JP6831779B2 - 修飾された第ｉｘ因子、並びに、細胞、器官及び組織への遺伝子導入のための組成物、方法及び使用 - Google Patents

修飾された第ｉｘ因子、並びに、細胞、器官及び組織への遺伝子導入のための組成物、方法及び使用 Download PDF

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JP6831779B2
JP6831779B2 JP2017518931A JP2017518931A JP6831779B2 JP 6831779 B2 JP6831779 B2 JP 6831779B2 JP 2017518931 A JP2017518931 A JP 2017518931A JP 2017518931 A JP2017518931 A JP 2017518931A JP 6831779 B2 JP6831779 B2 JP 6831779B2
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nucleic acid
sequence
aav
seq
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JP2018522529A (ja
JP2018522529A5 (OSRAM
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エー． ハイ，キャサリン
エー． ハイ，キャサリン
アンゲラ，ザビエル
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Childrens Hospital of Philadelphia CHOP
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    • 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
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    • 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
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    • 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)
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    • C12Y304/00—Hydrolases acting on peptide bonds, i.e. peptidases (3.4)
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    • C12Y304/21022—Coagulation factor IXa (3.4.21.22)
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    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
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JP2017518931A 2015-06-23 2016-06-23 修飾された第ｉｘ因子、並びに、細胞、器官及び組織への遺伝子導入のための組成物、方法及び使用 Active JP6831779B2 (ja)

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Application Number Priority Date Filing Date Title
JP2021012811A JP7361737B2 (ja) 2015-06-23 2021-01-29 修飾された第ｉｘ因子、並びに、細胞、器官及び組織への遺伝子導入のための組成物、方法及び使用
JP2023171763A JP2024009857A (ja) 2015-06-23 2023-10-03 修飾された第ｉｘ因子、並びに、細胞、器官及び組織への遺伝子導入のための組成物、方法及び使用

Applications Claiming Priority (11)

Application Number Priority Date Filing Date Title
US201562183599P 2015-06-23 2015-06-23
US62/183,599 2015-06-23
US201662315453P 2016-03-30 2016-03-30
US62/315,453 2016-03-30
US201662338315P 2016-05-18 2016-05-18
US62/338,315 2016-05-18
US201662348781P 2016-06-10 2016-06-10
US62/348,781 2016-06-10
US201662349572P 2016-06-13 2016-06-13
US62/349,572 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

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JP2018522529A JP2018522529A (ja) 2018-08-16
JP2018522529A5 JP2018522529A5 (OSRAM) 2019-07-25
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JP2017518931A Active JP6831779B2 (ja) 2015-06-23 2016-06-23 修飾された第ｉｘ因子、並びに、細胞、器官及び組織への遺伝子導入のための組成物、方法及び使用
JP2021012811A Active JP7361737B2 (ja) 2015-06-23 2021-01-29 修飾された第ｉｘ因子、並びに、細胞、器官及び組織への遺伝子導入のための組成物、方法及び使用
JP2023171763A Pending JP2024009857A (ja) 2015-06-23 2023-10-03 修飾された第ｉｘ因子、並びに、細胞、器官及び組織への遺伝子導入のための組成物、方法及び使用

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JP2023171763A Pending JP2024009857A (ja) 2015-06-23 2023-10-03 修飾された第ｉｘ因子、並びに、細胞、器官及び組織への遺伝子導入のための組成物、方法及び使用

