AR119552A2 - Producción de una n-acetilgalactosamina-6-sulfatasa humana activa altamente fosforilada y sus usos - Google Patents
Producción de una n-acetilgalactosamina-6-sulfatasa humana activa altamente fosforilada y sus usosInfo
- Publication number
- AR119552A2 AR119552A2 ARP200102165A ARP200102165A AR119552A2 AR 119552 A2 AR119552 A2 AR 119552A2 AR P200102165 A ARP200102165 A AR P200102165A AR P200102165 A ARP200102165 A AR P200102165A AR 119552 A2 AR119552 A2 AR 119552A2
- Authority
- AR
- Argentina
- Prior art keywords
- acetygalactosamine
- sulfatase
- production
- active human
- highly phosphorylated
- Prior art date
Links
- 238000004519 manufacturing process Methods 0.000 title abstract 2
- 101001066305 Homo sapiens N-acetylgalactosamine-6-sulfatase Proteins 0.000 abstract 3
- 102100031688 N-acetylgalactosamine-6-sulfatase Human genes 0.000 abstract 3
- 208000025797 Mucopolysaccharidosis type 4A Diseases 0.000 abstract 2
- 239000008194 pharmaceutical composition Substances 0.000 abstract 2
- 208000015439 Lysosomal storage disease Diseases 0.000 abstract 1
- 230000007812 deficiency Effects 0.000 abstract 1
- 238000003745 diagnosis Methods 0.000 abstract 1
- 201000010099 disease Diseases 0.000 abstract 1
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 abstract 1
- 238000009472 formulation Methods 0.000 abstract 1
- 238000000034 method Methods 0.000 abstract 1
- 239000000203 mixture Substances 0.000 abstract 1
- 208000012253 mucopolysaccharidosis IVA Diseases 0.000 abstract 1
- 238000011321 prophylaxis Methods 0.000 abstract 1
- 238000000746 purification Methods 0.000 abstract 1
- 208000011580 syndromic disease Diseases 0.000 abstract 1
- 238000011282 treatment Methods 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)
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
- C12N9/14—Hydrolases (3)
- C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
-
- 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
- A61K9/00—Medicinal preparations characterised by special physical form
- A61K9/08—Solutions
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P17/00—Drugs for dermatological disorders
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P19/00—Drugs for skeletal disorders
-
- 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
- A61P25/02—Drugs for disorders of the nervous system for peripheral neuropathies
-
- 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
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K1/00—General methods for the preparation of peptides, i.e. processes for the organic chemical preparation of peptides or proteins of any length
- C07K1/14—Extraction; Separation; Purification
-
- 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)
- C12Y301/06004—N-Acetylgalactosamine-6-sulfatase (3.1.6.4)
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
Landscapes
- Health & Medical Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Engineering & Computer Science (AREA)
- Organic Chemistry (AREA)
- General Health & Medical Sciences (AREA)
- Medicinal Chemistry (AREA)
- Genetics & Genomics (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- Biochemistry (AREA)
- General Engineering & Computer Science (AREA)
- Epidemiology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Biomedical Technology (AREA)
- Molecular Biology (AREA)
- Immunology (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Biotechnology (AREA)
- Microbiology (AREA)
- Gastroenterology & Hepatology (AREA)
- Neurosurgery (AREA)
- Neurology (AREA)
- Analytical Chemistry (AREA)
- Biophysics (AREA)
- Diabetes (AREA)
- Hematology (AREA)
- Physical Education & Sports Medicine (AREA)
- Dermatology (AREA)
- Obesity (AREA)
- Enzymes And Modification Thereof (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
Abstract
La presente proporciona composiciones de la N-aceltigalactosamina-6-sulfatasa (GALNS) humana activa altamente fosforilada y las composiciones y formulaciones farmacéuticas que la incluyen, los métodos para la producción y purificación de GALNS y su uso en el diagnóstico, profilaxis o tratamiento de enfermedades y condiciones, lo que en particular incluye las enfermedades por almacenamiento lisosomal que son ocasionadas por o están asociadas con una deficiencia en la enzima GALNS, por ejemplo, la Mucopolisacaridosis IVa (MPS IVa o síndrome de Morquio A).
