DK2723369T3 - Sammensætning og formulering omfattende rekombinant humant iduronat-2-sulfatase og fremgangsmåde til fremstilling deraf - Google Patents
Sammensætning og formulering omfattende rekombinant humant iduronat-2-sulfatase og fremgangsmåde til fremstilling deraf Download PDFInfo
- Publication number
- DK2723369T3 DK2723369T3 DK12803297.6T DK12803297T DK2723369T3 DK 2723369 T3 DK2723369 T3 DK 2723369T3 DK 12803297 T DK12803297 T DK 12803297T DK 2723369 T3 DK2723369 T3 DK 2723369T3
- Authority
- DK
- Denmark
- Prior art keywords
- ids
- composition
- formulation
- cells
- chromatography
- Prior art date
Links
Classifications
-
- 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)
-
- 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
- A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
-
- 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
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
- A61P1/16—Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P11/00—Drugs for disorders of the respiratory system
-
- 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
- A61P19/00—Drugs for skeletal disorders
- A61P19/02—Drugs for skeletal disorders for joint disorders, e.g. arthritis, arthrosis
-
- 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
- A61P19/04—Drugs for skeletal disorders for non-specific disorders of the connective tissue
-
- 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
- A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
- A61P31/12—Antivirals
-
- 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)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Organic Chemistry (AREA)
- General Health & Medical Sciences (AREA)
- Medicinal Chemistry (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Zoology (AREA)
- Genetics & Genomics (AREA)
- Wood Science & Technology (AREA)
- Gastroenterology & Hepatology (AREA)
- Immunology (AREA)
- General Engineering & Computer Science (AREA)
- Biochemistry (AREA)
- Epidemiology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Biomedical Technology (AREA)
- Physical Education & Sports Medicine (AREA)
- Microbiology (AREA)
- Molecular Biology (AREA)
- Biotechnology (AREA)
- Oncology (AREA)
- Communicable Diseases (AREA)
- Pulmonology (AREA)
- Virology (AREA)
- Rheumatology (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Neurosurgery (AREA)
- Neurology (AREA)
- Enzymes And Modification Thereof (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
Claims (14)
1. Iduronat-2-sulfatase (IDS) sammensætning med en aminosyresekvens af SEQ ID NO: 1, hvor en cysteinrest i position 59 i IDS-aminosyresekvensen omdannes til formylglycin (FGly) i et molærforhold på 65% eller derover, fortrinsvis i et molærforhold på 75% eller derover.
2. IDS-sammensætningen ifølge krav 1, hvor IDS med aminosyresekvensen af SEQ ID NO: 1 indeholder mannose-6-phosphat i en mængde på 2,0 til 4,0 mol pr. mol IDS, fortrinsvis i en mængde på 2,5 til 3,Omol pr. mol IDS.
3. Formulering, omfattende IDS-sammensætningen ifølge et hvilket som helst af kravene 1 og 2.
4. Formuleringen ifølge krav 4, yderligere omfattende: en farmaceutisk acceptabel bærer; og eventuelt ét eller flere stof(fer) valgt fra gruppen bestående afen buffer, et kulhydrat, et stabiliseringsmiddel, en antioxidant, et bakteriostatikum, et chelaterende stof, en adjuvans, et suspensionsmiddel, et fortykkelsesmiddel, og et konserveringsmiddel.
5. Fremgangsmåde til fremstilling af en sammensætning ifølge et hvilket som helst af kravene 1 og 2, omfattende: (1) at dyrke en rekombinant cellestamme transformeret med et gen der koder for IDS repræsenteret af SEQ ID NO: 1 og opnå kulturen; og (2) at oprense kulturen gennem anionombytter-kromatografi, hydrofob kromatografi, kationombytter-kromatografi, og affinitetskromatografi, hvor kationombytter-kromatografien udføres under anvendelse af en elueringsbuffer med en pH på 4,0 til 6,0.
6. Fremgangsmåden ifølge krav 5, omfattende: (1) at transformere en værtscelle med en eks press ion s vektor der bærer et IDS-gen for at opnå en rekombinant cellestamme; (2) at dyrke den rekombinante cellestamme i tilstedeværelse af et hydrolysat i et serumfrit medium og opnå kulturen; (3) at oprense IDS fra kulturen gennem anionombytter-kromatografi, hydrofob kromatografi, kationombytter-kromatografi og affinitetskromatografi; og (4) at kombinere det oprensede IDS med en farmaceutisk acceptabel bærer.
