LT3411478T - Optimizuoti viii faktoriaus genai - Google Patents

Optimizuoti viii faktoriaus genai

Info

Publication number
LT3411478T
LT3411478T LTEPPCT/US2017/015879T LTUS2017015879T LT3411478T LT 3411478 T LT3411478 T LT 3411478T LT US2017015879 T LTUS2017015879 T LT US2017015879T LT 3411478 T LT3411478 T LT 3411478T
Authority
LT
Lithuania
Prior art keywords
factor viii
optimized factor
viii genes
genes
optimized
Prior art date
Application number
LTEPPCT/US2017/015879T
Other languages
English (en)
Lithuanian (lt)
Inventor
Siyuan Tan
Tongyao 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 LT3411478T publication Critical patent/LT3411478T/lt

Links

Classifications

    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • 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
    • A61K48/0008—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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • 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
    • 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
    • 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
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/02—Fusion polypeptide containing a localisation/targetting motif containing a signal sequence
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/30—Non-immunoglobulin-derived peptide or protein having an immunoglobulin constant or Fc region, or a fragment thereof, attached thereto
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/31—Fusion polypeptide fusions, other than Fc, for prolonged plasma life, e.g. albumin
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2740/00—Reverse transcribing RNA viruses
    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
    • C12N2740/15011—Lentivirus, not HIV, e.g. FIV, SIV
    • C12N2740/15041—Use of virus, viral particle or viral elements as a vector
    • C12N2740/15043—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2740/00—Reverse transcribing RNA viruses
    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
    • C12N2740/16011—Human Immunodeficiency Virus, HIV
    • C12N2740/16041—Use of virus, viral particle or viral elements as a vector
    • C12N2740/16043—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2800/00—Nucleic acids vectors
    • C12N2800/22—Vectors comprising a coding region that has been codon optimised for expression in a respective host

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Biotechnology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Epidemiology (AREA)
  • Biophysics (AREA)
  • Hematology (AREA)
  • Microbiology (AREA)
  • Virology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Toxicology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Immunology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Diabetes (AREA)
  • Peptides Or Proteins (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
LTEPPCT/US2017/015879T 2016-02-01 2017-01-31 Optimizuoti viii faktoriaus genai LT3411478T (lt)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201662289696P 2016-02-01 2016-02-01
US201662409739P 2016-10-18 2016-10-18
PCT/US2017/015879 WO2017136358A1 (en) 2016-02-01 2017-01-31 Optimized factor viii genes

Publications (1)

Publication Number Publication Date
LT3411478T true LT3411478T (lt) 2022-09-26

Family

ID=58044178

Family Applications (1)

Application Number Title Priority Date Filing Date
LTEPPCT/US2017/015879T LT3411478T (lt) 2016-02-01 2017-01-31 Optimizuoti viii faktoriaus genai

Country Status (23)

Country Link
US (2) US11753461B2 (enExample)
EP (2) EP4137570A1 (enExample)
JP (3) JP7217630B2 (enExample)
KR (1) KR20180118659A (enExample)
CN (2) CN116218863A (enExample)
AU (3) AU2017214378B2 (enExample)
CA (1) CA3012695A1 (enExample)
CL (1) CL2018002054A1 (enExample)
CO (1) CO2018009120A2 (enExample)
DK (1) DK3411478T3 (enExample)
ES (1) ES2926585T3 (enExample)
HR (1) HRP20221089T1 (enExample)
HU (1) HUE059941T2 (enExample)
IL (2) IL320404A (enExample)
LT (1) LT3411478T (enExample)
MX (2) MX2018009375A (enExample)
PH (1) PH12018501628B1 (enExample)
PL (1) PL3411478T3 (enExample)
PT (1) PT3411478T (enExample)
RS (1) RS63548B1 (enExample)
SG (2) SG11201806398YA (enExample)
SI (1) SI3411478T1 (enExample)
WO (1) WO2017136358A1 (enExample)

