MX365396B - Polipéptidos modificados del factor ix y usos de los mismos. - Google Patents
Polipéptidos modificados del factor ix y usos de los mismos.Info
- Publication number
- MX365396B MX365396B MX2016010700A MX2016010700A MX365396B MX 365396 B MX365396 B MX 365396B MX 2016010700 A MX2016010700 A MX 2016010700A MX 2016010700 A MX2016010700 A MX 2016010700A MX 365396 B MX365396 B MX 365396B
- Authority
- MX
- Mexico
- Prior art keywords
- polypeptides
- modified
- fix
- modified factor
- activities
- Prior art date
Links
Classifications
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- 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
-
- 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)
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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
- A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
- A61K38/36—Blood coagulation or fibrinolysis factors
-
- 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
- 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
- C12N15/01—Preparation of mutants without inserting foreign genetic material therein; Screening processes therefor
-
- 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)
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Genetics & Genomics (AREA)
- General Health & Medical Sciences (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- Medicinal Chemistry (AREA)
- Biomedical Technology (AREA)
- General Engineering & Computer Science (AREA)
- Biochemistry (AREA)
- Gastroenterology & Hepatology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Biotechnology (AREA)
- Molecular Biology (AREA)
- Epidemiology (AREA)
- Immunology (AREA)
- Microbiology (AREA)
- Hematology (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Toxicology (AREA)
- Plant Pathology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Diabetes (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Peptides Or Proteins (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicinal Preparation (AREA)
- Preparation Of Compounds By Using Micro-Organisms (AREA)
- Enzymes And Modification Thereof (AREA)
Abstract
La presente invención se refiere a una molécula de ácido nucleico que codifica un polipéptido del factor IX (FIC) modificado, caracterizado porque comprende una sustitución de aminoácidos correspondiente a T343R o T343K en un polipéptido de FIX sin modificar, en donde: los residuos de aminoácidos correspondientes se identifican por alineación del polipéptido de FIX sin modificar con el polipéptido de SEQ ID NO: 3; el polipéptido FIX sin modificar comprende una secuencia de aminoácidos establecida en cualquiera de SEQ ID NOS: 2, 3, 20, 0325, o una secuencia de residuos de aminoácidos que tiene al menos 95% de identidad de secuencia con el polipéptido de FIX establecido en cualquiera de SEQ ID NOS: 2, 3, 20 O 325: o es un fragmento catalíticamente activo del mismo; el polipéptido de FIX modificado, cuando está en una forma activa, exhibe una o ambas de actividad catalítica aumenta y de actividad procoagulante aumentada en comparación el polipéptido de aminoácidos correspondientes a F342I/T343R/Y345T.
Applications Claiming Priority (2)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US45629810P | 2010-11-03 | 2010-11-03 | |
PCT/US2011/059233 WO2012061654A1 (en) | 2010-11-03 | 2011-11-03 | Modified factor ix polypeptides and uses thereof |
Publications (1)
Publication Number | Publication Date |
---|---|
MX365396B true MX365396B (es) | 2019-05-31 |
Family
ID=44936595
Family Applications (2)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
MX2016010700A MX365396B (es) | 2010-11-03 | 2011-11-03 | Polipéptidos modificados del factor ix y usos de los mismos. |
MX2013004828A MX345314B (es) | 2010-11-03 | 2011-11-03 | Polipeptidos modificados del factor ix y usos de los mismos. |
Family Applications After (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
MX2013004828A MX345314B (es) | 2010-11-03 | 2011-11-03 | Polipeptidos modificados del factor ix y usos de los mismos. |
Country Status (17)
Country | Link |
---|---|
US (4) | US8778870B2 (es) |
