PL2337849T3 - Zmutowany polipeptyd czynnika ix, jego zastosowania i sposób jego otrzymywania - Google Patents
Zmutowany polipeptyd czynnika ix, jego zastosowania i sposób jego otrzymywaniaInfo
- Publication number
- PL2337849T3 PL2337849T3 PL09748260T PL09748260T PL2337849T3 PL 2337849 T3 PL2337849 T3 PL 2337849T3 PL 09748260 T PL09748260 T PL 09748260T PL 09748260 T PL09748260 T PL 09748260T PL 2337849 T3 PL2337849 T3 PL 2337849T3
- Authority
- PL
- Poland
- Prior art keywords
- factor
- production
- polypeptide mutant
- mutant
- polypeptide
- Prior art date
Links
- 229960004222 factor ix Drugs 0.000 title 1
- 229920001184 polypeptide Polymers 0.000 title 1
- 102000004196 processed proteins & peptides Human genes 0.000 title 1
- 108090000765 processed proteins & peptides Proteins 0.000 title 1
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)
- C12N9/48—Hydrolases (3) acting on peptide bonds (3.4)
- C12N9/50—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25)
- C12N9/64—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue
- C12N9/6421—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue from mammals
- C12N9/6424—Serine endopeptidases (3.4.21)
- C12N9/644—Coagulation factor IXa (3.4.21.22)
-
- 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)
- A61K38/48—Hydrolases (3) acting on peptide bonds (3.4)
- A61K38/482—Serine endopeptidases (3.4.21)
- A61K38/4846—Factor VII (3.4.21.21); Factor IX (3.4.21.22); Factor Xa (3.4.21.6); Factor XI (3.4.21.27); Factor XII (3.4.21.38)
-
- 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
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y304/00—Hydrolases acting on peptide bonds, i.e. peptidases (3.4)
- C12Y304/21—Serine endopeptidases (3.4.21)
- C12Y304/21022—Coagulation factor IXa (3.4.21.22)
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)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- Genetics & Genomics (AREA)
- Medicinal Chemistry (AREA)
- Biomedical Technology (AREA)
- Biochemistry (AREA)
- General Engineering & Computer Science (AREA)
- Pharmacology & Pharmacy (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Animal Behavior & Ethology (AREA)
- Microbiology (AREA)
- Molecular Biology (AREA)
- Biotechnology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Gastroenterology & Hepatology (AREA)
- Immunology (AREA)
- Epidemiology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Hematology (AREA)
- Diabetes (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Peptides Or Proteins (AREA)
- Preparation Of Compounds By Using Micro-Organisms (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
Applications Claiming Priority (4)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| ITBO2008A000564A IT1394177B1 (it) | 2008-09-15 | 2008-09-15 | Polipeptide fattore ix modificato, sue utilizzazioni e metodo per la sua produzione |
| ITBO2009A000275A IT1395980B1 (it) | 2009-05-06 | 2009-05-06 | Polipeptide fattore ix modificato, sue utilizzazioni e metodo per la sua produzione |
| EP09748260.8A EP2337849B1 (en) | 2008-09-15 | 2009-09-15 | Factor ix polypeptide mutant, its uses and a method for its production |
| PCT/EP2009/061935 WO2010029178A1 (en) | 2008-09-15 | 2009-09-15 | Factor ix polypeptide mutant, its uses and a method for its production |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| PL2337849T3 true PL2337849T3 (pl) | 2018-11-30 |
