[go: up one dir, main page]

MX2018000307A - Factor ix modificado, y composiciones, metodos y usos para la transferencia de genes a celulas, organos y tejidos. - Google Patents

Factor ix modificado, y composiciones, metodos y usos para la transferencia de genes a celulas, organos y tejidos.

Info

Publication number
MX2018000307A
MX2018000307A MX2018000307A MX2018000307A MX2018000307A MX 2018000307 A MX2018000307 A MX 2018000307A MX 2018000307 A MX2018000307 A MX 2018000307A MX 2018000307 A MX2018000307 A MX 2018000307A MX 2018000307 A MX2018000307 A MX 2018000307A
Authority
MX
Mexico
Prior art keywords
organs
cells
methods
transfer
fabrics
Prior art date
Application number
MX2018000307A
Other languages
English (en)
Other versions
MX390848B (es
Inventor
A High Katherine
Anguela Xavier
Original Assignee
Childrens Hospital Philadelphia
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 Childrens Hospital Philadelphia filed Critical Childrens Hospital Philadelphia
Publication of MX2018000307A publication Critical patent/MX2018000307A/es
Publication of MX390848B publication Critical patent/MX390848B/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
    • A61K38/43—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
    • 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
    • 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
    • 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
    • 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
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/52—Genes encoding for enzymes or proenzymes
    • 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
    • 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
    • C12N15/864—Parvoviral vectors, e.g. parvovirus, densovirus
    • 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
    • C12N15/864—Parvoviral vectors, e.g. parvovirus, densovirus
    • C12N15/8645—Adeno-associated virus
    • 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
    • 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
    • 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
    • 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
    • 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

Landscapes

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

Abstract

La invención se refiere a una secuencia que codifica el Factor IX modificado, el casete de expresión, los vectores tales como vectores virales (por ejemplo, lenti- o adenoasociados virales), y métodos y usos de transferencia genética. En particular, se refiere a dirigir el ácido nucleico del Factor IX a las células, los tejidos u órganos para la expresión (transcripción) del Factor IX.
MX2018000307A 2015-06-23 2016-06-23 Factor ix modificado, y composiciones, metodos y usos para la transferencia de genes a celulas, organos y tejidos. MX390848B (es)

Applications Claiming Priority (6)

Application Number Priority Date Filing Date Title
US201562183599P 2015-06-23 2015-06-23
US201662315453P 2016-03-30 2016-03-30
US201662338315P 2016-05-18 2016-05-18
US201662348781P 2016-06-10 2016-06-10
US201662349572P 2016-06-13 2016-06-13
PCT/US2016/039075 WO2016210170A1 (en) 2015-06-23 2016-06-23 Modified factor ix, and compositions, methods and uses for gene transfer to cells, organs and tissues

Publications (2)

Publication Number Publication Date
MX2018000307A true MX2018000307A (es) 2018-05-02
MX390848B MX390848B (es) 2025-03-21

Family

ID=57586246

Family Applications (2)

Application Number Title Priority Date Filing Date
MX2018000307A MX390848B (es) 2015-06-23 2016-06-23 Factor ix modificado, y composiciones, metodos y usos para la transferencia de genes a celulas, organos y tejidos.
MX2022003116A MX2022003116A (es) 2015-06-23 2018-01-08 Factor ix modificado, y composiciones, metodos y usos para la transferencia de genes a celulas, organos y tejidos.

Family Applications After (1)

Application Number Title Priority Date Filing Date
MX2022003116A MX2022003116A (es) 2015-06-23 2018-01-08 Factor ix modificado, y composiciones, metodos y usos para la transferencia de genes a celulas, organos y tejidos.

