CN113396223A - 表达因子ix的慢病毒载体的用途 - Google Patents

表达因子ix的慢病毒载体的用途 Download PDF

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Publication number
CN113396223A
CN113396223A CN201980090827.7A CN201980090827A CN113396223A CN 113396223 A CN113396223 A CN 113396223A CN 201980090827 A CN201980090827 A CN 201980090827A CN 113396223 A CN113396223 A CN 113396223A
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lentiviral vector
seq
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nucleotide sequence
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刘彤瑶
S·帕塔罗育-怀特
D·德尔格
A·康托尔
L·纳迪尼
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Bioverativ Therapeutics Inc
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CN201980090827.7A 2018-12-06 2019-12-05 表达因子ix的慢病毒载体的用途 Pending CN113396223A (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201862776393P 2018-12-06 2018-12-06
US62/776,393 2018-12-06
PCT/US2019/064711 WO2020118069A2 (en) 2018-12-06 2019-12-05 Use of lentiviral vectors expressing factor ix

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CN113396223A true CN113396223A (zh) 2021-09-14

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US (1) US20200199626A1 (https=)
EP (1) EP3891289A2 (https=)
JP (2) JP7644007B2 (https=)
KR (1) KR20210100661A (https=)
CN (1) CN113396223A (https=)
AU (1) AU2019393880A1 (https=)
BR (1) BR112021010047A2 (https=)
CA (1) CA3121786A1 (https=)
CO (1) CO2021008877A2 (https=)
IL (1) IL283547A (https=)
MX (2) MX2021006648A (https=)
SG (1) SG11202105880TA (https=)
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WO (1) WO2020118069A2 (https=)

Families Citing this family (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
CA3012695A1 (en) 2016-02-01 2017-08-10 Bioverativ Therapeutics Inc. Optimized factor viii genes
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.
TW202246505A (zh) * 2021-03-05 2022-12-01 俄羅斯聯邦商亞那拜恩有限公司 編碼凝血因子ｉｘ蛋白的密碼子優化的核酸及其用途
GB202205615D0 (en) * 2022-04-14 2022-06-01 Genespire S R L Lentiviral vector
KR20250021306A (ko) * 2022-04-14 2025-02-12 제네스파이어 에스.알.엘. 렌티바이러스 벡터
WO2025226943A1 (en) 2024-04-25 2025-10-30 Masonic Medical Research Laboratory Polynucleotide for hepatocyte expression of vestigial like protein 4 and method of use thereof

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2016004113A1 (en) * 2014-06-30 2016-01-07 Biogen Ma Inc. Optimized factor ix gene

