PL3626257T3 - Sposoby i kompozycje do dostarczania arylosulfatazy a do oun - Google Patents
Sposoby i kompozycje do dostarczania arylosulfatazy a do ounInfo
- Publication number
- PL3626257T3 PL3626257T3 PL19192508T PL19192508T PL3626257T3 PL 3626257 T3 PL3626257 T3 PL 3626257T3 PL 19192508 T PL19192508 T PL 19192508T PL 19192508 T PL19192508 T PL 19192508T PL 3626257 T3 PL3626257 T3 PL 3626257T3
- Authority
- PL
- Poland
- Prior art keywords
- arylsulfatase
- compositions
- methods
- cns delivery
- cns
- Prior art date
Links
- 102100022146 Arylsulfatase A Human genes 0.000 title 1
- 108010036867 Cerebroside-Sulfatase Proteins 0.000 title 1
- 239000000203 mixture Substances 0.000 title 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)
- 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
- 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)
-
- 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
- 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
- A61K38/54—Mixtures of enzymes or proenzymes covered by more than a single one of groups A61K38/44 - A61K38/46 or A61K38/51 - A61K38/53
-
- 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
-
- 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
- A61K9/0012—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
-
- 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)
Landscapes
- Health & Medical Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- General Health & Medical Sciences (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Medicinal Chemistry (AREA)
- Pharmacology & Pharmacy (AREA)
- Engineering & Computer Science (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Epidemiology (AREA)
- Organic Chemistry (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Neurosurgery (AREA)
- Neurology (AREA)
- Biomedical Technology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Immunology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Gastroenterology & Hepatology (AREA)
- Biochemistry (AREA)
- Psychology (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Inorganic Chemistry (AREA)
- Oil, Petroleum & Natural Gas (AREA)
- Molecular Biology (AREA)
- Genetics & Genomics (AREA)
- Zoology (AREA)
- General Engineering & Computer Science (AREA)
- Wood Science & Technology (AREA)
- Hematology (AREA)
- Psychiatry (AREA)
- Diabetes (AREA)
- Hospice & Palliative Care (AREA)
- Obesity (AREA)
- Dermatology (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicinal Preparation (AREA)
Applications Claiming Priority (10)
| 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 | |
| PCT/US2011/041926 WO2011163650A2 (en) | 2010-06-25 | 2011-06-25 | Methods and compositions for cns delivery of arylsulfatase a |
| EP19192508.0A EP3626257B1 (en) | 2010-06-25 | 2011-06-25 | Methods and compositions for cns delivery of arylsulfatase a |
| EP11799037.4A EP2585104B1 (en) | 2010-06-25 | 2011-06-25 | Methods and compositions for cns delivery of arylsulfatase a |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| PL3626257T3 true PL3626257T3 (pl) | 2022-03-28 |
Family
ID=46888844
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| PL11799037T PL2585104T3 (pl) | 2010-06-25 | 2011-06-25 | Sposoby i kompozycje do dostarczania arylosulfatazy a do oun |
| PL19192508T PL3626257T3 (pl) | 2010-06-25 | 2011-06-25 | Sposoby i kompozycje do dostarczania arylosulfatazy a do oun |
