MX390902B - Composiciones y su uso para suministro de iduronato-2-sulfatasa al sistema nervioso central. - Google Patents
Composiciones y su uso para suministro de iduronato-2-sulfatasa al sistema nervioso central.Info
- Publication number
- MX390902B MX390902B MX2020001395A MX2020001395A MX390902B MX 390902 B MX390902 B MX 390902B MX 2020001395 A MX2020001395 A MX 2020001395A MX 2020001395 A MX2020001395 A MX 2020001395A MX 390902 B MX390902 B MX 390902B
- Authority
- MX
- Mexico
- Prior art keywords
- iduronate
- sulfatase
- compositions
- supplying
- nervous system
- Prior art date
Links
- 102100029199 Iduronate 2-sulfatase Human genes 0.000 title abstract 3
- 101710096421 Iduronate 2-sulfatase Proteins 0.000 title abstract 3
- 239000000203 mixture Substances 0.000 title abstract 3
- 210000003169 central nervous system Anatomy 0.000 title 1
- 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
- 238000000034 method Methods 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
- 102000004169 proteins and genes Human genes 0.000 abstract 1
- 108090000623 proteins and genes Proteins 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
- 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
- A61K9/0012—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
- A61K9/14—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
Landscapes
- Health & Medical Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- General Health & Medical Sciences (AREA)
- Medicinal Chemistry (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Engineering & Computer Science (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Epidemiology (AREA)
- Organic Chemistry (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Neurosurgery (AREA)
- Neurology (AREA)
- Biomedical Technology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Biochemistry (AREA)
- Immunology (AREA)
- Gastroenterology & Hepatology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Orthopedic Medicine & Surgery (AREA)
- General Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- Inorganic Chemistry (AREA)
- Molecular Biology (AREA)
- Oil, Petroleum & Natural Gas (AREA)
- Psychology (AREA)
- Psychiatry (AREA)
- Hospice & Palliative Care (AREA)
- Obesity (AREA)
- Hematology (AREA)
- Diabetes (AREA)
- Dermatology (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicinal Preparation (AREA)
Abstract
La presente invención se refiere a entre otras cosas, composiciones y métodos para suministro al SNC de enzimas lisosomales para tratamiento efectivo de enfermedades de almacenamiento lisosomal; en algunas modalidades, la presente invención incluye una formulación estable para administración intratecal directa al SNC que comprende una proteína Iduronato-2-sulfatasa (I2S), sal, y un tensoactivo de polisorbato para el tratamiento de Síndrome de Hunters.
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 (2)
| Publication Number | Publication Date |
|---|---|
| MX2020001395A MX2020001395A (es) | 2022-03-24 |
| MX390902B true MX390902B (es) | 2025-03-21 |
Family
ID=46888843
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| MX2020001395A MX390902B (es) | 2010-06-25 | 2011-06-25 | Composiciones y su uso para suministro de iduronato-2-sulfatasa al sistema nervioso central. |
| MX2013000322A MX2013000322A (es) | 2010-06-25 | 2011-06-25 | Composiciones y metodos para suministro al sistema nervioso central de iduronato-2-sulfatasa. |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| MX2013000322A MX2013000322A (es) | 2010-06-25 | 2011-06-25 | Composiciones y metodos para suministro al sistema nervioso central de iduronato-2-sulfatasa. |