Families Citing this family (24)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
FI3889173T3 (fi) 2013-02-15 2023-10-02 Bioverativ Therapeutics Inc Optimoitu tekijä viii:n geeni
WO2016004113A1 (en) 2014-06-30 2016-01-07 Biogen Ma Inc. Optimized factor ix gene
KR20180118659A (ko) 2016-02-01 2018-10-31 바이오버라티브 테라퓨틱스 인크. 최적화된 viii 인자 유전자
BR112020002394A2 (pt) * 2017-08-09 2020-07-28 Bioverativ Therapeutics Inc. moléculas de ácidos nucleicos e usos das mesmas
AU2019215063B2 (en) * 2018-02-01 2025-10-16 Bioverativ Therapeutics, Inc. Use of lentiviral vectors expressing Factor VIII
KR102245539B1 (ko) 2018-02-12 2021-04-29 주식회사 지앤피바이오사이언스 코어-쉘 구조의 마이크로 입자를 유효성분으로 포함하는 성장인자 유전자 발현 증가용 조성물
EP3759154A1 (en) 2018-03-02 2021-01-06 Sigilon Therapeutics, Inc. Biocompatible hydrogel capsules and process for preparing same
AU2019226587A1 (en) 2018-03-02 2020-09-03 Sigilon Therapeutics, Inc. Afibrotic compounds, devices, and uses thereof
WO2019195055A1 (en) 2018-04-04 2019-10-10 Sigilon Therapeutics, Inc. Implantable particles and related methods
KR102925832B1 (ko) * 2018-08-09 2026-02-11 바이오버라티브 테라퓨틱스 인크. 핵산 분자 및 비바이러스 유전자 치료를 위한 이의 용도
BR112021010047A2 (pt) * 2018-12-06 2021-08-24 Bioverativ Therapeutics Inc. Uso de vetores lentivirais expressando o fator ix
US10654911B1 (en) * 2019-04-02 2020-05-19 Beijing Neoletix Biological Technology Co., Ltd. Vector co-expressing truncated von Willebrand factor and factor VIII
US20220323519A1 (en) 2019-04-17 2022-10-13 Codiak Biosciences, Inc. Compositions of exosomes and aav
US12403164B2 (en) * 2019-09-30 2025-09-02 Bioverativ Therapeutics Inc. Lentiviral vector formulations
CN111647625A (zh) * 2019-12-25 2020-09-11 深圳三智医学科技有限公司 一种提高人凝血因子ix表达水平的方法
CN111808863B (zh) * 2020-06-23 2021-05-28 康霖生物科技（杭州）有限公司 一种密码子优化的凝血因子viii基因及其构建体
KR20230074703A (ko) 2020-06-24 2023-05-31 바이오버라티브 테라퓨틱스 인크. 유리 인자 viii을 상기 단백질이 발현되도록 변형된 렌티바이러스 벡터의 제제로부터 제거하는 방법
WO2023028441A1 (en) 2021-08-23 2023-03-02 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
IL310997A (en) 2021-08-23 2024-04-01 Bioverativ Therapeutics Inc Factor VIII gene optimization
EP4408874A1 (en) * 2021-09-30 2024-08-07 Bioverativ Therapeutics Inc. Nucleic acids encoding factor viii polypeptides with reduced immunogenicity
WO2023205300A2 (en) * 2022-04-20 2023-10-26 Asklepios Biopharmaceutical, Inc. Therapeutic adeno-associated virus using codon optimized nucleic acid encoding factor viii
PE20251064A1 (es) * 2022-04-28 2025-04-09 Biocad Joint Stock Co Acido nucleico aislado que codifica una proteina de fusion basada en fviii-bdd y en un peptido senal heterologo, y uso de la misma
WO2025219525A1 (en) 2024-04-19 2025-10-23 Fondazione Telethon Combinations, inhibitors, viral vectors, methods, and uses for in vivo gene therapy and gene editing

