RU2012122402A - Содержащие фактор фон виллебранда (vwf) препараты и способы, наборы и применения, связанные с ними - Google Patents
Содержащие фактор фон виллебранда (vwf) препараты и способы, наборы и применения, связанные с ними Download PDFInfo
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- RU2012122402A RU2012122402A RU2012122402/10A RU2012122402A RU2012122402A RU 2012122402 A RU2012122402 A RU 2012122402A RU 2012122402/10 A RU2012122402/10 A RU 2012122402/10A RU 2012122402 A RU2012122402 A RU 2012122402A RU 2012122402 A RU2012122402 A RU 2012122402A
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- 238000000034 method Methods 0.000 title claims abstract 12
- 239000003814 drug Substances 0.000 title 1
- 229940079593 drug Drugs 0.000 title 1
- 229920001184 polypeptide Polymers 0.000 claims abstract 35
- 102000004196 processed proteins & peptides Human genes 0.000 claims abstract 35
- 108090000765 processed proteins & peptides Proteins 0.000 claims abstract 35
- 125000003275 alpha amino acid group Chemical group 0.000 claims abstract 12
- 102000004169 proteins and genes Human genes 0.000 claims abstract 12
- 108090000623 proteins and genes Proteins 0.000 claims abstract 12
- 102100026735 Coagulation factor VIII Human genes 0.000 claims abstract 10
- 101000911390 Homo sapiens Coagulation factor VIII Proteins 0.000 claims abstract 10
- 239000002773 nucleotide Substances 0.000 claims abstract 8
- 125000003729 nucleotide group Chemical group 0.000 claims abstract 8
- 239000000203 mixture Substances 0.000 claims abstract 7
- 108060003951 Immunoglobulin Proteins 0.000 claims abstract 3
- 102000018358 immunoglobulin Human genes 0.000 claims abstract 3
- FWMNVWWHGCHHJJ-SKKKGAJSSA-N 4-amino-1-[(2r)-6-amino-2-[[(2r)-2-[[(2r)-2-[[(2r)-2-amino-3-phenylpropanoyl]amino]-3-phenylpropanoyl]amino]-4-methylpentanoyl]amino]hexanoyl]piperidine-4-carboxylic acid Chemical compound C([C@H](C(=O)N[C@H](CC(C)C)C(=O)N[C@H](CCCCN)C(=O)N1CCC(N)(CC1)C(O)=O)NC(=O)[C@H](N)CC=1C=CC=CC=1)C1=CC=CC=C1 FWMNVWWHGCHHJJ-SKKKGAJSSA-N 0.000 claims abstract 2
- 239000000539 dimer Substances 0.000 claims abstract 2
- 239000013604 expression vector Substances 0.000 claims abstract 2
- 208000031220 Hemophilia Diseases 0.000 claims 1
- 208000009292 Hemophilia A Diseases 0.000 claims 1
- 101710120037 Toxin CcdB Proteins 0.000 claims 1
- 230000015572 biosynthetic process Effects 0.000 claims 1
- 239000008280 blood Substances 0.000 claims 1
- 210000004369 blood Anatomy 0.000 claims 1
- 239000003937 drug carrier Substances 0.000 claims 1
- 230000002708 enhancing effect Effects 0.000 claims 1
- 230000002035 prolonged effect Effects 0.000 claims 1
- 238000003259 recombinant expression Methods 0.000 claims 1
- 239000011347 resin Substances 0.000 claims 1
- 229920005989 resin Polymers 0.000 claims 1
Classifications
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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K19/00—Hybrid peptides, i.e. peptides covalently bound to nucleic acids, or non-covalently bound protein-protein complexes
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
- C07K14/745—Blood coagulation or fibrinolysis factors
- C07K14/755—Factors VIII, e.g. factor VIII C (AHF), factor VIII Ag (VWF)
-
- 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
- A61K40/00—Cellular immunotherapy
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P7/00—Drugs for disorders of the blood or the extracellular fluid
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P7/00—Drugs for disorders of the blood or the extracellular fluid
- A61P7/02—Antithrombotic agents; Anticoagulants; Platelet aggregation inhibitors
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P7/00—Drugs for disorders of the blood or the extracellular fluid
