CN113227385B - 核酸分子及其用于非病毒基因疗法的用途 - Google Patents

核酸分子及其用于非病毒基因疗法的用途 Download PDF

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CN113227385B
CN113227385B CN201980065714.1A CN201980065714A CN113227385B CN 113227385 B CN113227385 B CN 113227385B CN 201980065714 A CN201980065714 A CN 201980065714A CN 113227385 B CN113227385 B CN 113227385B
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nucleic acid
itr
acid molecule
seq
nucleotides
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CN113227385A (zh
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P·扎卡斯
D·德尔格
S·帕塔罗育-怀特
刘彤瑶
A·塞雷金
R·T·彼得斯
J·刘
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Bioverativ Therapeutics Inc
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
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    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
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    • A61K31/7105—Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
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    • A61K48/0008—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • A61K48/0016—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the nucleic acid is delivered as a 'naked' nucleic acid, i.e. not combined with an entity such as a cationic lipid
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    • A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
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CN201980065714.1A 2018-08-09 2019-08-09 核酸分子及其用于非病毒基因疗法的用途 Active CN113227385B (zh)

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PCT/US2019/045957 WO2020033863A1 (en) 2018-08-09 2019-08-09 Nucleic acid molecules and uses thereof for non-viral gene therapy

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Families Citing this family (28)

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Publication number Priority date Publication date Assignee Title
RS61387B2 (sr) 2013-02-15 2024-06-28 Bioverativ Therapeutics Inc Gen optimizovanog faktora viii
MX2020001593A (es) 2017-08-09 2020-07-13 Bioverativ Therapeutics Inc Moleculas de acido nucleico y usos de las mismas.
WO2020019002A1 (en) 2018-07-20 2020-01-23 University Of Utah Research Foundation Gene therapy for macular degeneration
JP7602454B2 (ja) 2018-08-09 2024-12-18 バイオベラティブ セラピューティクス インコーポレイテッド 核酸分子及び非ウイルス遺伝子治療のためのそれらの使用
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
SG11202104634UA (en) * 2018-11-07 2021-06-29 Vivet Therapeutics Codon-optimized abcb11 transgene for the treatment of progressive familial intrahepatic cholestasis type 2 (pfic2)
CN111334528B (zh) * 2020-03-20 2022-02-18 山东农业大学 水貂肠炎细小病毒全基因组感染性克隆及其构建方法和应用
MX2023001109A (es) 2020-07-27 2023-04-24 Anjarium Biosciences Ag Composiciones de moleculas de adn, metodos para elaborar las mismas, y metodos de usos de las mismas.
AU2021330936A1 (en) * 2020-08-23 2023-05-04 Bioverativ Therapeutics Inc. MODIFIED BACULOVIRUS SYSTEM FOR IMPROVED PRODUCTION OF CLOSED-ENDED DNA (ceDNA)
CN112704729A (zh) * 2020-12-28 2021-04-27 江苏纳迪芯生命科技研究院有限公司 一种细胞因子颗粒
WO2022198025A2 (en) * 2021-03-19 2022-09-22 Generation Bio Co. Non-viral dna vectors and uses thereof for expressing pfic therapeutics
CN113325181A (zh) * 2021-04-25 2021-08-31 苏州市立医院(北区) 丝氨酸蛋白酶抑制剂在用于脓毒症早期预警的标志物中的应用
WO2022232029A2 (en) * 2021-04-26 2022-11-03 University Of Florida Research Foundation, Incorporated Synthetic aav vectors for repeated delivery of therapeutic genes
US20240287493A1 (en) * 2021-06-24 2024-08-29 University Of Utah Research Foundation Compositions and methods for treating pgm1 deficiency
WO2023028440A2 (en) 2021-08-23 2023-03-02 Bioverativ Therapeutics Inc. Baculovirus expression system
US20230084036A1 (en) * 2021-08-23 2023-03-16 Bioverativ Therapeutics Inc. Optimized factor viii genes
