CY1125885T1 - Τροποποιημενο πολυπεπτιδιο παραγοντα ix, οι χρησεις του και μια μεθοδος για την παραγωγη του - Google Patents
Τροποποιημενο πολυπεπτιδιο παραγοντα ix, οι χρησεις του και μια μεθοδος για την παραγωγη τουInfo
- Publication number
- CY1125885T1 CY1125885T1 CY20231100138T CY231100138T CY1125885T1 CY 1125885 T1 CY1125885 T1 CY 1125885T1 CY 20231100138 T CY20231100138 T CY 20231100138T CY 231100138 T CY231100138 T CY 231100138T CY 1125885 T1 CY1125885 T1 CY 1125885T1
- Authority
- CY
- Cyprus
- Prior art keywords
- polypeptide
- fix polypeptide
- modified fix
- production
- modified factor
- Prior art date
Links
- 229920001184 polypeptide Polymers 0.000 title abstract 5
- 102000004196 processed proteins & peptides Human genes 0.000 title abstract 5
- 108090000765 processed proteins & peptides Proteins 0.000 title abstract 5
- 229960004222 factor ix Drugs 0.000 title abstract 2
- 238000004519 manufacturing process Methods 0.000 title abstract 2
- DCXYFEDJOCDNAF-UHFFFAOYSA-N Asparagine Chemical group OC(=O)C(N)CC(N)=O DCXYFEDJOCDNAF-UHFFFAOYSA-N 0.000 abstract 1
- 102100022641 Coagulation factor IX Human genes 0.000 abstract 1
- 108010076282 Factor IX Proteins 0.000 abstract 1
- WHUUTDBJXJRKMK-UHFFFAOYSA-N Glutamic acid Chemical group OC(=O)C(N)CCC(O)=O WHUUTDBJXJRKMK-UHFFFAOYSA-N 0.000 abstract 1
- XUJNEKJLAYXESH-REOHCLBHSA-N L-Cysteine Chemical group SC[C@H](N)C(O)=O XUJNEKJLAYXESH-REOHCLBHSA-N 0.000 abstract 1
- DCXYFEDJOCDNAF-REOHCLBHSA-N L-asparagine Chemical group OC(=O)[C@@H](N)CC(N)=O DCXYFEDJOCDNAF-REOHCLBHSA-N 0.000 abstract 1
- CKLJMWTZIZZHCS-REOHCLBHSA-N L-aspartic acid Chemical group OC(=O)[C@@H](N)CC(O)=O CKLJMWTZIZZHCS-REOHCLBHSA-N 0.000 abstract 1
- WHUUTDBJXJRKMK-VKHMYHEASA-N L-glutamic acid Chemical group OC(=O)[C@@H](N)CCC(O)=O WHUUTDBJXJRKMK-VKHMYHEASA-N 0.000 abstract 1
- ZDXPYRJPNDTMRX-VKHMYHEASA-N L-glutamine Chemical group OC(=O)[C@@H](N)CCC(N)=O ZDXPYRJPNDTMRX-VKHMYHEASA-N 0.000 abstract 1
- HNDVDQJCIGZPNO-YFKPBYRVSA-N L-histidine Chemical group OC(=O)[C@@H](N)CC1=CN=CN1 HNDVDQJCIGZPNO-YFKPBYRVSA-N 0.000 abstract 1
- ROHFNLRQFUQHCH-YFKPBYRVSA-N L-leucine Chemical group CC(C)C[C@H](N)C(O)=O ROHFNLRQFUQHCH-YFKPBYRVSA-N 0.000 abstract 1
- KDXKERNSBIXSRK-YFKPBYRVSA-N L-lysine Chemical group NCCCC[C@H](N)C(O)=O KDXKERNSBIXSRK-YFKPBYRVSA-N 0.000 abstract 1
- ROHFNLRQFUQHCH-UHFFFAOYSA-N Leucine Natural products CC(C)CC(N)C(O)=O ROHFNLRQFUQHCH-UHFFFAOYSA-N 0.000 abstract 1
- KDXKERNSBIXSRK-UHFFFAOYSA-N Lysine Chemical group NCCCCC(N)C(O)=O KDXKERNSBIXSRK-UHFFFAOYSA-N 0.000 abstract 1
- 239000004472 Lysine Chemical group 0.000 abstract 1
- 235000009582 asparagine Nutrition 0.000 abstract 1
