NO2025003I1 - Fidanacogene elaparvovec - Google Patents

Fidanacogene elaparvovec

Info

Publication number
NO2025003I1
NO2025003I1 NO2025003C NO2025003C NO2025003I1 NO 2025003 I1 NO2025003 I1 NO 2025003I1 NO 2025003 C NO2025003 C NO 2025003C NO 2025003 C NO2025003 C NO 2025003C NO 2025003 I1 NO2025003 I1 NO 2025003I1
Authority
NO
Norway
Prior art keywords
fidanacogene
elaparvovec
fidanacogene elaparvovec
Prior art date
Application number
NO2025003C
Other languages
English (en)
Norwegian (no)
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 NO2025003I1 publication Critical patent/NO2025003I1/no

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
    • 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
    • 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)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Biophysics (AREA)
  • Epidemiology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Physics & Mathematics (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Plant Pathology (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)
NO2025003C 2015-06-23 2025-01-07 Fidanacogene elaparvovec NO2025003I1 (no)

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 (1)

Publication Number Publication Date
NO2025003I1 true NO2025003I1 (no) 2025-01-07

Family

ID=57586246

Family Applications (1)

Application Number Title Priority Date Filing Date
NO2025003C NO2025003I1 (no) 2015-06-23 2025-01-07 Fidanacogene elaparvovec

Country Status (25)

Country Link
US (6) US10799566B2 (enExample)
EP (2) EP4378487A3 (enExample)
JP (3) JP6831779B2 (enExample)
KR (2) KR102415896B1 (enExample)
CN (1) CN108138159A (enExample)
AU (2) AU2016282781A1 (enExample)
CA (1) CA2990193A1 (enExample)
CO (1) CO2018000134A2 (enExample)
DK (1) DK3313991T3 (enExample)
ES (1) ES2989774T3 (enExample)
FI (2) FI3313991T3 (enExample)
FR (1) FR25C1002I1 (enExample)
HR (1) HRP20241180T1 (enExample)
HU (2) HUE068603T2 (enExample)
IL (2) IL309741A (enExample)
MX (2) MX390848B (enExample)
NO (1) NO2025003I1 (enExample)
PE (1) PE20180675A1 (enExample)
PH (1) PH12017502424A1 (enExample)
PL (1) PL3313991T3 (enExample)
PT (1) PT3313991T (enExample)
SA (1) SA517390590B1 (enExample)
SG (1) SG10202100131WA (enExample)
SI (1) SI3313991T1 (enExample)
WO (1) WO2016210170A1 (enExample)

