TWI827560B - 用於製備病毒載體之手段及方法與其用途 - Google Patents

用於製備病毒載體之手段及方法與其用途 Download PDF

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TWI827560B
TWI827560B TW107138852A TW107138852A TWI827560B TW I827560 B TWI827560 B TW I827560B TW 107138852 A TW107138852 A TW 107138852A TW 107138852 A TW107138852 A TW 107138852A TW I827560 B TWI827560 B TW I827560B
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viral vector
aav
cells
patient
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TW201930590A (zh
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布萊恩 卡斯巴
詹姆士 哈福列德
喬瑟夫 巴列迪爾
艾倫 卡斯巴
羅伯特 哈吉
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瑞士商諾華股份有限公司
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TW107138852A 2017-11-08 2018-11-01 用於製備病毒載體之手段及方法與其用途 TWI827560B (zh)

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US201762583035P 2017-11-08 2017-11-08
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EP (2) EP3707264A1 (https=)
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KR (1) KR20200086292A (https=)
CN (4) CN120505370A (https=)
AU (2) AU2018365677C1 (https=)
BR (1) BR112019013268A2 (https=)
CA (1) CA3082136A1 (https=)
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Families Citing this family (49)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US11219696B2 (en) * 2008-12-19 2022-01-11 Nationwide Children's Hospital Delivery of polynucleotides using recombinant AAV9
US20200164008A1 (en) * 2017-07-17 2020-05-28 Spark Therapeutics, Inc. Apheresis methods and uses
WO2019094253A1 (en) 2017-11-08 2019-05-16 Avexis Inc. Means and method for preparing viral vectors and uses of same
EP3801638A1 (en) * 2018-06-08 2021-04-14 Novartis AG Cell-based assay for measuring drug product potency
US20210308281A1 (en) * 2018-08-15 2021-10-07 Biogen Ma Inc. Combination therapy for spinal muscular atrophy
CN120860258A (zh) * 2018-11-30 2025-10-31 北京诺华制药有限公司 Aav病毒载体及其用途
CA3136004A1 (en) * 2019-04-10 2020-10-15 Prevail Therapeutics, Inc. Gene therapies for lysosomal disorders
US10801042B1 (en) * 2019-07-15 2020-10-13 Vigene Biosciences, Inc. Use of ion concentrations to increase the packaging efficiency of recombinant adeno-associated virus
JP7759311B2 (ja) 2019-07-19 2025-10-23 フラッグシップ パイオニアリング イノベーションズ シックス，エルエルシー リコンビナーゼ組成物及び使用方法
EP4004213A1 (en) 2019-07-25 2022-06-01 Novartis AG Regulatable expression systems
BR112022002279A2 (pt) * 2019-08-08 2022-07-19 Biogen Ma Inc Ensaios de potência para produção de vetores virais
US20230043046A1 (en) * 2019-10-30 2023-02-09 Amicus Therapeutics, Inc. Recombinant CDKL5 Proteins, Gene Therapy and Production Methods
IL295154A (en) * 2020-01-28 2022-09-01 Lonza Walkersville Inc Methods for determining viral titer
US20230220402A1 (en) 2020-04-17 2023-07-13 Friedrich Miescher Institute For Biomedical Research Use of an orphan motif to increase expression of a heterologous transgene
RU2742837C1 (ru) * 2020-06-02 2021-02-11 Общество С Ограниченной Ответственностью "Анабион" Кодон-оптимизированная нуклеиновая кислота, которая кодирует белок SMN1, и ее применение
JP2023532544A (ja) * 2020-07-01 2023-07-28 メディミューン，エルエルシー 生物学的製剤を精製するための洗浄剤及び方法
CN112121179A (zh) * 2020-09-15 2020-12-25 山东兴瑞生物科技有限公司 一种组合物及其在治疗脊髓性肌萎缩症中的应用
MX2023004420A (es) * 2020-10-15 2023-07-05 Prevail Therapeutics Inc Composiciones de virus adenoasociados recombinantes y métodos para su producción.