Country Status (25)
| Country | Link |
|---|---|
| US (1) | US8545837B2 (es) |
| EP (3) | EP2593131B1 (es) |
| JP (1) | JP6045492B2 (es) |
| KR (5) | KR102603430B1 (es) |
| CN (1) | CN103179980B (es) |
| AU (4) | AU2011270669B2 (es) |
| CA (1) | CA2805448A1 (es) |
| CY (2) | CY1122307T1 (es) |
| DK (1) | DK2593131T3 (es) |
| ES (1) | ES2754234T3 (es) |
| HR (1) | HRP20191924T1 (es) |
| IL (3) | IL284599B2 (es) |
| LT (1) | LT2593131T (es) |
| ME (1) | ME03649B (es) |
| MX (2) | MX390902B (es) |
| NZ (2) | NZ605863A (es) |
| PL (2) | PL3626258T3 (es) |
| RS (1) | RS59469B1 (es) |
| RU (1) | RU2660348C2 (es) |
| SI (1) | SI2593131T1 (es) |
| SM (1) | SMT201900594T1 (es) |
| TW (1) | TWI611808B (es) |
| UA (1) | UA115649C2 (es) |
| WO (1) | WO2011163649A2 (es) |
| ZA (1) | ZA201300672B (es) |
Families Citing this family (46)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| AU2003222568B2 (en) | 2002-01-11 | 2009-05-07 | Bioasis Technologies, Inc. | Use of P97 as an enzyme delivery system for the delivery of therapeutic lysosomal enzymes |
| AU2008282496B2 (en) | 2007-07-27 | 2013-04-04 | Armagen Technologies, Inc. | Methods and compositions for increasing alpha-iduronidase activity in the CNS |
| JP2013507131A (ja) | 2009-10-09 | 2013-03-04 | アーメイゲン・テクノロジーズ・インコーポレイテッド | Cnsにおけるイズロン酸2−スルファターゼ活性を増加させるための方法および組成物 |
| RS62620B1 (sr) | 2010-06-25 | 2021-12-31 | Shire Human Genetic Therapies | Metode i kompozicije za isporuku cns akrilsulfataze a |
| NZ605874A (en) | 2010-06-25 | 2015-02-27 | Shire Human Genetic Therapies | Methods and compositions for cns delivery of heparan n-sulfatase |
| NZ605873A (en) | 2010-06-25 | 2015-02-27 | Shire Human Genetic Therapies | Methods and compositions for cns delivery of arylsulfatase a |
| SMT201600385T1 (it) | 2010-06-25 | 2017-03-08 | Shire Human Genetic Therapies | Trasporto di agenti terapeutici all’snc |
| CN103179980B (zh) | 2010-06-25 | 2016-09-28 | 夏尔人类遗传性治疗公司 | 艾杜糖醛酸-2-硫酸酯酶的cns递送的方法和组合物 |
| US20160145589A1 (en) | 2011-06-24 | 2016-05-26 | Green Cross Corporation | Composition and formulation comprising recombinant human iduronate-2-sulfatase and preparation method thereof |
| ES2582869T3 (es) | 2011-07-05 | 2016-09-15 | Bioasis Technologies Inc | Conjugados de P97-anticuerpo |
| EP2793933B1 (en) * | 2011-12-23 | 2019-04-17 | Shire Human Genetic Therapies, Inc. | Treatment of cognitive impairment of hunter syndrome by intrathecal delivery of iduronate-2-sulfatase |
| US20140377244A1 (en) | 2011-12-23 | 2014-12-25 | Shire Human Genetic Therapies, Inc. | Stable formulations for cns delivery of arylsulfatase a |
| EA201491578A1 (ru) * | 2012-03-30 | 2015-04-30 | Шир Хьюман Дженетик Терапис, Инк. | Подкожное введение идуронат-2-сульфатазы |
| 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 |
| US20140004097A1 (en) | 2012-06-29 | 2014-01-02 | Shire Human Genetic Therapies, Inc. | Method of producing recombinant iduronate-2-sulfatase |
| ES2647082T3 (es) | 2012-07-31 | 2017-12-19 | Bioasis Technologies Inc | Proteínas desfosforiladas de la enfermedad de depósito lisosómico y métodos de uso de las mismas |
| EA039246B1 (ru) * | 2012-12-06 | 2021-12-22 | Шир Хьюман Дженетик Терапис, Инк. | Способ лечения когнитивных нарушений, связанных с синдромом хантера |
| JP6586412B2 (ja) | 2013-03-13 | 2019-10-02 | バイオアシス テクノロジーズ インコーポレイテッド | p97のフラグメントおよびその使用 |
| SG11201509030TA (en) | 2013-05-07 | 2015-11-27 | Bio Blast Pharma Ltd | Treatment of protein aggregation myopathic and neurodegenerative diseases by parenteral administration of trehalose |
