HRP20191924T1 - Postupci i sastavi za isporuku iduronat-2-sulfataze u cns - Google Patents
Postupci i sastavi za isporuku iduronat-2-sulfataze u cns Download PDFInfo
- Publication number
- HRP20191924T1 HRP20191924T1 HRP20191924TT HRP20191924T HRP20191924T1 HR P20191924 T1 HRP20191924 T1 HR P20191924T1 HR P20191924T T HRP20191924T T HR P20191924TT HR P20191924 T HRP20191924 T HR P20191924T HR P20191924 T1 HRP20191924 T1 HR P20191924T1
- Authority
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- Croatia
- Prior art keywords
- stable aqueous
- concentration
- polysorbate
- optionally
- approximately
- Prior art date
Links
- 102100029199 Iduronate 2-sulfatase Human genes 0.000 title claims 8
- 101710096421 Iduronate 2-sulfatase Proteins 0.000 title claims 8
- 239000000203 mixture Substances 0.000 title claims 5
- 238000000034 method Methods 0.000 title claims 2
- 239000013011 aqueous formulation Substances 0.000 claims 8
- FAPWRFPIFSIZLT-UHFFFAOYSA-M Sodium chloride Chemical group [Na+].[Cl-] FAPWRFPIFSIZLT-UHFFFAOYSA-M 0.000 claims 6
- 238000007913 intrathecal administration Methods 0.000 claims 6
- 229920000136 polysorbate Polymers 0.000 claims 5
- 229950008882 polysorbate Drugs 0.000 claims 5
- 239000004094 surface-active agent Substances 0.000 claims 5
- 238000009472 formulation Methods 0.000 claims 4
- 229920001213 Polysorbate 20 Polymers 0.000 claims 3
- 238000001990 intravenous administration Methods 0.000 claims 3
- 239000000256 polyoxyethylene sorbitan monolaurate Substances 0.000 claims 3
- 235000010486 polyoxyethylene sorbitan monolaurate Nutrition 0.000 claims 3
- 229940068977 polysorbate 20 Drugs 0.000 claims 3
- 239000011780 sodium chloride Substances 0.000 claims 3
- 239000005388 borosilicate glass Substances 0.000 claims 2
- 229940071643 prefilled syringe Drugs 0.000 claims 2
- 150000003839 salts Chemical class 0.000 claims 2
- 229910019142 PO4 Inorganic materials 0.000 claims 1
- 229920001219 Polysorbate 40 Polymers 0.000 claims 1
- 229920001214 Polysorbate 60 Polymers 0.000 claims 1
- 125000003275 alpha amino acid group Chemical group 0.000 claims 1
- 239000003708 ampul Substances 0.000 claims 1
- 239000003085 diluting agent Substances 0.000 claims 1
- 239000002552 dosage form Substances 0.000 claims 1
- 230000001506 immunosuppresive effect Effects 0.000 claims 1
- 201000002273 mucopolysaccharidosis II Diseases 0.000 claims 1
- 208000022018 mucopolysaccharidosis type 2 Diseases 0.000 claims 1
- NBIIXXVUZAFLBC-UHFFFAOYSA-K phosphate Chemical compound [O-]P([O-])([O-])=O NBIIXXVUZAFLBC-UHFFFAOYSA-K 0.000 claims 1
- 239000010452 phosphate Substances 0.000 claims 1
- 239000000244 polyoxyethylene sorbitan monooleate Substances 0.000 claims 1
- 235000010482 polyoxyethylene sorbitan monooleate Nutrition 0.000 claims 1
- 235000010483 polyoxyethylene sorbitan monopalmitate Nutrition 0.000 claims 1
- 239000000249 polyoxyethylene sorbitan monopalmitate Substances 0.000 claims 1
- 239000001818 polyoxyethylene sorbitan monostearate Substances 0.000 claims 1
- 235000010989 polyoxyethylene sorbitan monostearate Nutrition 0.000 claims 1
- 229920001296 polysiloxane Polymers 0.000 claims 1
- 229940101027 polysorbate 40 Drugs 0.000 claims 1
- 229940113124 polysorbate 60 Drugs 0.000 claims 1
- 229920000053 polysorbate 80 Polymers 0.000 claims 1
- 229940068968 polysorbate 80 Drugs 0.000 claims 1
- 229920005989 resin Polymers 0.000 claims 1
- 239000011347 resin Substances 0.000 claims 1
- 239000004447 silicone coating Substances 0.000 claims 1
Classifications
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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
- A61K9/08—Solutions
-
- 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)
-
- 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
-
- 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
- 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
-
- 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
- A61K47/06—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
