CY1122307T1 - Μεθοδοι και συνθεσεις για παραδοση της ιδουρονικης-2-σουλφατασης στο κνς - Google Patents
Μεθοδοι και συνθεσεις για παραδοση της ιδουρονικης-2-σουλφατασης στο κνςInfo
- Publication number
- CY1122307T1 CY1122307T1 CY20191101211T CY191101211T CY1122307T1 CY 1122307 T1 CY1122307 T1 CY 1122307T1 CY 20191101211 T CY20191101211 T CY 20191101211T CY 191101211 T CY191101211 T CY 191101211T CY 1122307 T1 CY1122307 T1 CY 1122307T1
- Authority
- CY
- Cyprus
- Prior art keywords
- sulfatase
- compositions
- methods
- iduronic
- cns
- Prior art date
Links
- 239000000203 mixture Substances 0.000 title abstract 3
- 238000000034 method Methods 0.000 title abstract 2
- 102100029199 Iduronate 2-sulfatase Human genes 0.000 abstract 2
- 101710096421 Iduronate 2-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
- 238000009472 formulation Methods 0.000 abstract 1
- 238000007913 intrathecal administration Methods 0.000 abstract 1
- 230000002132 lysosomal effect Effects 0.000 abstract 1
- 201000002273 mucopolysaccharidosis II Diseases 0.000 abstract 1
- 208000022018 mucopolysaccharidosis type 2 Diseases 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
-
- 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
- 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
- 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
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 ιδουρονική-2-σουλφατάση (I2S), άλας και ένα πολυσορβικό επιφανειοδραστικό για την θεραπεία του συνδρόμου Hunter.
Applications Claiming Priority (8)
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/US2011/041925 WO2011163649A2 (en) | 2010-06-25 | 2011-06-25 | Methods and compositions for cns delivery of iduronate-2-sulfatase |
Publications (1)
Publication Number | Publication Date |
---|---|
CY1122307T1 true CY1122307T1 (el) | 2021-01-27 |
Family
ID=46888843
Family Applications (2)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
CY20191101211T CY1122307T1 (el) | 2010-06-25 | 2019-11-19 | Μεθοδοι και συνθεσεις για παραδοση της ιδουρονικης-2-σουλφατασης στο κνς |
CY20211100914T CY1124703T1 (el) | 2010-06-25 | 2021-10-20 | Μεθοδοι και συνθεσεις για παραδοση της ιδουρονικης-2-σουλφατασης στο κνς |
Family Applications After (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
CY20211100914T CY1124703T1 (el) | 2010-06-25 | 2021-10-20 | Μεθοδοι και συνθεσεις για παραδοση της ιδουρονικης-2-σουλφατασης στο κνς |
Country Status (24)
Country | Link |
---|---|
US (1) | US8545837B2 (el) |
EP (3) | EP3964229A1 (el) |
JP (1) | JP6045492B2 (el) |
KR (5) | KR102603430B1 (el) |
CN (1) | CN103179980B (el) |
AU (4) | AU2011270669B2 (el) |
CA (1) | CA2805448A1 (el) |
CY (2) | CY1122307T1 (el) |
DK (1) | DK2593131T3 (el) |
ES (1) | ES2754234T3 (el) |
HR (1) | HRP20191924T1 (el) |
IL (3) | IL284599B2 (el) |
LT (1) | LT2593131T (el) |
ME (1) | ME03649B (el) |
MX (2) | MX2020001395A (el) |
NZ (2) | NZ702808A (el) |
PL (2) | PL2593131T3 (el) |
RS (1) | RS59469B1 (el) |
RU (1) | RU2660348C2 (el) |
SI (1) | SI2593131T1 (el) |
TW (1) | TWI611808B (el) |
UA (1) | UA115649C2 (el) |
WO (1) | WO2011163649A2 (el) |
ZA (1) | ZA201300672B (el) |
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CA2472937C (en) | 2002-01-11 | 2014-06-17 | Biomarin Pharmaceutical, Inc. | Use of p97 as an enzyme delivery system for the delivery of therapeutic lysosomal enzymes |