AU2022334714A1 (en) * 2021-08-23 2024-04-04 Bioverativ Therapeutics Inc. Closed-end dna production with inverted terminal repeat sequences
JP2024532261A (ja) * 2021-08-23 2024-09-05 バイオベラティブ セラピューティクス インコーポレイテッド 操作ｉｔｒ配列および使用方法
CN122521785A (zh) * 2021-09-16 2026-08-07 北京锦篮基因科技股份有限公司 用于治疗ⅱ型粘多糖贮积症的重组腺相关病毒载体及其应用
KR20240067122A (ko) * 2021-09-30 2024-05-16 바이오버라티브 테라퓨틱스 인크. 감소된 면역원성을 갖는 인자 viii 폴리펩타이드를 암호화하는 핵산
CN114250224B (zh) * 2021-12-16 2024-02-27 福建和瑞基因科技有限公司 一种用于提取或检测样本中小分子rna的核酸组合物及其试剂盒和方法
WO2023135273A2 (en) * 2022-01-14 2023-07-20 Anjarium Biosciences Ag Compositions of dna molecules encoding factor viii, methods of making thereof, and methods of use thereof
FR3136367A1 (fr) * 2022-06-09 2023-12-15 Damien SULEIMAN Compositions et méthodes concernant des molécules d’ARN messager codant le facteur V encapsulées dans des nanoparticules lipidiques
CN119233998A (zh) * 2022-07-07 2024-12-31 深圳新诺微环生物科技有限公司 一种连接肽、包含连接肽的凝血八因子蛋白或其变体及其用途
JP2025525583A (ja) 2022-07-19 2025-08-05 ランパート バイオサイエンス インコーポレイテッド 非免疫原性環状非ウイルス性ｄｎａベクター
CN116064673A (zh) * 2022-10-19 2023-05-05 河南省人民医院 表达rs1的重组腺相关病毒载体及其应用
CN120981563A (zh) * 2023-03-24 2025-11-18 南京金斯瑞生物科技有限公司 一种提升质粒稳定性的宿主细胞

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN111247251A (zh) * 2017-08-09 2020-06-05 比奥维拉迪维治疗股份有限公司 核酸分子及其用途

Family Cites Families (216)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4683202A (en) 1985-03-28 1987-07-28 Cetus Corporation Process for amplifying nucleic acid sequences
US4965188A (en) 1986-08-22 1990-10-23 Cetus Corporation Process for amplifying, detecting, and/or cloning nucleic acid sequences using a thermostable enzyme
US4683195A (en) 1986-01-30 1987-07-28 Cetus Corporation Process for amplifying, detecting, and/or-cloning nucleic acid sequences
MX9203440A (es) 1985-04-12 1992-07-01 Genetics Inst Proteinas procoagulantes novedosas.
WO1986006408A1 (en) 1985-04-22 1986-11-06 Genetics Institute, Inc. HIGH YIElD PRODUCTION OF ACTIVE FACTOR IX
KR910006424B1 (ko) 1985-08-21 1991-08-24 인코텍스 비.브이 편성브리프(brief) 제조방법
ATE87663T1 (de) 1986-01-03 1993-04-15 Genetics Inst Verfahren zur herstellung von faktor-viii:c-typ- proteinen.
US5595886A (en) 1986-01-27 1997-01-21 Chiron Corporation Protein complexes having Factor VIII:C activity and production thereof
US4800159A (en) 1986-02-07 1989-01-24 Cetus Corporation Process for amplifying, detecting, and/or cloning nucleic acid sequences
US5543502A (en) 1986-06-24 1996-08-06 Novo Nordisk A/S Process for producing a coagulation active complex of factor VIII fragments
EP0279582A3 (en) 1987-02-17 1989-10-18 Pharming B.V. Dna sequences to target proteins to the mammary gland for efficient secretion
JP3101690B2 (ja) 1987-03-18 2000-10-23 エス・ビィ・２・インコーポレイテッド 変性抗体の、または変性抗体に関する改良
US6060447A (en) 1987-05-19 2000-05-09 Chiron Corporation Protein complexes having Factor VIII:C activity and production thereof
US6346513B1 (en) 1987-06-12 2002-02-12 Baxter Trading Gmbh Proteins with factor VIII activity: process for their preparation using genetically-engineered cells and pharmaceutical compositions containing them
IE69026B1 (en) 1987-06-12 1996-08-07 Immuno Ag Novel proteins with factor VIII activity process for their preparation using genetically-engineered cells and pharmaceutical compositions containing them
DE3720246A1 (de) 1987-06-19 1988-12-29 Behringwerke Ag Faktor viii:c-aehnliches molekuel mit koagulationsaktivitaet
FR2619314B1 (fr) 1987-08-11 1990-06-15 Transgene Sa Analogue du facteur viii, procede de preparation et composition pharmaceutique le contenant
US6780613B1 (en) 1988-10-28 2004-08-24 Genentech, Inc. Growth hormone variants
DE122010000006I2 (de) 1989-02-21 2012-06-21 Univ Washington Modifizierte formen von fortpflanzungshormonen.