-
- 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
- A61K9/14—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
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- 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
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P3/00—Drugs for disorders of the metabolism
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- 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
-
- 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)
-
- 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
- A61K9/0019—Injectable 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)
- Medicinal Chemistry (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Engineering & Computer Science (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Epidemiology (AREA)
- Organic Chemistry (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Neurology (AREA)
- Biomedical Technology (AREA)
- Neurosurgery (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Biochemistry (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Immunology (AREA)
- Gastroenterology & Hepatology (AREA)
- Genetics & Genomics (AREA)
- Zoology (AREA)
- Molecular Biology (AREA)
- Oil, Petroleum & Natural Gas (AREA)
- Inorganic Chemistry (AREA)
- Wood Science & Technology (AREA)
- General Engineering & Computer Science (AREA)
- Psychology (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Hospice & Palliative Care (AREA)
- Psychiatry (AREA)
- Obesity (AREA)
- Hematology (AREA)
- Diabetes (AREA)
- Dermatology (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicinal Preparation (AREA)
Claims (14)
1. Stabilna vodena formula za intratekalnu primjenu koja sadrži protein iduronat-2-sulfatazu (I2S) u koncentraciji od 10 mg/ml ili više, sol i polisorbatni surfaktant, pri čemu formula ima pH između 5,5 i 7,0 i sadrži fosfat u koncentraciji ne većoj od 5mM i pri čemu je sol NaCl.
2. Stabilna vodena formula prema patentnom zahtjevu 1, gdje je I2S protein prisutan u ili do koncentracije koja je izabrana od 15 mg/ml, 20 mg/ml, 25 mg/ml, 30 mg/ml, 35 mg/ml, 40 mg/ml, 45 mg/ml, 50 mg/ml, 60 mg/ml, 70 mg/ml, 80 mg/ml, 90 mg/ml, 100 mg/ml, 150 mg/ml, 200 mg/ml, 250 mg/ml, ili 300 mg/ml.
3. Stabilna vodena formula prema bilo kojem od prethodnih patentnih zahtjeva, gdje I2S protein sadrži aminokiselinsku sekvencu SEQ ID NO: 1.
4. Stabilna vodena formula prema bilo kojem od prethodnih patentnih zahtjeva, gdje je NaCl prisutan u koncentraciji u rasponu od približno 30-300 mM, opcionalno u koncentraciji u rasponu od približno 137-154 mM, opcionalno u koncentraciji od približno 154 mM.
5. Stabilna vodena formula prema bilo kojem od prethodnih patentnih zahtjeva, gdje je polisorbatni surfaktant izabran iz skupine koja se sastoji od polisorbata 20, polisorbata 40, polisorbata 60, polisorbata 80 i njihove kombinacije.
6. Stabilna vodena formula prema patentnom zahtjevu 5, gdje je polisorbatni surfaktant prisutan u koncentraciji u rasponu od 0,001 do 0,5%, opcionalno gdje je polisorbatni surfaktant polisorbat 20, opcionalno gdje je polisorbatni surfaktant prisutan u koncentraciji od približno 0,005%.
7. Stabilna vodena formula prema bilo kojem od prethodnih patentnih zahtjeva, pri čemu formula ima pH od približno 5,5-6,5 ili približno 6,0.
8. Stabilna vodena formula prema bilo kojem od prethodnih patentnih zahtjeva, pri čemu je formula rekonstituirana iz liofilizirane formule s razblaživačem.
9. Stabilna vodena formula prema bilo kojem od prethodnih patentnih zahtjeva, pri čemu formula sadrži protein iduronat-2-sulfatazu (I2S) u koncentraciji koja je u rasponu od približno 10-300 mg/ml, NaCl u koncentraciji od približno 154 mM, polisorbat 20 u koncentraciji od približno 0,005% i sa pH od približno 6,0, opcionalno gdje je protein I2S u koncentraciji od približno 10 mg/ml, 30 mg/ml, 50 mg/ml, 100 mg/ml ili 300 mg/ml.
10. Posuda za skladištenje koja sadrži jedinični dozni oblik stabilne vodene formule prema bilo kojem od prethodnih patentnih zahtjeva.