Family
ID=41397565
Family Applications (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| PL09748260T PL2337849T3 (pl) | 2008-09-15 | 2009-09-15 | Zmutowany polipeptyd czynnika ix, jego zastosowania i sposób jego otrzymywania |
| PL19174517.3T PL3581650T3 (pl) | 2008-09-15 | 2009-09-15 | Zmutowany polipeptyd czynnika ix, jego zastosowania i sposób jego wytwarzania |
| PL21200005.3T PL4032979T3 (pl) | 2008-09-15 | 2009-09-15 | Mutant polipeptydu czynnika ix, jego zastosowania i sposób jego otrzymywania |
Family Applications After (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| PL19174517.3T PL3581650T3 (pl) | 2008-09-15 | 2009-09-15 | Zmutowany polipeptyd czynnika ix, jego zastosowania i sposób jego wytwarzania |
| PL21200005.3T PL4032979T3 (pl) | 2008-09-15 | 2009-09-15 | Mutant polipeptydu czynnika ix, jego zastosowania i sposób jego otrzymywania |
Country Status (20)
| Country | Link |
|---|---|
| US (13) | USRE50288E1 (pl) |
| EP (5) | EP3252157A1 (pl) |
| CA (1) | CA2737094C (pl) |
| CY (3) | CY1125885T1 (pl) |
| DK (2) | DK2337849T3 (pl) |
| ES (3) | ES2687038T3 (pl) |
| FI (5) | FI3581650T3 (pl) |
| FR (4) | FR23C1028I1 (pl) |
| HK (1) | HK1244507A1 (pl) |
| HR (2) | HRP20181442T1 (pl) |
| HU (6) | HUE067487T2 (pl) |
| LT (5) | LT3581650T (pl) |
| LU (1) | LUC00311I2 (pl) |
| NO (2) | NO2023029I1 (pl) |
| PL (3) | PL2337849T3 (pl) |
| PT (1) | PT3581650T (pl) |
| SI (1) | SI3581650T1 (pl) |
| SM (2) | SMT202300084T1 (pl) |
| TR (1) | TR201813067T4 (pl) |
| WO (1) | WO2010029178A1 (pl) |
Families Citing this family (26)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| TR201813067T4 (tr) | 2008-09-15 | 2018-09-21 | Uniqure Biopharma B V | Faktör IX polipeptit mutantı, bunun kullanımları ve bunu üretmeye yönelik bir yöntem. |
| TWI557135B (zh) | 2010-11-03 | 2016-11-11 | 介控生化科技公司 | 經修飾之第九因子多胜肽及其用途 |
| WO2014018120A1 (en) | 2012-07-25 | 2014-01-30 | Catalyst Biosciences, Inc. | Modified factor x polypeptides and uses thereof |
| WO2014127215A1 (en) | 2013-02-15 | 2014-08-21 | Biogen Idec Ma Inc. | Optimized factor viii gene |
| US11008561B2 (en) | 2014-06-30 | 2021-05-18 | Bioverativ Therapeutics Inc. | Optimized factor IX gene |
| GB201420139D0 (en) | 2014-11-12 | 2014-12-24 | Ucl Business Plc | Factor IX gene therapy |
| MX390848B (es) | 2015-06-23 | 2025-03-21 | Childrens Hospital Philadelphia | Factor ix modificado, y composiciones, metodos y usos para la transferencia de genes a celulas, organos y tejidos. |
| CA3012695A1 (en) | 2016-02-01 | 2017-08-10 | Bioverativ Therapeutics Inc. | Optimized factor viii genes |
| IL305149A (en) | 2016-07-26 | 2023-10-01 | Biomarin Pharm Inc | Novel adeno-associated virus capsid proteins |
| CN106497949A (zh) * | 2016-10-14 | 2017-03-15 | 上海交通大学医学院附属瑞金医院 | 一种高活性凝血因子ix突变体、重组蛋白与融合蛋白的制备与应用 |
| CN110945127B (zh) | 2017-05-22 | 2024-07-12 | 武田药品工业株式会社 | 用于血友病b的基因疗法的编码具有增加的表达的重组fix的病毒载体 |
| JP7764129B2 (ja) | 2017-07-10 | 2025-11-05 | ユニキュアー アイピー ビー.ブイ. | ヒトにおけるaav遺伝子療法のための手段及び方法 |
| US11491212B1 (en) | 2017-09-27 | 2022-11-08 | Catalyst Biosciences, Inc. | Subcutaneous administration of modified factor IX polypeptides and treatment of hemophilia B |