US5633076A (en) 1989-12-01 1997-05-27 Pharming Bv Method of producing a transgenic bovine or transgenic bovine embryo
SE465222C5 (sv) 1989-12-15 1998-02-10 Pharmacia & Upjohn Ab Ett rekombinant, humant faktor VIII-derivat och förfarande för dess framställning
IE922437A1 (en) 1991-07-25 1993-01-27 Idec Pharma Corp Recombinant antibodies for human therapy
US5736137A (en) 1992-11-13 1998-04-07 Idec Pharmaceuticals Corporation Therapeutic application of chimeric and radiolabeled antibodies to human B lymphocyte restricted differentiation antigen for treatment of B cell lymphoma
CA2149326C (en) 1992-11-13 2007-04-17 Mitchell E. Reff Fully impaired consensus kozak sequences for mammalian expression
CA2162271A1 (en) 1993-05-26 1994-12-08 Robert Kotin Fusion proteins containing adeno-associated virus rep protein and bacterial protein
SE504074C2 (sv) 1993-07-05 1996-11-04 Pharmacia Ab Proteinberedning för subkutan, intramuskulär eller intradermal administrering
AU5351494A (en) 1993-10-07 1995-05-01 G.D. Searle & Co. Method of producing recombinant eukaryotic viruses in bacteria
US5827690A (en) 1993-12-20 1998-10-27 Genzyme Transgenics Corporatiion Transgenic production of antibodies in milk
FR2716893B1 (fr) 1994-03-03 1996-04-12 Rhone Poulenc Rorer Sa Virus recombinants, leur préparation et leur utilisation thérapeutique.
GB9422383D0 (en) 1994-11-05 1995-01-04 Wellcome Found Antibodies
US6485726B1 (en) 1995-01-17 2002-11-26 The Brigham And Women's Hospital, Inc. Receptor specific transepithelial transport of therapeutics
US6086875A (en) 1995-01-17 2000-07-11 The Brigham And Women's Hospital, Inc. Receptor specific transepithelial transport of immunogens
US6030613A (en) 1995-01-17 2000-02-29 The Brigham And Women's Hospital, Inc. Receptor specific transepithelial transport of therapeutics
US5869046A (en) 1995-04-14 1999-02-09 Genentech, Inc. Altered polypeptides with increased half-life
US6096871A (en) 1995-04-14 2000-08-01 Genentech, Inc. Polypeptides altered to contain an epitope from the Fc region of an IgG molecule for increased half-life
US5739277A (en) 1995-04-14 1998-04-14 Genentech Inc. Altered polypeptides with increased half-life
US6121022A (en) 1995-04-14 2000-09-19 Genentech, Inc. Altered polypeptides with increased half-life
SE9503380D0 (sv) 1995-09-29 1995-09-29 Pharmacia Ab Protein derivatives
GB9524973D0 (en) 1995-12-06 1996-02-07 Lynxvale Ltd Viral vectors
US6458563B1 (en) 1996-06-26 2002-10-01 Emory University Modified factor VIII
AU3968897A (en) 1996-08-02 1998-02-25 Bristol-Myers Squibb Company A method for inhibiting immunoglobulin-induced toxicity resulting from the use of immunoglobulins in therapy and in vivo diagnosis
WO1998023289A1 (en) 1996-11-27 1998-06-04 The General Hospital Corporation MODULATION OF IgG BINDING TO FcRn
US6277375B1 (en) 1997-03-03 2001-08-21 Board Of Regents, The University Of Texas System Immunoglobulin-like domains with increased half-lives
CA2225189C (en) 1997-03-06 2010-05-25 Queen's University At Kingston Canine factor viii gene, protein and methods of use
RO120148B1 (ro) 1997-03-14 2005-09-30 Idec Pharmaceuticals Corporation Sistem vector şi procedeu de inserţie a unui fragment adn, în genomul celulelor de mamifere
GB9722131D0 (en) 1997-10-20 1997-12-17 Medical Res Council Method
CA2323757C (en) 1998-04-02 2011-08-02 Genentech, Inc. Antibody variants and fragments thereof
US6242195B1 (en) 1998-04-02 2001-06-05 Genentech, Inc. Methods for determining binding of an analyte to a receptor
US6194551B1 (en) 1998-04-02 2001-02-27 Genentech, Inc. Polypeptide variants
US6528624B1 (en) 1998-04-02 2003-03-04 Genentech, Inc. Polypeptide variants
GB9809951D0 (en) 1998-05-08 1998-07-08 Univ Cambridge Tech Binding molecules
US6291214B1 (en) 1998-05-11 2001-09-18 Glaxo Wellcome Inc. System for generating recombinant viruses
JP2002522013A (ja) 1998-07-24 2002-07-23 ベイラ、カリッジ、アヴ、メダスン 部位特異的組換えを使用する迅速なサブクローニング
JP2002522063A (ja) 1998-08-17 2002-07-23 アブジェニックス インコーポレイテッド 増加した血清半減期を有する改変された分子の生成
US20020065236A1 (en) 1998-09-09 2002-05-30 Yew Nelson S. CpG reduced plasmids and viral vectors
US6221349B1 (en) 1998-10-20 2001-04-24 Avigen, Inc. Adeno-associated vectors for expression of factor VIII by target cells
