FIC20230026I1 - Etranakogeenidetsaparvoveekki (adenoassosioitu virusvektori, joka sisältää ihmisen hyytymistekijä IX:n geenin koodonioptimoitua Padua-johdannaista B-hemofilian hoitoon) - Google Patents
Etranakogeenidetsaparvoveekki (adenoassosioitu virusvektori, joka sisältää ihmisen hyytymistekijä IX:n geenin koodonioptimoitua Padua-johdannaista B-hemofilian hoitoon)Info
- Publication number
- FIC20230026I1 FIC20230026I1 FIC20230026C FIC20230026C FIC20230026I1 FI C20230026 I1 FIC20230026 I1 FI C20230026I1 FI C20230026 C FIC20230026 C FI C20230026C FI C20230026 C FIC20230026 C FI C20230026C FI C20230026 I1 FIC20230026 I1 FI C20230026I1
- Authority
- FI
- Finland
- Prior art keywords
- etranacogen
- dezaparvovec
- padua
- hemophilia
- adeno
- 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)
- C12N9/48—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)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Microbiology (AREA)
- Molecular Biology (AREA)
- Biotechnology (AREA)
- Epidemiology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Immunology (AREA)
- Gastroenterology & Hepatology (AREA)
- Diabetes (AREA)
- Hematology (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)
- Preparation Of Compounds By Using Micro-Organisms (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
Applications Claiming Priority (2)
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 |
Publications (1)
Publication Number | Publication Date |
---|---|
FIC20230026I1 true FIC20230026I1 (fi) | 2023-07-25 |
Family
ID=41397565
Family Applications (4)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
FIEP19174517.3T FI3581650T3 (fi) | 2008-09-15 | 2009-09-15 | IX-tekijän polypeptidimutantti, sen käyttöjä ja menetelmä sen valmistamiseksi |
FIC20230026C FIC20230026I1 (fi) | 2008-09-15 | 2023-07-25 | Etranakogeenidetsaparvoveekki (adenoassosioitu virusvektori, joka sisältää ihmisen hyytymistekijä IX:n geenin koodonioptimoitua Padua-johdannaista B-hemofilian hoitoon) |
FIC20240037C FIC20240037I1 (fi) | 2008-09-15 | 2024-10-29 | Fidanakogeenielaparvoveekki |
FIC20240036C FIC20240036I1 (fi) | 2008-09-15 | 2024-10-29 | Geeniterapia, joka ilmentää ihmisen hyytymistekijä IX:n muunnosta R338L |
Family Applications Before (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
FIEP19174517.3T FI3581650T3 (fi) | 2008-09-15 | 2009-09-15 | IX-tekijän polypeptidimutantti, sen käyttöjä ja menetelmä sen valmistamiseksi |
Family Applications After (2)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
FIC20240037C FIC20240037I1 (fi) | 2008-09-15 | 2024-10-29 | Fidanakogeenielaparvoveekki |
FIC20240036C FIC20240036I1 (fi) | 2008-09-15 | 2024-10-29 | Geeniterapia, joka ilmentää ihmisen hyytymistekijä IX:n muunnosta R338L |
Country Status (18)
Country | Link |
---|---|
US (11) | US9249405B2 (fi) |
EP (5) | EP3252157A1 (fi) |
CA (1) | CA2737094C (fi) |
CY (2) | CY1125885T1 (fi) |
DK (2) | DK3581650T3 (fi) |
ES (3) | ES2940323T3 (fi) |
FI (4) | FI3581650T3 (fi) |
FR (1) | FR23C1028I1 (fi) |
HK (1) | HK1244507A1 (fi) |
HR (2) | HRP20230259T1 (fi) |
HU (3) | HUE067487T2 (fi) |
LT (2) | LT3581650T (fi) |
NO (1) | NO2023029I1 (fi) |
PL (3) | PL2337849T3 (fi) |
PT (1) | PT3581650T (fi) |
SI (1) | SI3581650T1 (fi) |
TR (1) | TR201813067T4 (fi) |
WO (1) | WO2010029178A1 (fi) |
Families Citing this family (21)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
EP3252157A1 (en) | 2008-09-15 | 2017-12-06 | Paolo Simioni | Factor ix polypeptide mutant, its uses and a method for its production |
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 |
WO2014018120A1 (en) | 2012-07-25 | 2014-01-30 | Catalyst Biosciences, Inc. | Modified factor x polypeptides and uses thereof |
PL3889173T3 (pl) | 2013-02-15 | 2023-12-11 | Bioverativ Therapeutics Inc. | Zoptymalizowany gen czynnika viii |
WO2016004113A1 (en) | 2014-06-30 | 2016-01-07 | Biogen Ma Inc. | Optimized factor ix gene |