| TW202016314A (zh) | 2018-05-09 | 2020-05-01 | 美商拜奧馬林製藥公司 | 治療苯丙酮尿症之方法 |
| TW202016298A (zh) | 2018-05-14 | 2020-05-01 | 美商拜奧馬林製藥公司 | Aav 載體於幼年受試者中之穩定表現 |
| TW202005978A (zh) | 2018-05-14 | 2020-02-01 | 美商拜奧馬林製藥公司 | 新穎肝靶向腺相關病毒載體 |
| JP2021533799A (ja) * | 2018-08-20 | 2021-12-09 | ユーシーエル ビジネス リミテッド | 第ix因子をコードするヌクレオチド |
| GB201813528D0 (en) | 2018-08-20 | 2018-10-03 | Ucl Business Plc | Factor IX encoding nucleotides |
| US10842885B2 (en) | 2018-08-20 | 2020-11-24 | Ucl Business Ltd | Factor IX encoding nucleotides |
| UY38389A (es) * | 2018-09-27 | 2020-04-30 | Sigilon Therapeutics Inc | Dispositivos implantables para terapia celular y métodos relacionados |
| AU2019360270B2 (en) | 2018-10-18 | 2025-08-07 | Intellia Therapeutics, Inc. | Compositions and methods for expressing factor IX. |
| KR20220097891A (ko) | 2019-09-30 | 2022-07-08 | 바이오버라티브 테라퓨틱스 인크. | 렌티바이러스 벡터 제형 |
| WO2021154414A2 (en) | 2020-01-29 | 2021-08-05 | Catalyst Biosciences, Inc. | Gene therapy for hemophilia b with a chimeric aav capsid vector encoding modified factor ix polypeptides |
| MX2023000156A (es) | 2020-06-24 | 2023-02-16 | Bioverativ Therapeutics Inc | Metodos para la eliminacion de factor viii libre de preparaciones de vectores lentivirales modificados para expresar dicha proteina. |
| US20240293516A1 (en) * | 2021-07-01 | 2024-09-05 | CSL Behring Lengnau AG | Factor ix subcutaneous administration with enhanced safety |
| WO2025022005A1 (en) * | 2023-07-26 | 2025-01-30 | Le Quellec Sandra | Intranasal administration of factor ix polypeptides |
Family Cites Families (74)
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| EP0107278B1 (en) | 1982-08-04 | 1989-11-15 | National Research Development Corporation | Molecular cloning of the gene for human anti-haemophilic factor ix |
| US5171569A (en) | 1985-03-15 | 1992-12-15 | National Research Development Corporation | Factor IX preparations uncontaminated by plasma components or pox virus |
| US4994371A (en) | 1987-08-28 | 1991-02-19 | Davie Earl W | DNA preparation of Christmas factor and use of DNA sequences |
| US5583107A (en) | 1990-09-04 | 1996-12-10 | Cor Therapeutics, Inc. | Agents affecting thrombosis and hemostasis |
| US5223408A (en) | 1991-07-11 | 1993-06-29 | Genentech, Inc. | Method for making variant secreted proteins with altered properties |
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| US6315995B1 (en) | 1996-09-27 | 2001-11-13 | The Trustees Of Columbia University In The City Of New York | Methods for treating an ischemic disorder and improving stroke outcome |
| US6344596B1 (en) | 1997-02-14 | 2002-02-05 | American Red Cross | Expression of active human factor IX in mammary tissue and of milk non human transgenic mammals |
| US6531298B2 (en) | 1997-07-21 | 2003-03-11 | The University Of North Carolina At Chapel Hill | Factor IX antihemophilic factor with increased clotting activity |
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| US6610906B1 (en) | 1999-06-09 | 2003-08-26 | The Regents Of The University Of Michigan | Nucleotide sequences for gene regulation and methods of use thereof |
| US7109170B2 (en) | 1999-06-16 | 2006-09-19 | Saint Louis University | Region of factor IXa protease domain that interacts with factor VIIIa and methods therefor |