Country Status (25)

Country Link
US (6) US10799566B2 (es)
EP (2) EP4378487A3 (es)
JP (3) JP6831779B2 (es)
KR (2) KR102178322B1 (es)
CN (1) CN108138159A (es)
AU (2) AU2016282781A1 (es)
CA (1) CA2990193A1 (es)
CO (1) CO2018000134A2 (es)
DK (1) DK3313991T3 (es)
ES (1) ES2989774T3 (es)
FI (2) FI3313991T3 (es)
FR (1) FR25C1002I2 (es)
HR (1) HRP20241180T1 (es)
HU (2) HUE068603T2 (es)
IL (2) IL309741A (es)
MX (2) MX390848B (es)
NO (1) NO2025003I1 (es)
PE (1) PE20180675A1 (es)
PH (1) PH12017502424A1 (es)
PL (1) PL3313991T3 (es)
PT (1) PT3313991T (es)
SA (1) SA517390590B1 (es)
SG (1) SG10202100131WA (es)
SI (1) SI3313991T1 (es)
WO (1) WO2016210170A1 (es)

Families Citing this family (60)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2006110689A2 (en) * 2005-04-07 2006-10-19 The Trustees Of The University Of Pennsylvania Method of increasing the function of an aav vector
SMT202300084T1 (it) 2008-09-15 2023-05-12 Uniqure Biopharma B V Mutante del polipeptide del fattore ix, suoi usi ed un metodo per la sua produzione
AU2013221212B2 (en) * 2012-02-17 2018-08-09 The Children's Hospital Of Philadelphia AAV vector compositions and methods for gene transfer to cells, organs and tissues
CN105579465B (zh) 2013-07-22 2019-09-10 费城儿童医院 用于基因转移到细胞、器官和组织中的变异aav和组合物、方法及用途
CN107106689A (zh) 2014-11-05 2017-08-29 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
GB201508026D0 (en) 2015-05-11 2015-06-24 Ucl Business Plc Capsid
PL3313991T3 (pl) 2015-06-23 2024-11-04 The Children's Hospital Of Philadelphia Modyfikowany czynnik ix oraz kompozycje, sposoby i zastosowania do transferu genów do komórek, narządów i tkanek
JP7042263B2 (ja) 2016-10-20 2022-03-25 サンガモ セラピューティクス， インコーポレイテッド ファブリー病の治療のための方法および組成物
US10550405B2 (en) 2017-03-15 2020-02-04 The University Of North Carolina At Chapel Hill Rational polyploid adeno-associated virus vectors and methods of making and using the same
CN110770346B (zh) * 2017-03-15 2024-01-12 北卡罗来纳-查佩尔山大学 多倍体腺相关病毒载体及其制备和使用方法
TWI753168B (zh) * 2017-05-22 2022-01-21 日商武田藥品工業股份有限公司 用於血友病ｂ基因療法之編碼具有增強表現的重組ｆｉｘ變異體之病毒載體
MX2019014379A (es) * 2017-05-31 2020-01-23 Univ North Carolina Chapel Hill Casetes de expresion optimizados del gen del factor de coagulacion ix humano y su uso.
JOP20190269A1 (ar) 2017-06-15 2019-11-20 Voyager Therapeutics Inc بولي نوكليوتيدات aadc لعلاج مرض باركنسون
DK3652326T3 (da) * 2017-07-10 2024-10-28 Uniqure Ip Bv Midler og fremgangsmåder til AAV-genterapi i mennesker
IL319982A (en) * 2017-12-19 2025-05-01 Akouos Inc AAV virus-mediated delivery of therapeutic antibodies to the inner ear
JP2021523914A (ja) 2018-05-15 2021-09-09 ボイジャー セラピューティクス インコーポレイテッドＶｏｙａｇｅｒ Ｔｈｅｒａｐｅｕｔｉｃｓ，Ｉｎｃ． パーキンソン病を治療するための組成物および方法
CA3105953A1 (en) 2018-07-09 2020-01-16 Flagship Pioneering Innovations V, Inc. Fusosome compositions and uses thereof
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
GB2576508A (en) * 2018-08-20 2020-02-26 Ucl Business Plc Factor IX encoding nucleotides
GB201813528D0 (en) 2018-08-20 2018-10-03 Ucl Business Plc Factor IX encoding nucleotides
JP2021533799A (ja) * 2018-08-20 2021-12-09 ユーシーエル ビジネス リミテッド 第ｉｘ因子をコードするヌクレオチド
US10842885B2 (en) 2018-08-20 2020-11-24 Ucl Business Ltd Factor IX encoding nucleotides
BR112021003399A2 (pt) * 2018-08-24 2021-05-18 Spark Therapeutics, Inc. sequências de promotores otimizadas, construtos de expressão livres de íntron e métodos de uso
JP7528066B2 (ja) 2018-09-28 2024-08-05 ボイジャー セラピューティクス インコーポレイテッド 操作されたプロモータを有するフラタキシン発現コンストラクトおよびその使用方法
MX2021004277A (es) 2018-10-18 2021-09-08 Intellia Therapeutics Inc Composiciones y metodos para expresar el factor ix.
MA54188A (fr) * 2018-11-09 2021-09-15 Generation Bio Co Adn à extrémité fermée modifié (cedna) comprenant des répétitions terminales inversées modifiées symétriques