Family Applications Before (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| PL11799037T PL2585104T3 (pl) | 2010-06-25 | 2011-06-25 | Sposoby i kompozycje do dostarczania arylosulfatazy a do oun |
Country Status (26)
| Country | Link |
|---|---|
| US (1) | US9770410B2 (pl) |
| EP (3) | EP3964230A1 (pl) |
| JP (1) | JP6045493B2 (pl) |
| KR (5) | KR102318997B1 (pl) |
| CN (1) | CN103282046B (pl) |
| AU (5) | AU2011270670B2 (pl) |
| BR (1) | BR112012033216B1 (pl) |
| CA (1) | CA2803003C (pl) |
| CY (2) | CY1122282T1 (pl) |
| DK (1) | DK2585104T3 (pl) |
| ES (1) | ES2754776T3 (pl) |
| HR (1) | HRP20191923T1 (pl) |
| IL (3) | IL291556B2 (pl) |
| LT (1) | LT2585104T (pl) |
| ME (1) | ME03647B (pl) |
| MX (2) | MX344795B (pl) |
| NZ (2) | NZ605873A (pl) |
| PL (2) | PL2585104T3 (pl) |
| RS (1) | RS59468B1 (pl) |
| RU (1) | RU2671503C2 (pl) |
| SI (1) | SI2585104T1 (pl) |
| SM (1) | SMT201900589T1 (pl) |
| TW (1) | TWI667037B (pl) |
| UA (1) | UA115650C2 (pl) |
| WO (1) | WO2011163650A2 (pl) |
| ZA (1) | ZA201300675B (pl) |
Families Citing this family (19)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| PL2593131T3 (pl) | 2010-06-25 | 2020-01-31 | Shire Human Genetic Therapies, Inc. | Sposoby i kompozycje do dostarczania sulfatazy-2-iduronianiu do oun |
| RU2012154576A (ru) | 2010-06-25 | 2014-07-27 | Шир Хьюман Дженетик Терапис, Инк. | Способы и композиции для доставки к цнс гепаран-n-сульфатазы |
| UA115648C2 (uk) | 2010-06-25 | 2017-12-11 | Шае Хюмен Дженетік Терапіс, Інк. | Доставка терапевтичних агентів до цнс |
| CN104857504A (zh) | 2010-06-25 | 2015-08-26 | 夏尔人类遗传性治疗公司 | 芳基硫酸酯酶a cns递送的方法和组合物 |
| BR112012033216B1 (pt) | 2010-06-25 | 2022-10-11 | Shire Human Genetic Therapies, Inc | Uso de uma formulação estável compreendendo uma proteína arilsulfatase a (asa) e formulação estável para administração intratecal |
| WO2013096899A2 (en) | 2011-12-23 | 2013-06-27 | Shire Human Genetic Therapies, Inc. | Stable formulations for cns delivery of arylsulfatase a |
| EA030551B1 (ru) * | 2013-01-09 | 2018-08-31 | Шир Хьюман Дженетик Терапис, Инк. | Способы очистки арилсульфатазы а |
| CA2912678C (en) * | 2013-05-15 | 2023-10-10 | Regents Of The University Of Minnesota | Adeno-associated virus mediated gene transfer to the central nervous system |
| US10208734B2 (en) * | 2015-04-23 | 2019-02-19 | Continuum Dynamics, Inc. | Lift-driven wind turbine with force canceling blade configuration |
| EP3319597B1 (en) * | 2015-07-10 | 2021-02-17 | Byondis B.V. | Compositions comprising antibody-duocarmycin drug conjugates |
| JP2019509270A (ja) * | 2016-02-17 | 2019-04-04 | シャイアー ヒューマン ジェネティック セラピーズ インコーポレイテッド | 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 |
| WO2018017190A2 (en) * | 2016-06-01 | 2018-01-25 | Baxalta Incorporated | Formulations of polyalkylene oxide-asparaginase and methods of making and using the same |
| JP7042220B2 (ja) * | 2016-12-28 | 2022-03-25 | Jcrファーマ株式会社 | 凍結乾燥製剤 |
| WO2019060662A1 (en) | 2017-09-22 | 2019-03-28 | The Trustees Of The University Of Pennsylvania | GENE THERAPY FOR THE TREATMENT OF MUCOPOLYSACCHARIDOSE TYPE II |
| US20200384089A1 (en) | 2017-12-19 | 2020-12-10 | Shire Human Genetic Therapies, Inc. | Purified arylsulfatase a and compositons thereof |
| KR20210024082A (ko) | 2018-06-25 | 2021-03-04 | 제이씨알 파마 가부시키가이샤 | 단백질 함유 수성 액제 |
| AU2020270421A1 (en) * | 2019-05-03 | 2021-11-18 | The Trustees Of The University Of Pennsylvania | Compositions useful in treatment of metachromatic leukodystrophy |
| WO2021061517A1 (en) * | 2019-09-23 | 2021-04-01 | University Of Washington | Assay of arylsulfatase a enzymatic activity in dried blood spots by mass spectrometry and fluorometry |
