KR102625470B1 - 최적화된 인간 응고 인자 ix 유전자 발현 카세트 및 이의 사용 - Google Patents

최적화된 인간 응고 인자 ix 유전자 발현 카세트 및 이의 사용 Download PDF

Info

Publication number
KR102625470B1
KR102625470B1 KR1020197036397A KR20197036397A KR102625470B1 KR 102625470 B1 KR102625470 B1 KR 102625470B1 KR 1020197036397 A KR1020197036397 A KR 1020197036397A KR 20197036397 A KR20197036397 A KR 20197036397A KR 102625470 B1 KR102625470 B1 KR 102625470B1
Authority
KR
South Korea
Prior art keywords
polynucleotide
vector
sequence
aav
cells
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
KR1020197036397A
Other languages
English (en)
Korean (ko)
Other versions
KR20200014318A (ko
Inventor
시아오 시아오
주안 리
Original Assignee
더 유니버시티 오브 노쓰 캐롤라이나 엣 채플 힐
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 더 유니버시티 오브 노쓰 캐롤라이나 엣 채플 힐 filed Critical 더 유니버시티 오브 노쓰 캐롤라이나 엣 채플 힐
Priority to KR1020247001130A priority Critical patent/KR20240010098A/ko
Publication of KR20200014318A publication Critical patent/KR20200014318A/ko
Application granted granted Critical
Publication of KR102625470B1 publication Critical patent/KR102625470B1/ko
Active legal-status Critical Current
Anticipated expiration legal-status Critical

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
    • 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)
    • 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
    • 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
    • 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
    • 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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/16—Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
    • 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
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/81—Protease inhibitors
    • 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
    • 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
    • C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/06—Animal cells or tissues; Human cells or tissues
    • C12N5/0602—Vertebrate cells
    • C12N5/067—Hepatocytes
    • 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
    • C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/06—Animal cells or tissues; Human cells or tissues
    • C12N5/0602—Vertebrate cells
    • C12N5/0693—Tumour cells; Cancer cells
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12P—FERMENTATION OR ENZYME-USING PROCESSES TO SYNTHESISE A DESIRED CHEMICAL COMPOUND OR COMPOSITION OR TO SEPARATE OPTICAL ISOMERS FROM A RACEMIC MIXTURE
    • C12P21/00—Preparation of peptides or proteins
    • C12P21/02—Preparation of peptides or proteins having a known sequence of two or more amino acids, e.g. glutathione
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y304/00—Hydrolases acting on peptide bonds, i.e. peptidases (3.4)
    • C12Y304/21—Serine endopeptidases (3.4.21)
    • C12Y304/21022—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
    • C12N2510/00—Genetically modified cells
    • 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
    • C12N2800/10—Plasmid DNA
    • C12N2800/106—Plasmid DNA for vertebrates
    • C12N2800/107—Plasmid DNA for vertebrates for mammalian
    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Genetics & Genomics (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • General Health & Medical Sciences (AREA)
  • Wood Science & Technology (AREA)
  • Biomedical Technology (AREA)
  • Biochemistry (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Medicinal Chemistry (AREA)
  • Molecular Biology (AREA)
  • Microbiology (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Biophysics (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Epidemiology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Virology (AREA)
  • Diabetes (AREA)
  • Hematology (AREA)
  • Cell Biology (AREA)
  • Immunology (AREA)
  • Toxicology (AREA)
  • Oncology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
KR1020197036397A 2017-05-31 2018-05-31 최적화된 인간 응고 인자 ix 유전자 발현 카세트 및 이의 사용 Active KR102625470B1 (ko)

Priority Applications (1)

Application Number Priority Date Filing Date Title
KR1020247001130A KR20240010098A (ko) 2017-05-31 2018-05-31 최적화된 인간 응고 인자 ix 유전자 발현 카세트 및 이의 사용

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201762512833P 2017-05-31 2017-05-31
US62/512,833 2017-05-31
PCT/US2018/035267 WO2018222792A1 (en) 2017-05-31 2018-05-31 Optimized human clotting factor ix gene expression cassettes and their use

Related Child Applications (1)

Application Number Title Priority Date Filing Date
KR1020247001130A Division KR20240010098A (ko) 2017-05-31 2018-05-31 최적화된 인간 응고 인자 ix 유전자 발현 카세트 및 이의 사용

Publications (2)

Publication Number Publication Date
KR20200014318A KR20200014318A (ko) 2020-02-10
KR102625470B1 true KR102625470B1 (ko) 2024-01-16

Family

ID=64456331

Family Applications (2)

Application Number Title Priority Date Filing Date
KR1020197036397A Active KR102625470B1 (ko) 2017-05-31 2018-05-31 최적화된 인간 응고 인자 ix 유전자 발현 카세트 및 이의 사용
KR1020247001130A Ceased KR20240010098A (ko) 2017-05-31 2018-05-31 최적화된 인간 응고 인자 ix 유전자 발현 카세트 및 이의 사용

