JP7644007B2 - 第ｉｘ因子を発現するレンチウイルスベクターの使用 - Google Patents

第ｉｘ因子を発現するレンチウイルスベクターの使用 Download PDF

Info

Publication number
JP7644007B2
JP7644007B2 JP2021531954A JP2021531954A JP7644007B2 JP 7644007 B2 JP7644007 B2 JP 7644007B2 JP 2021531954 A JP2021531954 A JP 2021531954A JP 2021531954 A JP2021531954 A JP 2021531954A JP 7644007 B2 JP7644007 B2 JP 7644007B2
Authority
JP
Japan
Prior art keywords
fold
lentiviral vector
less
nucleotide sequence
seq
Prior art date
Legal status (The legal status 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 status listed.)
Active
Application number
JP2021531954A
Other languages
English (en)
Japanese (ja)
Other versions
JP2022514465A (ja
JP2022514465A5 (https=
Inventor
トンヤオ・リウ
スザンナ・パタロヨ－ホワイト
ダグラス・ドレイガー
アレッシオ・カントーレ
ルイージ・ナルディーニ
Original Assignee
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
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 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 filed Critical 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
Publication of JP2022514465A publication Critical patent/JP2022514465A/ja
Publication of JP2022514465A5 publication Critical patent/JP2022514465A5/ja
Priority to JP2024192744A priority Critical patent/JP2025020288A/ja
Application granted granted Critical
Publication of JP7644007B2 publication Critical patent/JP7644007B2/ja
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

Images

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
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • 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
    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • 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
    • A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
    • 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
    • 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
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • 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)
    • 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
    • C12N2740/00—Reverse transcribing RNA viruses
    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
    • C12N2740/16011—Human Immunodeficiency Virus, HIV
    • C12N2740/16051—Methods of production or purification of viral material
    • C12N2740/16052—Methods of production or purification of viral material relating to complementing cells and packaging systems for producing virus or viral particles
    • 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
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—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
    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination
    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/48—Vector 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)
JP2021531954A 2018-12-06 2019-12-05 第ｉｘ因子を発現するレンチウイルスベクターの使用 Active JP7644007B2 (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2024192744A JP2025020288A (ja) 2018-12-06 2024-11-01 第ｉｘ因子を発現するレンチウイルスベクターの使用

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201862776393P 2018-12-06 2018-12-06
US62/776,393 2018-12-06
PCT/US2019/064711 WO2020118069A2 (en) 2018-12-06 2019-12-05 Use of lentiviral vectors expressing factor ix

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2024192744A Division JP2025020288A (ja) 2018-12-06 2024-11-01 第ｉｘ因子を発現するレンチウイルスベクターの使用

Publications (3)

Publication Number Publication Date
JP2022514465A JP2022514465A (ja) 2022-02-14
JP2022514465A5 JP2022514465A5 (https=) 2022-11-16
JP7644007B2 true JP7644007B2 (ja) 2025-03-11

Family

ID=69024682

Family Applications (2)

Application Number Title Priority Date Filing Date
JP2021531954A Active JP7644007B2 (ja) 2018-12-06 2019-12-05 第ｉｘ因子を発現するレンチウイルスベクターの使用
JP2024192744A Pending JP2025020288A (ja) 2018-12-06 2024-11-01 第ｉｘ因子を発現するレンチウイルスベクターの使用

Family Applications After (1)

Application Number Title Priority Date Filing Date
JP2024192744A Pending JP2025020288A (ja) 2018-12-06 2024-11-01 第ｉｘ因子を発現するレンチウイルスベクターの使用

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

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2016004113A1 (en) 2014-06-30 2016-01-07 Biogen Ma Inc. Optimized factor ix gene

Family Cites Families (44)

* 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
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

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2016004113A1 (en) 2014-06-30 2016-01-07 Biogen Ma Inc. Optimized factor ix gene

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
CANTORE, A. et al.，Blood，2017年，Vol. 130, Suppl. 1，p. 605
SOSALE, N.G. et al.，Mol Ther Methods Clin Dev，2016年，Vol. 3, Article No. 16080，pp. 1-13

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
CA3121786A1 (en) 2020-06-11
AU2019393880A1 (en) 2021-07-15
SG11202105880TA (en) 2021-07-29
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
JP7644007B2 (ja) 第ｉｘ因子を発現するレンチウイルスベクターの使用
US12275970B2 (en) Optimized factor IX gene
US20240124555A1 (en) Optimized factor viii genes
AU2019215063B2 (en) Use of lentiviral vectors expressing Factor VIII
RU2816873C2 (ru) Применение лентивирусных векторов, экспрессрующих фактор ix
US20240350672A1 (en) Nucleic acids encoding factor viii polypeptides with reduced immunogenicity
HK40088478A (en) Optimized factor viii genes
BR122024018474A2 (pt) Molécula de ácido nucleico isolado, vetor, célula hospedeira, polipeptídeo, e seu método de produção
BR112018015659B1 (pt) Molécula de ácido nucleico isolada, vetor e método para a produção de um polipeptídeo com atividade de fviii
HK1257078B (en) Optimized factor viii genes

Legal Events

Date Code Title Description
A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20221108

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20221108

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20231010

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20231219

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20240311

A02 Decision of refusal

Free format text: JAPANESE INTERMEDIATE CODE: A02

Effective date: 20240702

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20241101

A911 Transfer to examiner for re-examination before appeal (zenchi)

Free format text: JAPANESE INTERMEDIATE CODE: A911

Effective date: 20241111

TRDD Decision of grant or rejection written
A01 Written decision to grant a patent or to grant a registration (utility model)

Free format text: JAPANESE INTERMEDIATE CODE: A01

Effective date: 20250204

A61 First payment of annual fees (during grant procedure)

Free format text: JAPANESE INTERMEDIATE CODE: A61

Effective date: 20250227

R150 Certificate of patent or registration of utility model

Ref document number: 7644007

Country of ref document: JP

Free format text: JAPANESE INTERMEDIATE CODE: R150