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MX2020001593A - Moleculas de acido nucleico y usos de las mismas. - Google Patents

Moleculas de acido nucleico y usos de las mismas.

Info

Publication number
MX2020001593A
MX2020001593A MX2020001593A MX2020001593A MX2020001593A MX 2020001593 A MX2020001593 A MX 2020001593A MX 2020001593 A MX2020001593 A MX 2020001593A MX 2020001593 A MX2020001593 A MX 2020001593A MX 2020001593 A MX2020001593 A MX 2020001593A
Authority
MX
Mexico
Prior art keywords
itr
nucleic acid
acid molecules
present disclosure
polypeptide
Prior art date
Application number
MX2020001593A
Other languages
English (en)
Inventor
Robert T Peters
Tongyao Liu
Alexey Seregin
Susannah Patarroyo-White
Douglas Drager
Jiayun Liu
Original Assignee
Bioverativ Therapeutics Inc
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Bioverativ Therapeutics Inc filed Critical Bioverativ Therapeutics Inc
Publication of MX2020001593A publication Critical patent/MX2020001593A/es

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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
    • 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
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/36—Blood coagulation or fibrinolysis factors
    • 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
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/36—Blood coagulation or fibrinolysis factors
    • A61K38/37—Factors VIII
    • 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
    • C07—ORGANIC CHEMISTRY
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    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • 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
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    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • 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)
    • C—CHEMISTRY; METALLURGY
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    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/775—Apolipopeptides
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    • 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)
    • C12N9/6432—Coagulation factor Xa (3.4.21.6)
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    • 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
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    • 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)
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    • 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)
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    • C12Y304/21—Serine endopeptidases (3.4.21)
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    • C12Y304/21—Serine endopeptidases (3.4.21)
    • C12Y304/21022—Coagulation factor IXa (3.4.21.22)
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    • C12Y304/21—Serine endopeptidases (3.4.21)
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Abstract

La presente descripción proporciona moléculas de ácido nucleico que comprenden una primera repetición terminal invertida (ITR), una segunda ITR y un casete génico que codifica un miARN y/o una proteína terapéutica. En ciertas modalidades, la proteína terapéutica comprende un factor de coagulación, por ejemplo, un polipéptido FVIII, un polipéptido FIX o un fragmento del mismo. En algunas modalidades, la primera ITR y/o la segunda ITR es una ITR de un virus no adenoasociado (AAV). La presente descripción también proporciona métodos para tratar trastornos hemorrágicos, tales como hemofilia, que comprenden administrar al sujeto la molécula de ácido nucleico o un polipéptido codificado por el mismo.
MX2020001593A 2017-08-09 2018-08-09 Moleculas de acido nucleico y usos de las mismas. MX2020001593A (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201762543297P 2017-08-09 2017-08-09
PCT/US2018/046110 WO2019032898A1 (en) 2017-08-09 2018-08-09 NUCLEIC ACID MOLECULES AND USES THEREOF

Publications (1)

Publication Number Publication Date
MX2020001593A true MX2020001593A (es) 2020-07-13

Family

ID=63638309

Family Applications (2)

Application Number Title Priority Date Filing Date
MX2020001593A MX2020001593A (es) 2017-08-09 2018-08-09 Moleculas de acido nucleico y usos de las mismas.
MX2024015674A MX2024015674A (es) 2017-08-09 2020-02-07 Moleculas de acido nucleico y usos de las mismas

Family Applications After (1)

Application Number Title Priority Date Filing Date
MX2024015674A MX2024015674A (es) 2017-08-09 2020-02-07 Moleculas de acido nucleico y usos de las mismas

Country Status (17)

Country Link
US (2) US12168776B2 (es)
EP (1) EP3665289A1 (es)
JP (3) JP7374883B2 (es)
KR (2) KR102780287B1 (es)
CN (2) CN111247251B (es)
AU (2) AU2018313921B2 (es)
BR (1) BR112020002394A2 (es)
CA (1) CA3072334A1 (es)
CO (1) CO2020002674A2 (es)
IL (2) IL322212A (es)
MX (2) MX2020001593A (es)
MY (1) MY203184A (es)
NZ (1) NZ762100A (es)
PH (1) PH12020500222A1 (es)
SG (1) SG11202000764RA (es)
TW (1) TWI904068B (es)
WO (1) WO2019032898A1 (es)