Family Cites Families (193)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4704362A (en) 1977-11-08 1987-11-03 Genentech, Inc. Recombinant cloning vehicle microbial polypeptide expression
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
AU5772886A (en) 1985-04-12 1986-11-05 Genetics Institute Inc. Novel procoagulant proteins
KR910006424B1 (ko) 1985-08-21 1991-08-24 인코텍스 비.브이 편성브리프(brief) 제조방법
JP2525022B2 (ja) 1986-01-03 1996-08-14 ジェネティックス・インスチチュ−ト・インコ−ポレ−テッド ▲ＶＩＩＩ▼：ｃ因子型タンパク質の改良生産方法
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
EP0307434B2 (en) 1987-03-18 1998-07-29 Scotgen Biopharmaceuticals, Inc. 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
ATE148171T1 (de) 1989-02-21 1997-02-15 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
MX9204374A (es) 1991-07-25 1993-03-01 Idec Pharma Corp Anticuerpo recombinante y metodo para su produccion.
ES2088838T3 (es) 1992-11-13 2004-01-01 Idec Pharmaceuticals Corporation Secuencias kozak de consenso totalmente alteradas destinadas a la expresion en los mamiferos.
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
SE504074C2 (sv) 1993-07-05 1996-11-04 Pharmacia Ab Proteinberedning för subkutan, intramuskulär eller intradermal administrering
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
US6818439B1 (en) * 1994-12-30 2004-11-16 Chiron Corporation Methods for administration of recombinant gene delivery vehicles for treatment of hemophilia and other disorders
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
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
US5739277A (en) 1995-04-14 1998-04-14 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
SE9503380D0 (sv) 1995-09-29 1995-09-29 Pharmacia Ab Protein derivatives
US6013516A (en) 1995-10-06 2000-01-11 The Salk Institute For Biological Studies Vector and method of use for nucleic acid delivery to non-dividing cells
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
WO1998005787A1 (en) 1996-08-02 1998-02-12 Bristol-Myers Squibb Company A method for inhibiting immunoglobulin-induced toxicity resulting from the use of immunoglobulins in therapy and in vivo diagnosis
AU723497C (en) 1996-09-06 2001-10-11 Trustees Of The University Of Pennsylvania, The Method for recombinant adeno-associated virus-directed gene therapy
GB9621680D0 (en) 1996-10-17 1996-12-11 Oxford Biomedica Ltd Lentiviral vectors
GB2325003B (en) 1996-10-17 2000-05-10 Oxford Biomedica Ltd Rectroviral vectors
GB9622500D0 (en) 1996-10-29 1997-01-08 Oxford Biomedica Ltd Therapeutic gene
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
BR9808584A (pt) 1997-03-14 2000-05-23 Idec Pharma Corp Processo para integrar genes em sìtios especìficos em células de mamìferos via recombinação homóloga e vetores para obter os mesmos
US6207455B1 (en) 1997-05-01 2001-03-27 Lung-Ji Chang Lentiviral vectors
WO1999003496A1 (en) 1997-07-21 1999-01-28 The University Of North Carolina At Chapel Hill Factor ix antihemophilic factor with increased clotting activity
GB9722131D0 (en) 1997-10-20 1997-12-17 Medical Res Council Method
US5994136A (en) 1997-12-12 1999-11-30 Cell Genesys, Inc. Method and means for producing high titer, safe, recombinant lentivirus vectors
US6194551B1 (en) 1998-04-02 2001-02-27 Genentech, Inc. Polypeptide variants
US6242195B1 (en) 1998-04-02 2001-06-05 Genentech, Inc. Methods for determining binding of an analyte to a receptor
US6528624B1 (en) 1998-04-02 2003-03-04 Genentech, Inc. Polypeptide variants
IL138608A0 (en) 1998-04-02 2001-10-31 Genentech Inc Antibody variants and fragments thereof
GB9809951D0 (en) 1998-05-08 1998-07-08 Univ Cambridge Tech Binding molecules