| AU2001249389A1 (en) | 2000-03-22 | 2001-10-03 | The Children's Hospital Of Philadelphia | Modified blood clotting factors and methods of use |
| AU2001269961A1 (en) | 2000-07-06 | 2002-01-21 | Guilford Pharmaceuticals | Improved polymers and polymerization processes |
| WO2002040544A2 (en) | 2000-11-14 | 2002-05-23 | Board Of Regents, University Of Texas Systems | Mutant human factor ix with an increased resistance to inhibition by heparin |
| US20040133930A1 (en) | 2002-03-11 | 2004-07-08 | Cooper Julian D. | Production of high levels of transgenic factor ix without gene rescue, and its therapeutic uses |
| WO2002096454A1 (en) | 2001-05-31 | 2002-12-05 | D. Collen Research Foundation Vzw | Recombinant molecules with reduced immunogenicity, methods and intermediates for obtaining them and their use in pharmaceutical compositions and diagnostic tools |
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| WO2006018204A1 (en) | 2004-08-17 | 2006-02-23 | Zlb Behring Gmbh | Modified vitamin k dependent polypeptides |
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| WO2006031226A1 (en) | 2004-09-13 | 2006-03-23 | Saint Louis University | Region of factor ixa protease domain that interacts with factor viiia and methods therefor |
| WO2006036502A2 (en) | 2004-09-22 | 2006-04-06 | St. Jude Children's Research Hospital | Improved expression of factor ix in gene therapy vectors |
| US7998930B2 (en) | 2004-11-04 | 2011-08-16 | Hanall Biopharma Co., Ltd. | Modified growth hormones |
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| WO2007101681A1 (en) | 2006-03-07 | 2007-09-13 | Baxter International Inc | Highly phosphorylated and sulfated recombinant factor ix |
| US20080038224A1 (en) | 2006-03-28 | 2008-02-14 | Thierry Guyon | Modified interferon-beta (IFN-beta) polypeptides |
| US7588772B2 (en) | 2006-03-30 | 2009-09-15 | Board Of Trustees Of The Leland Stamford Junior University | AAV capsid library and AAV capsid proteins |
| AU2007234698B2 (en) | 2006-04-07 | 2013-08-01 | The Board Of Regents Of The University Of Texas System | Methods and compositions related to adenoassociated virus-phage particles |
| KR20090013816A (ko) | 2006-05-24 | 2009-02-05 | 노보 노르디스크 헬스 케어 악티엔게젤샤프트 | 연장된 생체내 반감기를 갖는 인자 ⅸ 유사체 |
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| ES2535877T3 (es) | 2006-06-19 | 2015-05-18 | Asklepios Biopharmaceutical, Inc. | Genes de Factor VIII y Factor IX modificados y vectores para la terapia génica |
| US7700734B2 (en) | 2007-01-09 | 2010-04-20 | Shu-Wha Lin | Recombinant human factor IX and use thereof |
| AU2008209985A1 (en) * | 2007-02-01 | 2008-08-07 | Baxter Healthcare S.A. | Improved fix-mutant proteins for hemophilia B treatment |
| WO2008119815A1 (en) | 2007-04-02 | 2008-10-09 | Novo Nordisk A/S | Subcutaneous administration of coagulation factor ix |
| WO2008127654A2 (en) | 2007-04-11 | 2008-10-23 | The University Of North Carolina At Chapel Hill | Methods and compositions for intra-articular coagulation proteins |
| BRPI0705943A2 (pt) | 2007-04-19 | 2008-12-09 | Nautilus Biotech | formulaÇÕes de dosagem oral de polipeptÍdeos resistentes À protease e usos das mesmas para tratamento |
| US20080260820A1 (en) | 2007-04-19 | 2008-10-23 | Gilles Borrelly | Oral dosage formulations of protease-resistant polypeptides |