CN112251440A (zh) * 2020-10-29 2021-01-22 大连理工大学 一种CRISPR/Cas9基因载体、其制备方法及应用
CN116529381A (zh) 2020-12-03 2023-08-01 弗里德里克·米谢尔生物医学研究所 利用孤儿基序和CpG密度的组合来控制异源转基因的表达
CN117178054A (zh) 2021-04-15 2023-12-05 国立大学法人东京大学 病毒颗粒的制造方法
CA3227103A1 (en) 2021-07-30 2023-02-02 Matthew P. GEMBERLING Compositions and methods for modulating expression of frataxin (fxn)
US20240252684A1 (en) 2021-07-30 2024-08-01 Tune Therapeutics, Inc. Compositions and methods for modulating expression of methyl-cpg binding protein 2 (mecp2)
CN117980471A (zh) * 2021-08-04 2024-05-03 武田药品工业株式会社 阳离子交换剂上的腺相关病毒分离
WO2023042104A1 (en) 2021-09-16 2023-03-23 Novartis Ag Novel transcription factors
WO2023061499A1 (en) * 2021-10-15 2023-04-20 Shanghai Vitalgen Biopharma Co., Ltd. Recombinant adeno-associated viral vectors for treating spinal muscular atrophy
WO2023073526A1 (en) 2021-10-25 2023-05-04 Novartis Ag Methods for improving adeno-associated virus (aav) delivery
WO2023085382A1 (ja) * 2021-11-12 2023-05-19 Ａｇｃ株式会社 ウイルスの精製方法
WO2023095034A1 (en) 2021-11-24 2023-06-01 Novartis Ag Modulators of adeno-associated virus transduction and uses thereof
US20250353883A1 (en) 2022-05-06 2025-11-20 Novartis Ag Novel recombinant aav vp2 fusion polypeptides
CA3261865A1 (en) 2022-07-12 2024-01-18 Tune Therapeutics, Inc. TARGETED TRANSCRIPTIONAL ACTIVATION COMPOSITIONS, SYSTEMS AND METHODS
CN114990162A (zh) * 2022-07-18 2022-09-02 苏州吉纳星辰生物技术有限公司 一种降低aav病毒载体生产中质粒残留的方法
WO2024027632A1 (en) * 2022-08-01 2024-02-08 Skyline Therapeutics (Shanghai) Co., Ltd. Novel plasmid backbone to reduce dna impurities in raav preparation
CN115354071A (zh) * 2022-08-10 2022-11-18 上海药明生基医药科技有限公司 一种检测重组腺相关病毒宿主细胞dna残留的方法
CN116350801A (zh) * 2022-11-22 2023-06-30 四川至善唯新生物科技有限公司 一种重组腺相关病毒载体的药物组合物及其用途
WO2024163683A2 (en) 2023-02-01 2024-08-08 Tune Therapeutics, Inc. Systems, compositions, and methods for modulating expression of methyl-cpg binding protein 2 (mecp2) and x-inactive specific transcript (xist)
WO2024163678A2 (en) 2023-02-01 2024-08-08 Tune Therapeutics, Inc. Fusion proteins and systems for targeted activation of frataxin (fxn) and related methods
AU2024239386A1 (en) 2023-03-21 2025-08-14 F. Hoffmann-La Roche Ag Method for the production of recombinant aav particle preparations
WO2024252182A1 (en) * 2023-06-08 2024-12-12 Meiragtx Uk Ii Limited Cation exchange chromatography for aav capture
CN120677245A (zh) * 2024-01-25 2025-09-19 鼐济医药科技(杭州)有限公司 Aav载体、载体组合物、嵌合载体和治疗或预防sma的方法
WO2025168663A1 (en) 2024-02-09 2025-08-14 F. Hoffmann-La Roche Ag Method for producing recombinant adeno-associated viral particles
WO2025188316A1 (en) * 2024-03-08 2025-09-12 Jaguar Gene Therapy, Llc Aav pharmaceutical formulations and methods thereof
WO2025190867A1 (en) * 2024-03-11 2025-09-18 Sartorius Stedim Biotech Gmbh Two-step intensified cell lysis to release a viral vector
WO2025217224A1 (en) * 2024-04-08 2025-10-16 Advanced Medicine Partners, LLC Method of manufacture of aav particles
KR102915956B1 (ko) * 2024-04-17 2026-01-20 한국로봇융합연구원 3차원 오비탈 용접로봇