Family Applications After (1)

Application Number Title Priority Date Filing Date
KR1020247001130A Ceased KR20240010098A (ko) 2017-05-31 2018-05-31 최적화된 인간 응고 인자 ix 유전자 발현 카세트 및 이의 사용

Country Status (9)

Country Link
US (2) US11530402B2 (https=)
EP (1) EP3630978A4 (https=)
JP (3) JP7119009B2 (https=)
KR (2) KR102625470B1 (https=)
CN (2) CN114875051A (https=)
AU (1) AU2018275549B2 (https=)
CA (1) CA3064450A1 (https=)
MX (2) MX2019014379A (https=)
WO (1) WO2018222792A1 (https=)

Families Citing this family (16)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
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
KR20220097891A (ko) * 2019-09-30 2022-07-08 바이오버라티브 테라퓨틱스 인크. 렌티바이러스 벡터 제형
WO2021087007A1 (en) * 2019-10-28 2021-05-06 University Of Florida Research Foundation, Incorporated Gene therapy vectors
IL296986A (en) * 2020-04-06 2022-12-01 Homology Medicines Inc Adeno-associated virus preparations for ids gene transfer and methods of use thereof
CN113817759B (zh) * 2020-07-10 2023-06-02 南京吉迈生物技术有限公司 修饰的因子ix、组合物、方法及其在基因治疗中的应用
CN115109788A (zh) * 2021-03-23 2022-09-27 北京锦篮基因科技有限公司 Aav介导的人脂蛋白脂酶肝脏异位表达载体及其用途
JP2024521537A (ja) * 2021-06-23 2024-05-31 フロンテーラ セラピューティクス，インク． 血友病ｂを処置するための組成物および方法
CN114277057B (zh) * 2021-07-09 2023-10-13 上海天泽云泰生物医药有限公司 用于治疗或预防b型血友病的重组腺相关病毒载体和方法
US20230149563A1 (en) * 2021-10-27 2023-05-18 Regeneron Pharmaceuticals, Inc. Compositions and methods for expressing factor ix for hemophilia b therapy
CN118460618A (zh) * 2022-04-19 2024-08-09 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
CN115029360B (zh) * 2022-05-30 2024-08-02 上海勉亦生物科技有限公司 用于治疗粘多糖贮积症iiia型的转基因表达盒
CN115948408A (zh) * 2022-09-23 2023-04-11 上海信致医药科技有限公司 改进的人凝血因子viii基因表达盒及其应用
WO2025143742A1 (ko) * 2023-12-26 2025-07-03 삼성바이오에피스 주식회사 혈액응고인자 IX를 암호화하는 코돈-최적화된 폴리뉴클레오티드 및 이를 포함하는 rAAV 생산용 플라스미드

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20070042462A1 (en) 2005-08-21 2007-02-22 Markus Hildinger Super-size adeno-associated viral vector harboring a recombinant genome larger than 5.7 kb