- 229960001230 asparagine Drugs 0.000 abstract 1
- 235000003704 aspartic acid Nutrition 0.000 abstract 1
- OQFSQFPPLPISGP-UHFFFAOYSA-N beta-carboxyaspartic acid Chemical group OC(=O)C(N)C(C(O)=O)C(O)=O OQFSQFPPLPISGP-UHFFFAOYSA-N 0.000 abstract 1
- 235000018417 cysteine Nutrition 0.000 abstract 1
- XUJNEKJLAYXESH-UHFFFAOYSA-N cysteine Chemical group SCC(N)C(O)=O XUJNEKJLAYXESH-UHFFFAOYSA-N 0.000 abstract 1
- 235000013922 glutamic acid Nutrition 0.000 abstract 1
- 239000004220 glutamic acid Chemical group 0.000 abstract 1
- ZDXPYRJPNDTMRX-UHFFFAOYSA-N glutamine Chemical group OC(=O)C(N)CCC(N)=O ZDXPYRJPNDTMRX-UHFFFAOYSA-N 0.000 abstract 1
- 235000004554 glutamine Nutrition 0.000 abstract 1
- HNDVDQJCIGZPNO-UHFFFAOYSA-N histidine Chemical group OC(=O)C(N)CC1=CN=CN1 HNDVDQJCIGZPNO-UHFFFAOYSA-N 0.000 abstract 1
- 235000014304 histidine Nutrition 0.000 abstract 1
- 235000005772 leucine Nutrition 0.000 abstract 1
- 235000018977 lysine Nutrition 0.000 abstract 1
- 239000002773 nucleotide Substances 0.000 abstract 1
- 125000003729 nucleotide group Chemical group 0.000 abstract 1
- 239000008194 pharmaceutical composition Substances 0.000 abstract 1
- OUYCCCASQSFEME-UHFFFAOYSA-N tyrosine Chemical group OC(=O)C(N)CC1=CC=C(O)C=C1 OUYCCCASQSFEME-UHFFFAOYSA-N 0.000 abstract 1
- 235000002374 tyrosine Nutrition 0.000 abstract 1
- 125000001493 tyrosinyl group Chemical group [H]OC1=C([H])C([H])=C(C([H])=C1[H])C([H])([H])C([H])(N([H])[H])C(*)=O 0.000 abstract 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)
- Genetics & Genomics (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- Medicinal Chemistry (AREA)
- Biomedical Technology (AREA)
- General Engineering & Computer Science (AREA)
- Biochemistry (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Biotechnology (AREA)
- Microbiology (AREA)
- Molecular Biology (AREA)
- Immunology (AREA)
- Gastroenterology & Hepatology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Epidemiology (AREA)
- Hematology (AREA)
- Diabetes (AREA)
- Chemical Kinetics & Catalysis (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)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Preparation Of Compounds By Using Micro-Organisms (AREA)
Abstract
Στο παρόν κείμενο περιλαμβάνονται ένα τροποποιημένο πολυπεπτίδιο FIX (παράγοντας IX) που περιλαμβάνει λευκίνη, κυστεΐνη, ασπαρτικό οξύ, γλουταμικό οξύ, ιστιδίνη, λυσίνη, ασπαραγίνη, γλουταμίνη ή τυροσίνη στη θέση 338 · φαρμακευτικά σκευάσματα που περιέχουν το εν λόγω τροποποιημένο πολυπεπτίδιο FIX · μια νουκλεοτιδική αλληλουχία που κωδικοποιεί το τροποποιημένο πολυπεπτίδιο FIX · και μια μέθοδο για την παραγωγή του τροποποιημένου πολυπεπτιδίου FIX.