11. Posuda za skladištenje prema patentnom zahtjevu 10, pri čemu je posuda za skladištenje izabrana između ampule, bočice, patrone, rezervoara, lyo-injektora ili prethodno napunjene šprice, opcionalno gdje je posuda za skladištenje prethodno napunjena šprica i opcionalno je izabrana od borosilikatnih staklenih šprica s premazom od pečenog silikona, borosilikatnih staklenih šprica s raspršenim silikonom ili plastičnih šprica od smola bez silikona.
12. Posuda za skladištenje prema patentnom zahtjevu 10 ili 11, pri čemu je stabilna vodena formula prisutna u obujmu manjem od oko 5,0 mL, opcionalno manjem od oko 3,0 mL.
13. Stabilna vodena formula prema bilo kojem od patentnih zahtjeva 1-9, za upotrebu u postupku liječenja Hunterovog sindroma koji obuhvaća korak intratekalne primjene formule subjektu.
14. Stabilna vodena formula za upotrebu prema patentnom zahtjevu 13, pri čemu:
(i) intratekalna primjena se upotrebljava zajedno s intravenskom primjenom, opcionalno gdje intravenska primjena nije češća od jednom mjesečno ili nije češća od jednom u svaka dva mjeseca, ili
(ii) intratekalna primjena se upotrebljava u odsustvu intravenske primjene, i/ili
(iii) intratekalna primjena se vrši jednom u svaka dva tjedna, jednom mjesečno ili jednom u svaka dva mjeseca, i/ili
(iv) intratekalna primjena se upotrebljava u odsustvu istovremene imunosupresivne terapi02176je.
Applications Claiming Priority (9)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US35885710P | 2010-06-25 | 2010-06-25 | |
US36078610P | 2010-07-01 | 2010-07-01 | |
US38786210P | 2010-09-29 | 2010-09-29 | |
US201161435710P | 2011-01-24 | 2011-01-24 | |
US201161442115P | 2011-02-11 | 2011-02-11 | |
US201161476210P | 2011-04-15 | 2011-04-15 | |
US201161495268P | 2011-06-09 | 2011-06-09 | |
EP11799036.6A EP2593131B1 (en) | 2010-06-25 | 2011-06-25 | Methods and compositions for cns delivery of iduronate-2-sulfatase |
PCT/US2011/041925 WO2011163649A2 (en) | 2010-06-25 | 2011-06-25 | Methods and compositions for cns delivery of iduronate-2-sulfatase |
Publications (1)
Publication Number | Publication Date |
---|---|
HRP20191924T1 true HRP20191924T1 (hr) | 2020-01-10 |
Family
ID=46888843
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
HRP20191924TT HRP20191924T1 (hr) | 2010-06-25 | 2019-10-22 | Postupci i sastavi za isporuku iduronat-2-sulfataze u cns |
Country Status (24)
Country | Link |
---|---|
US (1) | US8545837B2 (hr) |
EP (3) | EP2593131B1 (hr) |
JP (1) | JP6045492B2 (hr) |
KR (5) | KR20230159646A (hr) |
CN (1) | CN103179980B (hr) |
AU (4) | AU2011270669B2 (hr) |
CA (1) | CA2805448A1 (hr) |
CY (2) | CY1122307T1 (hr) |
DK (1) | DK2593131T3 (hr) |
ES (1) | ES2754234T3 (hr) |
HR (1) | HRP20191924T1 (hr) |
IL (3) | IL284599B2 (hr) |
LT (1) | LT2593131T (hr) |
ME (1) | ME03649B (hr) |
MX (2) | MX2020001395A (hr) |
NZ (2) | NZ605863A (hr) |
PL (2) | PL2593131T3 (hr) |
RS (1) | RS59469B1 (hr) |
RU (1) | RU2660348C2 (hr) |
SI (1) | SI2593131T1 (hr) |
TW (1) | TWI611808B (hr) |
UA (1) | UA115649C2 (hr) |
WO (1) | WO2011163649A2 (hr) |
ZA (1) | ZA201300672B (hr) |
Families Citing this family (42)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
US20050158296A1 (en) | 2002-01-11 | 2005-07-21 | Starr Christopher M. | Use of p97 as an enzyme delivery system for the delivery of therapeutic lysosomal enzymes |