EP1105427A2 (en) 1998-08-17 2001-06-13 Abgenix, Inc. Generation of modified molecules with increased serum half-lives
US20090148906A1 (en) 1998-09-29 2009-06-11 Shire Human Genetic Therapies, Inc. A Delaware Corporation Optimized messenger rna
US6924365B1 (en) 1998-09-29 2005-08-02 Transkaryotic Therapies, Inc. Optimized messenger RNA
US6103526A (en) 1998-10-08 2000-08-15 Protein Sciences Corporation Spodoptera frugiperda single cell suspension cell line in serum-free media, methods of producing and using
EP1006183A1 (en) 1998-12-03 2000-06-07 Max-Planck-Gesellschaft zur Förderung der Wissenschaften e.V. Recombinant soluble Fc receptors
KR100940380B1 (ko) 1999-01-15 2010-02-02 제넨테크, 인크. 효과기 기능이 변화된 폴리펩티드 변이체
US6737056B1 (en) 1999-01-15 2004-05-18 Genentech, Inc. Polypeptide variants with altered effector function
AU778698B2 (en) 1999-04-29 2004-12-16 Miltenyi Biotec B.V. & Co. KG Method and means for producing high titer, safe, recombinant lentivirus vectors
CA2401327C (en) 2000-03-03 2014-05-06 Valentis, Inc. Nucleic acid formulations comprising poly-amino acids for gene delivery and methods of use
EP1274720A4 (en) 2000-04-12 2004-08-18 Human Genome Sciences Inc FUSION PROTEINS CONTAINING ALBUMIN
US7125841B2 (en) 2000-11-14 2006-10-24 The 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
JP4336498B2 (ja) 2000-12-12 2009-09-30 メディミューン，エルエルシー 延長した半減期を有する分子ならびにその組成物および用途
US20020159978A1 (en) 2001-02-06 2002-10-31 James Allen Muscle-directed gene transfer by use of recombinant AAV-1 and AAV-6 virions
US6888319B2 (en) 2001-03-01 2005-05-03 Palomar Medical Technologies, Inc. Flashlamp drive circuit
EP1377306A1 (en) 2001-03-09 2004-01-07 Dyax Corp. Serum albumin binding moieties
US20040106794A1 (en) 2001-04-16 2004-06-03 Schering Corporation 3,4-Di-substituted cyclobutene-1,2-diones as CXC-chemokine receptor ligands
EP1395293B1 (en) 2001-05-14 2009-07-22 Gbp Ip, Llc Lentiviral vectors encoding clotting factors for gene therapy
JP2005501547A (ja) 2001-09-04 2005-01-20 メルク　パテント　ゲゼルシャフト　ミット　ベシュレンクテル　ハフトング 修飾された第ｉｘ因子
AU2002360355B2 (en) 2001-11-09 2005-07-07 Government Of The United States Of America, Department Of Health And Human Services Production of adeno-associated virus in insect cells
SG10202108118RA (en) 2001-11-13 2021-08-30 Univ Pennsylvania A method of detecting and/or identifying adeno-associated virus (aav) sequences and isolating novel sequences identified thereby
ES2975413T3 (es) 2001-12-17 2024-07-05 Univ Pennsylvania Secuencias de serotipo 8 de virus adenoasociado (AAV), vectores que las contienen y usos de las mismas
KR101271635B1 (ko) 2001-12-21 2013-06-12 휴먼 게놈 사이언시즈, 인코포레이티드 알부민 융합 단백질
PT1463751E (pt) 2001-12-21 2013-08-26 Human Genome Sciences Inc Proteínas de fusão de albumina
US20080194481A1 (en) 2001-12-21 2008-08-14 Human Genome Sciences, Inc. Albumin Fusion Proteins
US6899917B2 (en) 2001-12-27 2005-05-31 3M Innovative Properties Company Powdered epoxy composition
US20040002587A1 (en) 2002-02-20 2004-01-01 Watkins Jeffry D. Fc region variants
AU2003209446B2 (en) 2002-03-01 2008-09-25 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
WO2003077834A2 (en) 2002-03-15 2003-09-25 The Brigham And Women's Hospital, Inc. Central airway administration for systemic delivery of therapeutics
US6615782B1 (en) 2002-04-12 2003-09-09 Delphi Technologies, Inc. Two-step finger follower rocker arm
US7211558B2 (en) 2002-05-22 2007-05-01 The Children's Hospital Of Philadelphia Compositions and methods for the treatment of hemophilia A
WO2004016750A2 (en) 2002-08-14 2004-02-26 Macrogenics, Inc. FcϜRIIB-SPECIFIC ANTIBODIES AND METHODS OF USE THEREOF
WO2004029207A2 (en) 2002-09-27 2004-04-08 Xencor Inc. Optimized fc variants and methods for their generation