Family Cites Families (18)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
FR2650838A1 (fr) * 1989-08-09 1991-02-15 Transgene Sa Vecteurs d'expression du facteur ix dans une cellule eucaryote superieure, procede de preparation de facteur ix par des animaux transgeniques et facteur ix obtenu
EP0710288B1 (en) * 1993-06-10 2006-04-05 Genetic Therapy, Inc. Adenoviral vectors for treatment of hemophilia
WO2006036502A2 (en) * 2004-09-22 2006-04-06 St. Jude Children's Research Hospital Improved expression of factor ix in gene therapy vectors
JP2009519710A (ja) 2005-12-16 2009-05-21 ザ ボード オブ トラスティーズ オブ ザ レランド スタンフォード ジュニア ユニバーシティー 遺伝子発現調節エレメントのハイスループットでの特徴付けのための機能性アレイ
ES2535877T3 (es) * 2006-06-19 2015-05-18 Asklepios Biopharmaceutical, Inc. Genes de Factor VIII y Factor IX modificados y vectores para la terapia génica
CN102628041B (zh) * 2008-02-14 2013-09-11 财团法人牧岩生命工学研究所 适于表达用于基因治疗的编码序列的表达载体
CA2722238C (en) * 2008-04-22 2017-11-28 Life Sciences Research Partners Vzw Liver-specific nucleic acid regulatory elements and methods and use thereof
US9700486B2 (en) * 2013-04-24 2017-07-11 Corning Incorporated Delamination resistant pharmaceutical glass containers containing active pharmaceutical ingredients
RU2585532C2 (ru) 2014-01-31 2016-05-27 Федеральное государственное учреждение "Федеральный исследовательский центр "Фундаментальные основы биотехнологии" Российской академии наук"(ФИЦ Биотехнологии РАН) Плазмида для экспрессии рекомбинантного фактора свёртываемости крови ix человека, клетка сно - продуцент рекомбинантного фактора свёртываемости крови ix человека и способ получения указанного фактора
SMT202000531T1 (it) * 2014-03-09 2020-11-10 Univ Pennsylvania Composizioni utili in trattamento di deficit di ornitina transcarbamilasi(otc)
KR102244434B1 (ko) * 2014-08-11 2021-04-23 삼성전자주식회사 재조합 벡터 및 이를 이용한 목적 폴리펩타이드의 생산 방법
GB201420139D0 (en) * 2014-11-12 2014-12-24 Ucl Business Plc Factor IX gene therapy
MX388739B (es) * 2015-02-06 2025-03-20 Univ North Carolina Chapel Hill Casetes optimizados de expresión del gen humano del factor viii de coagulación y su uso.
MX390848B (es) * 2015-06-23 2025-03-21 Childrens Hospital Philadelphia Factor ix modificado, y composiciones, metodos y usos para la transferencia de genes a celulas, organos y tejidos.
US10086089B2 (en) * 2015-09-18 2018-10-02 DNARx Systems and methods for nucleic acid expression in vivo
US11191847B2 (en) * 2016-04-15 2021-12-07 The Trustees Of The University Of Pennsylvania Gene therapy for treating hemophilia B
CN106497949A (zh) * 2016-10-14 2017-03-15 上海交通大学医学院附属瑞金医院 一种高活性凝血因子ix突变体、重组蛋白与融合蛋白的制备与应用
CN116392609A (zh) * 2017-03-23 2023-07-07 Dnarx公司 用于体内核酸表达的系统和方法

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20070042462A1 (en) 2005-08-21 2007-02-22 Markus Hildinger Super-size adeno-associated viral vector harboring a recombinant genome larger than 5.7 kb

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
GENBANK: X67082.1
HUMAN GENE THERAPY 26:69-81 (FEBRUARY 2015) [DOI: 10.1089/HUM.2014.106]

Also Published As

Publication number Publication date
EP3630978A4 (en) 2021-03-24
CN108977452B (zh) 2022-07-19
JP2020522259A (ja) 2020-07-30
JP2024097922A (ja) 2024-07-19
JP2022159358A (ja) 2022-10-17
KR20200014318A (ko) 2020-02-10
US20200087646A1 (en) 2020-03-19
CN114875051A (zh) 2022-08-09
US11530402B2 (en) 2022-12-20
KR20240010098A (ko) 2024-01-23
MX2019014379A (es) 2020-01-23
MX2024007348A (es) 2024-06-26
US12209263B2 (en) 2025-01-28
CA3064450A1 (en) 2018-12-06
AU2018275549B2 (en) 2024-12-19
CN108977452A (zh) 2018-12-11
WO2018222792A1 (en) 2018-12-06
JP7119009B2 (ja) 2022-08-16
US20230091502A1 (en) 2023-03-23
EP3630978A1 (en) 2020-04-08
AU2018275549A1 (en) 2019-11-21

Similar Documents

Publication Publication Date Title
KR102625470B1 (ko) 최적화된 인간 응고 인자 ix 유전자 발현 카세트 및 이의 사용
JP7437035B2 (ja) 最適化されたヒト凝固第ｖｉｉｉ因子遺伝子発現カセットおよびその使用
KR20210008561A (ko) 합성 간-향성 아데노-연관 바이러스 캡시드 및 그의 용도
WO2024060463A1 (zh) 改进的人凝血因子viii基因表达盒及其应用
HK40062724A (en) Optimized human clotting factor viii gene expression cassettes and their use
HK1249117B (zh) 优化的人类凝血因子viii基因表达盒及其用途

Legal Events

Date Code Title Description
PA0105 International application

Patent event date: 20191209

Patent event code: PA01051R01D

Comment text: International Patent Application

PG1501 Laying open of application
A201 Request for examination
PA0201 Request for examination

Patent event code: PA02012R01D

Patent event date: 20210531

Comment text: Request for Examination of Application

E902 Notification of reason for refusal
PE0902 Notice of grounds for rejection

Comment text: Notification of reason for refusal

Patent event date: 20230327

Patent event code: PE09021S01D

E701 Decision to grant or registration of patent right
PE0701 Decision of registration

Patent event code: PE07011S01D

Comment text: Decision to Grant Registration

Patent event date: 20231011

GRNT Written decision to grant
PR0701 Registration of establishment

Comment text: Registration of Establishment

Patent event date: 20240111

Patent event code: PR07011E01D

PR1002 Payment of registration fee

Payment date: 20240111

End annual number: 3

Start annual number: 1

PG1601 Publication of registration