| WO2024176112A1 (en) | 2023-02-21 | 2024-08-29 | Takeda Pharmaceutical Company Limited | Large scale production of recombinant arylsulfatase a and compositions thereof |
Family Cites Families (48)
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| 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. |
| KR20010101131A (ko) | 1998-12-07 | 2001-11-14 | 추후기재 | 폼페병의 치료 방법 |
| 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 |
| US6866844B2 (en) | 2002-11-07 | 2005-03-15 | Biomarin Pharmaceutical Inc. | Precursor N-acetylgalactosamine-4-sulfatase, methods of treatment using said enzyme and methods for producing and purifying said enzyme |
| AU2002256423B2 (en) | 2001-04-30 | 2008-07-24 | Zystor Therapeutics, Inc. | Subcellular targeting of therapeutic proteins |
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| ES2367257T3 (es) | 2002-04-25 | 2011-10-31 | Shire Human Genetic Therapies, Inc. | Tratamiento de deficit de alfa-galactosidasa a. |
| AU2003237314B2 (en) | 2002-05-29 | 2010-03-04 | Biomarin Pharmaceutical Inc. | Targeted therapeutic proteins |
| EP1587923B1 (en) | 2003-01-22 | 2011-08-24 | Duke University | Improved constructs for expressing lysosomal polypeptides |
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| BR112012033216B1 (pt) | 2010-06-25 | 2022-10-11 | Shire Human Genetic Therapies, Inc | Uso de uma formulação estável compreendendo uma proteína arilsulfatase a (asa) e formulação estável para administração intratecal |
| NZ702801A (en) | 2010-06-25 | 2016-08-26 | Shire Human Genetic Therapies | Treatment of sanfilippo syndrome type b |
| RU2012154576A (ru) | 2010-06-25 | 2014-07-27 | Шир Хьюман Дженетик Терапис, Инк. | Способы и композиции для доставки к цнс гепаран-n-сульфатазы |
| PL2593131T3 (pl) | 2010-06-25 | 2020-01-31 | Shire Human Genetic Therapies, Inc. | Sposoby i kompozycje do dostarczania sulfatazy-2-iduronianiu do oun |
| UA115648C2 (uk) | 2010-06-25 | 2017-12-11 | Шае Хюмен Дженетік Терапіс, Інк. | Доставка терапевтичних агентів до цнс |
| CN104857504A (zh) | 2010-06-25 | 2015-08-26 | 夏尔人类遗传性治疗公司 | 芳基硫酸酯酶a cns递送的方法和组合物 |
| WO2011163651A2 (en) | 2010-06-25 | 2011-12-29 | Shire Human Genetic Therapies, Inc. | METHODS AND COMPOSITIONS FOR CNS DELIVERY OF β-GALACTOCEREBROSIDASE |
| JP2012062312A (ja) | 2010-08-19 | 2012-03-29 | Yoshikatsu Eto | ハンター症候群の治療剤 |
-
2011
- 2011-06-25 BR BR112012033216-3A patent/BR112012033216B1/pt active IP Right Grant
- 2011-06-25 SM SM20190589T patent/SMT201900589T1/it unknown
- 2011-06-25 NZ NZ605873A patent/NZ605873A/en unknown
- 2011-06-25 UA UAA201214685A patent/UA115650C2/uk unknown
- 2011-06-25 PL PL11799037T patent/PL2585104T3/pl unknown
- 2011-06-25 EP EP21189310.2A patent/EP3964230A1/en active Pending
- 2011-06-25 CA CA2803003A patent/CA2803003C/en active Active
- 2011-06-25 KR KR1020207008105A patent/KR102318997B1/ko active Active
- 2011-06-25 PL PL19192508T patent/PL3626257T3/pl unknown
- 2011-06-25 IL IL291556A patent/IL291556B2/en unknown
- 2011-06-25 RU RU2012154574A patent/RU2671503C2/ru active
- 2011-06-25 RS RSP20191354 patent/RS59468B1/sr unknown
- 2011-06-25 MX MX2013000323A patent/MX344795B/es active IP Right Grant
- 2011-06-25 EP EP11799037.4A patent/EP2585104B1/en active Active
- 2011-06-25 NZ NZ702799A patent/NZ702799A/en unknown
- 2011-06-25 JP JP2013516846A patent/JP6045493B2/ja active Active
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