Families Citing this family (60)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP1866422B1 (en) 2005-04-07 2016-04-06 The Trustees of The University of Pennsylvania Method of increasing the function of an aav vector
EP2337849B1 (en) 2008-09-15 2018-06-13 uniQure biopharma B.V. Factor ix polypeptide mutant, its uses and a method for its production
RU2653444C2 (ru) * 2012-02-17 2018-05-08 Дзе Чилдрен'З Хоспитал Оф Филадельфия Композиции вектора aav и способы переноса генов в клетки, органы и ткани
SI3024498T1 (sl) 2013-07-22 2020-07-31 The Children's Hospital Of Philadelphia Različica AAV in sestavki, postopki in uporabe za prenos genov v celice, organe in tkiva
WO2016073693A2 (en) 2014-11-05 2016-05-12 Voyager Therapeutics, Inc. Aadc polynucleotides for the treatment of parkinson's disease
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
IL309741A (en) 2015-06-23 2024-02-01 Childrens Hospital Philadelphia Modified factor ix, and compositions, methods and uses for gene transfer to cells, organs and tissues
US11219695B2 (en) 2016-10-20 2022-01-11 Sangamo Therapeutics, Inc. Methods and compositions for the treatment of Fabry disease
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
ES2966692T3 (es) * 2017-03-15 2024-04-23 Univ North Carolina Chapel Hill Vectores de virus adenoasociados poliploides y métodos de fabricación y uso de los mismos
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
CN108977452B (zh) * 2017-05-31 2022-07-19 北卡罗来纳大学教堂山分校 优化的人凝血因子ix基因表达盒及其应用
JOP20190269A1 (ar) 2017-06-15 2019-11-20 Voyager Therapeutics Inc بولي نوكليوتيدات aadc لعلاج مرض باركنسون
PT3652326T (pt) * 2017-07-10 2024-12-26 Uniqure Ip Bv Meios e métodos para terapia génica de aav em humanos
BR112020012336A2 (pt) * 2017-12-19 2021-03-30 Akouos, Inc. Administração de anticorpos terapêuticos mediada por aav para o ouvido interno
EP3794126A1 (en) 2018-05-15 2021-03-24 Voyager Therapeutics, Inc. Compositions and methods for the treatment of parkinson's disease
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
GB201813528D0 (en) 2018-08-20 2018-10-03 Ucl Business Plc Factor IX encoding nucleotides
MX2021001901A (es) * 2018-08-20 2021-05-27 Ucl Business Ltd Nucleotidos de codificacion del factor ix.
US10842885B2 (en) 2018-08-20 2020-11-24 Ucl Business Ltd Factor IX encoding nucleotides
GB2576508A (en) * 2018-08-20 2020-02-26 Ucl Business Plc Factor IX encoding nucleotides
CA3111047A1 (en) 2018-08-24 2020-02-27 Spark Therapeutics, Inc. Optimized promoter sequences, intron-free expression constructs and methods of use
SG11202103151RA (en) 2018-09-28 2021-04-29 Voyager Therapeutics Inc Frataxin expression constructs having engineered promoters and methods of use thereof
SG11202103734PA (en) 2018-10-18 2021-05-28 Intellia Therapeutics Inc Compositions and methods for expressing factor ix
BR112021007102A2 (pt) * 2018-11-09 2021-08-03 Generation Bio Co. dna com extremidade fechada modificada (cedna) que compreende repetições terminais invertidas modificadas simétricas
MX2021008131A (es) * 2019-01-04 2021-10-13 Sangamo Therapeutics Inc Metodos y composiciones para el tratamiento de la enfermedad de fabry.
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
EP4022097A4 (en) * 2019-08-29 2022-12-14 Siemens Healthcare Diagnostics Inc. REAGENTS AND METHODS FOR DETECTING AAV EXCRETION
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
AU2021244555A1 (en) * 2020-03-24 2022-11-24 Generation Bio Co. Non-viral dna vectors and uses thereof for expressing factor ix therapeutics
JP2023521090A (ja) * 2020-04-07 2023-05-23 ザ・キュレーターズ・オブ・ザ・ユニバーシティ・オブ・ミズーリ ＡＡＶ遺伝子治療のためのＣｐＧフリー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
CA3200401A1 (en) 2020-11-03 2022-05-12 Pfizer Inc. Methods for purification of aav vectors by anion exchange chromatography
AU2021391433A1 (en) 2020-12-01 2023-06-22 Akouos, Inc. Anti-vegf antibody constructs and related methods for treating vestibular schwannoma associated symptoms
WO2022130172A1 (en) 2020-12-15 2022-06-23 Pfizer Inc. Hilic uplc-ms method for separating and analyzing intact adeno-associated virus capsid proteins
WO2022137076A1 (en) 2020-12-23 2022-06-30 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
WO2022208342A1 (en) 2021-04-01 2022-10-06 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
CA3216420A1 (en) 2021-04-26 2022-11-03 Seungil Han Adeno-associated viral vector capsids with improved tissue tropism
US20240218397A1 (en) * 2021-05-04 2024-07-04 Regenxbio Inc. Novel aav vectors and methods and uses thereof
CA3224755A1 (en) 2021-06-22 2022-12-29 Pfizer Inc. Production of adeno-associated virus vector in insect cells
CA3225312A1 (en) * 2021-06-23 2022-12-29 Inspirar Limited Composition and method for treating hemophilia b
CN118525089A (zh) 2021-10-20 2024-08-20 罗切斯特大学 年龄相关的白质损失的再生治疗
EP4423119A1 (en) * 2021-10-27 2024-09-04 Regeneron Pharmaceuticals, Inc. Compositions and methods for expressing factor ix for hemophilia b therapy
US20230270818A1 (en) 2021-11-02 2023-08-31 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
EP4509606A4 (en) * 2022-04-19 2025-12-10 Kanglin Biotech Hangzhou Co Ltd CONSTRUCTION OF NUCLEIC ACID FOR THE TREATMENT OF AN INHERITED COAGULATION FACTOR DEFICIENCY AND ITS USE
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
CA2264482A1 (en) 1996-09-06 1998-03-12 The Trustees Of The University Of Pennsylvania 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
WO1999003496A1 (en) 1997-07-21 1999-01-28 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