SG11202107325TA (en) * 2019-01-04 2021-08-30 Sangamo Therapeutics Inc Methods and compositions for the treatment of fabry disease
WO2020160303A1 (en) 2019-02-01 2020-08-06 Baxalta Incorporated Gene therapy of hemophilia b using viral vectors encoding recombinant fix variants with increased expression
US11857641B2 (en) * 2019-02-06 2024-01-02 Sangamo Therapeutics, Inc. Method for the treatment of mucopolysaccharidosis type I
CN110205297A (zh) * 2019-05-15 2019-09-06 华北理工大学 一种重组脂肪干细胞及其重组方法
WO2021042004A1 (en) * 2019-08-29 2021-03-04 Siemens Healthcare Diagnostics Inc. Reagents and methods for detecting aav shedding
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
MX2022011805A (es) * 2020-03-24 2023-01-11 Generation Bio Co Vectores de adn no virales y usos de estos para expresar agentes terapéuticos con factor ix.
CN115461465A (zh) * 2020-04-07 2022-12-09 密苏里大学管理机构 用于AAV基因治疗的无CpG的ITR
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
AU2021375404A1 (en) 2020-11-03 2023-06-08 Pfizer Inc. Methods for purification of aav vectors by anion exchange chromatography
CR20230228A (es) 2020-12-01 2023-07-18 Akouos Inc Construcciones de anticuerpos anti-vegf y métodos relacionados para el tratamiento de los síntomas asociados al schwannoma vestibular
WO2022130172A1 (en) 2020-12-15 2022-06-23 Pfizer Inc. Hilic uplc-ms method for separating and analyzing intact adeno-associated virus capsid proteins
EP4267726A1 (en) 2020-12-23 2023-11-01 Pfizer Inc. Methods for purification of aav vectors by affinity chromatography
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
US20240307553A1 (en) 2021-04-01 2024-09-19 Pfizer Inc. Pharmaceutical compositions containing adeno-associated viral vector
EP4079860A1 (en) * 2021-04-23 2022-10-26 uniQure biopharma B.V. Methods and means for the prevention and/or treatment of joint damage in hemophilia
EP4329820A1 (en) 2021-04-26 2024-03-06 Alexion Pharma International Operations Limited Adeno-associated viral vector capsids with improved tissue tropism
WO2022235614A2 (en) * 2021-05-04 2022-11-10 Regenxbio Inc. Novel aav vectors and methods and uses thereof
KR20240023638A (ko) 2021-06-22 2024-02-22 화이자 인코포레이티드 곤충 세포에서의 아데노-연관 바이러스 벡터의 생산
WO2022268110A1 (zh) * 2021-06-23 2022-12-29 英斯培瑞有限公司 用于治疗b型血友病的组合物和方法
AU2022368907A1 (en) 2021-10-20 2024-05-02 University Of Copenhagen Rejuvenation treatment of age-related white matter loss
KR20240099358A (ko) * 2021-10-27 2024-06-28 리제너론 파마슈티칼스 인코포레이티드 B형 혈우병 치료를 위한 인자 ix를 발현하기 위한 조성물 및 방법
CA3236365A1 (en) 2021-11-02 2023-05-11 University Of Rochester Tcf7l2 mediated remyelination in the brain
EP4198134A1 (en) * 2021-12-16 2023-06-21 Genethon Gamma-sarcoglycan gene transfer increase using modified itr sequences
WO2023202469A1 (zh) * 2022-04-19 2023-10-26 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及其用途
WO2024003687A1 (en) 2022-06-28 2024-01-04 Pfizer Inc. Nucleic acids encoding acid alpha-glucosidase (gaa) and vectors for gene therapy
WO2024038365A1 (en) 2022-08-16 2024-02-22 Pfizer Inc. Methods for purification of aav vectors by anion exchange chromatography
TW202426479A (zh) * 2022-10-10 2024-07-01 美商星火治療公司 Apoe基因療法
WO2024091824A1 (en) 2022-10-26 2024-05-02 Ada Forsyth Institute, Inc. Differentiation and reprogramming of chondrocyte
EP4658294A2 (en) 2023-02-02 2025-12-10 University of Rochester Competitive replacement of glial cells
WO2025090427A1 (en) 2023-10-23 2025-05-01 University Of Rochester Glial-targeted relief of hyperexcitability in neurodegenerative diseases
WO2025143742A1 (ko) * 2023-12-26 2025-07-03 삼성바이오에피스 주식회사 혈액응고인자 IX를 암호화하는 코돈-최적화된 폴리뉴클레오티드 및 이를 포함하는 rAAV 생산용 플라스미드
WO2025250457A1 (en) 2024-05-28 2025-12-04 University Of Rochester Enhanced brain transduction by gene therapeutics