- A61P7/04—Antihaemorrhagics; Procoagulants; Haemostatic agents; Antifibrinolytic agents
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- 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
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- C12N15/62—DNA sequences coding for fusion proteins
-
- 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
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
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- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Organic Chemistry (AREA)
- Engineering & Computer Science (AREA)
- General Health & Medical Sciences (AREA)
- Genetics & Genomics (AREA)
- Medicinal Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Zoology (AREA)
- Hematology (AREA)
- Molecular Biology (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Biochemistry (AREA)
- Biophysics (AREA)
- Pharmacology & Pharmacy (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Diabetes (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Biomedical Technology (AREA)
- Biotechnology (AREA)
- Wood Science & Technology (AREA)
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- Gastroenterology & Hepatology (AREA)
- Toxicology (AREA)
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- Epidemiology (AREA)
- Immunology (AREA)
- Peptides Or Proteins (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Preparation Of Compounds By Using Micro-Organisms (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
Abstract
1. Полипептид, содержащий первую аминокислотную последовательность, присутствующую в полипептиде vWF, и вторую аминокислотную последовательность, гетерологичную относительно первой, где этот полипептид способен связывать FVIII, при условии, что этот полипептид лишен домена vWF A1, A2, A3, D4, B1, B2, B3, C1, C2, CK или их комбинации.2. Полипептид по п.1, где эта первая аминокислотная последовательность представлена в SEQ ID NO:1, SEQ ID NO:2, SEQ ID NO:3, SEQ ID NO:4, SEQ ID NO:5, SEQ ID NO:6, SEQ ID NO:7, SEQ ID NO:8, SEQ ID NO:9, SEQ ID NO:10, SEQ ID NO:11, SEQ ID NO:12, SEQ ID NO:13, SEQ ID NO:14, SEQ ID NO:15, SEQ ID NO:33, SEQ ID NO:34 или SEQ ID NO:35.3. Полипептид по п.1, где этот полипептид содержит последовательность, представленную в SEQ ID NO:17, SEQ ID NO:18, SEQ ID NO:19, SEQ ID NO:20, SEQ ID NO:21, SEQ ID NO:22, SEQ ID NO:36, SEQ ID NO:38 или SEQ ID NO:39.4. Полипептид по п.1, где этот полипептид способен образовывать димер.5. Полипептид по п.1, где эта вторая аминокислотная последовательность соответствует Fc иммуноглобулина.6. Полипептид по п.1, где эта вторая аминокислотная последовательность имеет последовательность, представленную в SEQ ID NO:16.7. Композиция, содержащая полипептид по п.1.8. Белковый комплекс, содержащий полипептид по п.1 и FVIII.9. Композиция, содержащая белковый комплекс по п.8.10. Нуклеотидная последовательность, кодирующая полипептид по п.1.11. Нуклеотидная последовательность по п.10, где эта нуклеотидная последовательность представлена в SEQ ID NO:23, SEQ ID NO:24, SEQ ID NO:25, SEQ ID NO:26, SEQ ID NO:27, SEQ ID NO:28, SEQ ID NO:37, SEQ ID NO:42 или SEQ ID NO:43.12. Экспрессирующий вектор, содержащий нуклеотидную последовательность по п. 10.13. Клетка, экспрессирующая полипептид по п.1.14. Клетка, экспрессирующая белковый комплекс по п.8.15. Способ получения белкового комплекса по п.8, предусматривающий контактирование этого полипепт
Claims (25)
1. Полипептид, содержащий первую аминокислотную последовательность, присутствующую в полипептиде vWF, и вторую аминокислотную последовательность, гетерологичную относительно первой, где этот полипептид способен связывать FVIII, при условии, что этот полипептид лишен домена vWF A1, A2, A3, D4, B1, B2, B3, C1, C2, CK или их комбинации.