WO2002092786A2 (en) 2001-03-26 2002-11-21 The Board Of Trustees Of The Leland Stanford Junior University A 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
NZ618298A (en) 2001-11-13 2015-04-24 Univ Pennsylvania A method of detecting and/or identifying adeno-associated virus (aav) sequences and isolating novel sequences identified thereby
JP4810062B2 (ja) 2001-12-17 2011-11-09 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア アデノ随伴ウイルス（ａａｖ）血清型８の配列
AU2003233431A1 (en) 2002-03-22 2003-10-13 Board Of Regents, The University Of Texas System Protamine-adenoviral vector complexes and methods of use
NZ561656A (en) 2002-05-01 2009-03-31 Univ Florida 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
EP2438931B1 (en) 2004-09-22 2013-11-13 St. Jude Children's Research Hospital Improved expression of factor IX in gene therapy vectors
WO2006039253A2 (en) 2004-09-29 2006-04-13 Children's Memorial Hospital Sirna-mediated gene silencing of alpha synuclein
EP1866422B1 (en) 2005-04-07 2016-04-06 The Trustees of The University of Pennsylvania Method of increasing the function of an aav vector
US7588772B2 (en) 2006-03-30 2009-09-15 Board Of Trustees Of The Leland Stamford Junior University AAV capsid library and AAV capsid proteins
EP2016174A2 (en) 2006-04-28 2009-01-21 The Trustees of the University of Pennsylvania Modified aav vectors having reduced capsid immunogenicity and use thereof
US8198421B2 (en) * 2006-06-19 2012-06-12 Asklepios Biopharmaceutical, Inc. Modified factor VIII and factor IX genes and vectors for gene therapy
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
EP2337849B1 (en) * 2008-09-15 2018-06-13 uniQure biopharma B.V. Factor ix polypeptide mutant, its uses and a method for its production
GB0911870D0 (en) 2009-07-08 2009-08-19 Ucl Business Plc Optimised coding sequence and promoter
CN102869385A (zh) * 2009-08-18 2013-01-09 巴克斯特国际公司 针对组织因子途径抑制物的适体和其作为出血病症治疗剂的用途
JP5963743B2 (ja) 2010-04-23 2016-08-03 ユニバーシティ オブ マサチューセッツ Ｃｎｓターゲティングａａｖベクターおよびその使用方法
PT3486320T (pt) 2010-04-23 2022-03-30 Univ Florida Composições de ciclase raav-guanilato e métodos para tratar a amaurose congénita 1 de leber (acl1)
KR101853405B1 (ko) 2010-10-20 2018-05-02 주식회사 티움바이오 인자 ｉｘ 활성을 갖는 융합 단백질
AU2012340567B2 (en) 2011-11-22 2017-11-23 The Children's Hospital Of Philadelphia Virus vectors for highly efficient transgene delivery
WO2013078316A1 (en) * 2011-11-23 2013-05-30 Nationwide Children's Hospital, Inc. Recombinant adeno-associated virus delivery of alpha-sarcoglycan polynucleotides
HUE043537T2 (hu) 2012-02-15 2019-08-28 Bioverativ Therapeutics Inc Rekombináns VIII faktor fehérjék
RU2653444C2 (ru) 2012-02-17 2018-05-08 Дзе Чилдрен'З Хоспитал Оф Филадельфия Композиции вектора aav и способы переноса генов в клетки, органы и ткани
HUE054087T2 (hu) 2012-04-18 2021-09-28 Childrens Hospital Philadelphia Készítmények és eljárások nagy hatékonyságú géntranszferre AAV kapszidvariánsok alkalmazásával
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
US10398787B2 (en) 2012-10-26 2019-09-03 Vrije Universiteit Brussel Vectors 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
CN116004721A (zh) 2013-07-12 2023-04-25 费城儿童医院 Aav载体和用于抗aav（腺相关病毒）中和抗体的检测
SI3024498T1 (sl) 2013-07-22 2020-07-31 The Children's Hospital Of Philadelphia Različica AAV in sestavki, postopki in uporabe za prenos genov v celice, organe in tkiva
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
DK3270944T3 (da) 2015-03-17 2020-01-27 Univ Brussel Vrije Optimerede leverspecifikke ekspressionssystemer til FVIII og FIX
IL309741A (en) * 2015-06-23 2024-02-01 Childrens Hospital Philadelphia Modified factor ix, and compositions, methods and uses for gene transfer to cells, organs and tissues
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
EP3313991B1 (en) 2024-07-17
JP2021087431A (ja) 2021-06-10
AU2022268345A1 (en) 2022-12-15
CA2990193A1 (en) 2016-12-29
EP4378487A3 (en) 2024-09-04
JP2024009857A (ja) 2024-01-23
MX2018000307A (es) 2018-05-02
CO2018000134A2 (es) 2018-05-21
EP3313991A1 (en) 2018-05-02
SA517390590B1 (ar) 2022-02-10
KR20200128215A (ko) 2020-11-11
US20210330763A1 (en) 2021-10-28
FR25C1002I1 (fr) 2025-02-28
HUE068603T2 (hu) 2025-01-28
AU2022268345B2 (en) 2025-02-06
US20230142320A1 (en) 2023-05-11
US20160375110A1 (en) 2016-12-29
NZ738841A (en) 2024-10-25
US11896652B2 (en) 2024-02-13
HK1252351A1 (en) 2019-05-24
CN108138159A (zh) 2018-06-08
PH12017502424A1 (en) 2018-07-09
US11110153B2 (en) 2021-09-07
FIC20250001I1 (en) 2025-01-03
SG10202100131WA (en) 2021-02-25
DK3313991T3 (da) 2024-09-09
RU2018102242A3 (enExample) 2020-08-07
IL309741A (en) 2024-02-01
BR112017027956A2 (pt) 2018-08-28
MX390848B (es) 2025-03-21
US11491213B2 (en) 2022-11-08
RU2018102242A (ru) 2019-07-25
HUS2500003I1 (hu) 2025-01-28
EP3313991A4 (en) 2018-12-05
WO2016210170A9 (en) 2018-02-15
IL256517B1 (en) 2024-02-01
US20210008179A1 (en) 2021-01-14
PL3313991T3 (pl) 2024-11-04
JP2018522529A (ja) 2018-08-16
JP6831779B2 (ja) 2021-02-17
AU2016282781A1 (en) 2018-01-18
ES2989774T3 (es) 2024-11-27
US20200129598A1 (en) 2020-04-30
FI3313991T3 (fi) 2024-10-07
EP4378487A2 (en) 2024-06-05
JP7361737B2 (ja) 2023-10-16
PE20180675A1 (es) 2018-04-19
US20240131126A1 (en) 2024-04-25
AU2016282781A9 (en) 2019-08-01
US10799566B2 (en) 2020-10-13
KR102178322B1 (ko) 2020-11-13
IL256517A (en) 2018-02-28
SI3313991T1 (sl) 2024-11-29
HRP20241180T1 (hr) 2024-11-22
PT3313991T (pt) 2024-09-27
IL256517B2 (en) 2024-06-01
KR102415896B1 (ko) 2022-06-30
MX2022003116A (es) 2022-04-11
WO2016210170A1 (en) 2016-12-29
KR20180048580A (ko) 2018-05-10

