CO2021002965A2 - Moléculas de ácido nucleico y sus usos para la terapia génica no viral - Google Patents
Moléculas de ácido nucleico y sus usos para la terapia génica no viralInfo
- Publication number
- CO2021002965A2 CO2021002965A2 CONC2021/0002965A CO2021002965A CO2021002965A2 CO 2021002965 A2 CO2021002965 A2 CO 2021002965A2 CO 2021002965 A CO2021002965 A CO 2021002965A CO 2021002965 A2 CO2021002965 A2 CO 2021002965A2
- Authority
- CO
- Colombia
- Prior art keywords
- itr
- nucleic acid
- acid molecules
- gene therapy
- viral gene
- Prior art date
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- A—HUMAN NECESSITIES
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- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- 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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- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
- C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
- C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
- C12N15/86—Viral vectors
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- A61K31/70—Carbohydrates; Sugars; Derivatives thereof
- A61K31/7088—Compounds having three or more nucleosides or nucleotides
- 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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- A61K31/713—Double-stranded nucleic acids or oligonucleotides
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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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- A61K48/0025—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 non-active part clearly interacts with the delivered nucleic acid
- A61K48/0033—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 non-active part clearly interacts with the delivered nucleic acid the non-active part being non-polymeric
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- 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
- A61K48/0066—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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- C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
- C07K14/745—Blood coagulation or fibrinolysis factors
- C07K14/755—Factors VIII, e.g. factor VIII C (AHF), factor VIII Ag (VWF)
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- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; CARE OF BIRDS, FISHES, INSECTS; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K2227/00—Animals characterised by species
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- A01K—ANIMAL HUSBANDRY; CARE OF BIRDS, FISHES, INSECTS; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
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- C12N2750/14011—Parvoviridae
- C12N2750/14041—Use of virus, viral particle or viral elements as a vector
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- C12N2750/14311—Parvovirus, e.g. minute virus of mice
- C12N2750/14371—Demonstrated in vivo effect
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Abstract
La presente divulgación proporciona moléculas de ácido nucleico que comprenden una primera repetición terminal invertida (ITR), una segunda ITR, y un casete genético que codifica una secuencia diana. En algunas realizaciones, la secuencia diana codifica un miARN y/o una proteína terapéutica. En ciertas realizaciones, la proteína terapéutica comprende un factor de coagulación, un factor de crecimiento, una hormona, una citocina, un anticuerpo, un fragmento del mismo, y una combinación de los mismos. En algunas realizaciones, la primera ITR y/o la segunda ITR son una ITR de un virus no adenoasociado (AAV). La presente divulgación también proporciona métodos de tratamiento de un trastorno metabólico del hígado en un sujeto que comprenden administrar al sujeto la molécula de ácido nucleico o un polipéptido codificado de ese modo.
Applications Claiming Priority (2)