Family Cites Families (45)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6268213B1 (en) 1992-06-03 2001-07-31 Richard Jude Samulski Adeno-associated virus vector and cis-acting regulatory and promoter elements capable of expressing at least one gene and method of using same for gene therapy
AU722624B2 (en) 1996-09-06 2000-08-10 Trustees Of The University Of Pennsylvania, The An inducible method for production of recombinant adeno-associated viruses utilizing T7 polymerase
US6156303A (en) 1997-06-11 2000-12-05 University Of Washington Adeno-associated virus (AAV) isolates and AAV vectors derived therefrom
US6531298B2 (en) 1997-07-21 2003-03-11 The University Of North Carolina At Chapel Hill Factor IX antihemophilic factor with increased clotting activity
US6200560B1 (en) 1998-10-20 2001-03-13 Avigen, Inc. Adeno-associated virus vectors for expression of factor VIII by target cells
US6468524B1 (en) 2000-03-22 2002-10-22 The United States Of America, As Represented By The Secretary Of The Department Of Health And Human Services AAV4 vector and uses thereof
US7351813B2 (en) * 2000-06-20 2008-04-01 The Board Of Trustees Of The Leland Stanford Junior University Liver-specific gene expression cassettes, and methods of use
US6943012B2 (en) 2001-03-26 2005-09-13 The Board Of Trustees Of The Leland Stanford Junor University Helper dependent adenoviral vector system and methods for using the same
US20080220015A1 (en) 2001-08-03 2008-09-11 Nokad, S.A. Method of modulating neutralizing antibodies formation in mammals, and uses thereof in gene therapy, animal trangenesis and in functional inactivation of endogenous proteins
HU230406B1 (hu) 2001-11-13 2016-04-28 The Trustees Of The University Of Pennsylvania Eljárás adeno-asszociált vírus(AAV)szekvenciák detektálására és/vagy azonosítására és az azzal azonosított új szekvenciák izolálására
FI3517134T3 (fi) 2001-12-17 2024-04-03 Univ Pennsylvania Adeno-assosioituneen viruksen (aav) serotyypin 8 sekvenssejä, niitä sisältäviä vektoreita ja niiden käyttöjä
AU2003233431A1 (en) 2002-03-22 2003-10-13 Board Of Regents, The University Of Texas System Protamine-adenoviral vector complexes and methods of use
AU2003295312B2 (en) 2002-05-01 2008-08-14 University Of Florida Research Foundation, Inc. Improved rAAV expression systems for genetic modification of specific capsid proteins
WO2005037226A2 (en) * 2003-10-17 2005-04-28 Georgia Tech Research Corporation Genetically engineered enteroendocrine cells for treating glucose-related metabolic disorders
EP1804839B1 (en) 2004-09-22 2012-03-14 St. Jude Children's Research Hospital Improved expression of factor ix in gene therapy vectors
EP1799826B1 (en) 2004-09-29 2009-08-12 Children's Memorial Hospital siRNA-MEDIATED GENE SILENCING OF ALPHA SYNUCLEIN
WO2006110689A2 (en) 2005-04-07 2006-10-19 The Trustees Of The University Of Pennsylvania Method of increasing the function of an aav vector
EP2007795B1 (en) 2006-03-30 2016-11-16 The Board Of Trustees Of The Leland Stanford Junior University Aav capsid proteins
CN101495624A (zh) 2006-04-28 2009-07-29 宾夕法尼亚州立大学托管会 衣壳免疫原性降低的经修饰aav载体及其用途
CN104087591A (zh) * 2006-06-19 2014-10-08 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