Family Cites Families (44)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP3045539B2 (ja) 1989-02-21 2000-05-29 ワシントン ユニバーシティ 改変型生殖ホルモン
US6086875A (en) 1995-01-17 2000-07-11 The Brigham And Women's Hospital, Inc. Receptor specific transepithelial transport of immunogens
US6485726B1 (en) 1995-01-17 2002-11-26 The Brigham And Women's Hospital, Inc. Receptor specific transepithelial transport of therapeutics
US6030613A (en) 1995-01-17 2000-02-29 The Brigham And Women's Hospital, Inc. Receptor specific transepithelial transport of therapeutics
US6013516A (en) 1995-10-06 2000-01-11 The Salk Institute For Biological Studies Vector and method of use for nucleic acid delivery to non-dividing cells
GB9621680D0 (en) 1996-10-17 1996-12-11 Oxford Biomedica Ltd Lentiviral vectors
PT904392E (pt) 1996-10-17 2001-06-29 Oxford Biomedica Ltd Vectores retrovirais
GB9622500D0 (en) 1996-10-29 1997-01-08 Oxford Biomedica Ltd Therapeutic gene
US6207455B1 (en) 1997-05-01 2001-03-27 Lung-Ji Chang Lentiviral vectors
US5994136A (en) 1997-12-12 1999-11-30 Cell Genesys, Inc. Method and means for producing high titer, safe, recombinant lentivirus vectors
EP2213743A1 (en) 2000-04-12 2010-08-04 Human Genome Sciences, Inc. Albumin fusion proteins
ATE437221T1 (de) 2001-05-14 2009-08-15 Gbp Ip Llc Lentivirale vektoren kodierend für gerinnungsfaktoren für die gentherapie
AU2002364586A1 (en) 2001-12-21 2003-07-30 Delta Biotechnology Limited Albumin fusion proteins
KR101271635B1 (ko) 2001-12-21 2013-06-12 휴먼 게놈 사이언시즈, 인코포레이티드 알부민 융합 단백질
US20080194481A1 (en) 2001-12-21 2008-08-14 Human Genome Sciences, Inc. Albumin Fusion Proteins
JP2005526769A (ja) 2002-03-15 2005-09-08 ザ・ブリガーム・アンド・ウーメンズ・ホスピタル・インコーポレーテッド 治療剤を全身搬送するための中央気道投与
US6615782B1 (en) 2002-04-12 2003-09-09 Delphi Technologies, Inc. Two-step finger follower rocker arm
ES2298745T3 (es) 2003-04-24 2008-05-16 Fondazione Centro San Raffaele Del Monte Tabor Promotores bidireccionales de sintesis y utilizacion de los mismos.
EP1769078A1 (en) * 2004-07-01 2007-04-04 VIRxSYS Corporation Vector packaging cell line
CA2609142C (en) 2005-05-27 2016-02-09 Fondazione Centro San Raffaele Del Monte Tabor Therapeutic gene vectors comprising mirna target sequences
WO2007021494A2 (en) 2005-08-12 2007-02-22 Human Genome Sciences, Inc. Albumin fusion proteins
US8048848B2 (en) 2006-02-03 2011-11-01 Prolor Biotech Ltd. Long-acting interferons and derivatives thereof and methods thereof
GB0614780D0 (en) 2006-07-25 2006-09-06 Ucb Sa Biological products
WO2008033413A2 (en) 2006-09-14 2008-03-20 Human Genome Sciences, Inc. Albumin fusion proteins
EP2144930A1 (en) 2007-04-18 2010-01-20 ZymoGenetics, Inc. Single chain fc, methods of making and methods of treatment
EP2158318A2 (en) 2007-05-14 2010-03-03 Biogen Idec MA, Inc. Single-chain fc (scfc) regions, binding polypeptides comprising same, and methods related thereto
SI2173890T1 (sl) 2007-06-21 2011-06-30 Univ Muenchen Tech Biološko aktivni proteini, ki imajo povečano in vivo in/ali in vitro stabilnost
EP2364362B1 (en) 2008-11-12 2015-10-21 Ospedale San Raffaele S.r.l. Gene vector for inducing transgene-specific immune tolerance
ES2610356T3 (es) 2009-02-03 2017-04-27 Amunix Operating Inc. Polipéptidos recombinantes extendidos y composiciones que comprenden los mismos
US8680050B2 (en) 2009-02-03 2014-03-25 Amunix Operating Inc. Growth hormone polypeptides fused to extended recombinant polypeptides and methods of making and using same
US8703717B2 (en) 2009-02-03 2014-04-22 Amunix Operating Inc. Growth hormone polypeptides and methods of making and using same
US9050269B2 (en) 2009-03-10 2015-06-09 The Trustees Of The University Of Pennsylvania Protection of virus particles from phagocytosis by expression of CD47
PL2424571T3 (pl) 2009-04-30 2020-10-19 Ospedale San Raffaele S.R.L Wektor genowy
WO2010144508A1 (en) 2009-06-08 2010-12-16 Amunix Operating Inc. Glucose-regulating polypeptides and methods of making and using same
KR101813727B1 (ko) 2009-06-08 2018-01-02 아뮤닉스 오퍼레이팅 인코포레이티드 성장 호르몬 폴리펩타이드 및 이를 제조하고 사용하는 방법
CA2771999A1 (en) 2009-08-24 2011-03-10 Amunix Operating Inc. Coagulation factor vii compositions and methods of making and using same
WO2011028344A2 (en) 2009-08-25 2011-03-10 Amunix Operating Inc. Interleukin-1 receptor antagonist compositions and methods of making and using same
SI2506868T1 (en) 2009-12-06 2018-04-30 Bioverativ Therapeutics Inc. HYPER AND HYBRID POLYPETTIDES FACTOR VIII-FC AND METHODS FOR THEIR USE
EP3508573A1 (en) 2010-07-09 2019-07-10 Bioverativ Therapeutics Inc. Systems for factor viii processing and methods thereof
TW201217527A (en) 2010-07-09 2012-05-01 Biogen Idec Hemophilia Inc Processable single chain molecules and polypeptides made using same
CN104661674A (zh) 2012-07-11 2015-05-27 阿穆尼克斯运营公司 具有xten和血管性血友病因子蛋白的因子viii复合物、及其用途
TW202003554A (zh) 2013-08-14 2020-01-16 美商百歐維拉提夫治療公司 因子ｖｉｉｉ－ｘｔｅｎ融合物及其用途
EA201890423A1 (ru) 2015-08-03 2018-07-31 Биовератив Терапьютикс Инк. Слитые белки фактора ix, способы их получения и применения
CA3012695A1 (en) * 2016-02-01 2017-08-10 Bioverativ Therapeutics Inc. Optimized factor viii genes

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2016004113A1 (en) * 2014-06-30 2016-01-07 Biogen Ma Inc. Optimized factor ix gene
US20170260516A1 (en) * 2014-06-30 2017-09-14 Biogen Ma Inc. Optimized factor ix gene

Non-Patent Citations (3)

* Cited by examiner, † Cited by third party
Title
ALESSIO CANTORE等: "Liver-Directed Gene Therapy for Hemophilia B with Immune Stealth Lentiviral Vectors", 《BLOOD》, vol. 130, no. 605, pages 1 - 2 *
ALESSIO CANTORE等: "Liver-directed lentiviral gene therapy in a dog model of hemophilia B", 《SCI TRANSL MED》, vol. 7, no. 277, pages 1 - 27 *
NISHA G SOSALE等: ""Marker of Self" CD47 on lentiviral vectors decreases macrophage-mediated clearance and increases delivery to SIRPA-expressing lung carcinoma tumors", 《MOL THER METHODS CLIN DEV》, vol. 3, no. 16080, pages 1 - 13, XP055567980, DOI: 10.1038/mtm.2016.80 *

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WO2020118069A3 (en) 2020-07-16
IL283547A (en) 2021-07-29
JP2022514465A (ja) 2022-02-14
US20200199626A1 (en) 2020-06-25
JP7644007B2 (ja) 2025-03-11
CA3121786A1 (en) 2020-06-11
AU2019393880A1 (en) 2021-07-15
SG11202105880TA (en) 2021-07-29
CO2021008877A2 (es) 2021-07-30
MX2025015374A (es) 2026-02-03
BR112021010047A2 (pt) 2021-08-24
JP2025020288A (ja) 2025-02-12
WO2020118069A2 (en) 2020-06-11
EP3891289A2 (en) 2021-10-13
KR20210100661A (ko) 2021-08-17
MX2021006648A (es) 2021-07-07
TW202039855A (zh) 2020-11-01

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