Application Number | Priority Date | Filing Date | Title |
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US201862716826P | 2018-08-09 | 2018-08-09 | |
PCT/US2019/045957 WO2020033863A1 (en) | 2018-08-09 | 2019-08-09 | Nucleic acid molecules and uses thereof for non-viral gene therapy |
Publications (1)
Publication Number | Publication Date |
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CO2021002965A2 true CO2021002965A2 (es) | 2021-03-19 |
Family
ID=67766358
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
CONC2021/0002965A CO2021002965A2 (es) | 2018-08-09 | 2021-03-04 | Moléculas de ácido nucleico y sus usos para la terapia génica no viral |
Country Status (15)
Country | Link |
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US (1) | US20200069817A1 (es) |
EP (1) | EP3833766A1 (es) |
JP (1) | JP2021533753A (es) |
KR (1) | KR20210042128A (es) |
CN (1) | CN113227385A (es) |
AU (1) | AU2019319984A1 (es) |
BR (1) | BR112021002017A2 (es) |
CA (1) | CA3108799A1 (es) |
CO (1) | CO2021002965A2 (es) |
IL (1) | IL280652A (es) |
MX (1) | MX2021001599A (es) |
PH (1) | PH12021500010A1 (es) |
SG (1) | SG11202101157VA (es) |
TW (1) | TW202023630A (es) |
WO (1) | WO2020033863A1 (es) |
Families Citing this family (15)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
US10842885B2 (en) | 2018-08-20 | 2020-11-24 | Ucl Business Ltd | Factor IX encoding nucleotides |
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 |
JP2023535632A (ja) | 2020-07-27 | 2023-08-18 | アンジャリウム バイオサイエンシズ エージー | Dna分子の組成物、その作製方法、及びその使用方法 |
BR112023002395A2 (pt) * | 2020-08-23 | 2023-03-21 | Bioverativ Therapeutics Inc | Sistema de baculovírus modificado para produção aprimorada de dna com extremidades fechadas (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 |
CA3211687A1 (en) * | 2021-03-19 | 2022-09-22 | Ozan ALKAN | 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 |
WO2022272056A2 (en) * | 2021-06-24 | 2022-12-29 | University Of Utah Research Foundation | Compositions and methods for treating pgm1 deficiency |
IL310879A (en) | 2021-08-23 | 2024-04-01 | Bioverativ Therapeutics Inc | Expression system in colovirus |
WO2023028441A1 (en) | 2021-08-23 | 2023-03-02 | Bioverativ Therapeutics Inc. | Engineered itr sequences and methods of use |
WO2023056331A1 (en) * | 2021-09-30 | 2023-04-06 | Bioverativ Therapeutics Inc. | Nucleic acids encoding factor viii polypeptides with reduced immunogenicity |
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 |
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 |
Family Cites Families (131)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
US4965188A (en) | 1986-08-22 | 1990-10-23 | Cetus Corporation | Process for amplifying, detecting, and/or cloning nucleic acid sequences using a thermostable enzyme |
US4683202A (en) | 1985-03-28 | 1987-07-28 | Cetus Corporation | Process for amplifying nucleic acid sequences |
US4683195A (en) | 1986-01-30 | 1987-07-28 | Cetus Corporation | Process for amplifying, detecting, and/or-cloning nucleic acid sequences |
US4868112A (en) | 1985-04-12 | 1989-09-19 | Genetics Institute, Inc. | Novel procoagulant proteins |
US4770999A (en) | 1985-04-22 | 1988-09-13 | 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 |
WO1988007089A1 (en) | 1987-03-18 | 1988-09-22 | Medical Research Council | Altered antibodies |
US6060447A (en) | 1987-05-19 | 2000-05-09 | Chiron Corporation | Protein complexes having Factor VIII:C activity and production thereof |
US5171844A (en) | 1987-06-12 | 1992-12-15 | Gist-Brocades N.W. | Proteins with factor viii activity: process for their preparation using genetically-engineered cells and pharmaceutical compositions containing them |
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 |
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 |
AU648020B2 (en) | 1989-02-21 | 1994-04-14 | Washington University | Modified forms of reproductive hormones |