Similar Documents

Publication Publication Date Title
NO2025003I1 (no) Fidanacogene elaparvovec
DK3331869T3 (da) Muscarinagonister
DK3331528T3 (da) Muskarinagonister
DK3277719T3 (da) Polypeptider
EP3316375A4 (en) Redox flow cell
DK3360890T3 (da) Genterapi
DK3331529T3 (da) Muskarinagonister
DK3310450T3 (da) Olie-vand-separator
EP3290364A4 (en) Conveyor belt wear monitoring system
DK3270930T3 (da) Præeklampsi
EP3381654A4 (en) Liquid blow molding method
DK3394281T3 (da) Gærcelle
EP3363322A4 (en) Hair extender
DK3292136T3 (da) Penicillin-g-acylaser
DE112015006486A5 (de) Inkubationsrinne
EP3548009A4 (en) POLYMERIC NANOPARTICLES
DK3274482T3 (da) Stenborsknop
EP3377179A4 (en) DEUTERED EPI-743
DE102015206660A8 (de) Plattenaufteilanlage
DK3318308T3 (da) Badmintonketsjer
DE112015005904A5 (de) Gurtschlossbringer
DE112016005599A5 (de) Strahlungsgrill
EP3353789A4 (en) SpinRAM
EP3307242A4 (en) PICn-01
EP3308871A4 (en) CRIMPING DEVICE