| US20090030072A1 (en) | 2007-07-03 | 2009-01-29 | Sucampo Ag | Pharmaceutical combination of opioid and prostaglandin compound |
| CN102026653B (zh) | 2007-10-15 | 2014-06-18 | 北卡罗来纳-查佩尔山大学 | 半衰期延长的人因子ⅸ变体 |
| SG189790A1 (en) * | 2008-04-16 | 2013-05-31 | Bayer Healthcare Llc | Modified factor ix polypeptides and uses thereof |
| JP2011517950A (ja) | 2008-04-16 | 2011-06-23 | バイエル・ヘルスケア・エルエルシー | 第ix因子の部位特異的修飾 |
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| EP2149603A1 (en) | 2008-07-28 | 2010-02-03 | DRK-Blutspendedienst Baden-Württemberg-Hessen gGmbH | Factor IX variants with clotting activity in absence of their cofactor and their use for treating bleeding disorders |
| TR201813067T4 (tr) | 2008-09-15 | 2018-09-21 | Uniqure Biopharma B V | Faktör IX polipeptit mutantı, bunun kullanımları ve bunu üretmeye yönelik bir yöntem. |
| JP5909180B2 (ja) | 2009-06-25 | 2016-04-26 | ザ・ユニヴァーシティ・オヴ・ノース・キャロライナ・アト・チャペル・ヒル | キメラ第vii因子分子 |
| JP2013500726A (ja) | 2009-07-31 | 2013-01-10 | バイエル・ヘルスケア・エルエルシー | 改変第ix因子ポリペプチドおよびその使用 |
| TWI557135B (zh) | 2010-11-03 | 2016-11-11 | 介控生化科技公司 | 經修飾之第九因子多胜肽及其用途 |
| US10398787B2 (en) | 2012-10-26 | 2019-09-03 | Vrije Universiteit Brussel | Vectors for liver-directed gene therapy of hemophilia and methods and use thereof |
| EP2881463A1 (en) | 2013-12-09 | 2015-06-10 | DRK-Blutspendedienst Baden-Württemberg-Hessen gGmbH | Factor IX variants with clotting activity in absence of their cofactor and/or with increased F.IX clotting activity and their use for treating bleeding disorders |
| US11008561B2 (en) | 2014-06-30 | 2021-05-18 | Bioverativ Therapeutics Inc. | Optimized factor IX gene |
| US11007280B2 (en) | 2015-03-17 | 2021-05-18 | Vrije Universiteit Brussel | Optimized liver-specific expression systems for FVIII and FIX |
| EP3283126B1 (en) | 2015-04-16 | 2019-11-06 | Emory University | Recombinant promoters and vectors for protein expression in liver and use thereof |
| MX390848B (es) | 2015-06-23 | 2025-03-21 | Childrens Hospital Philadelphia | Factor ix modificado, y composiciones, metodos y usos para la transferencia de genes a celulas, organos y tejidos. |
| EA201890423A1 (ru) | 2015-08-03 | 2018-07-31 | Биовератив Терапьютикс Инк. | Слитые белки фактора ix, способы их получения и применения |
| US11191847B2 (en) | 2016-04-15 | 2021-12-07 | The Trustees Of The University Of Pennsylvania | Gene therapy for treating hemophilia B |
| MX2018012537A (es) | 2016-04-15 | 2019-02-25 | Univ Pennsylvania | Terapia de genes para tratar hemofilia a. |
| JP7764129B2 (ja) | 2017-07-10 | 2025-11-05 | ユニキュアー アイピー ビー.ブイ. | ヒトにおけるaav遺伝子療法のための手段及び方法 |
| US20210228738A1 (en) | 2017-07-17 | 2021-07-29 | INSERM (Institut National de la Santé et de la Recherche Médicale) | Compositions and methods for increasing or enhancing transduction of gene therapy vectors and for removing or reducing immunoglobulins |
-
2009
- 2009-09-15 TR TR2018/13067T patent/TR201813067T4/tr unknown
- 2009-09-15 ES ES09748260.8T patent/ES2687038T3/es active Active
- 2009-09-15 US US18/153,894 patent/USRE50288E1/en active Active
- 2009-09-15 EP EP17175191.0A patent/EP3252157A1/en not_active Ceased
- 2009-09-15 ES ES21200005T patent/ES2985035T3/es active Active
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