EP1135468B1 (en) 1998-11-10 2010-01-06 University Of North Carolina At Chapel Hill Virus vectors and methods of making and administering the same
EP1006183A1 (en) 1998-12-03 2000-06-07 Max-Planck-Gesellschaft zur Förderung der Wissenschaften e.V. Recombinant soluble Fc receptors
US6737056B1 (en) 1999-01-15 2004-05-18 Genentech, Inc. Polypeptide variants with altered effector function
HUP0104865A3 (en) 1999-01-15 2004-07-28 Genentech Inc Polypeptide variants with altered effector function
US6926898B2 (en) 2000-04-12 2005-08-09 Human Genome Sciences, Inc. Albumin fusion proteins
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
GB0029407D0 (en) 2000-12-01 2001-01-17 Affitech As Product
EP1355919B1 (en) 2000-12-12 2010-11-24 MedImmune, LLC Molecules with extended half-lives, compositions and uses thereof
US7211395B2 (en) 2001-03-09 2007-05-01 Dyax Corp. Serum albumin binding moieties
WO2002092134A1 (en) 2001-05-14 2002-11-21 Cell Genesys, Inc. Lentiviral vectors encoding clotting factors for gene therapy
CA2457429A1 (en) 2001-09-04 2003-03-13 Francis J. Carr Modified factor ix
US6723551B2 (en) 2001-11-09 2004-04-20 The United States Of America As Represented By The Department Of Health And Human Services Production of adeno-associated virus in insect cells
EP1572893B1 (en) 2001-11-09 2009-01-07 THE GOVERNMENT OF THE UNITED STATES OF AMERICA as represented by THE SECRETARY of the DEPARTMENT OF HEALTH AND HUMAN SERVICES Production of adeno-associated virus in insect cells
KR101271635B1 (ko) 2001-12-21 2013-06-12 휴먼 게놈 사이언시즈, 인코포레이티드 알부민 융합 단백질
CA2841097A1 (en) 2001-12-21 2003-07-24 Human Genome Sciences, Inc. Albumin and g-csf fusion proteins
US20080194481A1 (en) 2001-12-21 2008-08-14 Human Genome Sciences, Inc. Albumin Fusion Proteins
US20040002587A1 (en) 2002-02-20 2004-01-01 Watkins Jeffry D. Fc region variants
US7317091B2 (en) 2002-03-01 2008-01-08 Xencor, Inc. Optimized Fc variants
US20040018557A1 (en) 2002-03-01 2004-01-29 Immunomedics, Inc. Bispecific antibody point mutations for enhancing rate of clearance
US20040132101A1 (en) 2002-09-27 2004-07-08 Xencor Optimized Fc variants and methods for their generation
CA2479212A1 (en) 2002-03-15 2003-09-25 Brigham And Women's Hospital, Inc. Central airway administration for systemic delivery of therapeutics
PT1534335E (pt) 2002-08-14 2012-02-28 Macrogenics Inc Anticorpos específicos de fcγriib e processos para a sua utilização
CA2832136C (en) 2002-09-27 2015-11-17 Xencor Optimized fc variants and methods for their generation
ATE480562T1 (de) 2002-10-15 2010-09-15 Facet Biotech Corp Veränderung von fcrn-bindungsaffinitäten oder von serumhalbwertszeiten von antikörpern mittels mutagenese
GB2395337B (en) 2002-11-14 2005-12-28 Gary Michael Wilson Warning Unit
US7355008B2 (en) 2003-01-09 2008-04-08 Macrogenics, Inc. Identification and engineering of antibodies with variant Fc regions and methods of using same
US7041635B2 (en) 2003-01-28 2006-05-09 In2Gen Co., Ltd. Factor VIII polypeptide
US8388955B2 (en) 2003-03-03 2013-03-05 Xencor, Inc. Fc variants
US20090010920A1 (en) 2003-03-03 2009-01-08 Xencor, Inc. Fc Variants Having Decreased Affinity for FcyRIIb
WO2004094642A2 (en) 2003-04-24 2004-11-04 Fondazione Centro San Raffaele Del Monte Tabor Lentiviral vectors carrying synthetic bi-directional promoters and uses thereof
TWI353991B (en) 2003-05-06 2011-12-11 Syntonix Pharmaceuticals Inc Immunoglobulin chimeric monomer-dimer hybrids
GB0324368D0 (en) 2003-10-17 2003-11-19 Univ Cambridge Tech Polypeptides including modified constant regions
EP1697520A2 (en) 2003-12-22 2006-09-06 Xencor, Inc. Fc polypeptides with novel fc ligand binding sites
JP4762156B2 (ja) 2004-01-12 2011-08-31 アプライド モレキュラー エボリューション，インコーポレイテッド Ｆｃ領域変異体
US20080004228A1 (en) 2004-03-09 2008-01-03 Imre Berger New Expression Tools for Multiprotein Applications
US7276585B2 (en) 2004-03-24 2007-10-02 Xencor, Inc. Immunoglobulin variants outside the Fc region
WO2005123780A2 (en) 2004-04-09 2005-12-29 Protein Design Labs, Inc. Alteration of fcrn binding affinities or serum half-lives of antibodies by mutagenesis
