KR20170086537A - 중추 신경계에 표적화된 aav 벡터 - Google Patents

중추 신경계에 표적화된 aav 벡터 Download PDF

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KR20170086537A
KR20170086537A KR1020177014680A KR20177014680A KR20170086537A KR 20170086537 A KR20170086537 A KR 20170086537A KR 1020177014680 A KR1020177014680 A KR 1020177014680A KR 20177014680 A KR20177014680 A KR 20177014680A KR 20170086537 A KR20170086537 A KR 20170086537A
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aav
nucleic acid
particle
capsid
sequence
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Ceased
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English (en)
Korean (ko)
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스티븐 그레이
토마스 맥코운
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더 유니버시티 오브 노쓰 캐롤라이나 엣 채플 힐
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Priority to KR1020247013508A priority Critical patent/KR20240063169A/ko
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KR1020177014680A 2014-11-21 2015-11-20 중추 신경계에 표적화된 aav 벡터 Ceased KR20170086537A (ko)

Priority Applications (1)

Application Number Priority Date Filing Date Title
KR1020247013508A KR20240063169A (ko) 2014-11-21 2015-11-20 중추 신경계에 표적화된 aav 벡터

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201462082897P 2014-11-21 2014-11-21
US62/082,897 2014-11-21
US201562218857P 2015-09-15 2015-09-15
US62/218,857 2015-09-15
PCT/US2015/061788 WO2016081811A1 (en) 2014-11-21 2015-11-20 Aav vectors targeted to the central nervous system

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KR1020247013508A Division KR20240063169A (ko) 2014-11-21 2015-11-20 중추 신경계에 표적화된 aav 벡터

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KR1020247013508A Pending KR20240063169A (ko) 2014-11-21 2015-11-20 중추 신경계에 표적화된 aav 벡터

