MX386802B - Vectores del factor viii del virus adeno-asociado. - Google Patents

Vectores del factor viii del virus adeno-asociado.

Info

Publication number
MX386802B
MX386802B MX2016003262A MX2016003262A MX386802B MX 386802 B MX386802 B MX 386802B MX 2016003262 A MX2016003262 A MX 2016003262A MX 2016003262 A MX2016003262 A MX 2016003262A MX 386802 B MX386802 B MX 386802B
Authority
MX
Mexico
Prior art keywords
factor viii
adeno
associated virus
vectors
virus factor
Prior art date
Application number
MX2016003262A
Other languages
English (en)
Other versions
MX2016003262A (es
Inventor
Amit Nathwani
Andrew Davidoff
Edward Tuddenham
Jenny Mcintosh
Peter Cameron Colosi
Original Assignee
Biomarin Pharm Inc
St Jude Childrens Res Hospital
Univ London
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
Family has litigation
First worldwide family litigation filed litigation Critical https://patents.darts-ip.com/?family=51585244&utm_source=google_patent&utm_medium=platform_link&utm_campaign=public_patent_search&patent=MX386802(B) "Global patent litigation dataset” by Darts-ip is licensed under a Creative Commons Attribution 4.0 International License.
Application filed by Biomarin Pharm Inc, St Jude Childrens Res Hospital, Univ London filed Critical Biomarin Pharm Inc
Publication of MX2016003262A publication Critical patent/MX2016003262A/es
Publication of MX386802B publication Critical patent/MX386802B/es

Links

Classifications

    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/36—Blood coagulation or fibrinolysis factors
    • A61K38/37—Factors VIII
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • 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
    • 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
    • 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
    • C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • 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
    • C12N2750/14132—Use of virus as therapeutic agent, other than vaccine, e.g. as cytolytic agent
    • 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
    • C12N2750/14142—Use of virus, viral particle or viral elements as a vector virus or viral particle as vehicle, e.g. encapsulating small organic molecule
    • 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
    • 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
    • C12N2750/14151—Methods of production or purification of viral material
    • 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)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • Medicinal Chemistry (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • Wood Science & Technology (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Hematology (AREA)
  • General Engineering & Computer Science (AREA)
  • Epidemiology (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Virology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Immunology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Diabetes (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Toxicology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Peptides Or Proteins (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)

Abstract

La presente invención se refiere a vectores mejorados del Factor VIII (FVIII) del virus adeno-asociado (AAV), que incluyen vectores del FVIII del AAV que producen un polipéptido del Factor VIII funcional y vectores del FVIII del AAV con alta actividad de expresión.
MX2016003262A 2013-09-12 2014-09-10 Vectores del factor viii del virus adeno-asociado. MX386802B (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201361877042P 2013-09-12 2013-09-12
PCT/US2014/054960 WO2015038625A1 (en) 2013-09-12 2014-09-10 Adeno-associated virus factor viii vectors

Publications (2)

Publication Number Publication Date
MX2016003262A MX2016003262A (es) 2016-09-29
MX386802B true MX386802B (es) 2025-03-19

Family

ID=51585244

Family Applications (2)

Application Number Title Priority Date Filing Date
MX2016003262A MX386802B (es) 2013-09-12 2014-09-10 Vectores del factor viii del virus adeno-asociado.
MX2021012014A MX2021012014A (es) 2013-09-12 2016-03-11 Vectores del factor viii del virus adeno-asociado.

Family Applications After (1)

Application Number Title Priority Date Filing Date
MX2021012014A MX2021012014A (es) 2013-09-12 2016-03-11 Vectores del factor viii del virus adeno-asociado.