2. Полипептид по п.1, где эта первая аминокислотная последовательность представлена в SEQ ID NO:1, SEQ ID NO:2, SEQ ID NO:3, SEQ ID NO:4, SEQ ID NO:5, SEQ ID NO:6, SEQ ID NO:7, SEQ ID NO:8, SEQ ID NO:9, SEQ ID NO:10, SEQ ID NO:11, SEQ ID NO:12, SEQ ID NO:13, SEQ ID NO:14, SEQ ID NO:15, SEQ ID NO:33, SEQ ID NO:34 или SEQ ID NO:35.
3. Полипептид по п.1, где этот полипептид содержит последовательность, представленную в SEQ ID NO:17, SEQ ID NO:18, SEQ ID NO:19, SEQ ID NO:20, SEQ ID NO:21, SEQ ID NO:22, SEQ ID NO:36, SEQ ID NO:38 или SEQ ID NO:39.
4. Полипептид по п.1, где этот полипептид способен образовывать димер.
5. Полипептид по п.1, где эта вторая аминокислотная последовательность соответствует Fc иммуноглобулина.
6. Полипептид по п.1, где эта вторая аминокислотная последовательность имеет последовательность, представленную в SEQ ID NO:16.
7. Композиция, содержащая полипептид по п.1.
8. Белковый комплекс, содержащий полипептид по п.1 и FVIII.
9. Композиция, содержащая белковый комплекс по п.8.
10. Нуклеотидная последовательность, кодирующая полипептид по п.1.
11. Нуклеотидная последовательность по п.10, где эта нуклеотидная последовательность представлена в SEQ ID NO:23, SEQ ID NO:24, SEQ ID NO:25, SEQ ID NO:26, SEQ ID NO:27, SEQ ID NO:28, SEQ ID NO:37, SEQ ID NO:42 или SEQ ID NO:43.
12. Экспрессирующий вектор, содержащий нуклеотидную последовательность по п. 10.
13. Клетка, экспрессирующая полипептид по п.1.
14. Клетка, экспрессирующая белковый комплекс по п.8.
15. Способ получения белкового комплекса по п.8, предусматривающий контактирование этого полипептида с FVIII.
16. Способ по п.15, где это контактирование предусматривает рекомбинантную экспрессию этого полипептида в клетке, которая экспрессирует FVIII.
17. Способ получения FVIII, предусматривающий: контактирование FVIII с полипептидом по п.1 для образования белкового комплекса, содержащего этот полипептид и FVIII.
18. Способ по п.17, дополнительно предусматривающий прикрепление этого комплекса к смоле, содержащей партнер связывания, имеющий аффинность в отношении домена, определяемого второй аминокислотной последовательностью.
19. Способ по п.18, где эта аминокислотная последовательность соответствует Fc иммуноглобулина.
20. Способ по п.19, где этим партнером связывания является белок А или белок G.
21. Способ усиления фармакокинетического свойства FVIII в плазме, предусматривающий введение субъекту композиции, содержащей белковый комплекс по п.8.
22. Способ по п.21, где этим свойством является пролонгированный период полувыведения из плазмы.
23. Композиция, содержащая белковый комплекс по п.8 и фармацевтически приемлемый носитель.
24. Способ лечения связанного с кровью состояния, предусматривающий введение композиции по п.23 субъекту.
25. Способ по п.24, где этим состоянием является гемофилия.