Families Citing this family (23)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
TWI904068B (zh) 2017-08-09 2025-11-11 美商生物化學醫療公司 核酸分子及其用途
CA3108799A1 (en) 2018-08-09 2020-02-13 Bioverativ Therapeutics Inc. Nucleic acid molecules and uses thereof for non-viral gene therapy
US10842885B2 (en) 2018-08-20 2020-11-24 Ucl Business Ltd Factor IX encoding nucleotides
GB201813528D0 (en) 2018-08-20 2018-10-03 Ucl Business Plc Factor IX encoding nucleotides
CN113454232B (zh) * 2019-02-15 2024-10-29 世代生物公司 封闭端dna（cedna）产生中对rep蛋白活性的调节
BR112021017853A2 (pt) * 2019-03-13 2021-11-30 Generation Bio Co Vetores de dna não virais e usos dos mesmos para expressar terapêuticas de fviii
EP3938515A4 (en) * 2019-03-13 2022-12-28 Generation Bio Co. NON-VIRAL DNA VECTORS AND THEIR USES FOR EXPRESSING PHENYLALANINE HYDROXYLASE (PAH) THERAPEUTIC AGENTS
CA3136720A1 (en) 2019-04-17 2020-10-22 Codiak Biosciences, Inc. Compositions of exosomes and aav
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
WO2021078834A1 (en) * 2019-10-22 2021-04-29 Genethon Chimeric acid-alpha glucosidase polypeptides and uses thereof
DE102020111571A1 (de) 2020-03-11 2021-09-16 Immatics US, Inc. Wpre-mutantenkonstrukte, zusammensetzungen und zugehörige verfahren
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
US20230109504A1 (en) 2020-04-09 2023-04-06 University Of Sheffield Gene therapy treatment
US20230272432A1 (en) 2020-07-27 2023-08-31 Anjarium Biosciences Ag Compositions of dna molecules, methods of making therefor, and methods of use thereof
WO2022046665A1 (en) 2020-08-23 2022-03-03 Bioverativ Therapeutics Inc. MODIFIED BACULOVIRUS SYSTEM FOR IMPROVED PRODUCTION OF CLOSED-ENDED DNA (ceDNA)
JP2023542132A (ja) * 2020-09-16 2023-10-05 ジェネレーション バイオ カンパニー Ｆｖｉｉｉ治療薬を発現させるための非ウイルス性ｄｎａベクター及びその使用
EP4392444A1 (en) * 2021-08-23 2024-07-03 Bioverativ Therapeutics Inc. Engineered itr sequences and methods of use
JP2024534123A (ja) 2021-08-23 2024-09-18 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 バキュロウイルス発現システム
KR20230085335A (ko) 2021-12-07 2023-06-14 서제표 사용자 맞춤형 심리상담 전문가 매칭 장치
WO2023202469A1 (zh) * 2022-04-19 2023-10-26 康霖生物科技（杭州）有限公司 一种用于遗传性凝血因子缺乏病治疗的核酸构建体及其用途
TW202424194A (zh) * 2022-09-23 2024-06-16 美商安斯泰來基因治療股份有限公司 用於治療神經肌肉病症之組成物及方法
GB202215198D0 (en) 2022-10-14 2022-11-30 Univ Sheffield Gene therapy treatment
WO2025114699A1 (en) 2023-11-30 2025-06-05 Cytomos Limited Cell selection

Family Cites Families (208)