WO2004035752A2 (en) 2002-10-15 2004-04-29 Protein Design Labs, 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
EP2368578A1 (en) 2003-01-09 2011-09-28 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
US9715692B1 (en) 2003-04-11 2017-07-25 Facebook, Inc. System for managing bids for pay-per-click search engines
DK1616012T3 (da) 2003-04-24 2008-04-21 San Raffaele Centro Fond Lentivirale vektorer bærende syntetiske bidirektionelle promotorer og anvendelser deraf
TWI353991B (en) 2003-05-06 2011-12-11 Syntonix Pharmaceuticals Inc Immunoglobulin chimeric monomer-dimer hybrids
EP2530157B1 (en) 2003-07-31 2016-09-28 Regulus Therapeutics Inc. Oligomeric compounds and compositions for use in modulation of miRNAs
GB0324368D0 (en) 2003-10-17 2003-11-19 Univ Cambridge Tech Polypeptides including modified constant regions
GB0325379D0 (en) 2003-10-30 2003-12-03 Oxford Biomedica Ltd Vectors
WO2005077981A2 (en) 2003-12-22 2005-08-25 Xencor, Inc. Fc POLYPEPTIDES WITH NOVEL Fc LIGAND BINDING SITES
EP1706424B1 (en) 2004-01-12 2009-07-22 Applied Molecular Evolution, Inc. Fc region variants
WO2005092925A2 (en) 2004-03-24 2005-10-06 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
EP1769078A1 (en) 2004-07-01 2007-04-04 VIRxSYS Corporation Vector packaging cell line
WO2006085967A2 (en) 2004-07-09 2006-08-17 Xencor, Inc. OPTIMIZED ANTI-CD20 MONOCONAL ANTIBODIES HAVING Fc VARIANTS
EP3342782B1 (en) 2004-07-15 2022-08-17 Xencor, Inc. Optimized fc variants
DK1804839T3 (da) 2004-09-22 2012-04-10 St Jude Childrens Res Hospital Forbedret ekspression af faktor ix i genterapivektorer
WO2006047350A2 (en) 2004-10-21 2006-05-04 Xencor, Inc. IgG IMMUNOGLOBULIN VARIANTS WITH OPTIMIZED EFFECTOR FUNCTION
ES2564823T3 (es) 2005-05-27 2016-03-29 Ospedale San Raffaele S.R.L. Vector génico que comprende miARN
EP1908797B1 (en) 2005-07-05 2012-06-06 Kaneka Corporation Methacrylic resin composition
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
AU2006304997B2 (en) 2005-10-20 2012-03-01 Uniqure Ip B.V. Improved AAV vectors produced in insect cells
US8476234B2 (en) 2006-02-03 2013-07-02 Prolor Biotech Inc. Long-acting coagulation factors and methods of producing same
US8048848B2 (en) 2006-02-03 2011-11-01 Prolor Biotech Ltd. Long-acting interferons and derivatives thereof and methods thereof
KR20090019770A (ko) 2006-03-13 2009-02-25 티아이알 테크놀로지 엘피 고상 조명 시스템용 적응형 제어 장치 및 전압 결정 방법
CN101511373B (zh) 2006-06-19 2015-12-09 阿斯克肋匹奥生物制药公司 用于基因治疗的修饰的因子ⅷ和因子ⅸ基因和载体
WO2007149406A2 (en) 2006-06-19 2007-12-27 Nautilus Technology Llc Modified coagulation factor ix polypeptides and use thereof for treatment
ES2785223T3 (es) 2006-06-21 2020-10-06 Uniqure Ip Bv Células de insecto para la producción de vectores de AAV
WO2012170289A1 (en) 2011-06-03 2012-12-13 The Regents Of The University Of Michigan Method of producing factor viii proteins by recombinant methods
GB0614780D0 (en) 2006-07-25 2006-09-06 Ucb Sa Biological products
CA2663352A1 (en) 2006-09-14 2008-03-20 Human Genome Sciences, Inc. Albumin fusion proteins
US8167585B2 (en) 2006-09-28 2012-05-01 Siemens Industry, Inc. Devices and/or systems for mounting an auxiliary blower
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
EP2158318A2 (en) 2007-05-14 2010-03-03 Biogen Idec MA, Inc. Single-chain fc (scfc) regions, binding polypeptides comprising same, and methods related thereto
JP5351889B2 (ja) 2007-06-21 2013-11-27 テクニシェ ユニバーシタット ミュンヘン インビボおよび／またはインビトロ安定性が増大した生物学的に活性なタンパク質
EP2209487A4 (en) 2007-10-15 2012-06-20 Univ North Carolina Human factor ix variants with an extended half life
WO2009075772A1 (en) 2007-12-06 2009-06-18 Wyeth Crystal structure of human factor viii and uses thereof