EP2997976A1 (en) | 2007-07-27 | 2016-03-23 | Armagen Technologies, Inc. | Methods and compositions for increasing alpha-l-iduronidase activity in the cns |
HUE055963T2 (hu) | 2010-06-25 | 2022-01-28 | Shire Human Genetic Therapies | Eljárások és kompozíciók iduronát-2-szulfáz központi idegrendszerbe történõ leadásához |
KR102094094B1 (ko) | 2010-06-25 | 2020-03-27 | 샤이어 휴먼 지네틱 테라피즈 인크. | 아릴설파타제 a의 cns 전달을 위한 방법들 및 조성물들 |
ES2650689T3 (es) | 2010-06-25 | 2018-01-19 | Shire Human Genetic Therapies, Inc. | Administración de agentes terapéuticos al sistema nervioso central |
CA2805413A1 (en) * | 2010-06-25 | 2011-12-29 | Shire Human Genetic Therapies, Inc. | Methods and compositions for cns delivery of heparan n-sulfatase |
MX2020001395A (es) | 2010-06-25 | 2022-03-24 | Shire Human Genetic Therapies | Composiciones y su uso para suministro de iduronato-2-sulfatasa al sistema nervioso central. |
US20160145589A1 (en) | 2011-06-24 | 2016-05-26 | Green Cross Corporation | Composition and formulation comprising recombinant human iduronate-2-sulfatase and preparation method thereof |
HUE029855T2 (en) | 2011-07-05 | 2017-04-28 | Bioasis Technologies Inc | p97 antibody conjugates |
CA2859988A1 (en) * | 2011-12-23 | 2013-06-27 | Shire Human Genetic Therapies, Inc. | Treatment of cognitive impairment of hunter syndrome by intrathecal delivery of iduronate-2-sulfatase |
EP2793922B1 (en) | 2011-12-23 | 2019-10-30 | Shire Human Genetic Therapies, Inc. | Stable formulations for cns delivery of arylsulfatase a |
AU2013240306A1 (en) | 2012-03-30 | 2014-10-09 | Shire Human Genetic Therapies, Inc. | Subcutaneous administration of iduronate- 2-sulfatase |
US9150841B2 (en) | 2012-06-29 | 2015-10-06 | Shire Human Genetic Therapies, Inc. | Cells for producing recombinant iduronate-2-sulfatase |
US20140004097A1 (en) | 2012-06-29 | 2014-01-02 | Shire Human Genetic Therapies, Inc. | Method of producing recombinant iduronate-2-sulfatase |
KR101380740B1 (ko) | 2012-06-29 | 2014-04-11 | 쉐어 휴먼 제네텍 세러피스, 인코포레이티드 | 이듀로네이트-2-설파타제의 정제 |
CA2880162C (en) * | 2012-07-31 | 2023-04-04 | Bioasis Technologies, Inc. | Dephosphorylated lysosomal storage disease proteins and methods of use thereof |
EA039246B1 (ru) * | 2012-12-06 | 2021-12-22 | Шир Хьюман Дженетик Терапис, Инк. | Способ лечения когнитивных нарушений, связанных с синдромом хантера |
WO2014160438A1 (en) | 2013-03-13 | 2014-10-02 | Bioasis Technologies Inc. | Fragments of p97 and uses thereof |
KR20220119187A (ko) * | 2013-05-15 | 2022-08-26 | 리젠츠 오브 더 유니버시티 오브 미네소타 | 중추 신경계로의 아데노-연관 바이러스 매개 유전자 전달 |
AU2015210612B2 (en) | 2014-02-03 | 2020-04-09 | Bioasis Technologies Inc. | P97 fusion proteins |
DK3107562T3 (da) | 2014-02-19 | 2019-12-16 | Bioasis Technologies Inc | P97-ids-fusionsproteiner |
CN106413757B (zh) | 2014-05-01 | 2022-01-14 | 比奥阿赛斯技术有限公司 | p97-多核苷酸结合物 |
MA40954A (fr) * | 2014-11-14 | 2017-09-19 | Shire Human Genetic Therapies | Détermination de niveaux de glycosaminoglycane par spectrométrie de masse |
EP3292206B8 (en) | 2015-05-07 | 2022-02-09 | Takeda Pharmaceutical Company Limited | Glucocerebrosidase gene therapy for parkinson's disease |
AU2016263119B2 (en) * | 2015-05-15 | 2021-08-12 | Regents Of The University Of Minnesota | Adeno-associated for therapeutic delivery to central nervous system |