WO2006085967A2 (en) 2004-07-09 2006-08-17 Xencor, Inc. OPTIMIZED ANTI-CD20 MONOCONAL ANTIBODIES HAVING Fc VARIANTS
US7598071B2 (en) * 2004-07-09 2009-10-06 The United States Of America As Represented By The Department Of Health And Human Services Infectious clone of human parvovirus B19 and methods
EP3342782B1 (en) 2004-07-15 2022-08-17 Xencor, Inc. Optimized fc variants
EP2169072A1 (en) 2004-08-23 2010-03-31 Alnylam Pharmaceuticals, Inc Multiple RNA polymerase III promoter expression constructs
AU2005287047A1 (en) 2004-09-17 2006-03-30 University Of Utah Research Foundation Imaging reporters of transgene expression
WO2006047350A2 (en) 2004-10-21 2006-05-04 Xencor, Inc. IgG IMMUNOGLOBULIN VARIANTS WITH OPTIMIZED EFFECTOR FUNCTION
EP1828390B1 (en) 2004-12-15 2012-06-13 The University Of North Carolina At Chapel Hill Chimeric vectors
WO2006126972A1 (en) * 2005-05-27 2006-11-30 Agency For Science, Technology And Research Method of delivering nucleic acid molecules into embryonic stem cells using baculoviral vectors
DK2002003T3 (en) 2005-05-27 2016-03-21 Ospedale San Raffaele Srl Gene vector comprising miRNA
CA2618476A1 (en) 2005-08-12 2007-02-22 Human Genome Sciences, Inc. Albumin fusion proteins
AU2006314843B2 (en) 2005-11-17 2011-06-30 Karolinska Institutet Innovations Ab Human bocavirus and methods of diagnosis and treatment
WO2008016391A2 (en) * 2006-01-31 2008-02-07 The Board Of Trustees Of The Leland Stanford Junior University Self-complementary parvoviral vectors, and methods for making and using the same
US8048848B2 (en) 2006-02-03 2011-11-01 Prolor Biotech Ltd. Long-acting interferons and derivatives thereof and methods thereof
WO2007140011A2 (en) 2006-05-26 2007-12-06 The Government Of The United States Of America Erythroid progenitor cells and methods for producing parvovirus b19 therein
US7939632B2 (en) 2006-06-14 2011-05-10 Csl Behring Gmbh Proteolytically cleavable fusion proteins with high molar specific activity
EP2423307A1 (en) 2006-06-19 2012-02-29 Catalyst Biosciences, Inc. Modified coagulation factor IV polypeptides and use thereof for treatment
GB0614780D0 (en) 2006-07-25 2006-09-06 Ucb Sa Biological products
ES2385679T3 (es) 2006-08-24 2012-07-30 Virovek, Inc. Expresión de células de insecto de genes con marcos de lectura abiertos superpuestos, métodos y composiciones de éstos
WO2008033413A2 (en) 2006-09-14 2008-03-20 Human Genome Sciences, Inc. Albumin fusion proteins
US7700734B2 (en) 2007-01-09 2010-04-20 Shu-Wha Lin Recombinant human factor IX and use thereof
CA2682605A1 (en) 2007-04-18 2008-10-30 Zymogenetics, Inc. Single chain fc, methods of making and methods of treatment
MX2009012343A (es) 2007-05-14 2010-02-10 Biogen Idec Inc Regiones fc (sc fc) de cadena sencilla, polipeptidos de enlace que comprenden las mismas, y metodos relacionados con ello.
WO2008141400A1 (en) 2007-05-24 2008-11-27 Medvet Science Pty Ltd A novel aetiologic agent of acute gastroenteritis (age), diagnostic methods and therapeutic treatment thereof
US8563521B2 (en) 2007-06-21 2013-10-22 Technische Universitat Munchen Biological active proteins having increased in vivo and/or in vitro stability
US9115373B2 (en) 2007-09-19 2015-08-25 Uniqure Ip B.V. Use of AAV replication machinery for improved protein production
AU2008311973B2 (en) 2007-10-15 2013-10-03 Cangene Corporation Human Factor IX variants with an extended half life
KR20110015551A (ko) 2008-04-16 2011-02-16 바이엘 헬스케어 엘엘씨 제ｉｘ인자의 부위-지정 변형
KR20110005862A (ko) 2008-04-16 2011-01-19 바이엘 헬스케어 엘엘씨 변형된 제ｉｘ인자 폴리펩티드 및 이의 용도
EP2274420A4 (en) 2008-04-17 2013-09-25 Blood Systems Inc NEW HUMAN PARVOVIRUS: THE BOCAVIRUS
EP2268807A2 (en) 2008-04-21 2011-01-05 Novo Nordisk A/S Hyperglycosylated human coagulation factor ix
GB0820631D0 (en) 2008-11-11 2008-12-17 London School Hygiene & Tropical Medicine Vectors
US9938540B2 (en) 2008-11-12 2018-04-10 Ospedale San Raffaele S.R.L. Gene vector for inducing transgene-specific immune tolerance
DK3178835T3 (da) 2009-02-03 2019-06-24 Amunix Pharmaceuticals Inc Forlængede rekombinante polypeptider og sammensætninger omfattende samme
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
MX2011011508A (es) 2009-04-30 2012-02-13 San Raffaele Centro Fond Vector genico.
