CY1124910T1 - Μεθοδοι και συνθεσεις για παραδοση στο κνς της αρυλοσουλφατασης α - Google Patents
Μεθοδοι και συνθεσεις για παραδοση στο κνς της αρυλοσουλφατασης αInfo
- Publication number
- CY1124910T1 CY1124910T1 CY20211100942T CY211100942T CY1124910T1 CY 1124910 T1 CY1124910 T1 CY 1124910T1 CY 20211100942 T CY20211100942 T CY 20211100942T CY 211100942 T CY211100942 T CY 211100942T CY 1124910 T1 CY1124910 T1 CY 1124910T1
- Authority
- CY
- Cyprus
- Prior art keywords
- compositions
- methods
- cns
- arylosulfatase
- cns delivery
- Prior art date
Links
- 239000000203 mixture Substances 0.000 title abstract 3
- 238000000034 method Methods 0.000 title abstract 2
- 102100022146 Arylsulfatase A Human genes 0.000 abstract 2
- 108010036867 Cerebroside-Sulfatase Proteins 0.000 abstract 2
- 102000004190 Enzymes Human genes 0.000 abstract 1
- 108090000790 Enzymes Proteins 0.000 abstract 1
- 208000015439 Lysosomal storage disease Diseases 0.000 abstract 1
- 201000011442 Metachromatic leukodystrophy Diseases 0.000 abstract 1
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 abstract 1
- 238000009472 formulation Methods 0.000 abstract 1
- 238000007913 intrathecal administration Methods 0.000 abstract 1
- 230000002132 lysosomal effect Effects 0.000 abstract 1
- 229950008882 polysorbate Drugs 0.000 abstract 1
- 229920000136 polysorbate Polymers 0.000 abstract 1
- 150000003839 salts Chemical class 0.000 abstract 1
- 239000004094 surface-active agent Substances 0.000 abstract 1
Classifications
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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)
- A61K38/47—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
- A61K9/08—Solutions
-
- 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
- A61K9/14—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
Abstract
Η παρούσα εφεύρεση παρέχει, μεταξύ άλλων, συνθέσεις και μεθόδους για παράδοση στο ΚΝΣ λυσοσωματικών ενζύμων για αποτελεσματική θεραπεία νόσων λυσοσωματικής αποθήκευσης. Σε μερικές υλοποιήσεις, η παρούσα εφεύρεση περιλαμβάνει ένα σταθερό σκεύασμα για απευθείας ενδορραχιαία χορήγηση στο ΚΝΣ που περιλαμβάνει μία πρωτεΐνη αρυλοσουλφατάση Α (ASA), άλας και ένα πολυσορβικό επιφανειοδραστικό για την θεραπεία της Νόσου Μεταχρωματικής Λευκοδυστροφίας (Metachromatic Leukodystrophy Disease).
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 | |
EP11799037.4A EP2585104B1 (en) | 2010-06-25 | 2011-06-25 | Methods and compositions for cns delivery of arylsulfatase a |
PCT/US2011/041926 WO2011163650A2 (en) | 2010-06-25 | 2011-06-25 | Methods and compositions for cns delivery of arylsulfatase a |
Publications (1)
Publication Number | Publication Date |
---|---|
CY1124910T1 true CY1124910T1 (el) | 2023-01-05 |
Family
ID=46888844
Family Applications (2)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
CY20191101190T CY1122282T1 (el) | 2010-06-25 | 2019-11-13 | Μεθοδοι και συνθεσεις για παραδοση στο κνς της αρυλοσουλφατασης α |
CY20211100942T CY1124910T1 (el) | 2010-06-25 | 2021-11-02 | Μεθοδοι και συνθεσεις για παραδοση στο κνς της αρυλοσουλφατασης α |
Family Applications Before (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
CY20191101190T CY1122282T1 (el) | 2010-06-25 | 2019-11-13 | Μεθοδοι και συνθεσεις για παραδοση στο κνς της αρυλοσουλφατασης α |
Country Status (25)
Country | Link |
---|---|
US (1) | US9770410B2 (el) |
EP (3) | EP2585104B1 (el) |
JP (1) | JP6045493B2 (el) |
KR (5) | KR102094094B1 (el) |
CN (1) | CN103282046B (el) |
AU (4) | AU2011270670B2 (el) |
BR (1) | BR112012033216B1 (el) |
CA (1) | CA2803003C (el) |
CY (2) | CY1122282T1 (el) |
DK (1) | DK2585104T3 (el) |
ES (1) | ES2754776T3 (el) |
HR (1) | HRP20191923T1 (el) |
IL (3) | IL291556B2 (el) |
LT (1) | LT2585104T (el) |
ME (1) | ME03647B (el) |
MX (1) | MX344795B (el) |
NZ (2) | NZ702799A (el) |
PL (2) | PL2585104T3 (el) |
RS (1) | RS59468B1 (el) |
RU (1) | RU2671503C2 (el) |
SI (1) | SI2585104T1 (el) |
TW (1) | TWI667037B (el) |
UA (1) | UA115650C2 (el) |
WO (1) | WO2011163650A2 (el) |
ZA (1) | ZA201300675B (el) |
Families Citing this family (17)