Applications Claiming Priority (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US36671410P | 2010-07-22 | 2010-07-22 |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| AR119552A2 true AR119552A2 (es) | 2021-12-29 |
Family
ID=44543776
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| ARP110102649A AR082319A1 (es) | 2010-07-22 | 2011-07-21 | Produccion de una n-acetilgalactosamina-6-sulfatasa humana activa altamente fosforilada y sus usos |
| ARP200102165A AR119552A2 (es) | 2010-07-22 | 2020-07-31 | Producción de una n-acetilgalactosamina-6-sulfatasa humana activa altamente fosforilada y sus usos |
Family Applications Before (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| ARP110102649A AR082319A1 (es) | 2010-07-22 | 2011-07-21 | Produccion de una n-acetilgalactosamina-6-sulfatasa humana activa altamente fosforilada y sus usos |
Country Status (28)
| Country | Link |
|---|---|
| US (3) | US8765437B2 (es) |
| EP (1) | EP2595650B1 (es) |
| JP (2) | JP6030553B2 (es) |
| KR (2) | KR101949906B1 (es) |
| CN (1) | CN103037895B (es) |
| AR (2) | AR082319A1 (es) |
| AU (1) | AU2011280937B2 (es) |
| BR (1) | BR112013001558B1 (es) |
| CA (2) | CA3146151A1 (es) |
| CL (2) | CL2013000219A1 (es) |
| CY (1) | CY1118934T1 (es) |
| DK (1) | DK2595650T3 (es) |
| ES (1) | ES2616263T3 (es) |
| HR (1) | HRP20170245T1 (es) |
| HU (1) | HUE033163T2 (es) |
| IL (2) | IL224125B (es) |
| LT (1) | LT2595650T (es) |
| MX (1) | MX341838B (es) |
| PE (3) | PE20171798A1 (es) |
| PL (1) | PL2595650T3 (es) |
| PT (1) | PT2595650T (es) |
| RS (1) | RS55669B1 (es) |
| RU (1) | RU2607376C2 (es) |
| SG (1) | SG187580A1 (es) |
| SI (1) | SI2595650T1 (es) |
| SM (2) | SMT201700106T1 (es) |
| WO (1) | WO2012012718A2 (es) |
| ZA (1) | ZA201300286B (es) |
Families Citing this family (19)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP2245145B1 (en) * | 2008-01-18 | 2016-12-14 | BioMarin Pharmaceutical Inc. | Manufacture of active highly phosphorylated human lysosomal sulfatase enzymes and uses thereof |
| US8956859B1 (en) | 2010-08-13 | 2015-02-17 | Aviex Technologies Llc | Compositions and methods for determining successful immunization by one or more vaccines |
| US20130202633A1 (en) * | 2012-02-07 | 2013-08-08 | Saint Louis University, A Non-Profit Organization | Determination of immunogenic peptides in lysosomal enzymes and induction of oral tolerance |
| KR101380740B1 (ko) | 2012-06-29 | 2014-04-11 | 쉐어 휴먼 제네텍 세러피스, 인코포레이티드 | 이듀로네이트-2-설파타제의 정제 |
| US11529397B2 (en) | 2018-01-22 | 2022-12-20 | Saint Louis University | Method of treating mucopolysaccharidosis type IVA |
| US9290793B2 (en) | 2013-03-13 | 2016-03-22 | Shire Human Genetic Therapies, Inc. | Method of analyzing the charge profile of heparan N-sulfatase |
| DK2968494T3 (en) | 2013-03-13 | 2019-03-04 | Seattle Genetics Inc | ACTIVATED CARBON FILTERING FOR CLEANING BENZODIAZEPINE ADCs |
| KR20160010526A (ko) * | 2013-05-15 | 2016-01-27 | 리젠츠 오브 더 유니버시티 오브 미네소타 | 중추 신경계로의 아데노-연관 바이러스 매개 유전자 전달 |
| WO2014201220A1 (en) * | 2013-06-12 | 2014-12-18 | The Board Of Trustees Of The University Of Illinois | Methods for detecting prostate cancer |
| WO2015005961A1 (en) | 2013-07-12 | 2015-01-15 | Emd Millipore Corporation | A method of determining virus removal from a sample containing a target protein using activated carbon |
