KR102415896B1 - 변형된 인자 ix, 및 세포, 기관 및 조직으로 유전자를 전달하기 위한 조성물, 방법 및 용도 - Google Patents

변형된 인자 ix, 및 세포, 기관 및 조직으로 유전자를 전달하기 위한 조성물, 방법 및 용도 Download PDF

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KR102415896B1
KR102415896B1 KR1020207032060A KR20207032060A KR102415896B1 KR 102415896 B1 KR102415896 B1 KR 102415896B1 KR 1020207032060 A KR1020207032060 A KR 1020207032060A KR 20207032060 A KR20207032060 A KR 20207032060A KR 102415896 B1 KR102415896 B1 KR 102415896B1
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캐서린 에이. 하이
사비어 안구엘라
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더 칠드런스 호스피탈 오브 필라델피아
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KR1020207032060A 2015-06-23 2016-06-23 변형된 인자 ix, 및 세포, 기관 및 조직으로 유전자를 전달하기 위한 조성물, 방법 및 용도 Active KR102415896B1 (ko)

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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
KR1020187002040A KR102178322B1 (ko) 2015-06-23 2016-06-23 변형된 인자 ix, 및 세포, 기관 및 조직으로 유전자를 전달하기 위한 조성물, 방법 및 용도
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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KR102415896B1 true KR102415896B1 (ko) 2022-06-30

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KR1020187002040A Active KR102178322B1 (ko) 2015-06-23 2016-06-23 변형된 인자 ix, 및 세포, 기관 및 조직으로 유전자를 전달하기 위한 조성물, 방법 및 용도