CA2721362A1 (en) 2008-04-16 2009-11-19 Bayer Healthcare Llc Site-directed modification of factor ix
MX2010011345A (es) 2008-04-16 2011-02-23 Bayer Healthcare Llc Polipeptidos modificados del factor ix y usos de los mismos.
JP5665733B2 (ja) 2008-04-21 2015-02-04 ノボ ノルディスク ヘルス ケア アーゲー 高度にグリコシル化されたヒト凝固第ｉｘ因子
WO2009153563A1 (en) 2008-06-18 2009-12-23 Oxford Biomedica (Uk) Limited Virus purification
US10273502B2 (en) 2008-06-18 2019-04-30 Oxford Biomedica (Uk) Limited Virus purification
EP2149603A1 (en) 2008-07-28 2010-02-03 DRK-Blutspendedienst Baden-Württemberg-Hessen gGmbH Factor IX variants with clotting activity in absence of their cofactor and their use for treating bleeding disorders
FI3581650T3 (fi) 2008-09-15 2023-03-23 Uniqure Biopharma B V IX-tekijän polypeptidimutantti, sen käyttöjä ja menetelmä sen valmistamiseksi
US9938540B2 (en) 2008-11-12 2018-04-10 Ospedale San Raffaele S.R.L. Gene vector for inducing transgene-specific immune tolerance
CN108530543B (zh) 2009-02-03 2023-06-23 阿穆尼克斯制药公司 延伸重组多肽和包含该延伸重组多肽的组合物
US8680050B2 (en) 2009-02-03 2014-03-25 Amunix Operating Inc. Growth hormone polypeptides fused to extended recombinant polypeptides and methods of making and using same
US8703717B2 (en) 2009-02-03 2014-04-22 Amunix Operating Inc. Growth hormone polypeptides and methods of making and using same
US9050269B2 (en) 2009-03-10 2015-06-09 The Trustees Of The University Of Pennsylvania Protection of virus particles from phagocytosis by expression of CD47
US20120093840A1 (en) 2009-04-06 2012-04-19 Novo Nordisk A/S Targeted delivery of factor viii proteins to platelets
ES2786039T3 (es) 2009-04-30 2020-10-08 Ospedale San Raffaele Srl Vector génico
WO2010138263A2 (en) 2009-05-28 2010-12-02 University Of Massachusetts Novel aav 's and uses thereof
WO2010140148A1 (en) 2009-06-01 2010-12-09 Yeda Research And Development Co . Ltd Prodrugs containing albumin binding probe
DK2440241T3 (en) 2009-06-08 2017-10-02 Amunix Operating Inc GROWTH HORMON POLYPEPTIDES AND PROCEDURES FOR PREPARING AND USING THEREOF
JP5839597B2 (ja) 2009-06-08 2016-01-06 アムニクス オペレーティング インコーポレイテッド グルコース調節ポリペプチド並びにその作成及び使用方法
GB0911870D0 (en) 2009-07-08 2009-08-19 Ucl Business Plc Optimised coding sequence and promoter
CN102741275B (zh) 2009-08-24 2016-08-03 阿穆尼克斯运营公司 凝血因子ix组合物及其制备和使用方法
WO2011028344A2 (en) 2009-08-25 2011-03-10 Amunix Operating Inc. Interleukin-1 receptor antagonist compositions and methods of making and using same
SG10201405434VA (en) 2009-09-18 2014-10-30 Amgen Res Munich Gmbh Dosage regimen for administering an epcamxcd3 bispecific antibody
US8326547B2 (en) 2009-10-07 2012-12-04 Nanjingjinsirui Science & Technology Biology Corp. Method of sequence optimization for improved recombinant protein expression using a particle swarm optimization algorithm
WO2011069164A2 (en) 2009-12-06 2011-06-09 Biogen Idec Ma Inc. Factor viii-fc chimeric and hybrid polypeptides, and methods of use thereof
MX2012009809A (es) 2010-02-23 2012-11-23 Sanofi Sa Anticuerpos anti - integrina alfa2 y sus usos.
EP3567106A1 (en) 2010-04-23 2019-11-13 University of Massachusetts Cns targeting aav vectors and methods of use thereof
HRP20191305T1 (hr) 2010-07-09 2019-10-18 Bioverativ Therapeutics Inc. Preradive jednolančane molekule i polipeptidi izrađeni koristeći iste
PT3552619T (pt) 2010-07-09 2025-10-23 Bioverativ Therapeutics Inc Polipéptidos de fator ix e métodos de uso dos mesmos
EP3508573A1 (en) 2010-07-09 2019-07-10 Bioverativ Therapeutics Inc. Systems for factor viii processing and methods thereof
US20130017997A1 (en) 2010-08-19 2013-01-17 Amunix Operating Inc. Factor VIII Compositions and Methods of Making and Using Same
