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 PDFInfo
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- 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
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- 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
- A61K47/30—Macromolecular organic or inorganic compounds, e.g. inorganic polyphosphates
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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
- A61K9/0012—Galenical forms characterised by the site of application
- A61K9/0085—Brain, e.g. brain implants; Spinal cord
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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)
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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
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- A—HUMAN NECESSITIES
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- 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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- A—HUMAN NECESSITIES
- 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
- A61K47/30—Macromolecular organic or inorganic compounds, e.g. inorganic polyphosphates
- A61K47/36—Polysaccharides; Derivatives thereof, e.g. gums, starch, alginate, dextrin, hyaluronic acid, chitosan, inulin, agar or pectin
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- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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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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- A61P25/00—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
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- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P27/00—Drugs for disorders of the senses
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- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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- A—HUMAN NECESSITIES
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- 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
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- 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)
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- 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)
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- 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)
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y310/00—Hydrolases acting on sulfur-nitrogen bonds (3.10)
- C12Y310/01—Hydrolases acting on sulfur-nitrogen bonds (3.10) acting on sulfur-nitrogen bonds (3.10.1)
- C12Y310/01001—N-Sulfoglucosamine sulfohydrolase (3.10.1.1)
-
- A—HUMAN NECESSITIES
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- 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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- Engineering & Computer Science (AREA)
- General Health & Medical Sciences (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Medicinal Chemistry (AREA)
- Organic Chemistry (AREA)
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- Pharmacology & Pharmacy (AREA)
- Public Health (AREA)
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- Zoology (AREA)
- Wood Science & Technology (AREA)
- Biomedical Technology (AREA)
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- Chemical Kinetics & Catalysis (AREA)
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- Neurology (AREA)
- Neurosurgery (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Molecular Biology (AREA)
- Microbiology (AREA)
- Biotechnology (AREA)
- Inorganic Chemistry (AREA)
- Immunology (AREA)
- Gastroenterology & Hepatology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
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- Psychiatry (AREA)
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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 (enExample) |
| EP (1) | EP2588131B1 (enExample) |
| JP (1) | JP6073783B2 (enExample) |
| KR (1) | KR20140005842A (enExample) |
| CN (1) | CN103260637B (enExample) |
| AU (2) | AU2011270667B2 (enExample) |
| BR (1) | BR112012033197A2 (enExample) |
| CA (1) | CA2805413A1 (enExample) |
| MX (1) | MX2013000320A (enExample) |
| NZ (1) | NZ605874A (enExample) |
| RU (1) | RU2012154576A (enExample) |
| TW (1) | TWI546078B (enExample) |
| WO (1) | WO2011163647A2 (enExample) |
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| CA2694762A1 (en) | 2007-07-27 | 2009-02-05 | Armagen Technologies, Inc. | Methods and compositions for increasing alpha-l-iduronidase activity in the cns |
| EP2408474B1 (en) | 2009-03-18 | 2019-06-26 | Armagen, Inc. | Compositions and methods for blood-brain barrier delivery of igg-decoy receptor fusion proteins |