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US26114509P | 2009-11-13 | 2009-11-13 | |
| US61/261,145 | 2009-11-13 | ||
| PCT/US2010/056496 WO2011060242A2 (en) | 2009-11-13 | 2010-11-12 | Von willebrand factor (vwf)-containing preparations, and methods, kits, and uses related thereto |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| RU2012122402A true RU2012122402A (ru) | 2013-12-20 |
| RU2579977C2 RU2579977C2 (ru) | 2016-04-10 |
Family
ID=43992414
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| RU2012122402/10A RU2579977C2 (ru) | 2009-11-13 | 2010-11-12 | СОДЕРЖАЩИЕ ФАКТОР ФОН ВИЛЛЕБРАНДА (vWF) ПРЕПАРАТЫ И СПОСОБЫ, НАБОРЫ И ПРИМЕНЕНИЯ, СВЯЗАННЫЕ С НИМИ |
Country Status (19)
| Country | Link |
|---|---|
| US (1) | US8759293B2 (ru) |
| EP (1) | EP2499165B1 (ru) |
| JP (1) | JP5876416B2 (ru) |
| KR (1) | KR101808751B1 (ru) |
| CN (1) | CN102648212B (ru) |
| AU (1) | AU2010319425B2 (ru) |
| BR (1) | BR112012011740A2 (ru) |
| CA (1) | CA2780542C (ru) |
| CL (1) | CL2012001258A1 (ru) |
| ES (1) | ES2600879T3 (ru) |
| HU (1) | HUE029454T2 (ru) |
| IL (1) | IL219679A (ru) |
| MX (1) | MX2012005527A (ru) |
| NZ (1) | NZ600278A (ru) |
| PL (1) | PL2499165T3 (ru) |
| PT (1) | PT2499165T (ru) |
| RU (1) | RU2579977C2 (ru) |
| WO (1) | WO2011060242A2 (ru) |
| ZA (1) | ZA201203795B (ru) |
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| JPS6263355A (ja) * | 1985-09-13 | 1987-03-20 | Hitachi Ltd | バツテリバツクアツプramのチエツク方法 |
| CN103897063B (zh) | 2007-06-01 | 2017-04-12 | 马里兰大学巴尔的摩分校 | 免疫球蛋白恒定区Fc受体结合剂 |
| CN102741275B (zh) | 2009-08-24 | 2016-08-03 | 阿穆尼克斯运营公司 | 凝血因子ix组合物及其制备和使用方法 |
| KR101808751B1 (ko) | 2009-11-13 | 2017-12-13 | 그리폴스 테라퓨틱스 인코포레이티드 | 폰 빌레브란트 인자(vWF)-함유 제제, 및 그와 관련된 방법, 키트 및 용도 |
| EP3543257A1 (en) | 2010-07-28 | 2019-09-25 | Gliknik Inc. | Fusion proteins of natural human protein fragments to create orderly multimerized immunoglobulin fc compositions |
| AU2012324020A1 (en) | 2011-12-30 | 2013-07-18 | Grifols, S.A. | Method for purifying Factor VIII |
| EP2804623B1 (en) * | 2012-01-12 | 2019-08-07 | Bioverativ Therapeutics Inc. | Chimeric factor viii polypeptides and uses thereof |
| SMT201800586T1 (it) | 2012-01-12 | 2019-01-11 | Bioverativ Therapeutics Inc | Riduzione dell'immunogenicita' contro il fattore viii negli individui che si sottopongono alla terapia con fattore viii |
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| JP2013510581A (ja) | 2013-03-28 |
| RU2579977C2 (ru) | 2016-04-10 |
| CA2780542A1 (en) | 2011-05-19 |
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| NZ600278A (en) | 2014-04-30 |
| BR112012011740A2 (pt) | 2018-03-27 |
| US20120289468A1 (en) | 2012-11-15 |
| WO2011060242A2 (en) | 2011-05-19 |
| WO2011060242A3 (en) | 2011-10-13 |
| HK1170244A1 (zh) | 2013-02-22 |
| CN102648212B (zh) | 2014-12-03 |
| KR101808751B1 (ko) | 2017-12-13 |
| CL2012001258A1 (es) | 2012-09-14 |
| KR20120113214A (ko) | 2012-10-12 |
| HUE029454T2 (en) | 2017-03-28 |
| AU2010319425A1 (en) | 2012-06-14 |
| IL219679A (en) | 2016-11-30 |
| CA2780542C (en) | 2020-03-24 |
| CN102648212A (zh) | 2012-08-22 |
| EP2499165B1 (en) | 2016-09-14 |
| ES2600879T3 (es) | 2017-02-13 |
| JP5876416B2 (ja) | 2016-03-02 |
| US8759293B2 (en) | 2014-06-24 |
| ZA201203795B (en) | 2014-10-29 |
| IL219679A0 (en) | 2012-07-31 |
| EP2499165A2 (en) | 2012-09-19 |
| PL2499165T3 (pl) | 2017-04-28 |
| AU2010319425B2 (en) | 2015-07-02 |
| PT2499165T (pt) | 2016-10-28 |
| EP2499165A4 (en) | 2013-09-11 |
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