AU2012282875B2 (en) 2011-07-08 2016-04-21 Bioverativ Therapeutics Inc. Factor VIII chimeric and hybrid polypeptides, and methods of use thereof
WO2013093760A2 (en) 2011-12-19 2013-06-27 Grifols, S.A. Compositions, methods, and kits for preparing sialylated recombinant proteins
EP2804623B1 (en) 2012-01-12 2019-08-07 Bioverativ Therapeutics Inc. Chimeric factor viii polypeptides and uses thereof
LT3564260T (lt) 2012-02-15 2023-01-10 Bioverativ Therapeutics Inc. Viii faktoriaus kompozicijos ir jų gamybos bei panaudojimo būdai
EP2822577B1 (en) 2012-02-15 2019-02-06 Bioverativ Therapeutics Inc. Recombinant factor viii proteins
CN103316356B (zh) 2012-03-22 2016-08-17 北京三诺佳邑生物技术有限责任公司 一种重组慢病毒载体制剂
WO2013151663A1 (en) 2012-04-02 2013-10-10 modeRNA Therapeutics Modified polynucleotides for the production of membrane proteins
EP2863940A4 (en) * 2012-06-08 2016-08-10 Biogen Ma Inc Chimeric clotting factors
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
RU2500816C1 (ru) 2012-07-19 2013-12-10 Общество с ограниченной ответственностью "Международный биотехнологический центр "Генериум" ("МБЦ "Генериум") РЕКОМБИНАНТНАЯ ПЛАЗМИДНАЯ ДНК рАК380, КОДИРУЮЩАЯ ПОЛИПЕПТИД РЕКОМБИНАНТНОГО ФАКТОРА IХ СВЕРТЫВАЕМОСТИ КРОВИ ЧЕЛОВЕКА, ЛИНИЯ КЛЕТОК Cricetulus griseus CHO 1E6 - ПРОДУЦЕНТ РЕКОМБИНАНТНОГО ФАКТОРА IХ СВЕРТЫВАЕМОСТИ КРОВИ ЧЕЛОВЕКА И СПОСОБ ПОЛУЧЕНИЯ ПОЛИПЕПТИДА, ОБЛАДАЮЩЕГО АКТИВНОСТЬЮ РЕКОМБИНАНТНОГО ФАКТОРА IХ
AU2013336601B2 (en) * 2012-10-26 2018-01-25 Vrije Universiteit Brussel Vector for liver-directed gene therapy of hemophilia and methods and use thereof
FI3889173T3 (fi) * 2013-02-15 2023-10-02 Bioverativ Therapeutics Inc Optimoitu tekijä viii:n geeni
GB201304973D0 (en) * 2013-03-19 2013-05-01 Profactor Pharma Ltd Recombinant protein
TWI667255B (zh) 2013-08-14 2019-08-01 美商生物化學醫療公司 因子ｖｉｉｉ－ｘｔｅｎ融合物及其用途
EP2881463A1 (en) 2013-12-09 2015-06-10 DRK-Blutspendedienst Baden-Württemberg-Hessen gGmbH Factor IX variants with clotting activity in absence of their cofactor and/or with increased F.IX clotting activity and their use for treating bleeding disorders
SG11201605242YA (en) 2014-01-10 2016-07-28 Biogen Ma Inc Factor viii chimeric proteins and uses thereof
WO2016004113A1 (en) 2014-06-30 2016-01-07 Biogen Ma Inc. Optimized factor ix gene
GB201412494D0 (en) 2014-07-14 2014-08-27 Ospedale San Raffaele And Fond Telethon Vector production
TWI707951B (zh) 2015-04-08 2020-10-21 美商健臻公司 過大腺相關載體之製造
EP3283126B1 (en) 2015-04-16 2019-11-06 Emory University Recombinant promoters and vectors for protein expression in liver and use thereof
IL257231B (en) 2015-08-03 2022-09-01 Bioverativ Therapeutics Inc Factor ix fusion proteins and methods of making and using same
US10724006B2 (en) 2015-11-19 2020-07-28 Novartis Ag Buffers for stabilization of lentiviral preparations
KR20180118659A (ko) 2016-02-01 2018-10-31 바이오버라티브 테라퓨틱스 인크. 최적화된 viii 인자 유전자
US20190314291A1 (en) 2018-01-30 2019-10-17 Modernatx, Inc. Compositions and methods for delivery of agents to immune cells
AU2019215063B2 (en) 2018-02-01 2025-10-16 Bioverativ Therapeutics, Inc. Use of lentiviral vectors expressing Factor VIII
US20220193222A1 (en) 2018-11-30 2022-06-23 Vaccine Stabilization Institute Viral formulations containing amino acids
BR112021010047A2 (pt) 2018-12-06 2021-08-24 Bioverativ Therapeutics Inc. Uso de vetores lentivirais expressando o fator ix
US12403164B2 (en) 2019-09-30 2025-09-02 Bioverativ Therapeutics Inc. Lentiviral vector formulations
KR20230074703A (ko) 2020-06-24 2023-05-31 바이오버라티브 테라퓨틱스 인크. 유리 인자 viii을 상기 단백질이 발현되도록 변형된 렌티바이러스 벡터의 제제로부터 제거하는 방법
US20220090130A1 (en) 2020-08-23 2022-03-24 Bioverativ Therapeutics Inc. MODIFIED BACULOVIRUS SYSTEM FOR IMPROVED PRODUCTION OF CLOSED-ENDED DNA (ceDNA)