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US (6) US10799566B2 (https=)
EP (2) EP4378487A3 (https=)
JP (3) JP6831779B2 (https=)
KR (2) KR102415896B1 (https=)
CN (1) CN108138159A (https=)
AU (2) AU2016282781A1 (https=)
CA (1) CA2990193A1 (https=)
CO (1) CO2018000134A2 (https=)
DK (1) DK3313991T3 (https=)
ES (1) ES2989774T3 (https=)
FI (2) FI3313991T3 (https=)
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PT (1) PT3313991T (https=)
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Families Citing this family (62)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
ES2525067T3 (es) 2005-04-07 2014-12-17 The Trustees Of The University Of Pennsylvania Método de incremento de la función de un vector de AAV
TR201813067T4 (tr) 2008-09-15 2018-09-21 Uniqure Biopharma B V Faktör IX polipeptit mutantı, bunun kullanımları ve bunu üretmeye yönelik bir yöntem.
MX367100B (es) * 2012-02-17 2019-08-05 The Children´S Hospital Of Philadelphia Composiciones del vector aav y metodos para su transferencia genetica a celulas, organos y tejidos.
IL297919A (en) 2013-07-22 2023-01-01 Childrens Hospital Philadelphia Variant aav and compositions, methods and uses for gene transfer to cells, organs and tissues
RU2716991C2 (ru) 2014-11-05 2020-03-17 Вояджер Терапьютикс, Инк. Полинуклеотиды aadc для лечения болезни паркинсона
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
MX390848B (es) 2015-06-23 2025-03-21 Childrens Hospital Philadelphia Factor ix modificado, y composiciones, metodos y usos para la transferencia de genes a celulas, organos y tejidos.
KR102712926B1 (ko) 2016-10-20 2024-10-07 상가모 테라퓨틱스, 인코포레이티드 파브리병의 치료를 위한 방법 및 조성물
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
NZ756587A (en) * 2017-03-15 2025-12-19 Univ North Carolina Chapel Hill Polyploid adeno-associated virus vectors and methods of making and using the same
CN110945127B (zh) * 2017-05-22 2024-07-12 武田药品工业株式会社 用于血友病b的基因疗法的编码具有增加的表达的重组fix的病毒载体
JP7119009B2 (ja) 2017-05-31 2022-08-16 ザ ユニバーシティ オブ ノース カロライナ アット チャペル ヒル 最適化されたヒト凝固第ｉｘ因子遺伝子発現カセットおよびそれらの使用
JOP20190269A1 (ar) 2017-06-15 2019-11-20 Voyager Therapeutics Inc بولي نوكليوتيدات aadc لعلاج مرض باركنسون
JP7764129B2 (ja) 2017-07-10 2025-11-05 ユニキュアー アイピー ビー．ブイ． ヒトにおけるａａｖ遺伝子療法のための手段及び方法
EP3727468A4 (en) 2017-12-19 2021-09-22 Akouos, Inc. AAV-MEDIATED THERAPEUTIC ANTIBODIES DELIVERY TO THE INTERNAL EAR
KR20210019996A (ko) 2018-05-15 2021-02-23 보이저 테라퓨틱스, 인크. 파킨슨병의 치료를 위한 조성물 및 방법
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
EP3799569A1 (en) * 2018-07-27 2021-04-07 Zentrum für Forschungsförderung in der Pädiatrie GmbH Improved concepts for the treatment of genetic disorders with high-capacity plal-generated gold nanoparticles
US10842885B2 (en) 2018-08-20 2020-11-24 Ucl Business Ltd 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
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
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
EP3856762A1 (en) 2018-09-28 2021-08-04 Voyager Therapeutics, Inc. Frataxin expression constructs having engineered promoters and methods of use thereof
AU2019360270B2 (en) 2018-10-18 2025-08-07 Intellia Therapeutics, Inc. Compositions and methods for expressing factor IX.
KR20210090619A (ko) * 2018-11-09 2021-07-20 제너레이션 바이오 컴퍼니 대칭인 변형된 역말단반복을 포함하는 변형된 폐쇄형 DNA(ceDNA)
WO2020106916A1 (en) * 2018-11-21 2020-05-28 Stridebio, Inc. Recombinant viral vectors and nucleic acids for producing the same
CN113423419A (zh) * 2019-01-04 2021-09-21 桑格摩生物治疗股份有限公司 用于治疗法布里病的方法和组合物
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
AU2019428629A1 (en) * 2019-02-06 2021-01-28 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
US20230138409A1 (en) * 2020-03-24 2023-05-04 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
AU2021375404A1 (en) 2020-11-03 2023-06-08 Pfizer Inc. Methods for purification of aav vectors by anion exchange chromatography
IL303317A (en) 2020-12-01 2023-07-01 Akouos Inc ANTI-NATURAL ANTIBODY STRUCTURES AND RELATED METHODS FOR THE TREATMENT OF SYMPTOMS ASSOCIATED WITH VESTIBULAR SWANNOMA
US20240052322A1 (en) 2020-12-15 2024-02-15 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
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
US20250188126A1 (en) 2021-04-26 2025-06-12 Alexion Pharma International Operations Limited 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
WO2022269466A1 (en) 2021-06-22 2022-12-29 Pfizer Inc. Production of adeno-associated virus vector in insect cells
JP2024521537A (ja) * 2021-06-23 2024-05-31 フロンテーラ セラピューティクス，インク． 血友病ｂを処置するための組成物および方法
GB202110014D0 (en) * 2021-07-12 2021-08-25 Cytiva Bioprocess R & D Ab A method for separating adeno-associated virus capsids, compositions obtained by said method and uses thereof
CA3234809A1 (en) 2021-10-20 2023-04-27 Steven Goldman Isolated glial progenitor cells for use in the competition treatment of age-related white matter loss
US20230149563A1 (en) * 2021-10-27 2023-05-18 Regeneron Pharmaceuticals, Inc. Compositions and methods for expressing factor ix for hemophilia b therapy
EP4426331A1 (en) 2021-11-02 2024-09-11 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
CN118460618A (zh) * 2022-04-19 2024-08-09 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
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
WO2025250454A1 (en) 2024-05-28 2025-12-04 University Of Rochester Adeno-associated viruses evolved to specifically target human glial progenitor cells in vivo

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2001098482A2 (en) * 2000-06-20 2001-12-27 Board Of Trustees Of The Leland Stanford Junior University Liver-specific gene expression cassettes, and methods of use
WO2013078316A1 (en) 2011-11-23 2013-05-30 Nationwide Children's Hospital, Inc. Recombinant adeno-associated virus delivery of alpha-sarcoglycan polynucleotides
US20150023924A1 (en) 2013-07-22 2015-01-22 The Children's Hospital Of Philadelphia Variant aav and compositions, methods and uses for gene transfer to cells, organs and tissues

Family Cites Families (42)