* 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
US4683195A (en) 1986-01-30 1987-07-28 Cetus Corporation Process for amplifying, detecting, and/or-cloning 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
MX9203440A (es) 1985-04-12 1992-07-01 Genetics Inst Proteinas procoagulantes novedosas.
EP0218713B1 (en) 1985-04-22 1992-03-25 Genetics Institute, Inc. High yield production of active factor ix
KR910006424B1 (ko) 1985-08-21 1991-08-24 인코텍스 비.브이 편성브리프(brief) 제조방법
WO1987004187A1 (en) 1986-01-03 1987-07-16 Genetics Institute, Inc. METHOD FOR PRODUCING FACTOR VIII:c-TYPE PROTEINS
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
US5610278A (en) 1986-06-24 1997-03-11 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
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
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
CA2053864C (en) 1989-02-21 2001-11-20 Irving Boime Modified forms of reproductive hormones
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
DE669986T1 (de) 1992-11-13 1996-10-10 Idec Pharma Corp Völlig unfunktionelle konsensus-kozak-sequenzen zur säugetier-exprimierung.
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
JPH09501309A (ja) 1993-05-26 1997-02-10 アメリカ合衆国 アデノ関連性ウイルスレプタンパク質および細菌性タンパク質含有融合タンパク質
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
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
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
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
US6121022A (en) 1995-04-14 2000-09-19 Genentech, Inc. Altered polypeptides with increased half-life
US5869046A (en) 1995-04-14 1999-02-09 Genentech, Inc. Altered polypeptides with increased half-life
US5739277A (en) 1995-04-14 1998-04-14 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
DE69738522T2 (de) 1996-08-02 2009-04-02 Bristol-Myers Squibb Co. Ein verfahren zur inhibierung immunglobulininduzierter toxizität aufgrund von der verwendung von immunoglobinen in therapie und in vivo diagnostik
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
EP0981637B1 (en) 1997-03-14 2005-05-25 Biogen Idec Inc. Method for integrating genes at specific sites in mammalian cells via homologous recombination and vectors for accomplishing the same
GB9722131D0 (en) 1997-10-20 1997-12-17 Medical Res Council Method
US6194551B1 (en) 1998-04-02 2001-02-27 Genentech, Inc. Polypeptide variants
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
ATE375365T1 (de) 1998-04-02 2007-10-15 Genentech Inc Antikörper varianten und fragmente davon
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
AU8592298A (en) 1998-07-24 2000-02-14 Baylor College Of Medicine Rapid subcloning using site-specific recombination
AU770555B2 (en) 1998-08-17 2004-02-26 Abgenix, Inc. Generation of modified molecules with increased serum half-lives
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
DE69941905D1 (de) * 1998-11-10 2010-02-25 Univ North Carolina Virusvektoren und verfahren für ihre herstellung und verabreichung.
EP1006183A1 (en) 1998-12-03 2000-06-07 Max-Planck-Gesellschaft zur Förderung der Wissenschaften e.V. Recombinant soluble Fc receptors
EP1141024B1 (en) 1999-01-15 2018-08-08 Genentech, Inc. POLYPEPTIDE COMPRISING A VARIANT HUMAN IgG1 Fc REGION
US6737056B1 (en) 1999-01-15 2004-05-18 Genentech, Inc. Polypeptide variants with altered effector function
WO2001077137A1 (en) 2000-04-12 2001-10-18 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
EP1355919B1 (en) 2000-12-12 2010-11-24 MedImmune, LLC Molecules with extended half-lives, compositions and uses thereof
WO2002076489A1 (en) 2001-03-09 2002-10-03 Dyax Corp. Serum albumin binding moieties
EP1395293B1 (en) 2001-05-14 2009-07-22 Gbp Ip, Llc Lentiviral vectors encoding clotting factors for gene therapy
BR0212035A (pt) 2001-09-04 2004-08-03 Merck Patent Gmbh Fator ix modificado
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
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
EP2277889B1 (en) 2001-12-21 2014-07-09 Human Genome Sciences, Inc. Fusion proteins of albumin and interferon beta
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