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US (4) US10532110B2 (cg-RX-API-DMAC7.html)
EP (2) EP3221453A4 (cg-RX-API-DMAC7.html)
JP (4) JP2017536116A (cg-RX-API-DMAC7.html)
KR (2) KR20170086537A (cg-RX-API-DMAC7.html)
CN (2) CN107532173A (cg-RX-API-DMAC7.html)
AU (2) AU2015349759B2 (cg-RX-API-DMAC7.html)
BR (1) BR112017010447A2 (cg-RX-API-DMAC7.html)
CA (1) CA2967393A1 (cg-RX-API-DMAC7.html)
IL (1) IL252072B (cg-RX-API-DMAC7.html)
MX (1) MX2017006652A (cg-RX-API-DMAC7.html)
RU (1) RU2727015C2 (cg-RX-API-DMAC7.html)
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Families Citing this family (67)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US10577627B2 (en) 2014-06-09 2020-03-03 Voyager Therapeutics, Inc. Chimeric capsids
RU2020109343A (ru) 2014-11-05 2020-03-17 Вояджер Терапьютикс, Инк. Полинуклеотиды aadc для лечения болезни паркинсона
WO2016077687A1 (en) 2014-11-14 2016-05-19 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (als)
CN112375760A (zh) 2014-11-14 2021-02-19 沃雅戈治疗公司 调节性多核苷酸
KR20170086537A (ko) 2014-11-21 2017-07-26 더 유니버시티 오브 노쓰 캐롤라이나 엣 채플 힐 중추 신경계에 표적화된 aav 벡터
WO2016094783A1 (en) 2014-12-12 2016-06-16 Voyager Therapeutics, Inc. Compositions and methods for the production of scaav
WO2016100575A1 (en) 2014-12-16 2016-06-23 Board Of Regents Of The University Of Nebraska Gene therapy for juvenile batten disease
GB201508026D0 (en) 2015-05-11 2015-06-24 Ucl Business Plc Capsid
CN108289932A (zh) 2015-05-22 2018-07-17 D·D·格恩金 胞外dna作为神经变性的治疗靶点
CN114606267A (zh) 2015-09-28 2022-06-10 北卡罗来纳-查佩尔山大学 逃避抗体的病毒载体的方法和组合物
WO2017096162A1 (en) 2015-12-02 2017-06-08 Voyager Therapeutics, Inc. Assays for the detection of aav neutralizing antibodies
WO2017189959A1 (en) 2016-04-29 2017-11-02 Voyager Therapeutics, Inc. Compositions for the treatment of disease
US11299751B2 (en) 2016-04-29 2022-04-12 Voyager Therapeutics, Inc. Compositions for the treatment of disease
SG11201809643UA (en) 2016-05-18 2018-12-28 Voyager Therapeutics Inc Compositions and methods of treating huntington's disease
IL302748B1 (en) 2016-05-18 2025-09-01 Voyager Therapeutics Inc Modulatory polynucleotides
AU2017284198B2 (en) 2016-06-13 2023-09-28 The University Of North Carolina At Chapel Hill Optimized CLN1 genes and expression cassettes and their use
MA44546B1 (fr) 2016-06-15 2021-03-31 Univ California Virus adéno-associés variants et procédés d'utilisation
JP7226801B2 (ja) * 2016-08-16 2023-02-21 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 標的遺伝子導入のための方法および組成物
JP2019531787A (ja) 2016-08-30 2019-11-07 ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア 生物医学的ターゲティング及びデリバリーの方法並びにそれを実行するための装置及びシステム
EP3618839A4 (en) 2017-05-05 2021-06-09 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (als)
US11752181B2 (en) 2017-05-05 2023-09-12 Voyager Therapeutics, Inc. Compositions and methods of treating Huntington's disease
JOP20190269A1 (ar) 2017-06-15 2019-11-20 Voyager Therapeutics Inc بولي نوكليوتيدات aadc لعلاج مرض باركنسون
EP3654860A1 (en) 2017-07-17 2020-05-27 Voyager Therapeutics, Inc. Trajectory array guide system
CN111448308A (zh) 2017-08-03 2020-07-24 沃雅戈治疗公司 递送aav的组合物和方法
WO2019067840A1 (en) 2017-09-29 2019-04-04 Voyager Therapeutics, Inc. RESTRICTING CENTRAL AND PERIPHERAL NEUROLOGICAL PHENOTYPE FROM FRIEDREICH ATAXIA BY INTRAVENOUS ADMINISTRATION
CN111479924B (zh) 2017-10-16 2024-06-14 沃雅戈治疗公司 肌萎缩性侧索硬化症(als)的治疗
WO2019079242A1 (en) 2017-10-16 2019-04-25 Voyager Therapeutics, Inc. TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS)
EP3707265A4 (en) * 2017-11-07 2021-09-01 The University of North Carolina at Chapel Hill OPTIMIZED AGA GENES AND EXPRESSION CASSETTES AND THEIR USE
CN111867595A (zh) * 2017-12-20 2020-10-30 奥维德医疗公司 hM4Di在治疗癫痫发作紊乱中的用途
KR20200122320A (ko) 2018-01-16 2020-10-27 씨엘에스 테라퓨틱스 리미티드 데옥시리보뉴클레아제 (dnase) 활성을 갖는 효소의 간 발현에 의한 질환의 치료
BR112020020266A2 (pt) 2018-04-03 2021-01-19 Stridebio, Inc. Vetores de vírus com evasão de anticorpos
EP3774852A1 (en) 2018-04-03 2021-02-17 Stridebio, Inc. Antibody-evading virus vectors
EA202092362A1 (ru) 2018-04-03 2021-01-11 Страйдбайо, Инк. Вирусные векторы для нацеливания на ткани глаза
CA3099306A1 (en) 2018-05-15 2019-11-21 Voyager Therapeutics, Inc. Compositions and methods for the treatment of parkinson's disease
CN108676097B (zh) * 2018-05-24 2020-01-14 北京肽和生物科技有限公司 一种靶向肿瘤细胞的嵌合肽或嵌合蛋白及其应用