EP (2) | EP3663399B1 (es) |
JP (2) | JP2013545457A (es) |
KR (2) | KR101841906B1 (es) |
CN (1) | CN103282491B (es) |
AU (1) | AU2011323295B2 (es) |
BR (1) | BR112013010834B1 (es) |
CA (2) | CA2816325C (es) |
DK (2) | DK2635677T3 (es) |
ES (1) | ES2782078T3 (es) |
MX (2) | MX365396B (es) |
MY (2) | MY192475A (es) |
NZ (2) | NZ707439A (es) |
SG (2) | SG190089A1 (es) |
TW (2) | TWI595004B (es) |
WO (1) | WO2012061654A1 (es) |
ZA (1) | ZA201303134B (es) |
Families Citing this family (32)
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NZ590187A (en) * | 2002-10-02 | 2012-07-27 | Catalyst Biosciences Inc | Methods of generating and screening for proteases with altered specificity |
JP5629423B2 (ja) | 2004-04-12 | 2014-11-19 | カタリスト バイオサイエンシーズ, インコーポレイテッド | 野生型及び変異型mt−sp1による、vegf及びvegf受容体の切断 |
WO2007047995A2 (en) | 2005-10-21 | 2007-04-26 | Catalyst Biosciences, Inc. | Modified proteases that inhibit complement activation |
KR20150033726A (ko) | 2006-07-05 | 2015-04-01 | 카탈리스트 바이오사이언시즈, 인코포레이티드 | 프로테아제 스크리닝 방법 및 이에 의해 확인된 프로테아제 |
KR20100016462A (ko) * | 2007-04-13 | 2010-02-12 | 카탈리스트 바이오사이언시즈, 인코포레이티드 | 변형 제vii 인자 폴리펩타이드 및 이의 용도 |
TWI538916B (zh) | 2008-04-11 | 2016-06-21 | 介控生化科技公司 | 經修飾的因子vii多肽和其用途 |
FI3581650T3 (fi) | 2008-09-15 | 2023-03-23 | Uniqure Biopharma B V | IX-tekijän polypeptidimutantti, sen käyttöjä ja menetelmä sen valmistamiseksi |
TWI595004B (zh) | 2010-11-03 | 2017-08-11 | 介控生化科技公司 | 經修飾之第九因子多胜肽及其用途 |
IN2015DN01404A (es) | 2012-07-25 | 2015-07-03 | Catalyst Biosciences Inc | |
EP3513802B1 (en) * | 2012-10-26 | 2023-11-29 | Vrije Universiteit Brussel | Vector for liver-directed gene therapy of hemophilia and methods and use thereof |
JP6454643B2 (ja) | 2012-10-26 | 2019-01-16 | フリーイェ・ユニヴェルシテイト・ブリュッセルVrije Universieit Brussel | 血友病の肝臓指向性遺伝子治療のためのベクター並びにその方法及び使用 |
EP2922871B1 (en) * | 2012-11-20 | 2019-06-19 | The University of North Carolina At Chapel Hill | Methods and compositions for modified factor ix proteins |
WO2014099850A1 (en) * | 2012-12-19 | 2014-06-26 | Merck Sharp & Dohme Corp. | FACTOR IXa 318R/A MUTANT |
CN103060366A (zh) * | 2012-12-25 | 2013-04-24 | 武汉科技大学 | 人凝血因子ⅸ突变体毕赤酵母表达载体及构建方法和应用 |
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 |
CA2944909A1 (en) * | 2014-04-04 | 2015-10-08 | Bloodworks | Routine laboratory and point-of-care (poc) testing for hemostasis |
GB201420139D0 (en) | 2014-11-12 | 2014-12-24 | Ucl Business Plc | Factor IX gene therapy |
EA201890423A1 (ru) | 2015-08-03 | 2018-07-31 | Биовератив Терапьютикс Инк. | Слитые белки фактора ix, способы их получения и применения |
AU2016302335B2 (en) * | 2015-08-06 | 2022-08-04 | The Trustees Of The University Of Pennsylvania | GLP-1 and use thereof in compositions for treating metabolic diseases |
US11708570B2 (en) | 2016-07-27 | 2023-07-25 | The Children's Hospital Of Philadelphia | Compositions and methods for modulating Factor IX function |
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 |
WO2018199214A1 (ja) * | 2017-04-27 | 2018-11-01 | 中外製薬株式会社 | 薬物動態が改善された血液凝固第ix因子 |
KR102386890B1 (ko) | 2017-05-22 | 2022-04-15 | 다케다 야쿠힌 고교 가부시키가이샤 | B형 혈우병 유전자 요법을 위한 증가된 발현을 가지는 재조합 fix 변이체를 암호화하는 바이러스 벡터 |
US11491212B1 (en) * | 2017-09-27 | 2022-11-08 | Catalyst Biosciences, Inc. | Subcutaneous administration of modified factor IX polypeptides and treatment of hemophilia B |
US10842885B2 (en) | 2018-08-20 | 2020-11-24 | Ucl Business Ltd | Factor IX encoding nucleotides |
GB201817810D0 (en) * | 2018-10-31 | 2018-12-19 | Ucl Business Plc | Modified factor IX polypeptides |
US11596671B2 (en) | 2019-02-01 | 2023-03-07 | Takeda Pharmaceutical Company Limited | Gene therapy of hemophilia B using viral vectors encoding recombinant fix variants with increased expression |
CN113573726A (zh) * | 2019-03-19 | 2021-10-29 | 康诺贝林伦瑙有限公司 | 因子ix变体及其在治疗中的用途 |
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WO2023224992A2 (en) * | 2022-05-16 | 2023-11-23 | The Board Of Trustees Of The Leland Stanford Junior University | Targeted integration at alpha-globin locus in human hematopoietic stem and progenitor cells |
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