku cns akrilsulfataze a
NZ605873A (en) 2010-06-25 2015-02-27 Shire Human Genetic Therapies Methods and compositions for cns delivery of arylsulfatase a
EP2588132A4 (en) 2010-06-25 2014-10-15 Shire Human Genetic Therapies METHOD AND COMPOSITIONS FOR DELIVERING BETA GALACTOCEREBROSIDASE INTO THE CNS
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

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20090017005A1 (en) * 2003-08-29 2009-01-15 Biomarion Pharmaceutical Inc. Delivery of Therapeutic Compounds to the Brain and Other Tissues
CN101431960A (zh) * 2006-01-20 2009-05-13 建新公司 用于溶酶体贮积症的脑室内酶输注系统
CN101410408A (zh) * 2006-04-04 2009-04-15 希尔制药爱尔兰有限责任公司 用于浓缩多肽的方法

Also Published As

Publication number Publication date
AU2011270667B2 (en) 2017-01-05
RU2012154576A (ru) 2014-07-27
WO2011163647A3 (en) 2012-05-10
US9320711B2 (en) 2016-04-26
AU2017202216A1 (en) 2017-04-27
MX2013000320A (es) 2013-06-05
JP6073783B2 (ja) 2017-02-01
EP2588131B1 (en) 2019-08-14
NZ605874A (en) 2015-02-27
JP2013530988A (ja) 2013-08-01
CN103260637A (zh) 2013-08-21
TW201204385A (en) 2012-02-01
BR112012033197A2 (pt) 2017-06-06
CA2805413A1 (en) 2011-12-29
US20120014936A1 (en) 2012-01-19
KR20140005842A (ko) 2014-01-15
WO2011163647A2 (en) 2011-12-29
EP2588131A4 (en) 2014-04-09
AU2011270667A1 (en) 2013-02-14
TWI546078B (zh) 2016-08-21
EP2588131A2 (en) 2013-05-08

Similar Documents

Publication Publication Date Title
CN103260637B (zh) 乙酰肝素n-硫酸酯酶cns递送的方法和组合物
US12161702B2 (en) Methods and compositions for CNS delivery of heparan N-sulfatase
KR102356192B1 (ko) 이듀로네이트-2-설파타제의 cns 전달을 위한 방법들 및 조성물들
CN103282046B (zh) 芳基硫酸酯酶 a cns 递送的方法和组合物
JP2024102370A (ja) アリールスルファターゼaのcns送達のための方法および組成物

Legal Events

Date Code Title Description
C06 Publication
PB01 Publication
C10 Entry into substantive examination
SE01 Entry into force of request for substantive examination
C14 Grant of patent or utility model
GR01 Patent grant
CF01 Termination of patent right due to non-payment of annual fee

Granted publication date: 20160406

Termination date: 20170625

CF01 Termination of patent right due to non-payment of annual fee