KR20170004814A (ko) * | 2015-07-02 | 2017-01-11 | 주식회사 녹십자 | 헌터증후군 치료제 |
WO2017003270A1 (ko) * | 2015-07-02 | 2017-01-05 | 주식회사 녹십자 | 헌터증후군 치료제 및 치료방법 |
UA123704C2 (uk) * | 2015-12-30 | 2021-05-19 | Грін Кросс Корпорейшн | Спосіб лікування синдрому хантера |
AU2017207818B2 (en) * | 2016-01-15 | 2024-03-28 | Regents Of The University Of Minnesota | Methods and compositions for the treatment of neurologic disease |
CN117224659A (zh) * | 2016-02-17 | 2023-12-15 | 武田药品工业株式会社 | 用于芳基硫酸酯酶a的cns递送的方法和组合物 |
KR20180114199A (ko) * | 2016-02-24 | 2018-10-17 | 바이오마린 파머수티컬 인크. | 표적화된 치료적 리소좀 효소 융합 단백질들, 관련 제형들 및 이의 용도들 |
BR112018071156A2 (pt) | 2016-04-15 | 2019-03-12 | The Trustees Of The University Of Pennsylvania | terapia de gene para tratar mucopolissacaridose tipo ii |
US10940185B2 (en) | 2016-12-28 | 2021-03-09 | Jcr Pharmaceuticals Co., Ltd. | Lyophilized preparation |
BR112019021569A2 (pt) * | 2017-04-14 | 2020-05-12 | Regenxbio Inc. | Precursor de iduronato-2-sulfatase humana recombinante glicosilada (ids), método para tratar um sujeito humano diagnosticado com mucopolissacaridose tipo ii (mps ii) |
EP3684938A1 (en) | 2017-09-22 | 2020-07-29 | The Trustees of the University of Pennsylvania | Gene therapy for treating mucopolysaccharidosis type ii |
WO2019070577A1 (en) | 2017-10-02 | 2019-04-11 | Denali Therapeutics Inc. | FUSION PROTEINS COMPRISING SUBSTITUTE ENZYM THERAPY ENZYMES |
JP7060235B2 (ja) * | 2018-05-18 | 2022-04-26 | 国立大学法人広島大学 | 変異型イズロン酸-2-スルファターゼの機能回復薬剤のスクリーニング方法 |
BR112020024457A2 (pt) * | 2018-05-30 | 2021-03-23 | Medigenebio Corporation | método e composição para o tratamento de síndrome de hunter através de administração ventricular lateral cerebral |
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 |
KR20200047937A (ko) * | 2018-10-26 | 2020-05-08 | 재단법인 목암생명과학연구소 | Ids를 포함하는 융합 단백질 및 이의 용도 |
WO2020132452A1 (en) | 2018-12-20 | 2020-06-25 | Shire Human Genetic Therapies, Inc. | Purification of iduronate-2-sulfatase immunoglobulin fusion protein |
BR112023016075A2 (pt) | 2021-02-10 | 2023-11-21 | Regenxbio Inc | Método para tratar hepatoesplenomegalia, método de distribuição de um raav que codifica hids, método para tratar um sujeito humano, método de tratamento de um sujeito humano, método para determinar a eficácia do tratamento, método para identificar um sujeito como tendo mps ii |
Family Cites Families (35)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
US6118045A (en) * | 1995-08-02 | 2000-09-12 | Pharming B.V. | Lysosomal proteins produced in the milk of transgenic animals |
DE69808402T2 (de) * | 1997-08-22 | 2003-07-03 | Seikagaku Kogyo Co Ltd | Arzneimittel zur Behandlung von durch Hernie gestörter intervertebraler Bandscheibe |
ES2677343T3 (es) | 1998-12-07 | 2018-08-01 | Genzyme Corporation | Tratamiento de la enfermedad de Pompe |
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 |
US6534300B1 (en) | 1999-09-14 | 2003-03-18 | Genzyme Glycobiology Research Institute, Inc. | Methods for producing highly phosphorylated lysosomal hydrolases |
US20020099025A1 (en) * | 1999-12-30 | 2002-07-25 | Heywood James A. | Treatment of neurological disorders |
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 |
US7560424B2 (en) | 2001-04-30 | 2009-07-14 | Zystor Therapeutics, Inc. | Targeted therapeutic proteins |