US20040002587A1 (en) 2002-02-20 2004-01-01 Watkins Jeffry D. Fc region variants
EP1487879B1 (en) 2002-03-01 2012-12-26 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
US7317091B2 (en) 2002-03-01 2008-01-08 Xencor, Inc. Optimized Fc variants
EP1487992A4 (en) 2002-03-15 2007-10-31 Brigham & Womens Hospital ADMINISTRATION THROUGH THE CENTRAL AIRWAYS FOR THE SYSTEMATIC DELIVERY OF THERAPEUTICS
ES2381617T5 (es) 2002-08-14 2016-02-24 Macrogenics, Inc. Anticuerpos específicos frente a FcgammaRIIB y sus procedimientos de uso
WO2004029207A2 (en) 2002-09-27 2004-04-08 Xencor Inc. 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
AU2004204494B2 (en) 2003-01-09 2011-09-29 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
US20090010920A1 (en) 2003-03-03 2009-01-08 Xencor, Inc. Fc Variants Having Decreased Affinity for FcyRIIb
US8388955B2 (en) 2003-03-03 2013-03-05 Xencor, Inc. Fc variants
US8501464B2 (en) 2003-04-24 2013-08-06 Ospedale San Raffaele S.R.L. 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
WO2005077981A2 (en) 2003-12-22 2005-08-25 Xencor, Inc. Fc POLYPEPTIDES WITH NOVEL Fc LIGAND BINDING SITES
BRPI0506771A (pt) 2004-01-12 2007-05-22 Applied Molecular Evolution anticorpo, e, composição farmacêutica
JP4597188B2 (ja) 2004-03-09 2010-12-15 アイトゲネシシェ・テヒニーシェ・ホッホシューレ・チューリッヒ 多タンパク質適用のための新規発現ツール
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
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
CN103172731A (zh) 2004-07-15 2013-06-26 赞科股份有限公司 优化的Fc变体
WO2006033756A2 (en) 2004-08-23 2006-03-30 Nucleonics, Inc. Multiple rna polymerase iii promoter expression constructs
WO2006047350A2 (en) 2004-10-21 2006-05-04 Xencor, Inc. IgG IMMUNOGLOBULIN VARIANTS WITH OPTIMIZED EFFECTOR FUNCTION
ES2385837T3 (es) 2004-12-15 2012-08-01 The University Of North Carolina At Chapel Hill Vectores quiméricos
JP5956709B2 (ja) 2005-05-27 2016-07-27 オスペダーレ サン ラファエレ エス．アール．エル 遺伝子ベクター
JP5006312B2 (ja) 2005-05-27 2012-08-22 エイジェンシー フォー サイエンス， テクノロジー アンド リサーチ バキュロウイルスベクターを使用して核酸分子を胚性幹細胞に送達する方法
EP1924596A4 (en) 2005-08-12 2009-07-29 Human Genome Sciences Inc ALBUM INFUSION PROTEINS
US8110350B2 (en) 2005-11-17 2012-02-07 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
US20110190166A1 (en) * 2006-05-26 2011-08-04 Susan Wong 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
US8383388B2 (en) 2006-06-19 2013-02-26 Catalyst Biosciences, Inc. Modified coagulation factor IX polypeptides and use thereof for treatment
GB0614780D0 (en) 2006-07-25 2006-09-06 Ucb Sa Biological products
EP2061891B1 (en) 2006-08-24 2012-04-11 Virovek, Inc. Expression in insect cells of genes with overlapping open reading frames, methods and compositions therefor
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
WO2008131242A1 (en) 2007-04-18 2008-10-30 Zymogenetics, Inc. Single chain fc, methods of making and methods of treatment
CA2687117A1 (en) 2007-05-14 2008-11-27 Biogen Idec Ma Inc. Single-chain fc (scfc) regions, binding polypeptides comprising same, and methods related thereto
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
NZ580670A (en) 2007-06-21 2011-09-30 Univ Muenchen Tech Biological active proteins having increased in vivo and/or vitro stability
US9115373B2 (en) 2007-09-19 2015-08-25 Uniqure Ip B.V. Use of AAV replication machinery for improved protein production
US20110154516A1 (en) 2007-10-15 2011-06-23 Stafford Darrel W Human factor ix variants with an extended half life
KR20110005862A (ko) 2008-04-16 2011-01-19 바이엘 헬스케어 엘엘씨 변형된 제ｉｘ인자 폴리펩티드 및 이의 용도
CA2721362A1 (en) 2008-04-16 2009-11-19 Bayer Healthcare Llc Site-directed modification of factor ix
WO2009146210A2 (en) 2008-04-17 2009-12-03 Blood Systems, Inc. New human parvovirus: bocavirus
WO2009130198A2 (en) 2008-04-21 2009-10-29 Novo Nordisk A/S Hyperglycosylated human coagulation factor ix
GB0820631D0 (en) 2008-11-11 2008-12-17 London School Hygiene & Tropical Medicine Vectors
WO2010055413A1 (en) 2008-11-12 2010-05-20 Fondazione Centro San Raffaele Del Monte Tabor Gene vector for inducing transgene-specific immune tolerance
US8703717B2 (en) 2009-02-03 2014-04-22 Amunix Operating Inc. Growth hormone polypeptides and methods of making and using same
