SA517390590B1 - Ix عامل معدل، وتركيبات وطرق واستخدامات لنقل الجين إلى الخلايا، والأعضاء والأنسجة - Google Patents

Ix عامل معدل، وتركيبات وطرق واستخدامات لنقل الجين إلى الخلايا، والأعضاء والأنسجة

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SA517390590B1
SA517390590B1 SA517390590A SA517390590A SA517390590B1 SA 517390590 B1 SA517390590 B1 SA 517390590B1 SA 517390590 A SA517390590 A SA 517390590A SA 517390590 A SA517390590 A SA 517390590A SA 517390590 B1 SA517390590 B1 SA 517390590B1
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organs
tissues
cells
gene transfer
methods
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SA517390590A
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انجويلا زافير
ايه هاي كاثرين
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ذي شيلدرنـز هوسبيتال اوف فيلادلفيا
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Publication of SA517390590B1 publication Critical patent/SA517390590B1/ar

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    • 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)
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    • C12N9/64—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue
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    • C12N9/6424—Serine endopeptidases (3.4.21)
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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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Abstract

يتعلق الاختراع الحالي بمتوالية تشفير coding sequence عامل IX معدل modified Factor IX ، وقريص إظهار expression cassette ، ونواقل vectors مثل نواقل فيروسية viral على سبيل المثال، فيروسية مرتبطة بالعدسات أو الغدة lenti- or adeno-associated viral ، وطرق واستخدامات نقل الجين gene transfer . تحديدا، لاستهداف الحمض النووي nucleic acid لعامل IX إلى الخلايا أو الأنسجة أو الأعضاء للتعبير الوراثي عن (استنساخ transcription) العامل IX. شكل 19.
SA517390590A 2015-06-23 2017-12-21 Ix عامل معدل، وتركيبات وطرق واستخدامات لنقل الجين إلى الخلايا، والأعضاء والأنسجة SA517390590B1 (ar)

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

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SA517390590B1 true SA517390590B1 (ar) 2022-02-10

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US (6) US10799566B2 (ar)
EP (2) EP3313991B1 (ar)
JP (3) JP6831779B2 (ar)
KR (2) KR102178322B1 (ar)
CN (1) CN108138159A (ar)
AU (2) AU2016282781A1 (ar)
BR (1) BR112017027956A2 (ar)
CA (1) CA2990193A1 (ar)
CO (1) CO2018000134A2 (ar)
DK (1) DK3313991T3 (ar)
HK (1) HK1252351A1 (ar)
IL (2) IL256517B2 (ar)
MX (2) MX2018000307A (ar)
PE (1) PE20180675A1 (ar)
PH (1) PH12017502424A1 (ar)
SA (1) SA517390590B1 (ar)
SG (1) SG10202100131WA (ar)
WO (1) WO2016210170A1 (ar)

Families Citing this family (50)

* 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
FI3581650T3 (fi) 2008-09-15 2023-03-23 Uniqure Biopharma B V IX-tekijän polypeptidimutantti, sen käyttöjä ja menetelmä sen valmistamiseksi
BR112014020325A2 (pt) * 2012-02-17 2017-08-08 Childrens Hospital Philadelphia composições de vetor do aav e métodos para a transferência de gene para as células, órgãos e tecidos
HUE047996T2 (hu) 2013-07-22 2020-05-28 Childrens Hospital Philadelphia AAV variáns és készítmények, eljárások és alkalmazások sejtekbe, szervekbe és szövetekbe történõ géntranszferhez
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
EP3313991B1 (en) * 2015-06-23 2024-07-17 The Children's Hospital of Philadelphia Modified factor ix, and compositions, methods and uses for gene transfer to cells, organs and tissues
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
AU2018234695A1 (en) * 2017-03-15 2019-09-12 The University Of North Carolina At Chapel Hill Polyploid adeno-associated virus vectors and methods of making and using the same
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
KR102386890B1 (ko) * 2017-05-22 2022-04-15 다케다 야쿠힌 고교 가부시키가이샤 B형 혈우병 유전자 요법을 위한 증가된 발현을 가지는 재조합 fix 변이체를 암호화하는 바이러스 벡터
CN114875051A (zh) * 2017-05-31 2022-08-09 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
EA202090260A1 (ru) * 2017-07-10 2020-05-08 ЮНИКЬЮРЕ АйПи Б.В. Средства и способы для генотерапии aav у человека
EP3727468A4 (en) * 2017-12-19 2021-09-22 Akouos, Inc. AAV-MEDIATED DELIVERY OF THERAPEUTIC ANTIBODIES TO THE INNER EAR
KR20210043574A (ko) * 2018-07-09 2021-04-21 플래그쉽 파이어니어링 이노베이션스 브이, 인크. 푸소좀 조성물 및 이의 용도
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
EP3810770A1 (en) * 2018-08-20 2021-04-28 Ucl Business Ltd Factor ix encoding nucleotides
JP2021533805A (ja) * 2018-08-24 2021-12-09 スパーク セラピューティクス インコーポレイテッドSpark Therapeutics, Inc. 最適化されたプロモーター配列、イントロンを含まない発現構築物及び使用方法
CA3119310A1 (en) * 2018-11-09 2020-05-14 Generation Bio Co. Modified closed-ended dna (cedna) comprising symmetrical modified inverted terminal repeats
JP2022516317A (ja) * 2019-01-04 2022-02-25 サンガモ セラピューティクス， インコーポレイテッド ファブリー病の処置のための方法及び組成物
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
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
CN114302968A (zh) * 2019-08-29 2022-04-08 美国西门子医学诊断股份有限公司 用于检测aav脱落的试剂和方法
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
US20230250422A1 (en) * 2020-04-07 2023-08-10 The Curators Of The University Of Missouri Cpg-free itrs for aav gene therapy
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
EP4240861A1 (en) 2020-11-03 2023-09-13 Pfizer Inc. Methods for purification of aav vectors by anion exchange chromatography
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
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
CN117377500A (zh) 2021-04-26 2024-01-09 阿雷克森国际制药有限公司 具有改善的组织向性的腺相关病毒载体衣壳
WO2022235614A2 (en) * 2021-05-04 2022-11-10 Regenxbio Inc. Novel aav vectors and methods and uses thereof
JP2023002483A (ja) 2021-06-22 2023-01-10 ファイザー・インク 昆虫細胞におけるアデノ随伴ウイルスベクターの産生
CN117693593A (zh) * 2021-06-23 2024-03-12 英斯培瑞有限公司 用于治疗b型血友病的组合物和方法
WO2023069987A1 (en) 2021-10-20 2023-04-27 University Of Rochester Rejuvenation treatment of age-related white matter loss cross reference to related application
US20230149563A1 (en) * 2021-10-27 2023-05-18 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
CN117247973B (zh) * 2022-04-19 2024-05-10 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
WO2024081604A1 (en) * 2022-10-10 2024-04-18 Spark Therapeutics, Inc. Apoe gene therapy
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