| CN105377039A (zh) * | 2013-05-15 | 2016-03-02 | 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 |
| EP3292206B8 (en) | 2015-05-07 | 2022-02-09 | Takeda Pharmaceutical Company Limited | Glucocerebrosidase gene therapy for parkinson's disease |
| HK1256341A1 (zh) * | 2015-05-15 | 2019-09-20 | 明尼苏达大学董事会 | 用於治疗性递送到中枢神经系统的腺相关物 |
| 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 |
| WO2017116066A1 (en) | 2015-12-30 | 2017-07-06 | Green Cross Corporation | Methods and compositions for treating hunter syndrome |
| CA3010738A1 (en) * | 2016-01-15 | 2017-07-20 | Sangamo Therapeutics, Inc. | Methods and compositions for the treatment of neurologic disease |
| EP3416678A1 (en) * | 2016-02-17 | 2018-12-26 | Shire Human Genetic Therapies, Inc. | Methods and compositions for cns delivery of arylsulfatase a |
| EP4275751A3 (en) * | 2016-02-24 | 2023-12-27 | BioMarin Pharmaceutical Inc. | Improved naglu fusion protein formulation |
| IL305449B2 (en) | 2016-04-15 | 2026-01-01 | Univ Pennsylvania | Gene therapy for the treatment of mucocutaneous type ii diabetes |
| US10940185B2 (en) | 2016-12-28 | 2021-03-09 | Jcr Pharmaceuticals Co., Ltd. | Lyophilized preparation |
| JP2020516291A (ja) * | 2017-04-14 | 2020-06-11 | レジェンクスバイオ インコーポレーテッド | ヒト神経細胞又はグリア細胞により生産された組換えヒトイズロン酸−2−スルファターゼ(ids)によるムコ多糖症ii型の治療 |
| 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 |
| AU2018345303B2 (en) | 2017-10-02 | 2025-09-18 | Denali Therapeutics Inc. | Fusion proteins comprising enzyme replacement 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 |
| KR102671857B1 (ko) * | 2018-05-30 | 2024-06-04 | 주식회사 녹십자 | 대뇌 측뇌실 투여에 의해 헌터증후군을 치료하기 위한 방법 및 조성물 |
| 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 |
| KR102692543B1 (ko) * | 2018-10-26 | 2024-08-07 | 재단법인 목암생명과학연구소 | Ids를 포함하는 융합 단백질 및 이의 용도 |
| JP7458404B2 (ja) | 2018-12-20 | 2024-03-29 | アーマジェン・インコーポレイテッド | イズロン酸-2-スルファターゼ免疫グロブリン融合タンパク質の精製 |
| CA3150309A1 (en) * | 2019-10-01 | 2021-04-08 | Seelos Therapeutics, Inc. | Trehalose formulations and uses thereof |
| CA3167741A1 (en) * | 2020-01-29 | 2021-08-05 | Regenxbio Inc. | Treatment of mucopolysaccharidosis ii with recombinant human iduronate-2-sulfatase (ids) produced by human neural or glial cells |
| 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 |
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| US6118045A (en) | 1995-08-02 | 2000-09-12 | Pharming B.V. | Lysosomal proteins produced in the milk of transgenic animals |
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| TWI543768B (zh) * | 2007-11-30 | 2016-08-01 | 艾伯維生物技術有限責任公司 | 蛋白質調配物及製造其之方法 |
| US7722865B2 (en) | 2008-01-18 | 2010-05-25 | Biomarin Pharmaceutical Inc. | Manufacture of active highly phosphorylated human lysosomal sulfatase enzymes and uses thereof |
| HRP20170698T1 (hr) | 2008-05-07 | 2017-09-22 | Biomarin Pharmaceutical Inc. | Lizosomski usmjereni peptidi i njihove uporabe |
| NZ605874A (en) | 2010-06-25 | 2015-02-27 | Shire Human Genetic Therapies | Methods and compositions for cns delivery of heparan n-sulfatase |
| SMT201600385T1 (it) | 2010-06-25 | 2017-03-08 | Shire Human Genetic Therapies | Trasporto di agenti terapeutici all’snc |
| NZ605873A (en) | 2010-06-25 | 2015-02-27 | Shire Human Genetic Therapies | Methods and compositions for cns delivery of arylsulfatase a |
| EP2588132A4 (en) | 2010-06-25 | 2014-10-15 | Shire Human Genetic Therapies | METHOD AND COMPOSITIONS FOR DELIVERING BETA GALACTOCEREBROSIDASE INTO THE CNS |
| CN103179980B (zh) | 2010-06-25 | 2016-09-28 | 夏尔人类遗传性治疗公司 | 艾杜糖醛酸-2-硫酸酯酶的cns递送的方法和组合物 |
| 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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