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IL291556B2 (en) | 2010-06-25 | 2024-02-01 | Shire Human Genetic Therapies | Methods and compositions for administration of arylsulfatase A to the central nervous system |
RU2660348C2 (ru) | 2010-06-25 | 2018-07-05 | Шир Хьюман Дженетик Терапис, Инк. | Способы и композиции для доставки в цнс идуронат-2-сульфатазы |
EP2588130B1 (en) | 2010-06-25 | 2016-08-17 | Shire Human Genetic Therapies, Inc. | Cns delivery of therapeutic agents |
NZ702800A (en) | 2010-06-25 | 2017-03-31 | Shire Human Genetic Therapies | Methods and compositions for cns delivery of heparan n-sulfatase |
RU2012154576A (ru) | 2010-06-25 | 2014-07-27 | Шир Хьюман Дженетик Терапис, Инк. | Способы и композиции для доставки к цнс гепаран-n-сульфатазы |
US20140377244A1 (en) | 2011-12-23 | 2014-12-25 | Shire Human Genetic Therapies, Inc. | Stable formulations for cns delivery of arylsulfatase a |
BR112015015948B1 (pt) | 2013-01-09 | 2022-05-17 | Takeda Pharmaceutical Company Limited | Método de purificação da proteína arilsulfatase a (asa) recombinante |
CN115120746A (zh) * | 2013-05-15 | 2022-09-30 | 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 |
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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 |
EP3463308B1 (en) * | 2016-06-01 | 2021-12-01 | Servier IP UK Limited | Formulations of polyalkylene oxide-asparaginase and methods of making and using the same |
CN110114078B (zh) * | 2016-12-28 | 2024-01-30 | Jcr制药股份有限公司 | 冷冻干燥制剂 |
US11819539B2 (en) | 2017-09-22 | 2023-11-21 | The Trustees Of The University Of Pennsylvania | Gene therapy for treating Mucopolysaccharidosis type II |
CN111699249A (zh) | 2017-12-19 | 2020-09-22 | 夏尔人类遗传性治疗公司 | 纯化的芳基硫酸酯酶a及其组合物 |
US11738068B2 (en) | 2018-06-25 | 2023-08-29 | Jcr Pharmaceuticals Co., Ltd. | Protein-containing aqueous liquid formulation |
US20220348981A1 (en) * | 2019-09-23 | 2022-11-03 | University Of Washington | Assay of arylsulfatase a enzymatic activity in dried blood spots by mass spectrometry and fluorometry |
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NZ702800A (en) | 2010-06-25 | 2017-03-31 | Shire Human Genetic Therapies | Methods and compositions for cns delivery of heparan n-sulfatase |
IL291556B2 (en) | 2010-06-25 | 2024-02-01 | Shire Human Genetic Therapies | Methods and compositions for administration of arylsulfatase A to the central nervous system |
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2011
- 2011-06-25 IL IL291556A patent/IL291556B2/en unknown
- 2011-06-25 EP EP11799037.4A patent/EP2585104B1/en active Active
- 2011-06-25 KR KR1020187014958A patent/KR102094094B1/ko active IP Right Grant
- 2011-06-25 LT LT11799037T patent/LT2585104T/lt unknown
- 2011-06-25 JP JP2013516846A patent/JP6045493B2/ja active Active
- 2011-06-25 NZ NZ702799A patent/NZ702799A/en unknown
- 2011-06-25 MX MX2013000323A patent/MX344795B/es active IP Right Grant
- 2011-06-25 SI SI201131776T patent/SI2585104T1/sl unknown
- 2011-06-25 PL PL11799037T patent/PL2585104T3/pl unknown
- 2011-06-25 EP EP19192508.0A patent/EP3626257B1/en active Active
- 2011-06-25 KR KR1020237017347A patent/KR20230079472A/ko not_active Application Discontinuation
- 2011-06-25 PL PL19192508T patent/PL3626257T3/pl unknown
- 2011-06-25 CN CN201180040802.XA patent/CN103282046B/zh active Active
- 2011-06-25 BR BR112012033216-3A patent/BR112012033216B1/pt active IP Right Grant
- 2011-06-25 RS RSP20191354 patent/RS59468B1/sr unknown
- 2011-06-25 RU RU2012154574A patent/RU2671503C2/ru active
- 2011-06-25 ME MEP-2019-298A patent/ME03647B/me unknown
- 2011-06-25 US US13/168,963 patent/US9770410B2/en active Active
- 2011-06-25 KR KR1020207008105A patent/KR102318997B1/ko active IP Right Grant
- 2011-06-25 ES ES11799037T patent/ES2754776T3/es active Active
- 2011-06-25 AU AU2011270670A patent/AU2011270670B2/en active Active
- 2011-06-25 EP EP21189310.2A patent/EP3964230A1/en active Pending
- 2011-06-25 CA CA2803003A patent/CA2803003C/en active Active
- 2011-06-25 UA UAA201214685A patent/UA115650C2/uk unknown
- 2011-06-25 KR KR1020137001752A patent/KR101997935B1/ko active IP Right Grant
- 2011-06-25 WO PCT/US2011/041926 patent/WO2011163650A2/en active Application Filing
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