US7629309B2 (en) | 2002-05-29 | 2009-12-08 | Zystor Therapeutics, Inc. | Targeted therapeutic proteins |
US20030072761A1 (en) | 2001-10-16 | 2003-04-17 | Lebowitz Jonathan | Methods and compositions for targeting proteins across the blood brain barrier |
WO2003032727A1 (en) | 2001-10-16 | 2003-04-24 | Symbiontics Inc. | Methods and compositions for targeting underglycosylated proteins across the blood brain barrier |
ES2367257T3 (es) | 2002-04-25 | 2011-10-31 | Shire Human Genetic Therapies, Inc. | Tratamiento de deficit de alfa-galactosidasa a. |
CA2487815A1 (en) | 2002-05-29 | 2003-12-11 | Symbiontics, Inc. | Targeted therapeutic proteins |
US20040248262A1 (en) | 2003-01-22 | 2004-12-09 | Koeberl Dwight D. | Constructs for expressing lysomal polypeptides |
PT2444102E (pt) * | 2003-01-31 | 2015-09-17 | Sinai School Medicine | Terapia combinada para o tratamento de distúrbios de deficiência proteica |
US7442372B2 (en) * | 2003-08-29 | 2008-10-28 | Biomarin Pharmaceutical Inc. | Delivery of therapeutic compounds to the brain and other tissues |
WO2005074888A2 (en) | 2004-02-03 | 2005-08-18 | Biodelivery Sciences International, Inc. | Replacement enzyme cochleates |
WO2005077093A2 (en) | 2004-02-06 | 2005-08-25 | Biomarin Pharmaceutical Inc. | Manufacture of highly phosphorylated lysosomal enzymes and uses thereof |
JP4914224B2 (ja) | 2004-02-10 | 2012-04-11 | バイオマリン ファーマシューティカル インコーポレイテッド | 酸性αグルコシダーゼおよびそのフラグメント |
US20050208090A1 (en) | 2004-03-18 | 2005-09-22 | Medtronic, Inc. | Methods and systems for treatment of neurological diseases of the central nervous system |
JPWO2006121199A1 (ja) | 2005-05-11 | 2008-12-18 | 日本ケミカルリサーチ株式会社 | 脂質小胞体組成物 |
AR059089A1 (es) | 2006-01-20 | 2008-03-12 | Genzyme Corp | Administracion intraventricular de una enzima para enfermedades de almacenamiento lisosomal |
GB0611463D0 (en) | 2006-06-09 | 2006-07-19 | Novartis Ag | Organic compounds |
CA2680189A1 (en) | 2007-03-06 | 2008-09-12 | Saint Louis University | Modified enzyme and treatment method |
SG10201604258YA (en) * | 2007-11-30 | 2016-07-28 | Abbvie Biotechnology Ltd | Anti-tnf antibody formulations |
US7722865B2 (en) | 2008-01-18 | 2010-05-25 | Biomarin Pharmaceutical Inc. | Manufacture of active highly phosphorylated human lysosomal sulfatase enzymes and uses thereof |
AU2009244148B2 (en) | 2008-05-07 | 2014-10-09 | Biomarin Pharmaceutical Inc. | Lysosomal targeting peptides and uses thereof |
KR102094094B1 (ko) | 2010-06-25 | 2020-03-27 | 샤이어 휴먼 지네틱 테라피즈 인크. | 아릴설파타제 a의 cns 전달을 위한 방법들 및 조성물들 |
MX2020001395A (es) | 2010-06-25 | 2022-03-24 | Shire Human Genetic Therapies | Composiciones y su uso para suministro de iduronato-2-sulfatasa al sistema nervioso central. |
CA2805413A1 (en) | 2010-06-25 | 2011-12-29 | Shire Human Genetic Therapies, Inc. | Methods and compositions for cns delivery of heparan n-sulfatase |
US20110318324A1 (en) | 2010-06-25 | 2011-12-29 | Shire Human Genetic Therapies, Inc. | Methods and compositions for cns delivery of b-galactocerebrosidase |
ES2650689T3 (es) | 2010-06-25 | 2018-01-19 | Shire Human Genetic Therapies, Inc. | Administración de agentes terapéuticos al sistema nervioso central |
JP2012062312A (ja) * | 2010-08-19 | 2012-03-29 | Yoshikatsu Eto | ハンター症候群の治療剤 |
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