SE465222C5 (sv) | 1989-12-15 | 1998-02-10 | Pharmacia & Upjohn Ab | Ett rekombinant, humant faktor VIII-derivat och förfarande för dess framställning |
MX9204374A (es) | 1991-07-25 | 1993-03-01 | Idec Pharma Corp | Anticuerpo recombinante y metodo para su produccion. |
SE504074C2 (sv) | 1993-07-05 | 1996-11-04 | Pharmacia Ab | Proteinberedning för subkutan, intramuskulär eller intradermal administrering |
GB9422383D0 (en) | 1994-11-05 | 1995-01-04 | Wellcome Found | Antibodies |
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 |
US6485726B1 (en) | 1995-01-17 | 2002-11-26 | The Brigham And Women's Hospital, Inc. | Receptor specific transepithelial transport of therapeutics |
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 |
US5869046A (en) | 1995-04-14 | 1999-02-09 | 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 |
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 |
GB9722131D0 (en) | 1997-10-20 | 1997-12-17 | Medical Res Council | Method |
US6528624B1 (en) | 1998-04-02 | 2003-03-04 | Genentech, Inc. | Polypeptide variants |
US6242195B1 (en) | 1998-04-02 | 2001-06-05 | Genentech, Inc. | Methods for determining binding of an analyte to a receptor |
DK1068241T3 (da) | 1998-04-02 | 2008-02-04 | Genentech Inc | Antistofvarianter og fragmenter deraf |
US6194551B1 (en) | 1998-04-02 | 2001-02-27 | Genentech, Inc. | Polypeptide variants |
GB9809951D0 (en) | 1998-05-08 | 1998-07-08 | Univ Cambridge Tech | Binding molecules |
CA2341029A1 (en) | 1998-08-17 | 2000-02-24 | Abgenix, Inc. | Generation of modified molecules with increased serum half-lives |
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 |
ES2694002T3 (es) | 1999-01-15 | 2018-12-17 | Genentech, Inc. | Polipéptido que comprende una región Fc de IgG1 humana variante |
CA2405709A1 (en) | 2000-04-12 | 2001-10-25 | Human Genome Sciences, Inc. | Albumin fusion proteins |
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 |
GB0029407D0 (en) | 2000-12-01 | 2001-01-17 | Affitech As | Product |
ES2649037T3 (es) | 2000-12-12 | 2018-01-09 | Medimmune, Llc | Moléculas con semividas prolongadas, composiciones y usos de las mismas |
EP1377306A1 (en) | 2001-03-09 | 2004-01-07 | Dyax Corp. | Serum albumin binding moieties |
DE60233047D1 (de) | 2001-05-14 | 2009-09-03 | Gbp Ip Llc | Lentivirale vektoren kodierend für gerinnungsfaktoren für die gentherapie |
MXPA04001982A (es) | 2001-09-04 | 2004-06-07 | Merck Patent Gmbh | Factor ix modificado. |
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 |
CA2471363C (en) | 2001-12-21 | 2014-02-11 | Human Genome Sciences, Inc. | Albumin fusion proteins |
US20040002587A1 (en) | 2002-02-20 | 2004-01-01 | Watkins Jeffry D. | Fc region variants |
US20040018557A1 (en) | 2002-03-01 | 2004-01-29 | Immunomedics, Inc. | Bispecific antibody point mutations for enhancing rate of clearance |
US7317091B2 (en) | 2002-03-01 | 2008-01-08 | Xencor, Inc. | Optimized Fc variants |
US20040132101A1 (en) | 2002-09-27 | 2004-07-08 | Xencor | Optimized Fc variants and methods for their generation |
CN100343393C (zh) | 2002-03-15 | 2007-10-17 | 布赖汉姆妇女医院 | 适合治疗剂全身性递送的中央气道给药 |
CA2495251C (en) | 2002-08-14 | 2018-03-06 | Macrogenics, Inc. | Fc.gamma.riib-specific antibodies and methods of use thereof |
DK2345671T3 (en) | 2002-09-27 | 2016-02-15 | Xencor Inc | Optimized Fc variants and methods for their formation |
AU2003286467B2 (en) | 2002-10-15 | 2009-10-01 | Abbvie Biotherapeutics Inc. | Alteration of FcRn binding affinities or serum half-lives of antibodies by mutagenesis |
GB2395337B (en) | 2002-11-14 | 2005-12-28 | Gary Michael Wilson | Warning Unit |
JP2006524039A (ja) | 2003-01-09 | 2006-10-26 | マクロジェニクス,インコーポレーテッド | 変異型Fc領域を含む抗体の同定および作製ならびにその利用法 |
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 |
JP2006524051A (ja) | 2003-04-24 | 2006-10-26 | フォンダッチォーネ・セントロ・サン・ラファエル・デル・モンテ・タボール | 両方向性合成プロモーターを備えたレンチウイルスベクターおよびその使用 |