GB201420139D0 (en) | 2014-11-12 | 2014-12-24 | Ucl Business Plc | Factor IX gene therapy |
KR102415896B1 (ko) | 2015-06-23 | 2022-06-30 | 더 칠드런스 호스피탈 오브 필라델피아 | 변형된 인자 ix, 및 세포, 기관 및 조직으로 유전자를 전달하기 위한 조성물, 방법 및 용도 |
PL3411478T3 (pl) | 2016-02-01 | 2022-10-03 | Bioverativ Therapeutics Inc. | Geny zoptymalizowanego czynnika VIII |
IL263801B2 (en) | 2016-07-26 | 2024-01-01 | Biomarin Pharm Inc | Novel adeno-associated virus capsid proteins |
CN116732066A (zh) * | 2016-10-14 | 2023-09-12 | 四川至善唯新生物科技有限公司 | 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 |
CN118581121A (zh) | 2017-05-22 | 2024-09-03 | 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 |
EA202090260A1 (ru) | 2017-07-10 | 2020-05-08 | ЮНИКЬЮРЕ АйПи Б.В. | Средства и способы для генотерапии aav у человека |
US11491212B1 (en) | 2017-09-27 | 2022-11-08 | Catalyst Biosciences, Inc. | Subcutaneous administration of modified factor IX polypeptides and treatment of hemophilia B |
SG11202010830WA (en) | 2018-05-09 | 2020-11-27 | Biomarin Pharm Inc | Methods of treating phenylketonuria |
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 |
AU2019270972A1 (en) | 2018-05-14 | 2020-12-03 | Biomarin Pharmaceutical Inc. | Stable expression of AAV vectors in juvenile subjects |
AU2019323747A1 (en) * | 2018-08-20 | 2021-03-04 | Ucl Business Ltd | 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 |
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 |
WO2023275024A1 (en) * | 2021-07-01 | 2023-01-05 | 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 |
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 |
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WO1999003496A1 (en) * | 1997-07-21 | 1999-01-28 | The University Of North Carolina At Chapel Hill | Factor ix antihemophilic factor with increased clotting activity |
US6200560B1 (en) * | 1998-10-20 | 2001-03-13 | Avigen, Inc. | Adeno-associated virus vectors for expression of factor VIII by target cells |
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 |
WO2001070763A1 (en) | 2000-03-22 | 2001-09-27 | The Children's Hospital Of Philadelphia | Modified blood clotting factors and methods of use |
AU2002226028A1 (en) | 2000-11-14 | 2002-05-27 | Board Of Regents, Unversity 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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US20040009151A1 (en) | 2002-04-04 | 2004-01-15 | Kay Mark A. | Methods for delivering recombinant adeno-associated virus virions to the liver of a mammal |
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US20070280906A1 (en) * | 2006-06-03 | 2007-12-06 | Ognjen Petras | Method to generate mirrored adenoassociated viral vectors |
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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 |
US20080260820A1 (en) | 2007-04-19 | 2008-10-23 | Gilles Borrelly | Oral dosage formulations of protease-resistant polypeptides |
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 |
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AU2009244633A1 (en) * | 2008-04-16 | 2009-11-12 | Bayer Healthcare Llc | Modified Factor IX polypeptides and uses thereof |
WO2009140015A2 (en) | 2008-04-16 | 2009-11-19 | Bayer Healthcare Llc | Site-directed modification of factor ix |
EP2811017A2 (en) | 2008-04-21 | 2014-12-10 | Novo Nordisk Health Care AG | Hyperglycosylated human coagulation factor IX |
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 |
EP3252157A1 (en) | 2008-09-15 | 2017-12-06 | Paolo Simioni | Factor ix polypeptide mutant, its uses and a method for its production |
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 |
BR112012002072A2 (pt) | 2009-07-31 | 2016-11-08 | Bayer Healthcare Llc | polipeptídeos de fator ix modificados e usos dos mesmos |
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 |