JP5797551B2 (ja) 2008-04-22 2015-10-21 フエー・イー・ベー・フエー・ゼツト・ウエー 肝特異的核酸調節要素ならびにその方法および用途
SMT202300084T1 (it) 2008-09-15 2023-05-12 Uniqure Biopharma B V Mutante del polipeptide del fattore ix, suoi usi ed un metodo per la sua produzione
GB0911870D0 (en) 2009-07-08 2009-08-19 Ucl Business Plc Optimised coding sequence and promoter
AU2010284329B2 (en) * 2009-08-18 2015-04-16 Takeda Pharmaceutical Company Limited Aptamers to tissue factor pathway inhibitor and their use as bleeding disorder therapeutics
SG184876A1 (en) 2010-04-23 2012-11-29 Univ Florida Raav-guanylate cyclase compositions and methods for treating leber's congenital amaurosis-1 (lca1)
EP3514232A1 (en) 2010-04-23 2019-07-24 University of Massachusetts Cns targeting aav vectors and methods of use thereof
KR101853405B1 (ko) 2010-10-20 2018-05-02 주식회사 티움바이오 인자 ｉｘ 활성을 갖는 융합 단백질
HK1202434A1 (en) 2011-11-22 2015-10-02 The Children's Hospital Of Philadelphia Virus vectors for highly efficient transgene delivery
US9434928B2 (en) 2011-11-23 2016-09-06 Nationwide Children's Hospital, Inc. Recombinant adeno-associated virus delivery of alpha-sarcoglycan polynucleotides
BR112014019901A8 (pt) 2012-02-15 2018-01-02 Biogen Idec Inc Proteínas de fator viii recombinante
AU2013221212B2 (en) 2012-02-17 2018-08-09 The Children's Hospital Of Philadelphia AAV vector compositions and methods for gene transfer to cells, organs and tissues
DK2839014T3 (da) 2012-04-18 2021-03-08 Childrens Hospital Philadelphia Sammensætning og fremgangsmåder til højeffektiv genoverførsel ved anvendelse af aav-capsid-varianter
WO2014003553A1 (en) * 2012-06-27 2014-01-03 Arthrogen B.V. Combination for treating an inflammatory disorder
GB201213117D0 (en) * 2012-07-24 2012-09-05 Ucl Business Plc Transgene expression
EP3513802B1 (en) 2012-10-26 2023-11-29 Vrije Universiteit Brussel Vector for liver-directed gene therapy of hemophilia and methods and use thereof
US20140271550A1 (en) 2013-03-14 2014-09-18 The Trustees Of The University Of Pennsylvania Constructs and Methods for Delivering Molecules via Viral Vectors with Blunted Innate Immune Responses
US9700486B2 (en) 2013-04-24 2017-07-11 Corning Incorporated Delamination resistant pharmaceutical glass containers containing active pharmaceutical ingredients
JP2016525347A (ja) 2013-07-12 2016-08-25 ザ・チルドレンズ・ホスピタル・オブ・フィラデルフィアＴｈｅ Ｃｈｉｌｄｒｅｎ’Ｓ Ｈｏｓｐｉｔａｌ Ｏｆ Ｐｈｉｌａｄｅｌｐｈｉａ Ａａｖベクター、及び抗ａａｖ（アデノ関連ウイルス）中和抗体についてのアッセイ
CN105579465B (zh) 2013-07-22 2019-09-10 费城儿童医院 用于基因转移到细胞、器官和组织中的变异aav和组合物、方法及用途
NL2012981B1 (en) * 2014-06-11 2017-01-17 Heineken Supply Chain Bv Beverage dispensing system, beverage container and pressurizing system for use in a beverage dispensing system or container.
GB201420139D0 (en) 2014-11-12 2014-12-24 Ucl Business Plc Factor IX gene therapy
CA2979495A1 (en) 2015-03-17 2016-09-22 Vrije Universiteit Brussel Optimized liver-specific expression systems for fviii and fix
PL3313991T3 (pl) * 2015-06-23 2024-11-04 The Children's Hospital Of Philadelphia Modyfikowany czynnik ix oraz kompozycje, sposoby i zastosowania do transferu genów do komórek, narządów i tkanek
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
WO2017180861A1 (en) 2016-04-15 2017-10-19 The Trustees Of The University Of Pennsulvania Gene therapy for treating hemophilia b

