SG11202105880TA - Use of lentiviral vectors expressing factor ix - Google Patents

Use of lentiviral vectors expressing factor ix

Info

Publication number
SG11202105880TA
SG11202105880TA SG11202105880TA SG11202105880TA SG11202105880TA SG 11202105880T A SG11202105880T A SG 11202105880TA SG 11202105880T A SG11202105880T A SG 11202105880TA SG 11202105880T A SG11202105880T A SG 11202105880TA SG 11202105880T A SG11202105880T A SG 11202105880TA
Authority
SG
Singapore
Prior art keywords
lentiviral vectors
vectors expressing
expressing factor
factor
lentiviral
Prior art date
Application number
SG11202105880TA
Other languages
English (en)
Inventor
Tongyao Liu
Susannah Patarroyo-White
Douglas Drager
Alessio Cantore
Luigi Naldini
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 SG11202105880TA publication Critical patent/SG11202105880TA/en

Links

Classifications

    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005Medicinal 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
    • A61K48/0058Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00Drugs for disorders of the blood or the extracellular fluid
    • A61P7/04Antihaemorrhagics; Procoagulants; Haemostatic agents; Antifibrinolytic agents
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/745Blood coagulation or fibrinolysis factors
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14Hydrolases (3)
    • C12N9/48Hydrolases (3) acting on peptide bonds (3.4)
    • C12N9/50Proteinases, e.g. Endopeptidases (3.4.21-3.4.25)
    • C12N9/64Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue
    • C12N9/6421Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue from mammals
    • C12N9/6424Serine endopeptidases (3.4.21)
    • C12N9/644Coagulation factor IXa (3.4.21.22)
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005Medicinal 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
    • A61K48/0066Manipulation 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
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2740/00Reverse transcribing RNA viruses
    • C12N2740/00011Details
    • C12N2740/10011Retroviridae
    • C12N2740/16011Human Immunodeficiency Virus, HIV
    • C12N2740/16041Use of virus, viral particle or viral elements as a vector
    • C12N2740/16043Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2740/00Reverse transcribing RNA viruses
    • C12N2740/00011Details
    • C12N2740/10011Retroviridae
    • C12N2740/16011Human Immunodeficiency Virus, HIV
    • C12N2740/16051Methods of production or purification of viral material
    • C12N2740/16052Methods of production or purification of viral material relating to complementing cells and packaging systems for producing virus or viral particles
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2800/00Nucleic acids vectors
    • C12N2800/22Vectors comprising a coding region that has been codon optimised for expression in a respective host
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2830/00Vector systems having a special element relevant for transcription
    • C12N2830/008Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2830/00Vector systems having a special element relevant for transcription
    • C12N2830/48Vector systems having a special element relevant for transcription regulating transport or export of RNA, e.g. RRE, PRE, WPRE, CTE

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • Wood Science & Technology (AREA)
  • Molecular Biology (AREA)
  • Medicinal Chemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Hematology (AREA)
  • Virology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Diabetes (AREA)
  • Epidemiology (AREA)
  • Toxicology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
SG11202105880TA 2018-12-06 2019-12-05 Use of lentiviral vectors expressing factor ix SG11202105880TA (en)

Applications Claiming Priority (2)

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

Publications (1)

Publication Number Publication Date
SG11202105880TA true SG11202105880TA (en) 2021-07-29

Family

ID=69024682

Family Applications (1)

Application Number Title Priority Date Filing Date
SG11202105880TA SG11202105880TA (en) 2018-12-06 2019-12-05 Use of lentiviral vectors expressing factor ix

Country Status (14)

Country Link
US (1) US20200199626A1 (https=)
EP (1) EP3891289A2 (https=)
JP (2) JP7644007B2 (https=)
KR (1) KR20210100661A (https=)
CN (1) CN113396223A (https=)
AU (1) AU2019393880A1 (https=)
BR (1) BR112021010047A2 (https=)
CA (1) CA3121786A1 (https=)
CO (1) CO2021008877A2 (https=)
IL (1) IL283547A (https=)
MX (2) MX2021006648A (https=)
SG (1) SG11202105880TA (https=)
TW (1) TW202039855A (https=)
WO (1) WO2020118069A2 (https=)

Families Citing this family (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2014127215A1 (en) 2013-02-15 2014-08-21 Biogen Idec Ma Inc. Optimized factor viii gene
US11008561B2 (en) 2014-06-30 2021-05-18 Bioverativ Therapeutics Inc. Optimized factor IX gene
CA3012695A1 (en) 2016-02-01 2017-08-10 Bioverativ Therapeutics Inc. Optimized factor viii genes
KR20220097891A (ko) 2019-09-30 2022-07-08 바이오버라티브 테라퓨틱스 인크. 렌티바이러스 벡터 제형
MX2023000156A (es) 2020-06-24 2023-02-16 Bioverativ Therapeutics Inc Metodos para la eliminacion de factor viii libre de preparaciones de vectores lentivirales modificados para expresar dicha proteina.
TW202246505A (zh) * 2021-03-05 2022-12-01 俄羅斯聯邦商亞那拜恩有限公司 編碼凝血因子ix蛋白的密碼子優化的核酸及其用途
GB202205615D0 (en) * 2022-04-14 2022-06-01 Genespire S R L Lentiviral vector
KR20250021306A (ko) * 2022-04-14 2025-02-12 제네스파이어 에스.알.엘. 렌티바이러스 벡터
WO2025226943A1 (en) 2024-04-25 2025-10-30 Masonic Medical Research Laboratory Polynucleotide for hepatocyte expression of vestigial like protein 4 and method of use thereof