CN112566923B (zh) * 2018-06-12 2024-10-29 北卡罗来纳大学教堂山分校 合成嗜肝性腺相关病毒衣壳及其用途
FI3814513T3 (fi) 2018-06-28 2025-05-28 Univ North Carolina Chapel Hill Optimoidut cln5-geenit ja -ilmentymiskasetit sekä niiden käyttö
IL280300B2 (en) 2018-08-10 2025-06-01 Univ North Carolina Chapel Hill Optimized cln7 genes and expression cassettes and their use
WO2020068990A1 (en) 2018-09-26 2020-04-02 California Institute Of Technology Adeno-associated virus compositions for targeted gene therapy
EP3856762A1 (en) 2018-09-28 2021-08-04 Voyager Therapeutics, Inc. Frataxin expression constructs having engineered promoters and methods of use thereof
AU2019354995B2 (en) * 2018-10-02 2024-12-12 Voyager Therapeutics, Inc. Redirection of tropism of AAV capsids
WO2020097002A1 (en) 2018-11-05 2020-05-14 The University Of North Carolina At Chapel Hill Optimized fig4 genes and expression cassettes and their use
CA3121177A1 (en) * 2018-12-05 2020-06-11 Abeona Therapeutics Inc. Recombinant adeno-associated viral vector for gene delivery
WO2020132385A1 (en) 2018-12-21 2020-06-25 The University Of North Carolina At Chapel Hill Optimized galc genes and expression cassettes and their use
AU2020266523A1 (en) * 2019-04-29 2021-11-04 The Trustees Of The University Of Pennsylvania Novel AAV capsids and compositions containing same
MX2021014248A (es) 2019-05-22 2022-01-06 Univ North Carolina Chapel Hill Genes ube3a y casetes de expresion y su uso.
US20220241394A1 (en) * 2019-07-12 2022-08-04 Cls Therapeutics Limited TREATMENT OF DISEASES ASSOCIATED WITH PROTEIN MISFOLDING BY NERVOUS SYSTEM EXPRESSION OF AN ENZYME WHICH HAS A DEOXYRIBONUCLEASE (DNase) ACTIVITY
CA3151087A1 (en) * 2019-08-29 2021-03-04 The Board Of Trustees Of The Leland Stanford Junior University Adeno-associated viral vectors for crossing the human blood brain barrier
WO2021046169A1 (en) * 2019-09-03 2021-03-11 University Of Cincinnati Methods and compositions for the treatment of als
CN114787180A (zh) 2019-10-17 2022-07-22 斯特里迪比奥公司 用于治疗c型尼曼-匹克病的腺相关病毒载体
WO2021167919A1 (en) * 2020-02-18 2021-08-26 The University Of North Carolina At Chapel Hill Aav capsid-promoter interactions and cell selective gene expression
WO2021222831A2 (en) * 2020-05-01 2021-11-04 The Broad Institute, Inc. Engineered central nervous system compositions
EP4146672A4 (en) * 2020-05-04 2024-10-02 The University of North Carolina at Chapel Hill Modified adeno-associated virus vectors that evade neutralizing antibodies and uses thereof
EP4146673A4 (en) * 2020-05-05 2024-06-19 The University of North Carolina at Chapel Hill MODIFIED ADENO-ASSOCIATED VIRUS 5 CAPSIDS AND THEIR USES
CN111574595B (zh) * 2020-05-22 2022-10-04 中国医学科学院血液病医院(中国医学科学院血液学研究所) 腺相关病毒突变体及其应用
WO2022011390A1 (en) 2020-07-08 2022-01-13 Baylor College Of Medicine Gene therapy for stxbp1 encephalopathy
EP4182467A4 (en) * 2020-07-14 2024-08-21 Abeona Therapeutics Inc. RECOMBINANT ADENO-ASSOCIATED VIRAL VECTORS FOR MULTILATERAL GENE DELIVERY
US20230159949A1 (en) * 2020-07-22 2023-05-25 The Broad Institute, Inc. Engineered muscle targeting compositions
WO2022020697A1 (en) 2020-07-23 2022-01-27 The University Of North Carolina At Chapel Hill Optimized slc13a5 genes and expression cassettes and their use
PH12023550145A1 (en) * 2020-08-12 2024-03-18 UCB Biopharma SRL Gene therapy using nucleic acid constructs comprising methyl cpg binding protein 2 (mecp2) promoter sequences
US12104163B2 (en) 2020-08-19 2024-10-01 Sarepta Therapeutics, Inc. Adeno-associated virus vectors for treatment of Rett syndrome
CN113563430B (zh) * 2021-07-30 2022-05-31 上海信致医药科技有限公司 用于治疗眼部疾病的基因递送系统及其应用
IL311861A (en) 2021-11-02 2024-06-01 Voyager Therapeutics Inc Aav capsid variants and uses thereof
WO2023183304A2 (en) * 2022-03-21 2023-09-28 Capsigen Inc. Transcription-dependent directed evolution of aav capsids having enhanced tropism
WO2025015388A1 (en) * 2023-07-20 2025-01-23 Children's Medical Research Institute Modified cardiotropic aav capsid polypeptides and vectors
WO2025084951A1 (en) * 2023-10-16 2025-04-24 Joint Stock Company "Axelgen" Recombinant genetic construct, adeno-associated virus for the treatment of metachromatic leukodystrophy
WO2025147741A1 (en) * 2024-01-11 2025-07-17 Children's Medical Research Institute Aav capsids and vectors for transduction of cells