Also Published As

Publication number Publication date
US20240131126A1 (en) 2024-04-25
JP7361737B2 (ja) 2023-10-16
NZ738841A (en) 2024-10-25
US11896652B2 (en) 2024-02-13
FR25C1002I1 (fr) 2025-02-28
US20210330763A1 (en) 2021-10-28
US10799566B2 (en) 2020-10-13
BR112017027956A2 (pt) 2018-08-28
FI3313991T3 (fi) 2024-10-07
IL256517B2 (en) 2024-06-01
JP2024009857A (ja) 2024-01-23
FR25C1002I2 (fr) 2026-01-02
SG10202100131WA (en) 2021-02-25
US11110153B2 (en) 2021-09-07
KR102415896B1 (ko) 2022-06-30
US20160375110A1 (en) 2016-12-29
PT3313991T (pt) 2024-09-27
ES2989774T3 (es) 2024-11-27
FIC20250001I1 (en) 2025-01-03
HUE068603T2 (hu) 2025-01-28
KR20200128215A (ko) 2020-11-11
US20200129598A1 (en) 2020-04-30
NO2025003I1 (no) 2025-01-07
CO2018000134A2 (es) 2018-05-21
EP3313991A1 (en) 2018-05-02
EP4378487A3 (en) 2024-09-04
IL256517A (en) 2018-02-28
CN108138159A (zh) 2018-06-08
HRP20241180T1 (hr) 2024-11-22
SA517390590B1 (ar) 2022-02-10
US20210008179A1 (en) 2021-01-14
CA2990193A1 (en) 2016-12-29
AU2022268345B2 (en) 2025-02-06
KR20180048580A (ko) 2018-05-10
EP4378487A2 (en) 2024-06-05
AU2016282781A1 (en) 2018-01-18
DK3313991T3 (da) 2024-09-09
US20230142320A1 (en) 2023-05-11
PE20180675A1 (es) 2018-04-19
RU2018102242A (ru) 2019-07-25
IL309741A (en) 2024-02-01
JP2021087431A (ja) 2021-06-10
EP3313991B1 (en) 2024-07-17
WO2016210170A1 (en) 2016-12-29
HK1252351A1 (en) 2019-05-24
IL256517B1 (en) 2024-02-01
WO2016210170A9 (en) 2018-02-15
US11491213B2 (en) 2022-11-08
EP3313991A4 (en) 2018-12-05
KR102178322B1 (ko) 2020-11-13
JP6831779B2 (ja) 2021-02-17
PH12017502424A1 (en) 2018-07-09
HUS2500003I1 (hu) 2025-01-28
MX2022003116A (es) 2022-04-11
AU2016282781A9 (en) 2019-08-01
RU2018102242A3 (es) 2020-08-07
SI3313991T1 (sl) 2024-11-29
MX390848B (es) 2025-03-21
PL3313991T3 (pl) 2024-11-04
AU2022268345A1 (en) 2022-12-15
JP2018522529A (ja) 2018-08-16