Family Cites Families (45)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP3045539B2 (ja) 1989-02-21 2000-05-29 ワシントン ユニバーシティ 改変型生殖ホルモン
US6086875A (en) 1995-01-17 2000-07-11 The Brigham And Women's Hospital, Inc. Receptor specific transepithelial transport of immunogens
US6485726B1 (en) 1995-01-17 2002-11-26 The Brigham And Women's Hospital, Inc. Receptor specific transepithelial transport of therapeutics
US6030613A (en) 1995-01-17 2000-02-29 The Brigham And Women's Hospital, Inc. Receptor specific transepithelial transport of therapeutics
US6013516A (en) 1995-10-06 2000-01-11 The Salk Institute For Biological Studies Vector and method of use for nucleic acid delivery to non-dividing cells
GB9621680D0 (en) 1996-10-17 1996-12-11 Oxford Biomedica Ltd Lentiviral vectors
PT904392E (pt) 1996-10-17 2001-06-29 Oxford Biomedica Ltd Vectores retrovirais
GB9622500D0 (en) 1996-10-29 1997-01-08 Oxford Biomedica Ltd Therapeutic gene
US6207455B1 (en) 1997-05-01 2001-03-27 Lung-Ji Chang Lentiviral vectors
US5994136A (en) 1997-12-12 1999-11-30 Cell Genesys, Inc. Method and means for producing high titer, safe, recombinant lentivirus vectors
EP2213743A1 (en) 2000-04-12 2010-08-04 Human Genome Sciences, Inc. Albumin fusion proteins
ATE437221T1 (de) 2001-05-14 2009-08-15 Gbp Ip Llc Lentivirale vektoren kodierend für gerinnungsfaktoren für die gentherapie
AU2002364586A1 (en) 2001-12-21 2003-07-30 Delta Biotechnology Limited Albumin fusion proteins
KR101271635B1 (ko) 2001-12-21 2013-06-12 휴먼 게놈 사이언시즈, 인코포레이티드 알부민 융합 단백질
US20080194481A1 (en) 2001-12-21 2008-08-14 Human Genome Sciences, Inc. Albumin Fusion Proteins
JP2005526769A (ja) 2002-03-15 2005-09-08 ザ・ブリガーム・アンド・ウーメンズ・ホスピタル・インコーポレーテッド 治療剤を全身搬送するための中央気道投与
US6615782B1 (en) 2002-04-12 2003-09-09 Delphi Technologies, Inc. Two-step finger follower rocker arm
ES2298745T3 (es) 2003-04-24 2008-05-16 Fondazione Centro San Raffaele Del Monte Tabor Promotores bidireccionales de sintesis y utilizacion de los mismos.
EP1769078A1 (en) * 2004-07-01 2007-04-04 VIRxSYS Corporation Vector packaging cell line
CA2609142C (en) 2005-05-27 2016-02-09 Fondazione Centro San Raffaele Del Monte Tabor Therapeutic gene vectors comprising mirna target sequences
WO2007021494A2 (en) 2005-08-12 2007-02-22 Human Genome Sciences, Inc. Albumin fusion proteins
US8048848B2 (en) 2006-02-03 2011-11-01 Prolor Biotech Ltd. Long-acting interferons and derivatives thereof and methods thereof
GB0614780D0 (en) 2006-07-25 2006-09-06 Ucb Sa Biological products
WO2008033413A2 (en) 2006-09-14 2008-03-20 Human Genome Sciences, Inc. Albumin fusion proteins
EP2144930A1 (en) 2007-04-18 2010-01-20 ZymoGenetics, Inc. Single chain fc, methods of making and methods of treatment
EP2158318A2 (en) 2007-05-14 2010-03-03 Biogen Idec MA, Inc. Single-chain fc (scfc) regions, binding polypeptides comprising same, and methods related thereto
SI2173890T1 (sl) 2007-06-21 2011-06-30 Univ Muenchen Tech Biološko aktivni proteini, ki imajo povečano in vivo in/ali in vitro stabilnost
EP2364362B1 (en) 2008-11-12 2015-10-21 Ospedale San Raffaele S.r.l. Gene vector for inducing transgene-specific immune tolerance
ES2610356T3 (es) 2009-02-03 2017-04-27 Amunix Operating Inc. Polipéptidos recombinantes extendidos y composiciones que comprenden los mismos
US8680050B2 (en) 2009-02-03 2014-03-25 Amunix Operating Inc. Growth hormone polypeptides fused to extended recombinant polypeptides and methods of making and using same
US8703717B2 (en) 2009-02-03 2014-04-22 Amunix Operating Inc. Growth hormone polypeptides and methods of making and using same
US9050269B2 (en) 2009-03-10 2015-06-09 The Trustees Of The University Of Pennsylvania Protection of virus particles from phagocytosis by expression of CD47
PL2424571T3 (pl) 2009-04-30 2020-10-19 Ospedale San Raffaele S.R.L Wektor genowy
WO2010144508A1 (en) 2009-06-08 2010-12-16 Amunix Operating Inc. Glucose-regulating polypeptides and methods of making and using same
KR101813727B1 (ko) 2009-06-08 2018-01-02 아뮤닉스 오퍼레이팅 인코포레이티드 성장 호르몬 폴리펩타이드 및 이를 제조하고 사용하는 방법
CA2771999A1 (en) 2009-08-24 2011-03-10 Amunix Operating Inc. Coagulation factor vii compositions and methods of making and using same
WO2011028344A2 (en) 2009-08-25 2011-03-10 Amunix Operating Inc. Interleukin-1 receptor antagonist compositions and methods of making and using same
SI2506868T1 (en) 2009-12-06 2018-04-30 Bioverativ Therapeutics Inc. HYPER AND HYBRID POLYPETTIDES FACTOR VIII-FC AND METHODS FOR THEIR USE
EP3508573A1 (en) 2010-07-09 2019-07-10 Bioverativ Therapeutics Inc. Systems for factor viii processing and methods thereof
TW201217527A (en) 2010-07-09 2012-05-01 Biogen Idec Hemophilia Inc Processable single chain molecules and polypeptides made using same
CN104661674A (zh) 2012-07-11 2015-05-27 阿穆尼克斯运营公司 具有xten和血管性血友病因子蛋白的因子viii复合物、及其用途
TW202003554A (zh) 2013-08-14 2020-01-16 美商百歐維拉提夫治療公司 因子viii-xten融合物及其用途
US11008561B2 (en) * 2014-06-30 2021-05-18 Bioverativ Therapeutics Inc. Optimized factor IX gene
EA201890423A1 (ru) 2015-08-03 2018-07-31 Биовератив Терапьютикс Инк. Слитые белки фактора ix, способы их получения и применения
CA3012695A1 (en) * 2016-02-01 2017-08-10 Bioverativ Therapeutics Inc. Optimized factor viii genes