Family Cites Families (59)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4474893A (en) 1981-07-01 1984-10-02 The University of Texas System Cancer Center Recombinant monoclonal antibodies
US5023243A (en) 1981-10-23 1991-06-11 Molecular Biosystems, Inc. Oligonucleotide therapeutic agent and method of making same
US4501729A (en) 1982-12-13 1985-02-26 Research Corporation Aerosolized amiloride treatment of retained pulmonary secretions
US4816567A (en) 1983-04-08 1989-03-28 Genentech, Inc. Recombinant immunoglobin preparations
US4676980A (en) 1985-09-23 1987-06-30 The United States Of America As Represented By The Secretary Of The Department Of Health And Human Services Target specific cross-linked heteroantibodies
AU7966987A (en) 1986-09-08 1988-04-07 Applied Biotechnology, Inc. Empty viral capsid vaccines
US4987071A (en) 1986-12-03 1991-01-22 University Patents, Inc. RNA ribozyme polymerases, dephosphorylases, restriction endoribonucleases and methods
US4968603A (en) 1986-12-31 1990-11-06 The Regents Of The University Of California Determination of status in neoplastic disease
EP0442926A1 (en) 1988-11-10 1991-08-28 Imperial Cancer Research Technology Limited Polypeptides
US5916563A (en) 1988-11-14 1999-06-29 United States Of America Parvovirus protein presenting capsids
US5399346A (en) 1989-06-14 1995-03-21 The United States Of America As Represented By The Department Of Health And Human Services Gene therapy
US5149797A (en) 1990-02-15 1992-09-22 The Worcester Foundation For Experimental Biology Method of site-specific alteration of rna and production of encoded polypeptides
ES2026826A6 (es) 1991-03-26 1992-05-01 Ercros Sa Procedimiento para la produccion de una vacuna subunidad contra el parvovirus canino y otros virus relacionados.
US5478745A (en) 1992-12-04 1995-12-26 University Of Pittsburgh Recombinant viral vector system
US5869248A (en) 1994-03-07 1999-02-09 Yale University Targeted cleavage of RNA using ribonuclease P targeting and cleavage sequences
US5658785A (en) 1994-06-06 1997-08-19 Children's Hospital, Inc. Adeno-associated virus materials and methods
US6204059B1 (en) 1994-06-30 2001-03-20 University Of Pittsburgh AAV capsid vehicles for molecular transfer
US5599706A (en) 1994-09-23 1997-02-04 Stinchcomb; Dan T. Ribozymes targeted to apo(a) mRNA
US6093570A (en) 1995-06-07 2000-07-25 The University Of North Carolina At Chapel Hill Helper virus-free AAV production
US6040183A (en) 1995-06-07 2000-03-21 University Of North Carloina At Chapel Hill Helper virus-free AAV production
US6083702A (en) 1995-12-15 2000-07-04 Intronn Holdings Llc Methods and compositions for use in spliceosome mediated RNA trans-splicing
WO1997022250A1 (en) 1995-12-15 1997-06-26 Intronn Llc Therapeutic molecules generated by trans-splicing
WO1998011244A2 (en) 1996-09-11 1998-03-19 The Government Of The United States Of America, Represented By The Secretary, Department Of Health And Human Services Aav4 vector and uses thereof
US6156303A (en) 1997-06-11 2000-12-05 University Of Washington Adeno-associated virus (AAV) isolates and AAV vectors derived therefrom
ES2313784T3 (es) 1998-05-28 2009-03-01 The Government Of The Usa, As Represented By The Secretary, Department Of Health And Human Services Vector aav5 y usos del mismo.
EP1115880A2 (en) 1998-09-22 2001-07-18 University of Florida Methods for large-scale production of recombinant aav vectors
EP1127150B1 (en) 1998-11-05 2007-05-16 The Trustees Of The University Of Pennsylvania Adeno-associated virus serotype 1 nucleic acid sequences, vectors and host cells containing same
WO2000028004A1 (en) 1998-11-10 2000-05-18 The University Of North Carolina At Chapel Hill Virus vectors and methods of making and administering the same
JP2000325085A (ja) 1999-05-21 2000-11-28 Masafumi Matsuo デュシェンヌ型筋ジストロフィー治療剤
AU1775901A (en) 1999-11-17 2001-05-30 Avigen, Inc. Recombinant adeno-associated virus virions for the treatment of lysosomal disorders