| WO2016007909A2 (en) * | 2014-07-11 | 2016-01-14 | Biostrategies LC | Materials and methods for treating disorders associated with sulfatase enzymes |
| US10472615B2 (en) | 2016-01-21 | 2019-11-12 | Saint Louis University | Reduced immunogenic proteins for lysosomal storage disorders |
| CN109071634A (zh) | 2016-04-26 | 2018-12-21 | R.P.谢勒技术有限责任公司 | 抗体偶联物及其制备和使用方法 |
| US11584777B2 (en) | 2017-08-31 | 2023-02-21 | Green Cross Corporation | Method for purifying a sulfatase protein |
| US12146169B2 (en) * | 2018-05-30 | 2024-11-19 | Esteve Pharmaceuticals, S.A. | Adenoassociated virus vectors for the treatment of mucopolysaccharidoses type IV A |
| RU2763990C2 (ru) * | 2020-02-19 | 2022-01-12 | Акционерное общество "ГЕНЕРИУМ" | Клетка, продуцирующая с высокой эффективностью активный белок арилсульфатазу в, и способ получения этой клетки |
| WO2021216460A1 (en) * | 2020-04-19 | 2021-10-28 | Figene, Llc | Gene modified fibroblasts for therapeutic applications |
| US20250134967A1 (en) * | 2022-02-07 | 2025-05-01 | M6P Therapeutics, Inc. | Compositions comprising acid alpha glucosidase and methods of use thereof |
| TW202403043A (zh) * | 2022-03-18 | 2024-01-16 | 美商健臻公司 | 重組人類酸性鞘磷脂酶醫藥組合物及方法 |
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| US4179337A (en) | 1973-07-20 | 1979-12-18 | Davis Frank F | Non-immunogenic polypeptides |
| US4394448A (en) | 1978-02-24 | 1983-07-19 | Szoka Jr Francis C | Method of inserting DNA into living cells |
| US4391904A (en) | 1979-12-26 | 1983-07-05 | Syva Company | Test strip kits in immunoassays and compositions therein |
| US4554101A (en) | 1981-01-09 | 1985-11-19 | New York Blood Center, Inc. | Identification and preparation of epitopes on antigens and allergens on the basis of hydrophilicity |
| US5186941A (en) | 1983-05-06 | 1993-02-16 | Vestar, Inc. | Vesicle formulation for the controlled release of therapeutic agents |
| JPS62122588A (ja) | 1985-11-22 | 1987-06-03 | Taiyo Fishery Co Ltd | 純コンドロイチナ−ゼの製造法 |
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| PT101031B (pt) | 1991-11-05 | 2002-07-31 | Transkaryotic Therapies Inc | Processo para o fornecimento de proteinas por terapia genetica |
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| RU2195492C2 (ru) * | 2000-09-04 | 2002-12-27 | Институт физиологии Коми научного центра Уральского отделения РАН | Способ получения пектолитического ферментного препарата |
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| JP2006508643A (ja) * | 2002-07-01 | 2006-03-16 | アーキオン ライフ サイエンシーズ エルエルシー ディー／ビー／エー バイオ−テクニカル リソーセズ ディビジョン | グルコサミンおよびｎ−アセチルグルコサミン製造のためのプロセスおよび材料 |
| CN101444621B (zh) | 2003-02-11 | 2013-11-20 | 夏尔人类遗传性治疗公司 | 使用甲酰-甘氨酸生成酶(fge)对多种硫酸酯酶缺乏症和其它病症进行诊断和治疗 |
| JP2007277094A (ja) * | 2004-06-29 | 2007-10-25 | Chemo Sero Therapeut Res Inst | 改変ダニ主要アレルゲン含有医薬組成物 |
| AU2005323106B2 (en) * | 2004-12-22 | 2010-07-29 | Ambrx, Inc. | Methods for expression and purification of recombinant human growth hormone |
| JP2009534390A (ja) * | 2006-04-21 | 2009-09-24 | アムジェン インコーポレイテッド | 生物医薬品製剤のための緩衝剤 |
| US20080231439A1 (en) | 2007-03-22 | 2008-09-25 | Inventec Corporation | Activity Detection Circuit for a Storage Device |