EA020843B1 (ru) 2009-02-03 2015-02-27 Амуникс Оперейтинг Инк. Удлиненные рекомбинантные полипептиды и содержащие их композиции
US8716448B2 (en) 2009-02-03 2014-05-06 Amunix Operating Inc. Coagulation factor VII compositions and methods of making and using same
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
PT2424571T (pt) 2009-04-30 2020-05-06 Ospedale San Raffaele Srl Vetor génico
WO2010140148A1 (en) 2009-06-01 2010-12-09 Yeda Research And Development Co . Ltd Prodrugs containing albumin binding probe
EP2440241B1 (en) 2009-06-08 2017-08-09 Amunix Operating Inc. Growth hormone polypeptides and methods of making and using same
ES2705249T3 (es) 2009-06-08 2019-03-22 Amunix Operating Inc Polipéptidos reguladores de glucosa y métodos para su producción y uso
EP2292781A1 (en) 2009-08-17 2011-03-09 Genethon Baculovirus-based production of biopharmaceuticals free of contaminating baculoviral virions
WO2011028344A2 (en) 2009-08-25 2011-03-10 Amunix Operating Inc. Interleukin-1 receptor antagonist compositions and methods of making and using same
ES2683695T3 (es) 2010-01-12 2018-09-27 The University Of North Carolina At Chapel Hill Repeticiones terminales invertidas restrictivas para vectores virales
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
WO2011112090A2 (en) 2010-03-11 2011-09-15 Amsterdam Molecular Therapeutics (Amt) Ip B.V. Method for identifying variant rep protein encoding nucleic acids
TW201217527A (en) 2010-07-09 2012-05-01 Biogen Idec Hemophilia Inc Processable single chain molecules and polypeptides made using same
CN103140237A (zh) 2010-07-09 2013-06-05 比奥根艾迪克依蒙菲利亚公司 因子ix多肽及其使用方法
US8865881B2 (en) 2011-02-22 2014-10-21 California Institute Of Technology Delivery of proteins using adeno-associated virus (AAV) vectors
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
ES2899848T3 (es) 2012-01-12 2022-03-15 Bioverativ Therapeutics Inc Reducción de la inmunogenicidad contra el factor VIII en individuos sometidos a una terapia con factor VIII
US11370827B2 (en) 2012-01-12 2022-06-28 Bioverativ Therapeutics Inc. Chimeric factor VIII polypeptides and uses thereof
EP2822577B1 (en) 2012-02-15 2019-02-06 Bioverativ Therapeutics Inc. Recombinant factor viii proteins
PT3564260T (pt) 2012-02-15 2023-01-18 Bioverativ Therapeutics Inc Composições de fator viii e métodos de produção e utilização das mesmas
US20150111955A1 (en) 2012-02-17 2015-04-23 The Children's Hospital Of Philadelphia Aav vector compositions and methods for gene transfer to cells, organs and tissues
CN110054699A (zh) 2012-07-11 2019-07-26 比奥贝拉蒂治疗公司 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
US10391152B2 (en) 2012-10-18 2019-08-27 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
JP6330026B2 (ja) 2013-03-15 2018-05-23 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 第ｖｉｉｉ因子ポリペプチド製剤
EP3517612A1 (en) 2013-03-15 2019-07-31 The University of North Carolina At Chapel Hill Synthetic adeno-associated virus inverted terminal repeats
CA2909085C (en) 2013-04-08 2023-08-29 University Of Iowa Research Foundation Chimeric adeno-associated virus/ bocavirus parvovirus vector
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
DK3091997T5 (da) 2014-01-10 2024-10-14 Bioverativ Therapeutics Inc Kimære faktor viii-proteiner og anvendelser deraf
PL3116900T3 (pl) 2014-03-09 2021-03-08 The Trustees Of The University Of Pennsylvania Kompozycje użyteczne w leczeniu niedoboru transkarbamylazy ornitynowej (otc)
UA123986C2 (uk) 2014-05-13 2021-07-07 Дзе Трастіз Оф Дзе Юніверсіті Оф Пенсильванія Композиція, яка містить aav, який експресує конструкцію антитіл з подвійною специфічністю, і її застосування
EP3151866B1 (en) 2014-06-09 2023-03-08 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
WO2016094783A1 (en) 2014-12-12 2016-06-16 Voyager Therapeutics, Inc. Compositions and methods for the production of scaav
WO2016127057A1 (en) 2015-02-06 2016-08-11 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
TWI707951B (zh) 2015-04-08 2020-10-21 美商健臻公司 過大腺相關載體之製造
EP3294309A4 (en) 2015-05-14 2019-01-16 St. Jude Children's Research Hospital, Inc. NUCLEIC ACID MOLECULES WITH SPACERS AND METHOD FOR 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