Country Status (34)

Country Link
US (6) US9504762B2 (es)
EP (2) EP3770169A1 (es)
JP (5) JP6735672B2 (es)
KR (2) KR102484396B1 (es)
CN (2) CN105636981B (es)
AR (1) AR097657A1 (es)
AU (1) AU2014318890B2 (es)
CA (3) CA2921232C (es)
CL (1) CL2016000502A1 (es)
CY (2) CY1124213T1 (es)
DK (1) DK3044231T3 (es)
ES (1) ES2813698T3 (es)
FI (1) FIC20230007I1 (es)
FR (1) FR23C1007I2 (es)
HR (1) HRP20201324T1 (es)
HU (2) HUE050484T2 (es)
IL (2) IL244404B (es)
LT (2) LT3044231T (es)
LU (1) LUC00298I2 (es)
MX (2) MX386802B (es)
NL (1) NL301222I2 (es)
NO (1) NO2023003I1 (es)
NZ (1) NZ716549A (es)
PE (1) PE20160769A1 (es)
PH (1) PH12016500328A1 (es)
PL (1) PL3044231T3 (es)
PT (1) PT3044231T (es)
RS (1) RS61039B1 (es)
RU (1) RU2710307C2 (es)
SG (1) SG11201601932UA (es)
SI (1) SI3044231T1 (es)
SM (1) SMT202000458T1 (es)
TW (1) TWI632239B (es)
WO (1) WO2015038625A1 (es)