US20120134977A1 (en) 2009-06-01 2012-05-31 Yeda Research And Development Co. Ltd. Prodrugs containing albumin binding probe
PE20121539A1 (es) 2009-06-08 2012-12-06 Amunix Operating Inc Polipeptidos de la hormona de crecimiento y metodos de preparacion
WO2010144508A1 (en) 2009-06-08 2010-12-16 Amunix Operating Inc. Glucose-regulating polypeptides and methods of making and using same
GB0911870D0 (en) * 2009-07-08 2009-08-19 Ucl Business Plc Optimised coding sequence and promoter
EP2292781A1 (en) 2009-08-17 2011-03-09 Genethon Baculovirus-based production of biopharmaceuticals free of contaminating baculoviral virions
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
US9169494B2 (en) * 2010-01-12 2015-10-27 The University Of North Carolina At Chapel Hill Restrictive inverted terminal repeats for viral vectors
EP2534172A2 (en) 2010-02-12 2012-12-19 The U.S.A. as represented by the Secretary, Department of Health and Human Services Compositions and methods for preventing or treating a human parvovirus infection
WO2011112090A2 (en) 2010-03-11 2011-09-15 Amsterdam Molecular Therapeutics (Amt) Ip B.V. Method for identifying variant rep protein encoding nucleic acids
MX375112B (es) 2010-07-09 2025-03-06 Biogen Hemophilia Inc Polipéptidos de factor ix y métodos para usarlos.
PT2591006T (pt) 2010-07-09 2019-07-29 Bioverativ Therapeutics Inc Moléculas de cadeia simples processáveis e polipéptidos preparados utilizando as mesmas
JP6091435B2 (ja) 2011-02-22 2017-03-08 カリフォルニア インスティチュート オブ テクノロジー アデノ随伴ウイルス（ａａｖ）ベクターを用いたタンパク質の送達
EP2500434A1 (en) * 2011-03-12 2012-09-19 Association Institut de Myologie Capsid-free AAV vectors, compositions, and methods for vector production and gene delivery
EP2508607A1 (en) 2011-04-07 2012-10-10 Helmholtz-Zentrum für Infektionsforschung GmbH Medicament for liver regeneration and for treatment of liver failure
HRP20191920T1 (hr) 2012-01-12 2020-01-10 Bioverativ Therapeutics Inc. Kimerni polipeptidi faktora viii i njihova uporaba
EA038705B1 (ru) 2012-01-12 2021-10-07 Биовератив Терапьютикс Инк. Способы снижения иммуногенности фактора свёртывания крови viii у пациентов, получающих лечение фактором viii
CN111548418A (zh) 2012-02-15 2020-08-18 比奥贝拉蒂治疗公司 因子viii组合物及其制备和使用方法
WO2013123457A1 (en) 2012-02-15 2013-08-22 Biogen Idec Ma Inc. Recombinant factor viii proteins
BR112014020325A2 (pt) 2012-02-17 2017-08-08 Childrens Hospital Philadelphia composições de vetor do aav e métodos para a transferência de gene para as células, órgãos e tecidos
ES2770501T3 (es) 2012-07-11 2020-07-01 Bioverativ Therapeutics Inc Complejo del factor VIII con XTEN y proteína del factor de Von Willebrand y sus usos
AU2013331000B2 (en) * 2012-10-18 2018-04-19 Bioverativ Therapeutics Inc. Methods of using a fixed dose of a clotting factor
EP2914731B1 (en) 2012-11-01 2019-09-11 California Institute of Technology Reversible gene expression
CN103215308B (zh) 2013-02-01 2015-12-23 中国科学院苏州生物医学工程技术研究所 表达重组人fviii的整合质粒、细胞株及其构建方法和应用
RS61387B2 (sr) 2013-02-15 2024-06-28 Bioverativ Therapeutics Inc Gen optimizovanog faktora viii
ES2926798T3 (es) 2013-03-15 2022-10-28 Bioverativ Therapeutics Inc Formulaciones de polipéptido de factor VIII
EP2970966A4 (en) * 2013-03-15 2016-10-26 Univ North Carolina SYNTHETIC ADENOASSOZED VIRUS INVERTED REPEAT SEQUENCES
AU2014251099B2 (en) 2013-04-08 2019-01-17 The University Of Kansas Chimeric adeno-associated virus/ bocavirus parvovirus vector
US10548953B2 (en) 2013-08-14 2020-02-04 Bioverativ Therapeutics Inc. Factor VIII-XTEN fusions and uses thereof