* 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
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
CA2442144A1 (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
MX359371B (es) 2001-11-13 2018-09-25 Univ Pennsylvania Un metodo para detectar y/o identificar secuencias del virus adeno-asociado y aislamiento de secuencias novedosas identificadas de ese modo.
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 PROTAMINE-ADENOVIRAL VECTOR COMPLEXES AND APPLICATION METHODS
SI1496944T1 (sl) 2002-05-01 2009-02-28 Univ Florida Izboljšani sistemi ekspresije RAAV za genetsko modifikacijo specifičnih kapsidnih proteinov
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
CA2580189C (en) 2004-09-29 2013-05-21 Children's Memorial Hospital Sirna-mediated gene silencing of alpha synuclein
ES2525067T3 (es) 2005-04-07 2014-12-17 The Trustees Of The University Of Pennsylvania Método de incremento de la función de un vector de AAV
US7588772B2 (en) 2006-03-30 2009-09-15 Board Of Trustees Of The Leland Stamford Junior University AAV capsid library and AAV capsid proteins
WO2008027084A2 (en) 2006-04-28 2008-03-06 The Trustees Of The University Of Pennsylvania Modified aav vectors having reduced capsid immunogenicity and use thereof
ES2535877T3 (es) * 2006-06-19 2015-05-18 Asklepios Biopharmaceutical, Inc. Genes de Factor VIII y Factor IX modificados y vectores para la terapia génica
CA2722238C (en) 2008-04-22 2017-11-28 Life Sciences Research Partners Vzw Liver-specific nucleic acid regulatory elements and methods and use thereof
TR201813067T4 (tr) 2008-09-15 2018-09-21 Uniqure Biopharma B V Faktör IX polipeptit mutantı, bunun kullanımları ve bunu üretmeye yönelik bir yöntem.
GB0911870D0 (en) 2009-07-08 2009-08-19 Ucl Business Plc Optimised coding sequence and promoter
JP2013502218A (ja) * 2009-08-18 2013-01-24 バクスター・インターナショナル・インコーポレイテッド 組織因子経路阻害剤に対するアプタマーおよび出血障害治療薬としてのそれらの使用
EP2561073B1 (en) 2010-04-23 2016-08-24 University of Massachusetts Cns targeting aav vectors and methods of use thereof
SG10201800541SA (en) 2010-04-23 2018-03-28 Univ Florida Raav-guanylate cyclase compositions and methods for treating leber's congenital amaurosis-1 (lca1)
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
EP2822577B1 (en) 2012-02-15 2019-02-06 Bioverativ Therapeutics Inc. Recombinant factor viii proteins
MX367100B (es) 2012-02-17 2019-08-05 The Children´S Hospital Of Philadelphia Composiciones del vector aav y metodos para su transferencia genetica a celulas, organos y tejidos.
NZ701693A (en) 2012-04-18 2017-02-24 The Children’S Hospital Of Philadelphia Composition and methods for highly efficient gene transfer using aav capsid variants
US9943574B2 (en) * 2012-06-27 2018-04-17 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
HK1222673A1 (zh) 2013-07-12 2017-07-07 The Children's Hospital Of Philadelphia Aav载体和用於抗aav(腺相关病毒)中和抗体的检测
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
US11007280B2 (en) 2015-03-17 2021-05-18 Vrije Universiteit Brussel Optimized liver-specific expression systems for FVIII and FIX
MX390848B (es) 2015-06-23 2025-03-21 Childrens Hospital Philadelphia Factor ix modificado, y composiciones, metodos y usos para la transferencia de genes a celulas, organos y tejidos.
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

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2001098482A2 (en) * 2000-06-20 2001-12-27 Board Of Trustees Of The Leland Stanford Junior University Liver-specific gene expression cassettes, and methods of use
WO2013078316A1 (en) 2011-11-23 2013-05-30 Nationwide Children's Hospital, Inc. Recombinant adeno-associated virus delivery of alpha-sarcoglycan polynucleotides
US20150023924A1 (en) 2013-07-22 2015-01-22 The Children's Hospital Of Philadelphia Variant aav and compositions, methods and uses for gene transfer to cells, organs and tissues

Non-Patent Citations (3)

* Cited by examiner, † Cited by third party
Title
Grimm D. et al, JOURNAL OF VIROLOGY, 82(12):pp.5887~5911 (2008. 6.)*
Hodges B.L. et al, MOLECULAR THERAPY 10(2):pp.269~278. (2004. 8.)*
Rogers G. et al, Jour. of Translational Medicine 12(1): e25 (2014.)*

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