Families Citing this family (61)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
GB201210357D0 (en) 2012-06-12 2012-07-25 Ucl Business Plc Factor VIII sequences
ES2813698T3 (es) 2013-09-12 2021-03-24 Biomarin Pharm Inc Vectores de AAV que comprenden un gen que codifica el factor VIII
US11008561B2 (en) 2014-06-30 2021-05-18 Bioverativ Therapeutics Inc. Optimized factor IX gene
CN112553229A (zh) 2014-11-05 2021-03-26 沃雅戈治疗公司 用于治疗帕金森病的aadc多核苷酸
GB201420139D0 (en) 2014-11-12 2014-12-24 Ucl Business Plc Factor IX gene therapy
JP6879486B2 (ja) * 2015-03-17 2021-06-02 フリーイェ・ユニヴェルシテイト・ブリュッセルＶｒｉｊｅ Ｕｎｉｖｅｒｓｉｅｉｔ Ｂｒｕｓｓｅｌ Ｆｖｉｉｉおよびｆｉｘ用の最適化された肝臓特異的発現系
TWI707951B (zh) 2015-04-08 2020-10-21 美商健臻公司 過大腺相關載體之製造
EP3626274B1 (en) * 2015-04-16 2025-01-22 Emory University Recombinant promoters and vectors for protein expression in liver and use thereof
BR112018006074A2 (pt) * 2015-09-24 2018-10-09 Biomarin Pharm Inc vetores de fator viii de vírus adeno-associado, partículas virais associadas e formulações terapêuticas compreendendo as mesmas
KR102766526B1 (ko) * 2015-10-28 2025-02-13 상가모 테라퓨틱스, 인코포레이티드 간-특이적 작제물인, 인자 viii 발현 카세트 및 이의 사용 방법
PE20231949A1 (es) * 2015-10-30 2023-12-05 Spark Therapeutics Inc VARIANTES DEL FACTOR VIII REDUCIDO CON CpG, COMPOSICIONES Y METODOS Y USOS PARA EL TRATAMIENTO DE TRASTORNOS DE LA HEMOSTASIA
SG11201804070XA (en) 2015-11-13 2018-06-28 Baxalta Inc Viral vectors encoding recombinant fviii variants with increased expression for gene therapy of hemophilia a
EP3374388A1 (en) * 2015-11-13 2018-09-19 Baxalta Incorporated Viral vectors encoding recombinant fviii variants with increased expression for gene therapy of hemophilia a
WO2017180857A1 (en) * 2016-04-15 2017-10-19 The Trustees Of The University Of Pennsylvania Gene therapy for treating hemophilia a
WO2017205573A1 (en) * 2016-05-25 2017-11-30 Lonza Houston Inc. Methods for isolating adeno-associated virus using a polydiallyldialkylammonium salt
IL263801B2 (en) 2016-07-26 2024-01-01 Biomarin Pharm Inc Novel adeno-associated virus capsid proteins
JOP20190269A1 (ar) 2017-06-15 2019-11-20 Voyager Therapeutics Inc بولي نوكليوتيدات aadc لعلاج مرض باركنسون
BR112020001979A2 (pt) * 2017-08-01 2020-08-18 Spark Therapeutics, Inc. métodos de terapia genética do fator viii (fviii)
JP2021522811A (ja) 2018-05-09 2021-09-02 ビオマリン プハルマセウトイカル インコーポレイテッド フェニルケトン尿症の治療方法
AR117427A1 (es) 2018-05-14 2021-08-04 Biomarin Pharm Inc Expresión estable de vectores de aav en sujetos jóvenes
TW202005978A (zh) 2018-05-14 2020-02-01 美商拜奧馬林製藥公司 新穎肝靶向腺相關病毒載體
AU2019268330A1 (en) 2018-05-15 2020-11-26 Voyager Therapeutics, Inc. Compositions and methods for the treatment of Parkinson's disease
AU2019306194A1 (en) 2018-07-16 2021-02-04 Baxalta GmbH Gene therapy of hemophilia A using viral vectors encoding recombinant FVIII variants with increased expression
WO2020020475A1 (en) * 2018-07-27 2020-01-30 Zentrum Für Forschungsförderung In Der Pädiatrie Gmbh Improved concepts for the treatment of genetic disorders with high-capacity plal-generated gold nanoparticles
JP7684212B2 (ja) 2018-08-03 2025-05-27 サンガモ セラピューティクス， インコーポレイテッド 第ｖｉｉｉ因子の発現による改善された臨床パラメーター
WO2020033863A1 (en) 2018-08-09 2020-02-13 Bioverativ Therapeutics Inc. Nucleic acid molecules and uses thereof for non-viral gene therapy
US10842885B2 (en) 2018-08-20 2020-11-24 Ucl Business Ltd Factor IX encoding nucleotides
GB201813528D0 (en) 2018-08-20 2018-10-03 Ucl Business Plc Factor IX encoding nucleotides
EP3856762A1 (en) 2018-09-28 2021-08-04 Voyager Therapeutics, Inc. Frataxin expression constructs having engineered promoters and methods of use thereof