Also Published As

Publication number Publication date
WO2020118069A3 (en) 2020-07-16
IL283547A (en) 2021-07-29
JP2022514465A (ja) 2022-02-14
US20200199626A1 (en) 2020-06-25
JP7644007B2 (ja) 2025-03-11
CA3121786A1 (en) 2020-06-11
AU2019393880A1 (en) 2021-07-15
CO2021008877A2 (es) 2021-07-30
CN113396223A (zh) 2021-09-14
MX2025015374A (es) 2026-02-03
BR112021010047A2 (pt) 2021-08-24
JP2025020288A (ja) 2025-02-12
WO2020118069A2 (en) 2020-06-11
EP3891289A2 (en) 2021-10-13
KR20210100661A (ko) 2021-08-17
MX2021006648A (es) 2021-07-07
TW202039855A (zh) 2020-11-01

Similar Documents

Publication Publication Date Title
IL276402A (en) Use of a lentiviral vector for FVIII expression
IL283547A (en) Using lentiviral vectors expressing factor xi
IL253820A0 (en) Retrovirus and lentivirus vectors
GB201706477D0 (en) Modification of polypeptides
SG11202001367QA (en) Retroviral vectors
GB201804514D0 (en) Treatment of pyroptosis
SG11201803546WA (en) Compositions comprising lentiviral vectors expressing il-12 and methods of use thereof
PT3661491T (pt) 3-metilmetcatinona para uso no tratamento de um indivíduo sob uma intervenção psicoterapêutica
ZA201906238B (en) New therapeutical use of h3-ligands
GB201804515D0 (en) Treatment of necroptosis
SG11202102112QA (en) Ship
IL252758A0 (en) An electrochemical reaction of oxygen with hydrocarbons
ZA201701908B (en) Adenoviral vector encoding human atonal homolog-1 (hath1)
SG11202102012SA (en) Improved lentiviral vector
ZA202105399B (en) Use of spiropidion
PL3449931T3 (pl) Zastosowanie cerebrolizyny
EP3648789C0 (en) MTMR2-S POLYPEPTIDE FOR USE IN THE TREATMENT OF MYOPATHY
GB201710024D0 (en) Electrochemical impedance spectroscopy of protein-protein interaction
HK40062234A (en) Use of lentiviral vectors expressing factor ix
SG11202105136WA (en) Application of chidamide
GB201806638D0 (en) Maintaining state of speculation
LT3597169T (lt) Sauna
HK40043577A (en) Use of lentiviral vectors expressing factor viii
GB201810835D0 (en) Methods of use
GB2577737B (en) Determination of material