JP5901877B2 (ja) | 2007-07-27 | 2016-04-13 | アーメイゲン・テクノロジーズ・インコーポレイテッドArmagen Technologies, Inc. | 中枢神経系のα−L−イデュロニダーゼ活性を増加させるための方法および組成物 |
EP2588130B1 (en) | 2010-06-25 | 2016-08-17 | Shire Human Genetic Therapies, Inc. | Cns delivery of therapeutic agents |
RU2012154576A (ru) | 2010-06-25 | 2014-07-27 | Шир Хьюман Дженетик Терапис, Инк. | Способы и композиции для доставки к цнс гепаран-n-сульфатазы |
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 |
NZ702800A (en) | 2010-06-25 | 2017-03-31 | Shire Human Genetic Therapies | Methods and compositions for cns delivery of heparan n-sulfatase |
US20160145589A1 (en) | 2011-06-24 | 2016-05-26 | Green Cross Corporation | Composition and formulation comprising recombinant human iduronate-2-sulfatase and preparation method thereof |
JP6208658B2 (ja) | 2011-07-05 | 2017-10-04 | バイオアシス テクノロジーズ インコーポレイテッド | p97−抗体結合体および使用方法 |
US20140377244A1 (en) | 2011-12-23 | 2014-12-25 | Shire Human Genetic Therapies, Inc. | Stable formulations for cns delivery of arylsulfatase a |
AU2012358223B2 (en) * | 2011-12-23 | 2018-01-18 | Takeda Pharmaceutical Company Limited | Treatment of cognitive impairment of Hunter syndrome by intrathecal delivery of iduronate-2-sulfatase |
WO2013148277A1 (en) | 2012-03-30 | 2013-10-03 | Shire Human Genetic Therapies, Inc. | Subcutaneous administration of 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 |
US9150841B2 (en) | 2012-06-29 | 2015-10-06 | Shire Human Genetic Therapies, Inc. | Cells for producing recombinant iduronate-2-sulfatase |
EP2880156B1 (en) | 2012-07-31 | 2017-08-23 | 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 |
CN105263958B (zh) | 2013-03-13 | 2019-09-27 | 比奥阿赛斯技术有限公司 | p97片段及其应用 |
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 |
JP6603227B2 (ja) | 2014-02-03 | 2019-11-06 | バイオアシス テクノロジーズ インコーポレイテッド | P97融合タンパク質 |
DK3107562T3 (da) | 2014-02-19 | 2019-12-16 | Bioasis Technologies Inc | P97-ids-fusionsproteiner |
US10058619B2 (en) | 2014-05-01 | 2018-08-28 | Bioasis Technologies, Inc. | P97-polynucleotide conjugates |
MA40954A (fr) * | 2014-11-14 | 2017-09-19 | Shire Human Genetic Therapies | Détermination de niveaux de glycosaminoglycane par spectrométrie de masse |
US10967073B2 (en) | 2015-05-07 | 2021-04-06 | The Mclean Hospital Corporation | Glucocerebrosidase gene therapy for Parkinson's disease |
KR20220082100A (ko) * | 2015-05-15 | 2022-06-16 | 리젠츠 오브 더 유니버시티 오브 미네소타 | 중추 신경계로의 아데노-연관 치료적 전달 |
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RU2660348C2 (ru) | 2010-06-25 | 2018-07-05 | Шир Хьюман Дженетик Терапис, Инк. | Способы и композиции для доставки в цнс идуронат-2-сульфатазы |
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2011
- 2011-06-25 RU RU2012154575A patent/RU2660348C2/ru active
- 2011-06-25 JP JP2013516845A patent/JP6045492B2/ja active Active
- 2011-06-25 MX MX2020001395A patent/MX2020001395A/es unknown
- 2011-06-25 KR KR1020227002252A patent/KR102603430B1/ko active IP Right Grant
- 2011-06-25 MX MX2013000322A patent/MX2013000322A/es active IP Right Grant
- 2011-06-25 UA UAA201214667A patent/UA115649C2/uk unknown
- 2011-06-25 KR KR1020237039076A patent/KR20230159646A/ko not_active Application Discontinuation
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