EP4461814A3 (en) * 2018-10-12 2025-02-26 Genzyme Corporation Generation of improved human pah for treatment of severe pku by liver-directed gene replacement therapy
TWI851647B (zh) 2019-01-16 2024-08-11 日商武田藥品工業股份有限公司 用於a型血友病基因治療之編碼表現增加之重組fviii變異體的病毒載體
WO2020168362A1 (en) 2019-02-15 2020-08-20 Crispr Therapeutics Ag Gene editing for hemophilia a with improved factor viii expression
JP7795357B2 (ja) 2019-03-13 2026-01-07 ジェネレーション バイオ カンパニー Ｆｖｉｉｉ治療薬を発現するための非ウイルス性ｄｎａベクターおよびその使用
TW202126284A (zh) 2019-09-30 2021-07-16 美商百歐維拉提夫治療公司 慢病毒載體配製物
JP6831943B1 (ja) 2019-10-24 2021-02-17 三ツ星ベルト株式会社 摩擦伝動ベルトおよびその製造方法
US20220396610A1 (en) * 2019-11-01 2022-12-15 Freeline Therapeutics Limited Factor viii polypeptide
CA3161154A1 (en) * 2019-11-14 2021-05-20 Biomarin Pharmaceutical Inc. Treatment of hereditary angioedema with liver-specific gene therapy vectors
EP4069269A4 (en) * 2019-12-06 2023-12-27 The Children's Hospital Of Philadelphia COMPOSITIONS AND METHODS FOR MODULATING FACTOR VIII FUNCTION
US20230023826A1 (en) 2019-12-12 2023-01-26 Takeda Pharmaceutical Company Limited Gene therapy of hemophilia a using viral vectors encoding recombinant fviii variants with increased expression
WO2021236908A2 (en) 2020-05-20 2021-11-25 Biomarin Pharmaceutical Inc. Use of regulatory proteins for the production of adeno-associated virus
WO2021262963A1 (en) 2020-06-24 2021-12-30 Bioverativ Therapeutics Inc. Methods for the removal of free factor viii from preparations of lentiviral vectors modified to express said protein
US20230407328A1 (en) 2020-11-02 2023-12-21 Biomarin Pharmaceutical Inc. Process for enriching adeno-associated virus
AU2021391433A1 (en) * 2020-12-01 2023-06-22 Akouos, Inc. Anti-vegf antibody constructs and related methods for treating vestibular schwannoma associated symptoms
CN116710149A (zh) * 2020-12-18 2023-09-05 桑格摩生物治疗股份有限公司 含有腺相关病毒载体的改善的药物组合物
JP2024527252A (ja) 2021-06-14 2024-07-24 武田薬品工業株式会社 発現の増加した組み換えｆｖｉｉｉバリアントをコードするウイルスベクターを用いた、血友病ａの遺伝子療法
EP4359547A1 (en) * 2021-06-22 2024-05-01 Pfizer Inc. Production of adeno-associated virus vector in insect cells
EP4377466A4 (en) * 2021-07-28 2025-11-05 Biomarin Pharm Inc LARGE-SCALE ADENE-ASSOCIATED VIRUS PRODUCTION SYSTEMS
KR20240049332A (ko) * 2021-08-23 2024-04-16 바이오버라티브 테라퓨틱스 인크. 최적화된 인자 viii 유전자
WO2023036054A2 (en) * 2021-09-08 2023-03-16 Inspirar Limited Composition and method for treating hemophilia
WO2023135273A2 (en) 2022-01-14 2023-07-20 Anjarium Biosciences Ag Compositions of dna molecules encoding factor viii, methods of making thereof, and methods of use thereof
WO2023173078A2 (en) * 2022-03-11 2023-09-14 Homology Medicines, Inc. Bidirectional dual promoter expression vectors and uses thereof
US20250276093A1 (en) * 2022-04-20 2025-09-04 AskBio Inc. Therapeutic adeno-associated virus using codon optimized nucleic acid encoding factor viii
TW202417631A (zh) 2022-09-22 2024-05-01 美商拜奧馬林製藥公司 用ａａｖ基因治療載體治療心肌病
TW202421788A (zh) 2022-09-22 2024-06-01 美商拜奧馬林製藥公司 用ａａｖ基因療法載體治療致心律不整性心肌病
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
WO2024074143A1 (en) * 2022-10-08 2024-04-11 Lingyi Biotech Co., Ltd. Construct for enhancing gene expression
CN116555266B (zh) * 2023-06-09 2024-11-12 呈诺再生医学科技(北京)有限公司 肝脏特异性表达的重组启动子和载体及其在肝脏相关疾病治疗中的应用
WO2025008774A1 (en) 2023-07-05 2025-01-09 Takeda Pharmaceutical Company Limited Viral vectors encoding recombinant fviii variants with increased expression for gene therapy of hemophilia a
WO2025214477A1 (en) 2024-04-12 2025-10-16 Skyline Therapeutics (Shanghai) Co., Ltd. Treatment of genetic cardiomyopathies with aav gene therapy vectors
CN118064436B (zh) * 2024-04-18 2024-07-26 上海凌医生物科技有限公司 一种增强基因表达的嵌合启动子
WO2025239324A1 (ja) * 2024-05-13 2025-11-20 中外製薬株式会社 遺伝子改変非ヒト動物