Family Cites Families (44)

* 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
EP0931158A1 (en) 1996-09-06 1999-07-28 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
NZ532635A (en) 2001-11-13 2007-05-31 Univ Pennsylvania A method of identifying unknown adeno-associated virus (AAV) sequences and a kit for the method
PT1453547T (pt) 2001-12-17 2016-12-28 Univ Pennsylvania Sequências do vírus adeno-associado (aav) do serotipo 8, vetores contendo as mesmas, e utilizações destas
EP1496979A4 (en) 2002-03-22 2007-10-31 Univ Texas ADENOVIRAL-PROTAMINE VECTOR COMPLEXES AND METHODS OF USE
DE60323078D1 (de) 2002-05-01 2008-10-02 Univ Florida Verbesserte raav-expressionssysteme für die genetische modifikation spezifischer capsidproteine
WO2005037226A2 (en) * 2003-10-17 2005-04-28 Georgia Tech Research Corporation Genetically engineered enteroendocrine cells for treating glucose-related metabolic disorders
WO2006036502A2 (en) * 2004-09-22 2006-04-06 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
JP2009535339A (ja) 2006-04-28 2009-10-01 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 低下されたキャプシド免疫原性を有する改変ａａｖベクターおよびその使用
EP2037892B1 (en) * 2006-06-19 2015-03-18 Asklepios Biopharmaceutical, Inc. Modified factor viii and factor ix genes and vectors for gene therapy
DK2282764T3 (da) 2008-04-22 2019-10-14 Vib Vzw Leverspecifikke nukleinsyreregulerende elementer samt fremgangsmåder og anvendelser heraf
FI3581650T3 (fi) 2008-09-15 2023-03-23 Uniqure Biopharma B V IX-tekijän polypeptidimutantti, sen käyttöjä ja menetelmä sen valmistamiseksi
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
EP2826860B1 (en) 2010-04-23 2018-08-22 University of Massachusetts CNS targeting AAV vectors and methods of use thereof
KR20190060881A (ko) 2010-04-23 2019-06-03 유니버시티 오브 플로리다 리서치 파운데이션, 인크. 레베르 선천성 흑내장-1(lca1)의 치료를 위한 재조합 아데노-관련된 바이러스-구아닐레이트 사이클라제 조성물 및 방법
KR101853405B1 (ko) 2010-10-20 2018-05-02 주식회사 티움바이오 인자 ｉｘ 활성을 갖는 융합 단백질
JP6348064B2 (ja) 2011-11-22 2018-06-27 ザ チルドレンズ ホスピタル オブ フィラデルフィア 効率の高いトランスジーン送達のためのウイルスベクター
US9434928B2 (en) 2011-11-23 2016-09-06 Nationwide Children's Hospital, Inc. Recombinant adeno-associated virus delivery of alpha-sarcoglycan polynucleotides
CA2864126A1 (en) 2012-02-15 2013-08-22 Biogen Idec Ma Inc. Recombinant factor viii proteins
BR112014020325A2 (pt) 2012-02-17 2017-08-08 Childrens Hospital Philadelphia composições de vetor do aav e métodos para a transferência de gene para as células, órgãos e tecidos
DK2839014T3 (da) 2012-04-18 2021-03-08 Childrens Hospital Philadelphia Sammensætning og fremgangsmåder til højeffektiv genoverførsel ved anvendelse af aav-capsid-varianter
AU2013281328B2 (en) * 2012-06-27 2017-11-23 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
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
HUE047996T2 (hu) * 2013-07-22 2020-05-28 Childrens Hospital Philadelphia AAV variáns és készítmények, eljárások és alkalmazások sejtekbe, szervekbe és szövetekbe történõ géntranszferhez
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
EP3313991B1 (en) * 2015-06-23 2024-07-17 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
US11191847B2 (en) 2016-04-15 2021-12-07 The Trustees Of The University Of Pennsylvania Gene therapy for treating hemophilia B

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