Also Published As

Publication number Publication date
HRP20221089T1 (hr) 2022-11-25
JP7217630B2 (ja) 2023-02-03
CN116218863A (zh) 2023-06-06
IL320404A (en) 2025-06-01
US20190185543A1 (en) 2019-06-20
DK3411478T3 (da) 2022-09-12
US11753461B2 (en) 2023-09-12
AU2026200420A1 (en) 2026-02-12
CO2018009120A2 (es) 2018-10-22
EP3411478A1 (en) 2018-12-12
WO2017136358A1 (en) 2017-08-10
ES2926585T3 (es) 2022-10-27
SG11201806398YA (en) 2018-08-30
MX2018009375A (es) 2018-09-05
IL260796B2 (en) 2026-03-01
IL260796B1 (en) 2025-11-01
JP2024114805A (ja) 2024-08-23
KR20180118659A (ko) 2018-10-31
CA3012695A1 (en) 2017-08-10
HK1257078A1 (en) 2019-10-11
PT3411478T (pt) 2022-09-13
CN109072214B (zh) 2023-02-17
MX2023001394A (es) 2023-03-03
PH12018501628B1 (en) 2023-11-10
SI3411478T1 (sl) 2022-10-28
RS63548B1 (sr) 2022-09-30
IL260796A (en) 2018-10-31
NZ745831A (en) 2025-07-25
PH12018501628A1 (en) 2019-06-03
AU2017214378A1 (en) 2018-09-20
SG10201913278PA (en) 2020-02-27
AU2017214378B2 (en) 2023-05-04
JP2022089920A (ja) 2022-06-16
JP2019506166A (ja) 2019-03-07
PL3411478T3 (pl) 2022-10-03
AU2023202218A1 (en) 2023-05-04
EP4137570A1 (en) 2023-02-22
HUE059941T2 (hu) 2023-01-28
CN109072214A (zh) 2018-12-21
US20240124555A1 (en) 2024-04-18
BR112018015659A2 (en) 2018-12-26
AU2023202218B2 (en) 2025-10-30
CL2018002054A1 (es) 2019-01-25
EP3411478B1 (en) 2022-06-08

Similar Documents

Publication Publication Date Title
SG10201913278PA (en) Optimized factor viii genes
GB201721459D0 (en) No details
PL3368678T3 (pl) Konstrukt genetyczny
GB201715843D0 (en) No details
GB201714253D0 (en) No details
GB201715887D0 (en) No details
GB201622033D0 (en) No details
GB201717272D0 (en) No details
GB201717622D0 (en) No details
GB201802686D0 (en) No Details
GB201802603D0 (en) No details
GB201802295D0 (en) No Details
GB201702043D0 (en) No details
GB201709809D0 (en) No details
GB201800519D0 (en) No details
GB201705484D0 (en) Genetic construct
GB201801948D0 (en) No details
PL3630961T3 (pl) Konstrukt genetyczny
GB201802121D0 (en) No details
GB201801950D0 (en) No details
GB201703185D0 (en) No details
IL257500A (en) Altered cullin1 gene
IL246713A0 (en) Conjugates of factor viii
GB201805865D0 (en) Genes
GB201801252D0 (en) No details