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 |
US20080089892A1 (en) | 2004-01-12 | 2008-04-17 | Eli Lilly And Co. | Fc Region Variants |
EP1737890A2 (en) | 2004-03-24 | 2007-01-03 | 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 |
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 |
WO2006085967A2 (en) | 2004-07-09 | 2006-08-17 | Xencor, Inc. | OPTIMIZED ANTI-CD20 MONOCONAL ANTIBODIES HAVING Fc VARIANTS |
EP2471813B1 (en) | 2004-07-15 | 2014-12-31 | Xencor, Inc. | Optimized Fc variants |
WO2006047350A2 (en) | 2004-10-21 | 2006-05-04 | Xencor, Inc. | IgG IMMUNOGLOBULIN VARIANTS WITH OPTIMIZED EFFECTOR FUNCTION |
CA2591544A1 (en) * | 2004-12-15 | 2006-06-22 | The University Of North Carolina At Chapel Hill | Chimeric vectors |
US20080213233A1 (en) * | 2005-05-27 | 2008-09-04 | Shu Wang | Method of Delivering Nucleic Acid Molecules Into Embryonic Stem Cells Using Baculoviral Vectors |
KR20130114758A (ko) | 2005-05-27 | 2013-10-17 | 오스페달레 산 라파엘 에스.알.엘. | Mi-rna를 포함하는 유전자 벡터 |
JP2009504157A (ja) | 2005-08-12 | 2009-02-05 | ヒューマン ジノーム サイエンシーズ, インコーポレイテッド | アルブミン融合タンパク質 |
EP1979485A2 (en) * | 2006-01-31 | 2008-10-15 | 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 |
US7939632B2 (en) | 2006-06-14 | 2011-05-10 | Csl Behring Gmbh | Proteolytically cleavable fusion proteins with high molar specific activity |
WO2007149406A2 (en) | 2006-06-19 | 2007-12-27 | Nautilus Technology Llc | Modified coagulation factor ix polypeptides and use thereof for treatment |
GB0614780D0 (en) | 2006-07-25 | 2006-09-06 | Ucb Sa | Biological products |
BRPI0716744A2 (pt) | 2006-09-14 | 2016-10-04 | Human Genome Sciences Inc | proteínas de fusão de albumina |
US7700734B2 (en) | 2007-01-09 | 2010-04-20 | Shu-Wha Lin | Recombinant human factor IX and use thereof |
EP2144930A1 (en) | 2007-04-18 | 2010-01-20 | ZymoGenetics, Inc. | Single chain fc, methods of making and methods of treatment |
NZ581395A (en) | 2007-05-14 | 2012-08-31 | Biogen Idec Inc | Single-chain fc (scfc) regions, binding polypeptides comprising same, and methods related thereto |
CN101970678B (zh) | 2007-06-21 | 2014-08-20 | 慕尼黑科技大学 | 具有增加的体内和/或体外稳定性的生物学活性蛋白 |
CA2702363A1 (en) | 2007-10-15 | 2009-04-23 | The University Of North Carolina At Chapel Hill | Human factor ix variants with an extended half life |
AU2009244633A1 (en) | 2008-04-16 | 2009-11-12 | Bayer Healthcare Llc | Modified Factor IX polypeptides and uses thereof |
KR20110015551A (ko) | 2008-04-16 | 2011-02-16 | 바이엘 헬스케어 엘엘씨 | 제ix인자의 부위-지정 변형 |
EP2268807A2 (en) | 2008-04-21 | 2011-01-05 | Novo Nordisk A/S | Hyperglycosylated human coagulation factor ix |
DK2364362T3 (en) | 2008-11-12 | 2016-01-25 | Ospedale San Raffaele Srl | Gene vector FOR INDUCTION OF IMMUNE TOLERANCE transgene |
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 |
CN102348715B (zh) | 2009-02-03 | 2017-12-08 | 阿穆尼克斯运营公司 | 延伸重组多肽和包含该延伸重组多肽的组合物 |
US8716448B2 (en) | 2009-02-03 | 2014-05-06 | Amunix Operating Inc. | Coagulation factor VII compositions 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 |
ES2786039T3 (es) | 2009-04-30 | 2020-10-08 | Ospedale San Raffaele Srl | Vector génico |
WO2010140148A1 (en) | 2009-06-01 | 2010-12-09 | Yeda Research And Development Co . Ltd | Prodrugs containing albumin binding probe |
PT2440228T (pt) | 2009-06-08 | 2018-12-24 | Amunix Operating Inc | Polipéptidos de regulação da glicose e métodos de preparação e utilização dos mesmos |
EP2440241B1 (en) | 2009-06-08 | 2017-08-09 | Amunix Operating Inc. | Growth hormone polypeptides and methods of making and using same |
GB0911870D0 (en) * | 2009-07-08 | 2009-08-19 | Ucl Business Plc | Optimised coding sequence and promoter |
WO2011028344A2 (en) | 2009-08-25 | 2011-03-10 | Amunix Operating Inc. | Interleukin-1 receptor antagonist compositions and methods of making and using same |