Similar Documents

Publication Publication Date Title
MX2018000307A (es) Factor ix modificado, y composiciones, metodos y usos para la transferencia de genes a celulas, organos y tejidos.
CL2023000294A1 (es) Expresión selectiva de un transgene de tejidos
CO2017004728A2 (es) Composiciones y métodos para inhibir la expresión del gen hao1 (hidroxiácido-oxidasa 1 (glicolato-oxidasa)
MX2016001044A (es) Aav variante y composiciones, metodos y usos para transferencia genica a celulas, organos y tejidos.
AR132094A2 (es) Composiciones detergentes, variantes de lipasa y polinucleótidos que las codifican
CL2017002820A1 (es) Variantes de alfa-amilasa y polinucleótidos que codifican las mismas
MX2021004282A (es) Construcciones de ácido nucleico y métodos de uso.
AR134186A2 (es) Un anticuerpo anti-csf-1r, un proceso para producirlo, una composición farmacéutica que lo comprende, una secuencia de adn aislado, un vector de expresión o clonación y una célula hospedadora
MX2020001309A (es) Anticuerpos anti-cd47 y sus usos.
CL2018001107A1 (es) Variantes del factor viii cpg reducido, composiciones y métodos y usos para el tratamiento de trastornos de la hemostasis.
CO2019000395A2 (es) Genes de mini–distrofina optimizados y casetes de expresión y su uso
MX2021011676A (es) Polimorfos de sepiapterina y sales de los mismos.
BR112018011881A2 (pt) proteínas capsidiais modificadas para liberação aumentada de vetores de parvovírus
CL2017002731A1 (es) Adenovirus oncolítico que codifica una proteína b7
MX2018013139A (es) Composiciones detergentes y sus usos.
MX2017016688A (es) Nuevas enzimas y sistemas crispr.
MX2016014129A (es) Composiciones para el control de mosquitos y usos de las mismas.
MX368723B (es) Composiciones y metodos para inhibir la expresion del gen alas1.
MX2017008190A (es) Metodos y composiciones para la modificacion genetica dirigida a traves de direccionamiento multiple de una sola etapa.
MX2018005392A (es) Acidos nucleicos modificados dirigidos a acidos nucleicos.
CL2019000033A1 (es) Variantes de xilanasa y polinucleótidos que las codifican.
MX2019015143A (es) Rna guias modificados, complejos crispr-ribonucleoproteina y metodos de uso.
MX2021013913A (es) Composiciones y metodos para el tratamiento de enfermedades mediadas por atpasa.
CR20160195A (es) Composiciones y métodos para inhibir la expresión del gen alas 1
AR105416A1 (es) VARIANTES DE a-AMILASA Y POLINUCLEÓTIDOS QUE LAS CODIFICAN