HRP20221089T1 (hr) 2016-02-01 2022-11-25 Bioverativ Therapeutics Inc. Optimizirani geni faktora viii
MX2018010633A (es) 2016-03-03 2019-06-13 Univ Massachusetts Acido desoxirribonucleico (adn) lineal duplex de extremos cerrados para transferencia genica no viral.
WO2017205739A1 (en) 2016-05-26 2017-11-30 University Of Iowa Research Foundation cis AND trans REQUIREMENTS FOR TERMINAL RESOLUTION OF HUMAN BOCAVIRUS 1
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
CA3069821A1 (en) 2017-07-14 2019-01-17 Oncorus, Inc. Encapsulated polynucleotides and methods of use
TWI904068B (zh) 2017-08-09 2025-11-11 美商生物化學醫療公司 核酸分子及其用途
EP3665290A1 (en) 2017-08-09 2020-06-17 The U.S.A. As Represented By The Secretary, Department Of Health And Human Services Closed-ended, linear, duplex adenoassociated virus dna, and uses thereof
MX2020002500A (es) 2017-09-08 2020-09-17 Generation Bio Co Adn de extremo cerrado modificado (adnec).
SG11202000765PA (en) 2017-09-08 2020-03-30 Generation Bio Co Lipid nanoparticle formulations of non-viral, capsid-free dna vectors
BR112020006149A2 (pt) 2017-09-27 2020-10-20 Sigilon Therapeutics, Inc. célula ativa manipulada, elemento implantável, e, composição farmacêutica.
CA3084185A1 (en) 2017-12-06 2019-06-13 Generation Bio Co. Gene editing using a modified closed-ended dna (cedna)
MX2020005790A (es) 2018-01-19 2020-10-28 Generation Bio Co Vectores de adn de extremo cerrado que pueden obtenerse de síntesis libre de células y un proceso para obtener vectores de ceadn.
US20220042035A1 (en) 2018-02-14 2022-02-10 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. REGULATED EXPRESSION OF TRANSGENES USING CLOSED-ENDED DNA VECTOR (CEDNA)
MA52116A (fr) 2018-03-02 2021-01-06 Generation Bio Co Vecteurs d'adn à extrémité fermée (cedna) pour l'insertion de transgènes au niveau de havres génomiques sécuritaires (gsh) dans des génomes humains et murins
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
EP3810782A2 (en) 2018-06-22 2021-04-28 Asklepios Biopharmaceutical, Inc. Vectors for gene delivery that persist within cells
CA3108799A1 (en) 2018-08-09 2020-02-13 Bioverativ Therapeutics Inc. Nucleic acid molecules and uses thereof for non-viral gene therapy
EP3853368A1 (en) 2018-09-17 2021-07-28 Universität für Bodenkultur Wien Dual vector system for improved production of proteins in animal cells
SG11202103614RA (en) 2018-10-12 2021-05-28 Genzyme Corp Generation of improved human pah for treatment of severe pku by liver-directed gene replacement therapy
CN113316640A (zh) 2018-11-09 2021-08-27 世代生物公司 包含对称的被修饰的反向末端重复序列的被修饰的封闭端dna(cedna)
CA3127799A1 (en) 2019-01-24 2020-07-30 Generation Bio Co. Close-ended dna (cedna) and use in methods of reducing gene or nucleic acid therapy related immune response
FR3092522B1 (fr) 2019-02-07 2021-09-10 Rubix S&I Système et procédé de nettoyage d’un habitacle de véhicule
CN113454232B (zh) 2019-02-15 2024-10-29 世代生物公司 封闭端dna（cedna）产生中对rep蛋白活性的调节
MA55223A (fr) 2019-03-06 2022-01-12 Generation Bio Co Adn à extrémité fermée (cedna) et composés de modulation immunitaire
BR112021017853A2 (pt) 2019-03-13 2021-11-30 Generation Bio Co Vetores de dna não virais e usos dos mesmos para expressar terapêuticas de fviii
AU2020272557A1 (en) 2019-04-12 2021-12-09 Ascend Gene And Cell Therapies Ltd Plasmid system
ES2928689T3 (es) 2019-04-12 2022-11-22 Freeline Therapeutics Ltd Sistema de plásmidos
EP3736286A1 (en) 2019-05-09 2020-11-11 Biotest AG Single chain factor viii molecule
WO2020257590A1 (en) 2019-06-21 2020-12-24 Asklepios Biopharmaceutical, Inc. Production of vectors using phage origin of replication
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
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
WO2022046665A1 (en) 2020-08-23 2022-03-03 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
EP4392443A1 (en) 2021-08-23 2024-07-03 Bioverativ Therapeutics Inc. Optimized factor viii genes
EP4392444A1 (en) 2021-08-23 2024-07-03 Bioverativ Therapeutics Inc. Engineered itr sequences and methods of use

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