WO2011088081A1 (en) * | 2010-01-12 | 2011-07-21 | The University Of North Carolina At Chapel Hill | Restrictive inverted terminal repeats for viral vectors |
US20130052226A1 (en) * | 2010-02-12 | 2013-02-28 | The Government Of The Usa, As Represented By The Secretary, Department Of Health & Human Services | Compositions and methods for preventing or treating a human parvovirus infection |
KR20230156435A (ko) | 2010-07-09 | 2023-11-14 | 바이오버라티브 테라퓨틱스 인크. | 인자 ix 폴리펩티드 및 이들의 사용 방법 |
EP2591006B1 (en) | 2010-07-09 | 2019-04-24 | Bioverativ Therapeutics Inc. | Processable single chain molecules and polypeptides made using same |
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 |
NZ626945A (en) | 2012-01-12 | 2016-10-28 | Biogen Ma Inc | Chimeric factor viii polypeptides and uses thereof |
CA2863329C (en) | 2012-01-12 | 2023-01-03 | Biogen Idec Ma Inc. | Methods of reducing immunogenicity against factor viii in individuals undergoing factor viii therapy |
KR20190094480A (ko) | 2012-02-15 | 2019-08-13 | 바이오버라티브 테라퓨틱스 인크. | 재조합 인자 viii 단백질 |
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 |
PT2882450T (pt) | 2012-07-11 | 2020-02-19 | Bioverativ Therapeutics Inc | Complexo de fator viii com xten e a proteína fator de von willebrand, e utilizações do mesmo |
HUE047933T2 (hu) | 2013-03-15 | 2020-05-28 | Bioverativ Therapeutics Inc | Faktor VIII polipeptid készítmények |
EP2970966A4 (en) * | 2013-03-15 | 2016-10-26 | Univ North Carolina | SYNTHETIC ADENOASSOZED VIRUS INVERTED REPEAT SEQUENCES |
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 |
EP4108254A1 (en) * | 2013-08-14 | 2022-12-28 | Bioverativ Therapeutics Inc. | Recombinant factor viii proteins |
SI3091997T1 (sl) | 2014-01-10 | 2022-10-28 | Bioverativ Therapeutics Inc. | Himerni proteini faktorja VIII in njihova uporaba |
MY186389A (en) * | 2014-05-13 | 2021-07-22 | Univ Pennsylvania | Compositions comprising aav expressing dual antibody constructs and uses thereof |
WO2015191508A1 (en) * | 2014-06-09 | 2015-12-17 | Voyager Therapeutics, Inc. | Chimeric capsids |
WO2016004113A1 (en) | 2014-06-30 | 2016-01-07 | Biogen Ma Inc. | Optimized factor ix gene |
EP3221456B1 (en) * | 2014-11-21 | 2021-09-22 | University of Florida Research Foundation, Inc. | Genome-modified recombinant adeno-associated virus vectors |
US10308705B2 (en) * | 2015-02-06 | 2019-06-04 | The University Of North Carolina At Chapel Hill | Optimized human clotting factor VIII gene expression cassettes and their use |
WO2016134338A1 (en) * | 2015-02-19 | 2016-08-25 | University Of Florida Research Foundation, Inc. | Recombinant aav vectors for gene therapy of human hematopoietic disorders |
JP7217630B2 (ja) * | 2016-02-01 | 2023-02-03 | 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 | 最適化第viii因子遺伝子 |
KR20200035130A (ko) * | 2017-08-09 | 2020-04-01 | 바이오버라티브 테라퓨틱스 인크. | 핵산 분자 및 이의 용도 |
-
2019
- 2019-08-09 BR BR112021002017-9A patent/BR112021002017A2/pt unknown
- 2019-08-09 US US16/537,192 patent/US20200069817A1/en active Pending
- 2019-08-09 CN CN201980065714.1A patent/CN113227385A/zh active Pending
- 2019-08-09 CA CA3108799A patent/CA3108799A1/en active Pending
- 2019-08-09 WO PCT/US2019/045957 patent/WO2020033863A1/en unknown
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- 2019-08-09 AU AU2019319984A patent/AU2019319984A1/en active Pending
- 2019-08-09 SG SG11202101157VA patent/SG11202101157VA/en unknown
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CA3108799A1 (en) | 2020-02-13 |
TW202023630A (zh) | 2020-07-01 |
MX2021001599A (es) | 2021-07-02 |
WO2020033863A1 (en) | 2020-02-13 |
US20200069817A1 (en) | 2020-03-05 |
SG11202101157VA (en) | 2021-03-30 |
AU2019319984A1 (en) | 2021-03-04 |
JP2021533753A (ja) | 2021-12-09 |
PH12021500010A1 (en) | 2021-10-11 |
KR20210042128A (ko) | 2021-04-16 |
EP3833766A1 (en) | 2021-06-16 |
IL280652A (en) | 2021-03-25 |
BR112021002017A2 (pt) | 2021-05-11 |
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