JP2020010717A5 - - Google Patents

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JP2020010717A5
JP2020010717A5 JP2019176968A JP2019176968A JP2020010717A5 JP 2020010717 A5 JP2020010717 A5 JP 2020010717A5 JP 2019176968 A JP2019176968 A JP 2019176968A JP 2019176968 A JP2019176968 A JP 2019176968A JP 2020010717 A5 JP2020010717 A5 JP 2020010717A5
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JP2019176968A
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JP2020010717A (ja
JP7000394B2 (ja
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Priority claimed from EP13196284.7A external-priority patent/EP2881463A1/en
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JP2019176968A 2013-12-09 2019-09-27 補因子の非存在下で凝固活性を有する、及び/又は、第ix因子凝固活性が増加した第ix因子変異体、並びに、出血性疾患を処置するためのその使用 Active JP7000394B2 (ja)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
EP13196284.7 2013-12-09
EP13196284.7A EP2881463A1 (en) 2013-12-09 2013-12-09 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

Related Parent Applications (1)

Application Number Title Priority Date Filing Date
JP2016537997A Division JP6655536B2 (ja) 2013-12-09 2014-12-03 補因子の非存在下で凝固活性を有する、及び/又は、第ix因子凝固活性が増加した第ix因子変異体、並びに、出血性疾患を処置するためのその使用

Publications (3)

Publication Number Publication Date
JP2020010717A JP2020010717A (ja) 2020-01-23
JP2020010717A5 true JP2020010717A5 (https=) 2020-04-02
JP7000394B2 JP7000394B2 (ja) 2022-02-04

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ID=49765821

Family Applications (2)

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JP2016537997A Active JP6655536B2 (ja) 2013-12-09 2014-12-03 補因子の非存在下で凝固活性を有する、及び/又は、第ix因子凝固活性が増加した第ix因子変異体、並びに、出血性疾患を処置するためのその使用
JP2019176968A Active JP7000394B2 (ja) 2013-12-09 2019-09-27 補因子の非存在下で凝固活性を有する、及び/又は、第ix因子凝固活性が増加した第ix因子変異体、並びに、出血性疾患を処置するためのその使用

Family Applications Before (1)

Application Number Title Priority Date Filing Date
JP2016537997A Active JP6655536B2 (ja) 2013-12-09 2014-12-03 補因子の非存在下で凝固活性を有する、及び/又は、第ix因子凝固活性が増加した第ix因子変異体、並びに、出血性疾患を処置するためのその使用

Country Status (4)

Country Link
US (3) US10000748B2 (https=)
EP (2) EP2881463A1 (https=)
JP (2) JP6655536B2 (https=)
WO (1) WO2015086406A2 (https=)

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* Cited by examiner, † Cited by third party
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.
WO2014127215A1 (en) 2013-02-15 2014-08-21 Biogen Idec Ma Inc. Optimized factor viii gene
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
GB201420139D0 (en) 2014-11-12 2014-12-24 Ucl Business Plc Factor IX gene therapy
CA3012695A1 (en) 2016-02-01 2017-08-10 Bioverativ Therapeutics Inc. Optimized factor viii genes
KR102625416B1 (ko) 2016-05-16 2024-01-15 다케다 파머수티컬 컴패니 리미티드 항-인자 ix 파두아 항체
EP3538659A4 (en) * 2016-11-09 2020-07-15 Engene, Inc. INTESTINAL EXPRESSION OF THE SCHEDULED DEATH LIGAND 1
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
EP3687580A1 (en) 2017-09-27 2020-08-05 Sigilon Therapeutics, Inc. Methods, compositions, and implantable elements comprising active cells
BR112020020084A2 (pt) 2018-04-04 2021-01-05 Sigilon Therapeutics, Inc. Partícula, preparação de uma pluralidade de partículas, método de preparação de uma partícula, e, composição de partículas.
US20210145889A1 (en) 2018-04-04 2021-05-20 Sigilon Therapeutics, Inc. Methods, compositions, and implantable elements comprising stem cells
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
WO2020104480A1 (en) * 2018-11-19 2020-05-28 Uniqure Biopharma B.V. Adeno-associated virus vectors for expressing fviii mimetics and uses thereof
CN110684798A (zh) * 2019-09-03 2020-01-14 深圳新诺微环生物科技有限公司 肌肉靶向的微环dna基因治疗
KR20220097891A (ko) 2019-09-30 2022-07-08 바이오버라티브 테라퓨틱스 인크. 렌티바이러스 벡터 제형
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.
JP6883899B1 (ja) * 2020-12-07 2021-06-09 株式会社血栓トランスレーショナルリサーチラボ 血液凝固検査試薬、および血液凝固検査方法
US20230149563A1 (en) 2021-10-27 2023-05-18 Regeneron Pharmaceuticals, Inc. Compositions and methods for expressing factor ix for hemophilia b therapy
WO2025160324A2 (en) 2024-01-26 2025-07-31 Regeneron Pharmaceuticals, Inc. Methods and compositions for using plasma cell depleting agents and/or b cell depleting agents to suppress host anti-aav antibody response and enable aav transduction and re-dosing
US20250276092A1 (en) 2024-03-01 2025-09-04 Regeneron Pharmaceuticals, Inc. Methods and compositions for re-dosing aav using anti-cd40 antagonistic antibody to suppress host anti-aav antibody response

Family Cites Families (11)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6458563B1 (en) 1996-06-26 2002-10-01 Emory University Modified factor VIII
US6531298B2 (en) 1997-07-21 2003-03-11 The University Of North Carolina At Chapel Hill Factor IX antihemophilic factor with increased clotting activity
JP2004505016A (ja) 2000-05-10 2004-02-19 ノボ ノルディスク アクティーゼルスカブ 第VIIa因子及び第XIII因子を含んで成る医薬組成物
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
EP2423307A1 (en) * 2006-06-19 2012-02-29 Catalyst Biosciences, Inc. Modified coagulation factor IV polypeptides and use thereof for treatment
US7700734B2 (en) 2007-01-09 2010-04-20 Shu-Wha Lin Recombinant human factor IX and use thereof
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
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
CA2892038C (en) * 2012-11-20 2021-12-28 Darrel W. Stafford Methods and compositions for modified factor ix proteins
PT3068869T (pt) * 2013-11-15 2020-10-08 Univ Pennsylvania Composições para supressão da formação de inibidores contra fator viii em pacientes de hemofilia a
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

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