US20160311885A1 (en) * 2013-08-14 2016-10-27 Biogen Ma Inc. Recombinant factor viii proteins
ES2813698T3 (es) 2013-09-12 2021-03-24 Biomarin Pharm Inc Vectores de AAV que comprenden un gen que codifica el factor VIII
EA201691111A1 (ru) 2014-01-10 2017-01-30 Байоджен Ма Инк. Химерные белки фактора viii и их применение
BR112016020688B1 (pt) 2014-03-09 2024-01-30 The Trustees Of The University Of Pennsylvania Vetores virais recombinantes, vírus adenoassociado recombinante, composição farmacêutica, bem como uso dos mesmos
CR20160519A (es) * 2014-05-13 2017-01-27 The Trustees Of The Univ Of Pennysylvania Composiciones que comprenden avv que expresa constructos de anticuerpos duales y sus usos
US10577627B2 (en) * 2014-06-09 2020-03-03 Voyager Therapeutics, Inc. Chimeric capsids
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
EP3221456B1 (en) 2014-11-21 2021-09-22 University of Florida Research Foundation, Inc. Genome-modified recombinant adeno-associated virus vectors
US11697825B2 (en) 2014-12-12 2023-07-11 Voyager Therapeutics, Inc. Compositions and methods for the production of scAAV
KR102554850B1 (ko) 2015-02-06 2023-07-13 더 유니버시티 오브 노쓰 캐롤라이나 엣 채플 힐 최적화된 인간 응고 인자 viii 유전자 발현 카세트 및 그의 용도
WO2016134338A1 (en) 2015-02-19 2016-08-25 University Of Florida Research Foundation, Inc. Recombinant aav vectors for gene therapy of human hematopoietic disorders
TWI707951B (zh) 2015-04-08 2020-10-21 美商健臻公司 過大腺相關載體之製造
AU2016260401B2 (en) 2015-05-14 2022-05-19 St. Jude Children's Research Hospital, Inc. Nucleic acid molecules containing spacers and methods of use thereof
PE20231949A1 (es) 2015-10-30 2023-12-05 Spark Therapeutics Inc VARIANTES DEL FACTOR VIII REDUCIDO CON CpG, COMPOSICIONES Y METODOS Y USOS PARA EL TRATAMIENTO DE TRASTORNOS DE LA HEMOSTASIA
KR20180118659A (ko) 2016-02-01 2018-10-31 바이오버라티브 테라퓨틱스 인크. 최적화된 viii 인자 유전자
IL261524B2 (en) 2016-03-03 2023-10-01 Univ Massachusetts End-capped linear double-stranded DNA for non-viral gene transfer
US12173305B2 (en) 2016-05-26 2024-12-24 University Of Iowa Research Foundation cis and trans requirements for terminal resolution of human bocavirus 1
KR102219859B1 (ko) 2016-06-24 2021-02-25 재단법인 목암생명과학연구소 재조합 단쇄 fviii 및 그 화학 접합물
FR3054841B1 (fr) 2016-08-05 2021-01-29 Centre Nat Rech Scient Systeme d'expression baculovirus
US11142775B2 (en) 2017-01-13 2021-10-12 University Of Iowa Research Foundation Bocaparvovirus small noncoding RNA and uses thereof
RU2020106730A (ru) 2017-07-14 2021-08-16 Онкорус, Инк. Инкапсулированные полинуклеотиды и способы их применения
US11549125B2 (en) 2017-08-09 2023-01-10 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Closed-ended, linear, duplex adenoassociated virus DNA, and uses thereof
CA3075168A1 (en) 2017-09-08 2019-03-14 Generation Bio Co. Modified closed-ended dna (cedna)
EP3679148A4 (en) 2017-09-08 2021-06-09 Generation Bio Co. LIPID NANOPARTICLE FORMULATIONS FROM NONVIRAL, CAPSID-FREE DNA VECTORS
KR20200057051A (ko) 2017-09-27 2020-05-25 시질론 테라퓨틱스, 인크. 활성 세포를 포함하는 방법, 조성물 및 이식 가능한 요소
JP2021505159A (ja) 2017-12-06 2021-02-18 ジェネレーション バイオ カンパニー 修飾型閉端ｄｎａ（ｃｅｄｎａ）を使用する遺伝子編集
JP2021511047A (ja) 2018-01-19 2021-05-06 ジェネレーション バイオ カンパニー 無細胞合成から得ることができる閉端ＤＮＡベクターおよびｃｅＤＮＡベクターを得るためのプロセス
AU2019221642A1 (en) 2018-02-14 2020-07-09 Generation Bio Co. Non-viral DNA vectors and uses thereof for antibody and fusion protein production
EP3755803A4 (en) 2018-02-22 2022-01-19 Generation Bio Co. CONTROLLED EXPRESSION OF TRANSGENES USING DNA (CEDNA) VECTORS