7. Fremgangsmåden ifølge et hvilket som helst af kravene 5 og 6, hvor værtscellen er en CHO celle (Chinese hamster ovary cell).
8. Fremgangsmåden ifølge et hvilket som helst af kravene 5 til 7, hvor den hydrofobe kromatografi udføres under anvendelse af en elueringsbuffer med en pH på 5,0 til 7,0.
9. Fremgangsmåden ifølge et hvilket som helst af kravene 5 til 7, hvor affinitetskromatografien udføres under anvendelse af en elueringsbuffer med en pH på 6,0 til 8,0.
10. Fremgangsmåden ifølge et hvilket som helst af kravene 5 til 7, hvor anionombytter-kromatografien udføres under anvendelse afen elueringsbuffer med en pH på 5,5 til 7,5.
11. Fremgangsmåden ifølge et hvilket som helst af kravene 5 til 10, yderligere omfattende inaktivering af virus ved en pH på 3,0 til 4,0.
12. Sammensætning fremstillet ved fremgangsmåden ifølge et hvilket som helst af kravene 5 til 11.
13. Fremgangsmåde til fremstilling afen formulering, omfattende formulering af sammensætningen ifølge kravene 1 eller 2 med en farmaceutisk acceptabel bærer, og eventuelt med ét eller flere stof(fer) valgt fra gruppen bestående af en buffer, et kulhydrat, et stabiliseringsmiddel, en antioxidant, et bakteriostatikum, et chelerende stof, en adjuvans, et suspensionsmiddel, et fortykningsmiddel, og et konserveringsmiddel.
14. Sammensætning ifølge et hvilket som helst af kravene 1, 2 og 12, eller formulering ifølge et hvilket som helst af kravene 3 og 4, til anvendelse i behandlingen af Hunter's syndrom.
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201161500994P | 2011-06-24 | 2011-06-24 | |
| KR1020120012718A KR101158673B1 (ko) | 2011-06-24 | 2012-02-08 | 재조합 인간 이듀로네이트-2-설파타아제를 포함하는 조성물, 제제 및 이의 제조방법 |
| PCT/KR2012/004734 WO2012177020A2 (en) | 2011-06-24 | 2012-06-15 | Composition and formulation comprising recombinant human iduronate-2-sulfatase and preparation method thereof |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| DK2723369T3 true DK2723369T3 (da) | 2016-06-13 |
| DK2723369T4 DK2723369T4 (da) | 2023-07-24 |
Family
ID=46716009
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| DK12803297.6T DK2723369T4 (da) | 2011-06-24 | 2012-06-15 | Sammensætning og formulering omfattende rekombinant humant iduronat-2-sulfatase og fremgangsmåde til fremstilling deraf |
Country Status (28)
| Country | Link |
|---|---|
| US (2) | US9206402B2 (da) |
| EP (1) | EP2723369B2 (da) |
| JP (2) | JP5844459B2 (da) |
| KR (2) | KR101158673B1 (da) |
| CN (1) | CN103635203B (da) |
| AU (2) | AU2012274215B2 (da) |
| BR (1) | BR112013032193A2 (da) |
| CA (1) | CA2839674C (da) |
| CO (1) | CO6862105A2 (da) |
| CY (1) | CY1117861T1 (da) |
| DK (1) | DK2723369T4 (da) |
| EA (1) | EA026499B1 (da) |
| ES (1) | ES2575377T5 (da) |
| FI (1) | FI2723369T4 (da) |
| HK (1) | HK1195731A1 (da) |
| HR (1) | HRP20160490T4 (da) |
| HU (1) | HUE027431T2 (da) |
| IL (1) | IL230120B (da) |
| MX (1) | MX341946B (da) |
| MY (1) | MY166647A (da) |
| PE (1) | PE20140734A1 (da) |
| PL (1) | PL2723369T5 (da) |
| PT (1) | PT2723369E (da) |
| RS (1) | RS54822B2 (da) |
| SI (1) | SI2723369T2 (da) |
| SM (1) | SMT201600194B (da) |
| UA (1) | UA109949C2 (da) |