WO2025252480A1 (en) 2024-06-07 2025-12-11 F. Hoffmann-La Roche Ag Method for purifying plasmid dna
WO2026022064A1 (en) 2024-07-22 2026-01-29 F. Hoffmann-La Roche Ag Novel aav rep orfs and rep polypeptides
WO2026028089A1 (en) 2024-07-29 2026-02-05 Novartis Ag Compositions and methods for the treatment of lysosomal acid lipase deficiency (lal-d)
WO2026037757A1 (en) 2024-08-13 2026-02-19 F. Hoffmann-La Roche Ag Modified aav particles

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2011094198A1 (en) * 2010-01-28 2011-08-04 The Children's Hospital Of Philadelphia Research Institute, Abramson Research Center A scalable manufacturing platform for viral vector purification and viral vectors so purified for use in gene therapy
CN105087650A (zh) * 2007-10-05 2015-11-25 吉尼松公司 使用外周注射aav载体向运动神经元进行广泛基因投送

Family Cites Families (57)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5173418A (en) 1985-05-10 1992-12-22 Benzon Pharma, A/S Production in Escherichia coli of extracellular Serratia spp. hydrolases
US5139941A (en) 1985-10-31 1992-08-18 University Of Florida Research Foundation, Inc. AAV transduction vectors
US5173414A (en) 1990-10-30 1992-12-22 Applied Immune Sciences, Inc. Production of recombinant adeno-associated virus vectors
PT728214E (pt) 1993-11-09 2004-11-30 Ohio Med College Linhas celulares estaveis capazes de expressar o gene de replicacao do virus adeno-associado
WO1995013365A1 (en) 1993-11-09 1995-05-18 Targeted Genetics Corporation Generation of high titers of recombinant aav vectors
US5658785A (en) 1994-06-06 1997-08-19 Children's Hospital, Inc. Adeno-associated virus materials and methods
US5856152A (en) 1994-10-28 1999-01-05 The Trustees Of The University Of Pennsylvania Hybrid adenovirus-AAV vector and methods of use therefor
CA2207927A1 (en) 1994-12-06 1996-06-13 Targeted Genetics Corporation Packaging cell lines for generation of high titers of recombinant aav vectors
US6001650A (en) 1995-08-03 1999-12-14 Avigen, Inc. High-efficiency wild-type-free AAV helper functions
FR2737730B1 (fr) 1995-08-10 1997-09-05 Pasteur Merieux Serums Vacc Procede de purification de virus par chromatographie
WO1997008298A1 (en) 1995-08-30 1997-03-06 Genzyme Corporation Chromatographic purification of adenovirus and aav
US6632670B1 (en) 1995-09-08 2003-10-14 Genzyme Corporation AAV vectors for gene therapy
US5910434A (en) 1995-12-15 1999-06-08 Systemix, Inc. Method for obtaining retroviral packaging cell lines producing high transducing efficiency retroviral supernatant
JP2001500497A (ja) 1996-09-06 2001-01-16 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 組換えアデノ随伴ウイルスに指図される遺伝子治療の方法
US6756207B1 (en) 1997-02-27 2004-06-29 Cellomics, Inc. System for cell-based screening
DK1009808T3 (da) 1997-09-05 2013-01-21 Genzyme Corp Fremgangsmåder til generering af hjælper-frie præparater af rekombinante aav-vektorer med høj titer
US6258595B1 (en) 1999-03-18 2001-07-10 The Trustees Of The University Of Pennsylvania Compositions and methods for helper-free production of recombinant adeno-associated viruses
JP2003518380A (ja) * 1999-12-29 2003-06-10 ジェンザイム・コーポレイション 核酸および他の細胞性汚染混入物質からウイルスベクターを分離するための濾過助剤の使用方法