AU2001253255A1 (en) 2000-04-07 2001-10-23 Large Scale Biology Corporation Compositions and methods for inhibiting gene expression
US6653467B1 (en) 2000-04-26 2003-11-25 Jcr Pharmaceutical Co., Ltd. Medicament for treatment of Duchenne muscular dystrophy
CA2410828C (en) 2000-06-01 2012-01-24 University Of North Carolina At Chapel Hill Duplexed parvovirus vectors
US7201898B2 (en) 2000-06-01 2007-04-10 The University Of North Carolina At Chapel Hill Methods and compounds for controlled release of recombinant parvovirus vectors
US6727355B2 (en) 2000-08-25 2004-04-27 Jcr Pharmaceuticals Co., Ltd. Pharmaceutical composition for treatment of Duchenne muscular dystrophy
US20030083299A1 (en) 2000-11-04 2003-05-01 Ferguson Ian A. Non-invasive delivery of polypeptides through the blood-brain barrier
US6623729B2 (en) 2001-07-09 2003-09-23 Korea Advanced Institute Of Science And Technology Process for preparing sustained release micelle employing conjugate of anticancer drug and biodegradable polymer
ES2526341T3 (es) * 2001-12-17 2015-01-09 The Trustees Of The University Of Pennsylvania Secuencias de serotipo 9 de virus adeno-asociado (AAV), vectores que las contienen, y usos de las mismas
ITRM20020253A1 (it) 2002-05-08 2003-11-10 Univ Roma Molecole chimeriche di snrna con sequenze antisenso per le giunzioni di splicing del gene della distrofina e applicazioni terapeutiche.
US20070026076A1 (en) 2003-02-24 2007-02-01 Tzyy-Choou Wu Molecular vaccines employing nucleic acid encoding anti-apoptotic proteins
EP3513814A1 (en) 2003-05-01 2019-07-24 Genzyme Corporation Gene therapy for neurometabolic disorders
US9233131B2 (en) 2003-06-30 2016-01-12 The Regents Of The University Of California Mutant adeno-associated virus virions and methods of use thereof
CN102199626B (zh) 2003-09-30 2015-06-24 宾夕法尼亚大学托管会 腺伴随病毒(aav)进化支、序列、含有这些序列的载体及它们的应用
CA2551100A1 (en) 2003-12-23 2005-07-14 The Trustees Of The University Of Pennsylvania Compositions and methods for combined therapy of disease
FR2874384B1 (fr) 2004-08-17 2010-07-30 Genethon Vecteur viral adeno-associe pour realiser du saut d'exons dans un gene codant une proteine a domaines dispensables
US20070059288A1 (en) 2005-03-31 2007-03-15 Dinsmore Jonathan H Treatment for heart disease
EP2383346B1 (en) 2005-04-07 2014-10-08 The Trustees of the University of Pennsylvania Modified AAVrh.64 capsids, compositions containing same and uses thereof
US7867484B2 (en) * 2006-01-27 2011-01-11 University Of North Carolina At Chapel Hill Heparin and heparan sulfate binding chimeric vectors
ES2785223T3 (es) * 2006-06-21 2020-10-06 Uniqure Ip Bv Células de insecto para la producción de vectores de AAV
EP2019143A1 (en) 2007-07-23 2009-01-28 Genethon CNS gene delivery using peripheral administration of AAV vectors
EP2058401A1 (en) 2007-10-05 2009-05-13 Genethon Widespread gene delivery to motor neurons using peripheral injection of AAV vectors
US8632764B2 (en) * 2008-04-30 2014-01-21 University Of North Carolina At Chapel Hill Directed evolution and in vivo panning of virus vectors
US9415121B2 (en) * 2008-12-19 2016-08-16 Nationwide Children's Hospital Delivery of MECP2 polynucleotide using recombinant AAV9
CA2833908C (en) 2010-04-23 2021-02-09 University Of Massachusetts Cns targeting aav vectors and methods of use thereof
US8663624B2 (en) 2010-10-06 2014-03-04 The Regents Of The University Of California Adeno-associated virus virions with variant capsid and methods of use thereof
CN103189507A (zh) * 2010-10-27 2013-07-03 学校法人自治医科大学 用于向神经系统细胞导入基因的腺相关病毒粒子
KR101830651B1 (ko) 2011-01-04 2018-02-21 엘지전자 주식회사 정보 표시 장치 및 그 방법
DK2900686T3 (da) * 2012-09-28 2020-08-17 Univ North Carolina Chapel Hill Aav-vektorer targeteret til oligodendrocytter
KR20170086537A (ko) * 2014-11-21 2017-07-26 더 유니버시티 오브 노쓰 캐롤라이나 엣 채플 힐 중추 신경계에 표적화된 aav 벡터

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