| WO (1) | WO2012177020A2 (da) |
Families Citing this family (20)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2012101671A1 (en) * | 2011-01-25 | 2012-08-02 | Jcr Pharmaceuticals Co., Ltd. | Method for production of human recombinant iduronate 2-sulfatase |
| KR101158673B1 (ko) * | 2011-06-24 | 2012-07-03 | 주식회사 지씨바이오 | 재조합 인간 이듀로네이트-2-설파타아제를 포함하는 조성물, 제제 및 이의 제조방법 |
| US20160145589A1 (en) | 2011-06-24 | 2016-05-26 | Green Cross Corporation | Composition and formulation comprising recombinant human iduronate-2-sulfatase and preparation method thereof |
| US9150841B2 (en) | 2012-06-29 | 2015-10-06 | Shire Human Genetic Therapies, Inc. | Cells for producing recombinant iduronate-2-sulfatase |
| KR101380740B1 (ko) | 2012-06-29 | 2014-04-11 | 쉐어 휴먼 제네텍 세러피스, 인코포레이티드 | 이듀로네이트-2-설파타제의 정제 |
| US20140004097A1 (en) * | 2012-06-29 | 2014-01-02 | Shire Human Genetic Therapies, Inc. | Method of producing recombinant iduronate-2-sulfatase |
| CN106153746B (zh) * | 2015-03-31 | 2020-11-06 | 三生国健药业（上海）股份有限公司 | IgG2型单抗二硫键配对分析方法 |
| CN106153745B (zh) * | 2015-03-31 | 2020-11-10 | 三生国健药业（上海）股份有限公司 | 抗体蛋白二硫键配对分析方法 |
| CN106153744B (zh) * | 2015-03-31 | 2020-11-10 | 三生国健药业（上海）股份有限公司 | 融合蛋白二硫键配对分析方法 |
| WO2017003270A1 (ko) * | 2015-07-02 | 2017-01-05 | 주식회사 녹십자 | 헌터증후군 치료제 및 치료방법 |
| KR20170004814A (ko) | 2015-07-02 | 2017-01-11 | 주식회사 녹십자 | 헌터증후군 치료제 |
| KR102272399B1 (ko) * | 2015-12-30 | 2021-07-05 | 주식회사 녹십자 | 헌터증후군을 치료하기 위한 방법 및 조성물 |
| WO2017146646A1 (en) * | 2016-02-22 | 2017-08-31 | Agency For Science, Technology And Research | Cell culture medium |
| AU2018253303A1 (en) | 2017-04-14 | 2019-10-31 | Regenxbio Inc. | Treatment of mucopolysaccharidosis II with recombinant human iduronate-2 sulfatase (IDS) produced by human neural or glial cells |
| WO2019022563A2 (ko) * | 2017-07-28 | 2019-01-31 | 한미약품 주식회사 | 이두로네이트 2-설파타제 결합체 |
| KR20200058510A (ko) | 2017-10-02 | 2020-05-27 | 데날리 테라퓨틱스 인크. | 효소 대체 요법 효소를 포함하는 융합 단백질 |
| KR20190136271A (ko) | 2018-05-30 | 2019-12-10 | 주식회사 녹십자 | 대뇌 측뇌실 투여에 의해 헌터증후군을 치료하기 위한 방법 및 조성물 |
| WO2020050947A2 (en) * | 2018-09-05 | 2020-03-12 | Sangamo Therapeutics, Inc. | Enzymatic assays for quantifying therapy in subjects with mucopolysaccharidosis type i or ii |
| CN112805026A (zh) | 2019-02-06 | 2021-05-14 | 桑格摩生物治疗股份有限公司 | 用于治疗i型黏多糖贮积症的方法 |
| WO2022173605A2 (en) | 2021-02-10 | 2022-08-18 | Regenxbio Inc. | Treatment of mucopolysaccharidosis ii with recombinant human iduronate-2-sulfatase (ids) |
Family Cites Families (6)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5932211A (en) | 1991-11-12 | 1999-08-03 | Women's And Children's Hospital | Glycosylation variants of iduronate 2-sulfatase |
| IL155588A0 (en) * | 2003-04-27 | 2003-11-23 | Metabogal Ltd | Methods for expression of enzymatically active recombinant lysosomal enzymes in transgenic plant root cells and vectors used thereby |
| DK2325302T3 (da) * | 2003-02-11 | 2016-03-14 | Shire Human Genetic Therapies | Celler der co-udtrykker et sulfatase og et C-formylglycin-genererende enzym og fremgangsmåder og anvendelser deraf |