CA3092459A1 (en) 2018-03-02 2019-09-06 Generation Bio Co. Closed-ended dna (cedna) vectors for insertion of transgenes at genomic safe harbors (gsh) in humans and murine genomes
CA3092832A1 (en) 2018-03-02 2019-09-06 Generation Bio Co. Identifying and characterizing genomic safe harbors (gsh) in humans and murine genomes, and viral and non-viral vector compositions for targeted integration at an identified gsh loci
EP3762500A1 (en) 2018-03-06 2021-01-13 Voyager Therapeutics, Inc. Insect cell manufactured partial self-complementary aav genomes
CN112639110A (zh) 2018-06-22 2021-04-09 阿斯克肋匹奥生物制药公司 用于基因递送以在细胞内持续存在的载体
JP7602454B2 (ja) 2018-08-09 2024-12-18 バイオベラティブ セラピューティクス インコーポレイテッド 核酸分子及び非ウイルス遺伝子治療のためのそれらの使用
US20220042040A1 (en) 2018-09-17 2022-02-10 Universität Für Bodenkultur Wien Dual vector system for improved production of proteins in animal cells
UY38389A (es) 2018-09-27 2020-04-30 Sigilon Therapeutics Inc Dispositivos implantables para terapia celular y métodos relacionados
CN120349985A (zh) 2018-10-12 2025-07-22 建新公司 通过肝导向基因替代疗法治疗严重pku的改良的人pah的产生
WO2020097417A1 (en) 2018-11-09 2020-05-14 Generation Bio Co. Modified closed-ended dna (cedna) comprising symmetrical modified inverted terminal repeats
KR20210119416A (ko) 2019-01-24 2021-10-05 제너레이션 바이오 컴퍼니 폐쇄-말단 dna (cedna), 및 유전자 또는 핵산 치료 관련 면역 반응을 감소시키는 방법에서의 이의 용도
FR3092522B1 (fr) 2019-02-07 2021-09-10 Rubix S&I Système et procédé de nettoyage d’un habitacle de véhicule
CA3129321A1 (en) 2019-02-15 2020-08-20 Generation Bio Co. Modulation of rep protein activity in closed-ended dna (cedna) production
SG11202108864UA (en) 2019-03-06 2021-09-29 Generation Bio Co Closed-ended dna (cedna) and immune modulating compounds
KR20210151785A (ko) 2019-03-13 2021-12-14 제너레이션 바이오 컴퍼니 비바이러스성 dna 벡터 및 fviii 치료제 발현을 위한 이의 용도
PL3722434T3 (pl) 2019-04-12 2022-12-12 Freeline Therapeutics Limited Układ plazmidowy
CA3136545A1 (en) 2019-04-12 2020-10-15 Freeline Therapeutics Limited Plasmid system
EP3736286A1 (en) 2019-05-09 2020-11-11 Biotest AG Single chain factor viii molecule
EP3987043A4 (en) 2019-06-21 2023-07-19 Asklepios Biopharmaceutical, Inc. PRODUCTION OF VECTORS USING THE PHAGEN REPLICATION ORIGIN
US20220228171A1 (en) 2019-07-17 2022-07-21 Generation Bio Co. Compositions and production of nicked closed-ended dna vectors
WO2021216975A1 (en) 2020-04-23 2021-10-28 Aav Gene Therapeutics, Inc. Aav native-neuro platform and use for neuronal disease gene therapy
MX2023001109A (es) 2020-07-27 2023-04-24 Anjarium Biosciences Ag Composiciones de moleculas de adn, metodos para elaborar las mismas, y metodos de usos de las mismas.
US20220356490A1 (en) 2020-08-23 2022-11-10 Bioverativ Therapeutics Inc. Baculovirus expression system
US20220243201A1 (en) 2020-08-23 2022-08-04 Bioverativ Therapeutics Inc. Engineered itr sequences and methods of use
AU2021330936A1 (en) 2020-08-23 2023-05-04 Bioverativ Therapeutics Inc. MODIFIED BACULOVIRUS SYSTEM FOR IMPROVED PRODUCTION OF CLOSED-ENDED DNA (ceDNA)
AU2022334714A1 (en) 2021-08-23 2024-04-04 Bioverativ Therapeutics Inc. Closed-end dna production with inverted terminal repeat sequences
WO2023028440A2 (en) 2021-08-23 2023-03-02 Bioverativ Therapeutics Inc. Baculovirus expression system
US20230084036A1 (en) 2021-08-23 2023-03-16 Bioverativ Therapeutics Inc. Optimized factor viii genes
JP2024532261A (ja) 2021-08-23 2024-09-05 バイオベラティブ セラピューティクス インコーポレイテッド 操作ｉｔｒ配列および使用方法

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN111247251A (zh) * 2017-08-09 2020-06-05 比奥维拉迪维治疗股份有限公司 核酸分子及其用途

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