Applications Claiming Priority (5)
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 |
PCT/EP2009/061935 WO2010029178A1 (en) | 2008-09-15 | 2009-09-15 | Factor ix polypeptide mutant, its uses and a method for its production |
EP17175191.0A EP3252157A1 (en) | 2008-09-15 | 2009-09-15 | Factor ix polypeptide mutant, its uses and a method for its production |
EP09748260.8A EP2337849B1 (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 |
---|---|
CY1125885T1 true CY1125885T1 (el) | 2024-02-16 |
Family
ID=41397565
Family Applications (2)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
CY20231100138T CY1125885T1 (el) | 2008-09-15 | 2023-03-14 | Τροποποιημενο πολυπεπτιδιο παραγοντα ix, οι χρησεις του και μια μεθοδος για την παραγωγη του |
CY2023016C CY2023016I2 (el) | 2008-09-15 | 2023-07-27 | Τροποποιημενο πολυπεπτιδιο παραγοντα ix, οι χρησεις του και μια μεθοδος για την παραγωγη του |
Family Applications After (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
CY2023016C CY2023016I2 (el) | 2008-09-15 | 2023-07-27 | Τροποποιημενο πολυπεπτιδιο παραγοντα ix, οι χρησεις του και μια μεθοδος για την παραγωγη του |
Country Status (18)
Country | Link |
---|---|
US (11) | US9249405B2 (el) |
EP (5) | EP2337849B1 (el) |
CA (1) | CA2737094C (el) |
CY (2) | CY1125885T1 (el) |
DK (2) | DK3581650T3 (el) |
ES (2) | ES2940323T3 (el) |
FI (2) | FI3581650T3 (el) |
FR (1) | FR23C1028I1 (el) |
HK (1) | HK1244507A1 (el) |
HR (2) | HRP20230259T1 (el) |
HU (2) | HUE061345T2 (el) |
LT (2) | LT3581650T (el) |
NO (1) | NO2023029I1 (el) |
PL (3) | PL2337849T3 (el) |
PT (1) | PT3581650T (el) |
SI (1) | SI3581650T1 (el) |
TR (1) | TR201813067T4 (el) |
WO (1) | WO2010029178A1 (el) |
Families Citing this family (21)
Publication number | Priority date | Publication date | Assignee | Title |
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FI3581650T3 (fi) | 2008-09-15 | 2023-03-23 | Uniqure Biopharma B V | IX-tekijän polypeptidimutantti, sen käyttöjä ja menetelmä sen valmistamiseksi |
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 |
JP6363600B2 (ja) | 2012-07-25 | 2018-07-25 | 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Catalyst Biosciences, Inc. | 修飾第x因子ポリペプチドおよびその使用 |
HRP20231183T1 (hr) | 2013-02-15 | 2024-01-05 | Bioverativ Therapeutics Inc. | Optimizirani gen faktora viii |
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 |
CN108138159A (zh) * | 2015-06-23 | 2018-06-08 | 费城儿童医院 | 经修饰的因子ix、以及用于基因转移到细胞、器官和组织的组合物、方法和用途 |
EP3411478B1 (en) | 2016-02-01 | 2022-06-08 | Bioverativ Therapeutics Inc. | Optimized factor viii genes |
EP3491008A2 (en) | 2016-07-26 | 2019-06-05 | BioMarin Pharmaceutical 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 |
JP2020526203A (ja) | 2017-07-10 | 2020-08-31 | 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 |
KR20210008501A (ko) | 2018-05-09 | 2021-01-22 | 바이오마린 파머수티컬 인크. | 페닐케톤뇨증을 치료하는 방법 |
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 |
JP2021523198A (ja) | 2018-05-14 | 2021-09-02 | ビオマリン プハルマセウトイカル インコーポレイテッド | 幼若対象におけるaavベクターの安定的発現 |
US10842885B2 (en) | 2018-08-20 | 2020-11-24 | Ucl Business Ltd | Factor IX encoding nucleotides |
BR112021002998A2 (pt) * | 2018-08-20 | 2021-05-11 | Ucl Business Ltd | polinucleotídeos, partícula viral e composição |
UY38389A (es) * | 2018-09-27 | 2020-04-30 | Sigilon Therapeutics Inc | Dispositivos implantables para terapia celular y métodos relacionados |
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 |
US20240293516A1 (en) * | 2021-07-01 | 2024-09-05 | CSL Behring Lengnau AG | Factor ix subcutaneous administration with enhanced safety |
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US5171569A (en) | 1985-03-15 | 1992-12-15 | National Research Development Corporation | Factor IX preparations uncontaminated by plasma components or pox virus |
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FI3581650T3 (fi) | 2008-09-15 | 2023-03-23 | Uniqure Biopharma B V | IX-tekijän polypeptidimutantti, sen käyttöjä ja menetelmä sen valmistamiseksi |
AU2010266065B2 (en) | 2009-06-25 | 2016-01-14 | The University Of North Carolina At Chapel Hill | Chimeric Factor VII molecules |
PE20121643A1 (es) | 2009-07-31 | 2012-11-25 | Bayer Healthcare Llc | Polipeptidos del factor ix modificados y usos de los mismos |
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 |
EP3513802B1 (en) | 2012-10-26 | 2023-11-29 | Vrije Universiteit Brussel | Vector 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 |
AU2016232146B2 (en) | 2015-03-17 | 2021-11-04 | Vrije Universiteit Brussel | Optimized liver-specific expression systems for FVIII and FIX |
CN108138159A (zh) | 2015-06-23 | 2018-06-08 | 费城儿童医院 | 经修饰的因子ix、以及用于基因转移到细胞、器官和组织的组合物、方法和用途 |
UA126016C2 (uk) | 2015-08-03 | 2022-08-03 | Біовератів Терапеутікс Інк. | Злитий білок фактора іх |
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