| WO2005113765A2 (en) | 2004-05-06 | 2005-12-01 | Biomarin Pharmaceutical Inc. | Methods of activation of sulfatases and methods and compositions of using the same |
| KR101158673B1 (ko) * | 2011-06-24 | 2012-07-03 | 주식회사 지씨바이오 | 재조합 인간 이듀로네이트-2-설파타아제를 포함하는 조성물, 제제 및 이의 제조방법 |
| KR101380740B1 (ko) | 2012-06-29 | 2014-04-11 | 쉐어 휴먼 제네텍 세러피스, 인코포레이티드 | 이듀로네이트-2-설파타제의 정제 |
-
2012
- 2012-02-08 KR KR1020120012718A patent/KR101158673B1/ko active IP Right Grant
- 2012-05-24 KR KR1020120055450A patent/KR101910124B1/ko active IP Right Grant
- 2012-06-15 JP JP2014516901A patent/JP5844459B2/ja active Active
- 2012-06-15 MX MX2013015127A patent/MX341946B/es active IP Right Grant
- 2012-06-15 HU HUE12803297A patent/HUE027431T2/en unknown
- 2012-06-15 DK DK12803297.6T patent/DK2723369T4/da active
- 2012-06-15 FI FIEP12803297.6T patent/FI2723369T4/fi active
- 2012-06-15 US US14/128,918 patent/US9206402B2/en active Active
- 2012-06-15 EP EP12803297.6A patent/EP2723369B2/en active Active
- 2012-06-15 BR BR112013032193A patent/BR112013032193A2/pt not_active Application Discontinuation
- 2012-06-15 WO PCT/KR2012/004734 patent/WO2012177020A2/en active Application Filing
- 2012-06-15 EA EA201490149A patent/EA026499B1/ru not_active IP Right Cessation
- 2012-06-15 PL PL12803297.6T patent/PL2723369T5/pl unknown
- 2012-06-15 HR HRP20160490TT patent/HRP20160490T4/hr unknown
- 2012-06-15 SI SI201230565T patent/SI2723369T2/sl unknown
- 2012-06-15 CA CA2839674A patent/CA2839674C/en active Active
- 2012-06-15 RS RS20160383A patent/RS54822B2/sr unknown
- 2012-06-15 MY MYPI2013004575A patent/MY166647A/en unknown
- 2012-06-15 AU AU2012274215A patent/AU2012274215B2/en active Active
- 2012-06-15 CN CN201280030629.XA patent/CN103635203B/zh active Active
- 2012-06-15 UA UAA201400664A patent/UA109949C2/ru unknown
- 2012-06-15 PE PE2013002881A patent/PE20140734A1/es active IP Right Grant
- 2012-06-15 PT PT128032976T patent/PT2723369E/pt unknown
- 2012-06-15 ES ES12803297T patent/ES2575377T5/es active Active
-
2013
- 2013-12-23 IL IL230120A patent/IL230120B/en active IP Right Grant
-
2014
- 2014-01-21 CO CO14010749A patent/CO6862105A2/es unknown
- 2014-09-09 HK HK14109110.0A patent/HK1195731A1/zh unknown
-
2015
- 2015-07-27 US US14/809,856 patent/US9249397B2/en active Active
- 2015-11-18 JP JP2015225934A patent/JP6000432B2/ja active Active
-
2016
- 2016-03-08 AU AU2016201487A patent/AU2016201487B2/en active Active
- 2016-05-30 CY CY20161100468T patent/CY1117861T1/el unknown
- 2016-06-22 SM SM201600194T patent/SMT201600194B/it unknown
Also Published As
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| DK2723369T3 (da) | Sammensætning og formulering omfattende rekombinant humant iduronat-2-sulfatase og fremgangsmåde til fremstilling deraf | |
| AU2016200460B2 (en) | Cells for producing recombinant iduronate-2-sulfatase | |
| US10174299B2 (en) | Composition and formulation comprising recombinant human iduronate-2-sulfatase and preparation method thereof | |
| NZ620284B2 (en) | Composition and formulation comprising recombinant human iduronate-2-sulfatase and preparation method thereof | |
| US20240167007A1 (en) | Mutated arylsulfatase a with increased stability |