Family Cites Families (20)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4745051A (en) 1983-05-27 1988-05-17 The Texas A&M University System Method for producing a recombinant baculovirus expression vector
EP0127839B1 (en) 1983-05-27 1992-07-15 THE TEXAS A&M UNIVERSITY SYSTEM Method for producing a recombinant baculovirus expression vector
ZA848495B (en) 1984-01-31 1985-09-25 Idaho Res Found Production of polypeptides in insect cells
KR100356615B1 (ko) * 1993-07-13 2003-04-03 아방티 파르마 소시에테 아노님 결함아데노바이러스벡터및유전자치료에서그의사용
US6204059B1 (en) 1994-06-30 2001-03-20 University Of Pittsburgh AAV capsid vehicles for molecular transfer
US6521225B1 (en) 1996-09-06 2003-02-18 Chiron Corporation AAV vectors
WO2000011149A1 (en) * 1998-08-24 2000-03-02 Uab Research Foundation Methods of producing high titer recombinant adeno-associated virus
US6200560B1 (en) 1998-10-20 2001-03-13 Avigen, Inc. Adeno-associated virus vectors for expression of factor VIII by target cells
US6221349B1 (en) 1998-10-20 2001-04-24 Avigen, Inc. Adeno-associated vectors for expression of factor VIII by target cells
US6723551B2 (en) 2001-11-09 2004-04-20 The United States Of America As Represented By The Department Of Health And Human Services Production of adeno-associated virus in insect cells
WO2003074714A1 (en) 2002-03-05 2003-09-12 Stichting Voor De Technische Wetenschappen Baculovirus expression system
ES2436741T3 (es) 2004-09-22 2014-01-07 St. Jude Children's Research Hospital Expresión mejorada de Factor IX en vectores de terapia génica
CN1286981C (zh) * 2004-11-30 2006-11-29 华中科技大学同济医学院附属同济医院 表达人类cyp2j2反义基因的重组腺相关病毒及其制备方法
EP1739179A1 (en) * 2005-06-30 2007-01-03 Octapharma AG Serum-free stable transfection and production of recombinant human proteins in human cell lines
US7943374B2 (en) * 2005-08-21 2011-05-17 Markus Hildinger Super-size adeno-associated viral vector harboring a recombinant genome larger than 5.7 kb
US7943379B2 (en) 2008-04-30 2011-05-17 Nationwide Children's Hospital, Inc. Production of rAAV in vero cells using particular adenovirus helpers
GB0911870D0 (en) * 2009-07-08 2009-08-19 Ucl Business Plc Optimised coding sequence and promoter
KR20140083035A (ko) * 2011-10-18 2014-07-03 체에스엘 베링 게엠베하 인자 ｖｉｉｉ의 생체이용률을 증가시키기 위한 황산화 글리코사미노글리칸과 하이알루로니다제의 병용 사용
CN103215308B (zh) * 2013-02-01 2015-12-23 中国科学院苏州生物医学工程技术研究所 表达重组人fviii的整合质粒、细胞株及其构建方法和应用
ES2813698T3 (es) 2013-09-12 2021-03-24 Biomarin Pharm Inc Vectores de AAV que comprenden un gen que codifica el factor VIII