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US (6) US10799566B2 (OSRAM)
EP (2) EP3313991B1 (OSRAM)
JP (3) JP6831779B2 (OSRAM)
KR (2) KR102415896B1 (OSRAM)
CN (1) CN108138159A (OSRAM)
AU (2) AU2016282781A1 (OSRAM)
CA (1) CA2990193A1 (OSRAM)
CO (1) CO2018000134A2 (OSRAM)
DK (1) DK3313991T3 (OSRAM)
ES (1) ES2989774T3 (OSRAM)
FI (2) FI3313991T3 (OSRAM)
FR (1) FR25C1002I1 (OSRAM)
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HU (2) HUE068603T2 (OSRAM)
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PT (1) PT3313991T (OSRAM)
SA (1) SA517390590B1 (OSRAM)
SG (1) SG10202100131WA (OSRAM)
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Families Citing this family (60)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3409296A1 (en) * 2005-04-07 2018-12-05 The Trustees of the University of Pennsylvania Method of increasing the function of an aav vector
US9249405B2 (en) 2008-09-15 2016-02-02 Paolo Simioni Factor IX polypeptide mutant, its uses and a method for its production
CN104520428B (zh) * 2012-02-17 2018-09-21 费城儿童医院 将基因转移到细胞、器官和组织的aav载体组合物和方法
PH12016500162B1 (en) 2013-07-22 2024-02-21 Childrens Hospital Philadelphia Variant aav and compositions, methods and uses for gene trnsfer to cells, organs, and tissues
KR20170096998A (ko) 2014-11-05 2017-08-25 보이저 테라퓨틱스, 인크. 파킨슨병의 치료를 위한 aadc 폴리뉴클레오티드
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
CA2990193A1 (en) * 2015-06-23 2016-12-29 The Children's Hospital Of 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
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
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
MX2019013836A (es) 2017-05-22 2020-01-30 Baxalta Inc Vectores virales que codifican variantes del factor de coagulacion ix (fix) recombinantes con mayor expresion para la terapia genica de hemofilia b.
KR20240010098A (ko) * 2017-05-31 2024-01-23 더 유니버시티 오브 노쓰 캐롤라이나 엣 채플 힐 최적화된 인간 응고 인자 ix 유전자 발현 카세트 및 이의 사용
JOP20190269A1 (ar) 2017-06-15 2019-11-20 Voyager Therapeutics Inc بولي نوكليوتيدات aadc لعلاج مرض باركنسون
IL271957B2 (en) 2017-07-10 2024-02-01 Uniqure Ip Bv Means and methods for AAV-based gene therapy
MX2020006435A (es) 2017-12-19 2021-02-09 Akouos Inc Administracion de anticuerpos terapeuticos mediada por vector de virus adenoasociado (aav) en el oido interno.
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
CN112955174A (zh) 2018-07-09 2021-06-11 旗舰先锋创新V股份有限公司 融合剂脂质体组合物和其用途
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
US10842885B2 (en) 2018-08-20 2020-11-24 Ucl Business Ltd Factor IX encoding nucleotides
CN112739818A (zh) * 2018-08-20 2021-04-30 Ucl商业有限责任公司 编码因子ix的核苷酸
GB201813528D0 (en) 2018-08-20 2018-10-03 Ucl Business Plc Factor IX encoding nucleotides
MX2021002198A (es) * 2018-08-24 2021-07-15 Spark Therapeutics Inc Secuencias de promotores optimizadas, construcciones de expresion libres de intrones y metodos 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
EP3867380A2 (en) 2018-10-18 2021-08-25 Intellia Therapeutics, Inc. Compositions and methods for expressing factor ix
JP7590963B2 (ja) * 2018-11-09 2024-11-27 ジェネレーション バイオ カンパニー 対称的な修飾型逆位末端反復配列を含む修飾型閉端ｄｎａ（ｃｅｄｎａ）
WO2020142752A1 (en) * 2019-01-04 2020-07-09 Sangamo Therapeutics, Inc. Methods and compositions for the treatment of fabry disease
US11596671B2 (en) 2019-02-01 2023-03-07 Takeda Pharmaceutical Company Limited Gene therapy of hemophilia B using viral vectors encoding recombinant fix variants with increased expression
CA3106693A1 (en) * 2019-02-06 2020-08-13 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
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
CA3200401A1 (en) 2020-11-03 2022-05-12 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
IL303959A (en) 2020-12-23 2023-08-01 Pfizer 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
JP2024515902A (ja) 2021-04-26 2024-04-10 アレクシオン ファーマ インターナショナル オペレーションズ リミテッド 組織向性が改善されたアデノ随伴ウイルスベクターカプシド
US20240218397A1 (en) * 2021-05-04 2024-07-04 Regenxbio Inc. Novel aav vectors and methods and uses thereof
MX2023015329A (es) 2021-06-22 2024-01-23 Pfizer Produccion de vector de virus adeno-asociado en celulas de insecto.
US20240287490A1 (en) * 2021-06-23 2024-08-29 Inspirar Limited Composition and method for treating hemophilia b
WO2023069987A1 (en) 2021-10-20 2023-04-27 University Of Rochester Rejuvenation treatment of age-related white matter loss cross reference to related application
WO2023077012A1 (en) * 2021-10-27 2023-05-04 Regeneron Pharmaceuticals, Inc. Compositions and methods for expressing factor ix for hemophilia b therapy
WO2023081633A1 (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
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
WO2024163747A2 (en) 2023-02-02 2024-08-08 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
WO2025250454A1 (en) 2024-05-28 2025-12-04 University Of Rochester Adeno-associated viruses evolved to specifically target human glial progenitor cells in vivo