| HUE044865T2 (hu) | 2009-10-09 | 2019-11-28 | Armagen Inc | Eljárások és készítmények a központi idegrendszerben iduronát-2-szulfatáz-aktivitás növelésére |
| HUE031073T2 (en) | 2010-05-14 | 2017-06-28 | Dana Farber Cancer Inst Inc | Thieno triazolo-diazepine compounds for the treatment of neoplasia |
| CA2799403C (en) | 2010-05-14 | 2020-01-21 | Dana-Farber Cancer Institute, Inc. | Compositions and methods for treating leukemia |
| MX2013000320A (es) | 2010-06-25 | 2013-06-05 | Shire Human Genetic Therapies | Composiciones y metodos para suministro al sistema nervioso central de heparan n-sulfatasa. |
| PT3626258T (pt) | 2010-06-25 | 2021-10-19 | Shire Human Genetic Therapies | Métodos e composições para fornecimento ao snc de iduronato-2-sulfatase |
| US20110318327A1 (en) * | 2010-06-25 | 2011-12-29 | Shire Human Genetic Therapies, Inc. | Treatment of sanfilippo syndrome type b |
| DK2588130T3 (en) | 2010-06-25 | 2016-10-24 | Shire Human Genetic Therapies | Cns delivery of therapeutic agents |
| HRP20191924T1 (hr) | 2010-06-25 | 2020-01-10 | Shire Human Genetic Therapies, Inc. | Postupci i sastavi za isporuku iduronat-2-sulfataze u cns |
| NZ605873A (en) | 2010-06-25 | 2015-02-27 | Shire Human Genetic Therapies | Methods and compositions for cns delivery of arylsulfatase a |
| WO2012085622A1 (en) * | 2010-12-22 | 2012-06-28 | 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 |
| EP2548594B1 (en) * | 2011-07-22 | 2023-07-12 | Piramal Critical Care Inc. | Intrathecal baclofen pharmaceutical dosage forms and related delivery system |
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| WO2013096899A2 (en) | 2011-12-23 | 2013-06-27 | Shire Human Genetic Therapies, Inc. | Stable formulations for cns delivery of arylsulfatase a |
| MX367395B (es) | 2011-12-23 | 2019-08-20 | Shire Human Genetic Therapies | Iduronato-2-sulfatasa para usarse en el tratamiento del deterioro cognitivo del síndrome de hunter mediante suministro intratecal. |
| CA2880162C (en) * | 2012-07-31 | 2023-04-04 | Bioasis Technologies, Inc. | Dephosphorylated lysosomal storage disease proteins and methods of use thereof |
| BR112015012767A2 (pt) * | 2012-12-07 | 2017-09-12 | Shire Human Genetic Therapies | métodos e composições para tratamento intratecal administrado de mucupolisacaridose tipo iiia |
| CN105051056A (zh) * | 2013-03-13 | 2015-11-11 | 夏尔人类遗传性治疗公司 | 表征溶酶体酶的方法 |
| RU2692251C2 (ru) * | 2013-05-15 | 2019-06-24 | Риджентс Оф Зэ Юниверсити Оф Миннесота | Опосредованный аденоассоциированным вирусом перенос генов в центральную нервную систему |
| EP3730516A1 (en) * | 2013-07-22 | 2020-10-28 | Armagen, Inc. | Methods and compositions for increasing enzyme activity in the cns |
| RU2016122654A (ru) | 2013-11-08 | 2017-12-14 | Дана-Фарбер Кэнсер Инститьют, Инк. | Комбинированная терапия злокачественной опухоли с использованием ингибиторов бромодоменового и экстратерминального (вет) белка |
| CA2940472A1 (en) | 2014-02-28 | 2015-09-03 | Tensha Therapeutics, Inc. | Treatment of conditions associated with hyperinsulinaemia |
| EA033325B1 (ru) | 2014-10-27 | 2019-09-30 | Тэнша Терапеутикс, Инк. | Ингибиторы бромодомена |
| WO2016080367A1 (ja) | 2014-11-18 | 2016-05-26 | 塩野義製薬株式会社 | 安定化されたペプチド組成物 |
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| CN108883162A (zh) * | 2016-02-17 | 2018-11-23 | 夏尔人类遗传性治疗公司 | 用于芳基硫酸酯酶a的cns递送的方法和组合物 |
| WO2017181113A1 (en) | 2016-04-15 | 2017-10-19 | The Trustees Of The University Of Pennsyvania | Gene therapy for treating mucopolysaccharidosis type ii |
| KR102471458B1 (ko) * | 2016-12-28 | 2022-11-25 | 제이씨알 파마 가부시키가이샤 | 동결건조 제제 |
| 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 |
| UY38238A (es) * | 2018-05-25 | 2019-12-31 | 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 |
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| JP2023522853A (ja) * | 2020-04-07 | 2023-06-01 | ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア | 中枢神経系及び非中枢神経系への治療薬の送達のための合成エキソソームの製造 |
| 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 |
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| JP6073783B2 (ja) | 2017-02-01 |
| US9320711B2 (en) | 2016-04-26 |
| JP2013530988A (ja) | 2013-08-01 |
| AU2017202216A1 (en) | 2017-04-27 |
| EP2588131A2 (en) | 2013-05-08 |
| NZ605874A (en) | 2015-02-27 |
| MX2013000320A (es) | 2013-06-05 |
| AU2011270667A1 (en) | 2013-02-14 |
| CN103260637A (zh) | 2013-08-21 |
| BR112012033197A2 (pt) | 2017-06-06 |
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| CA2805413A1 (en) | 2011-12-29 |
| RU2012154576A (ru) | 2014-07-27 |
| WO2011163647A3 (en) | 2012-05-10 |
| WO2011163647A2 (en) | 2011-12-29 |
| AU2011270667B2 (en) | 2017-01-05 |
| TW201204385A (en) | 2012-02-01 |
| US20120014936A1 (en) | 2012-01-19 |
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