| KR20210049186A (ko) * | 2007-11-30 | 2021-05-04 | 애브비 바이오테크놀로지 리미티드 | 단백질 제형 및 이의 제조방법 |
| EP2245145B1 (en) * | 2008-01-18 | 2016-12-14 | BioMarin Pharmaceutical Inc. | Manufacture of active highly phosphorylated human lysosomal sulfatase enzymes and uses thereof |
| US7722865B2 (en) * | 2008-01-18 | 2010-05-25 | Biomarin Pharmaceutical Inc. | Manufacture of active highly phosphorylated human lysosomal sulfatase enzymes and uses thereof |
| EP2196476A1 (en) * | 2008-12-10 | 2010-06-16 | Novartis Ag | Antibody formulation |
-
2011
- 2011-07-21 AR ARP110102649A patent/AR082319A1/es not_active Application Discontinuation
- 2011-07-22 KR KR1020157007141A patent/KR101949906B1/ko active Active
- 2011-07-22 US US13/188,907 patent/US8765437B2/en active Active
- 2011-07-22 PE PE2017001625A patent/PE20171798A1/es unknown
- 2011-07-22 JP JP2013520881A patent/JP6030553B2/ja active Active
- 2011-07-22 WO PCT/US2011/045011 patent/WO2012012718A2/en not_active Ceased
- 2011-07-22 SM SM20170106T patent/SMT201700106T1/it unknown
- 2011-07-22 KR KR1020137004487A patent/KR101598897B1/ko active Active
- 2011-07-22 DK DK11751689.8T patent/DK2595650T3/en active
- 2011-07-22 EP EP11751689.8A patent/EP2595650B1/en active Active
- 2011-07-22 SG SG2013004841A patent/SG187580A1/en unknown
- 2011-07-22 PE PE2013000110A patent/PE20131140A1/es not_active Application Discontinuation
- 2011-07-22 CN CN201180035900.4A patent/CN103037895B/zh active Active
- 2011-07-22 PL PL11751689T patent/PL2595650T3/pl unknown
- 2011-07-22 MX MX2013000908A patent/MX341838B/es active IP Right Grant
- 2011-07-22 PT PT117516898T patent/PT2595650T/pt unknown
- 2011-07-22 RS RS20170165A patent/RS55669B1/sr unknown
- 2011-07-22 HR HRP20170245TT patent/HRP20170245T1/hr unknown
- 2011-07-22 ES ES11751689.8T patent/ES2616263T3/es active Active
- 2011-07-22 AU AU2011280937A patent/AU2011280937B2/en active Active
- 2011-07-22 CA CA3146151A patent/CA3146151A1/en active Pending
- 2011-07-22 LT LTEP11751689.8T patent/LT2595650T/lt unknown
- 2011-07-22 CA CA2805673A patent/CA2805673C/en active Active
- 2011-07-22 PE PE2017001626A patent/PE20171799A1/es unknown
- 2011-07-22 BR BR112013001558-6A patent/BR112013001558B1/pt active IP Right Grant
- 2011-07-22 SI SI201131104A patent/SI2595650T1/sl unknown
- 2011-07-22 HU HUE11751689A patent/HUE033163T2/en unknown
- 2011-07-22 RU RU2013107765A patent/RU2607376C2/ru active
-
2013
- 2013-01-07 IL IL224125A patent/IL224125B/en active IP Right Grant
- 2013-01-11 ZA ZA2013/00286A patent/ZA201300286B/en unknown
- 2013-01-22 CL CL2013000219A patent/CL2013000219A1/es unknown
-
2014
- 2014-05-19 US US14/281,538 patent/US8940513B2/en active Active
- 2014-12-17 US US14/573,907 patent/US9567572B2/en active Active
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2016
- 2016-07-15 JP JP2016140213A patent/JP2016178950A/ja not_active Withdrawn
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2017
- 2017-02-15 CY CY20171100207T patent/CY1118934T1/el unknown
- 2017-02-16 SM SM201700106T patent/SMT201700106B/it unknown
- 2017-03-02 CL CL2017000498A patent/CL2017000498A1/es unknown
-
2018
- 2018-06-26 IL IL260282A patent/IL260282B/en active IP Right Grant
-
2020
- 2020-07-31 AR ARP200102165A patent/AR119552A2/es unknown
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