Also Published As

Publication number Publication date
FR23C1007I1 (fr) 2023-03-24
EP3770169A1 (en) 2021-01-27
CA3178379A1 (en) 2015-03-19
AU2014318890B2 (en) 2019-03-07
US20200101140A1 (en) 2020-04-02
JP6735672B2 (ja) 2020-08-05
KR20160049015A (ko) 2016-05-04
US20220339262A1 (en) 2022-10-27
US9504762B2 (en) 2016-11-29
RU2710307C2 (ru) 2019-12-26
IL244404B (en) 2021-10-31
JP2019193675A (ja) 2019-11-07
TW201546285A (zh) 2015-12-16
AU2014318890A1 (en) 2016-02-25
JP2021072871A (ja) 2021-05-13
HUE050484T2 (hu) 2020-12-28
RS61039B1 (sr) 2020-12-31
CA2921232A1 (en) 2015-03-19
HUS2300003I1 (hu) 2023-01-28
CN112538501A (zh) 2021-03-23
NL301222I1 (es) 2023-03-01
RU2019141647A3 (es) 2020-05-18
IL244404A0 (en) 2016-04-21
SI3044231T1 (sl) 2020-12-31
LT3044231T (lt) 2020-11-10
NL301222I2 (nl) 2023-10-19
US12239692B2 (en) 2025-03-04
HRP20201324T1 (hr) 2020-11-27
US10463718B2 (en) 2019-11-05
KR102484396B1 (ko) 2023-01-04
US20170095538A1 (en) 2017-04-06
AR097657A1 (es) 2016-04-06
ES2813698T8 (es) 2022-01-26
CY2023006I1 (el) 2023-06-09
LUC00298I2 (es) 2025-09-22
BR112016005576A8 (pt) 2022-10-18
LTPA2023508I1 (es) 2023-04-11
BR112016005576A2 (pt) 2017-09-12
CA3178384A1 (en) 2015-03-19
CN105636981B (zh) 2020-11-06
SMT202000458T1 (it) 2020-11-10
RU2019141647A (ru) 2020-01-27
SG11201601932UA (en) 2016-04-28
NZ716549A (en) 2022-10-28
CN105636981A (zh) 2016-06-01
FIC20230007I1 (fi) 2023-02-08
US20180161403A1 (en) 2018-06-14
PH12016500328A1 (en) 2016-05-02
TWI632239B (zh) 2018-08-11
CY2023006I2 (el) 2023-11-15
ES2813698T3 (es) 2021-03-24
JP2025113388A (ja) 2025-08-01
DK3044231T3 (da) 2020-09-07
JP7523211B2 (ja) 2024-07-26
RU2016113702A (ru) 2017-10-17
US20250332224A1 (en) 2025-10-30
EP3044231B1 (en) 2020-08-05
US20150071883A1 (en) 2015-03-12
JP2016534739A (ja) 2016-11-10
MX2021012014A (es) 2021-10-22
CA2921232C (en) 2023-03-14
IL286859A (en) 2021-10-31
US11406690B2 (en) 2022-08-09
MX2016003262A (es) 2016-09-29
LTC3044231I2 (es) 2025-04-10
HK1226416A1 (en) 2017-09-29
FR23C1007I2 (fr) 2024-06-14
CY1124213T1 (el) 2022-03-24
NO2023003I1 (no) 2023-01-12
KR20230006058A (ko) 2023-01-10
PL3044231T3 (pl) 2021-01-11
JP2023133597A (ja) 2023-09-22
WO2015038625A1 (en) 2015-03-19
PT3044231T (pt) 2020-08-27
EP3044231A1 (en) 2016-07-20
CL2016000502A1 (es) 2016-10-21
PE20160769A1 (es) 2016-08-12

Similar Documents

Publication Publication Date Title
MX386802B (es) Vectores del factor viii del virus adeno-asociado.
CY1122653T1 (el) Αντισωματα αντι-τιgιτ, αντισωματα αντι-ρvrig και συνδυασμοι εξ αυτων
IL268644A (en) Adeno-associated virus (aav) clade f vector and uses therefor
LT3428194T (lt) Pagerinta dvejopo savitumo polipeptido molekulė
FR3021319B1 (fr) Peptides, compositions les comprenant et utilisations notamment cosmetiques
EP3221456A4 (en) Genome-modified recombinant adeno-associated virus vectors
PL3587417T3 (pl) Benzamidy podstawione 1,3-tiazol-2-ilem
EP3802852A4 (en) MANIPULATED CYCLIC PEPTIDES
MX373035B (es) Trastornos neurodegenerativos.
ZA201803551B (en) Personal care composition comprising taurine, arginine, glycine
HUE050362T2 (hu) Javított rekombináns fehérje expresszió hibrid CHEF1 promótert alkalmazva
MX2015012905A (es) Formulaciones de factor viii recombinantes.
UY4241Q (es) Mesa
EP3800199C0 (en) H3.3 CTL PEPTIDES AND THEIR USES
NZ756883A (en) Adeno-associated virus factor viii vectors
BR112016007816A2 (pt) composições peptídicas inovadoras
IT201700030700A1 (it) Punta multifunzione.
Gzelev SMETKOVODNA DELOVA PISMENOST OT B⁄ LGARSKOTO I EVROPEISKOTO SREDNOVEKOVIE
若梅 We are family
庞泽欣 Think New
TR201719150U5 (tr) Koltuklarda sirt mekani̇zmasi
EA201991576A1 (ru) Частичные агонисты инсулинового рецептора
UA97535U (xx) Сорбційно-фільтруючий матеріал
TN2014000522A1 (fr) لوح مدرسي بوجهين
UA118271U (uk) Інформаційна система