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 |
EP3270944B1 (en) | 2015-03-17 | 2019-10-23 | Vrije Universiteit Brussel | Optimized liver-specific expression systems for fviii and fix |
KR102415896B1 (ko) | 2015-06-23 | 2022-06-30 | 더 칠드런스 호스피탈 오브 필라델피아 | 변형된 인자 ix, 및 세포, 기관 및 조직으로 유전자를 전달하기 위한 조성물, 방법 및 용도 |
EA201890423A1 (ru) | 2015-08-03 | 2018-07-31 | Биовератив Терапьютикс Инк. | Слитые белки фактора ix, способы их получения и применения |
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2009
- 2009-09-15 EP EP17175191.0A patent/EP3252157A1/en not_active Ceased
- 2009-09-15 PT PT191745173T patent/PT3581650T/pt unknown
- 2009-09-15 ES ES19174517T patent/ES2940323T3/es active Active
- 2009-09-15 EP EP21200005.3A patent/EP4032979B1/en active Active
- 2009-09-15 PL PL09748260T patent/PL2337849T3/pl unknown
- 2009-09-15 ES ES21200005T patent/ES2985035T3/es active Active
- 2009-09-15 DK DK19174517.3T patent/DK3581650T3/da active
- 2009-09-15 HU HUE21200005A patent/HUE067487T2/hu unknown
- 2009-09-15 EP EP22214327.3A patent/EP4219547A3/en active Pending
- 2009-09-15 EP EP09748260.8A patent/EP2337849B1/en not_active Revoked
- 2009-09-15 HU HUE19174517A patent/HUE061345T2/hu unknown
- 2009-09-15 EP EP19174517.3A patent/EP3581650B1/en active Active
- 2009-09-15 TR TR2018/13067T patent/TR201813067T4/tr unknown
- 2009-09-15 PL PL21200005.3T patent/PL4032979T3/pl unknown
- 2009-09-15 DK DK09748260.8T patent/DK2337849T3/en active
- 2009-09-15 US US13/063,898 patent/US9249405B2/en active Active
- 2009-09-15 PL PL19174517.3T patent/PL3581650T3/pl unknown
- 2009-09-15 FI FIEP19174517.3T patent/FI3581650T3/fi active
- 2009-09-15 HR HRP20230259TT patent/HRP20230259T1/hr unknown
- 2009-09-15 WO PCT/EP2009/061935 patent/WO2010029178A1/en active Application Filing
- 2009-09-15 SI SI200932179T patent/SI3581650T1/sl unknown
- 2009-09-15 LT LTEP19174517.3T patent/LT3581650T/lt unknown
- 2009-09-15 ES ES09748260.8T patent/ES2687038T3/es active Active
- 2009-09-15 CA CA2737094A patent/CA2737094C/en active Active
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2015
- 2015-12-29 US US14/981,981 patent/US20160122740A1/en not_active Abandoned
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2017
- 2017-07-14 US US15/650,070 patent/US9982248B2/en active Active
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2018
- 2018-03-21 HK HK18103924.5A patent/HK1244507A1/zh unknown
- 2018-05-25 US US15/989,665 patent/US10465180B2/en active Active
- 2018-09-10 HR HRP20181442TT patent/HRP20181442T1/hr unknown
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2019
- 2019-10-01 US US16/589,851 patent/US20200231958A1/en not_active Abandoned
- 2019-12-09 US US16/707,414 patent/US20200190498A1/en not_active Abandoned
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2020
- 2020-02-11 US US16/787,456 patent/US20200199564A1/en not_active Abandoned
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2021
- 2021-03-26 US US17/213,897 patent/US20210214704A1/en active Pending
- 2021-03-26 US US17/213,848 patent/US20210238573A1/en active Pending
- 2021-09-10 US US17/447,372 patent/US20220002702A1/en not_active Abandoned
- 2021-12-08 US US17/643,326 patent/US20220090042A1/en not_active Abandoned
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2023
- 2023-03-14 CY CY20231100138T patent/CY1125885T1/el unknown
- 2023-07-25 FI FIC20230026C patent/FIC20230026I1/fi unknown
- 2023-07-25 HU HUS2300024C patent/HUS2300024I1/hu unknown
- 2023-07-26 FR FR23C1028C patent/FR23C1028I1/fr active Active
- 2023-07-27 CY CY2023016C patent/CY2023016I2/el unknown
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- 2023-07-31 NO NO2023029C patent/NO2023029I1/no unknown
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2024
- 2024-10-29 FI FIC20240037C patent/FIC20240037I1/fi unknown
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