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
JP2001500015A (ja) 1996-09-06 2001-01-09 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 Ｔ７ポリメラーゼを利用する組換えアデノ随伴ウイルスの誘導可能な製造方法
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
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
SG168422A1 (en) 2001-11-13 2011-02-28 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
CA2479759A1 (en) 2002-03-22 2003-10-09 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
DK2438931T3 (da) 2004-09-22 2013-12-02 St Jude Childrens Res Hospital Forbedret ekspression af faktor ix i genterapivektorer
WO2006039253A2 (en) 2004-09-29 2006-04-13 Children's Memorial Hospital Sirna-mediated gene silencing of alpha synuclein
EP3409296A1 (en) 2005-04-07 2018-12-05 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
EP2037892B1 (en) * 2006-06-19 2015-03-18 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
US9249405B2 (en) 2008-09-15 2016-02-02 Paolo Simioni 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
RU2012110221A (ru) * 2009-08-18 2013-09-27 Бакстер Интернэшнл Инк. Аптамеры к ингибитору пути тканевого фактора и их использование в качестве терапевтических агентов при нарушениях свертываемости
AU2011242527B2 (en) 2010-04-23 2016-05-19 University Of Florida Research Foundation, Inc. rAAV-guanylate cyclase compositions and methods for treating Leber's congenital amaurosis-1 (LCA1)
EP3540055A1 (en) 2010-04-23 2019-09-18 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
US10640785B2 (en) 2011-11-22 2020-05-05 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
WO2013123457A1 (en) 2012-02-15 2013-08-22 Biogen Idec Ma Inc. Recombinant factor viii proteins
CN104520428B (zh) 2012-02-17 2018-09-21 费城儿童医院 将基因转移到细胞、器官和组织的aav载体组合物和方法
CA2870736C (en) 2012-04-18 2021-11-02 The Children's Hospital Of Philadelphia Composition and methods for highly efficient gene transfer using aav capsid variants
EP2866826B1 (en) 2012-06-27 2025-08-06 Meiragtx UK II Limited 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
WO2015006743A1 (en) 2013-07-12 2015-01-15 The Children's Hospital Of Philadelphia Aav vector and assay for anti-aav (adeno-associated virus) neutralizing antibodies
PH12016500162B1 (en) 2013-07-22 2024-02-21 Childrens Hospital Philadelphia Variant aav and compositions, methods and uses for gene trnsfer to cells, organs, and tissues
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
ES2764453T3 (es) 2015-03-17 2020-06-03 Univ Brussel Vrije Sistemas de expresión específica de hígado optimizados para FVIII y FIX
CA2990193A1 (en) * 2015-06-23 2016-12-29 The Children's Hospital Of 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

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