US7056502B2 (en) 2000-04-28 2006-06-06 The Trustees Of The University Of Pennsylvania Recombinant aav vectors with AAV5 capsids and AAV5 vectors pseudotyped in heterologous capsids
DK2573170T3 (en) 2001-12-17 2018-04-09 Univ Pennsylvania Sequences of adeno-associated virus (AAV) serotype 9, vectors containing them, and their use
US7261544B2 (en) 2003-05-21 2007-08-28 Genzyme Corporation Methods for producing preparations of recombinant AAV virions substantially free of empty capsids
US9441244B2 (en) 2003-06-30 2016-09-13 The Regents Of The University Of California Mutant adeno-associated virus virions and methods of use thereof
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
US20050153446A1 (en) 2003-11-07 2005-07-14 Steindler Dennis A. Culturing and differentiating neural precursor cells
BRPI0511764B8 (pt) * 2004-06-01 2021-05-25 Avigen Inc método de prevenção de agregação de vírions de vírus adeno-associado recombinante (raav) em uma preparação purificada de virions raav
EP3470072A1 (en) 2005-06-23 2019-04-17 Biogen MA Inc. Compositions and methods for modulation of smn2 splicing
EP2942392B1 (en) 2008-06-30 2018-10-03 Janssen Biotech, Inc. Differentiation of pluripotent stem cells
US9415121B2 (en) 2008-12-19 2016-08-16 Nationwide Children's Hospital Delivery of MECP2 polynucleotide using recombinant AAV9
US11219696B2 (en) 2008-12-19 2022-01-11 Nationwide Children's Hospital Delivery of polynucleotides using recombinant AAV9
WO2010071832A1 (en) 2008-12-19 2010-06-24 Nationwide Children's Hospital Delivery of polynucleotides across the blood brain barrier using recombinant aav9
HRP20250897T1 (hr) 2009-05-02 2025-09-26 Genzyme Corporation Genska terapija za neurodegenerativne poremećaje
ES2699827T3 (es) 2009-06-17 2019-02-13 Biogen Ma Inc Composiciones y métodos para la modulación de corte y empalme de SMN2 en un sujeto
CN102712902B (zh) 2009-12-23 2019-01-08 詹森生物科技公司 人胚胎干细胞的分化
DE102012007232B4 (de) 2012-04-07 2014-03-13 Susanne Weller Verfahren zur Herstellung von rotierenden elektrischen Maschinen
EP2861723A4 (en) 2012-06-14 2016-01-20 Janssen Biotech Inc DIFFERENTIATION OF HUMAN EMBRYONAL STEM CELLS IN ENDOCRINE PANCREAS CELLS
US20150182637A1 (en) * 2012-06-21 2015-07-02 Association Institut De Myologie Widespread gene delivery of gene therapy vectors
AU2013296425B2 (en) * 2012-08-01 2018-06-07 Nationwide Children's Hospital Intrathecal delivery of recombinant adeno-associated virus 9
MX380973B (es) 2013-05-01 2025-03-12 Genzyme Corp Composiciones y metodos para tratar la atrofia muscular espinal.
CN105339495A (zh) 2013-07-11 2016-02-17 宝生物工程株式会社 制备无包膜病毒的方法
EP3024497B1 (en) 2013-07-26 2021-01-13 University of Iowa Research Foundation Methods and compositions for treating brain diseases
SI3702466T1 (sl) 2013-08-27 2023-04-28 Research Institute At Nationwide Children's Hospital Proizvodi in postopki za zdravljenje amiotrofične lateralne skleroze
JP2015092462A (ja) 2013-09-30 2015-05-14 Tdk株式会社 正極及びそれを用いたリチウムイオン二次電池
WO2015141521A1 (ja) 2014-03-21 2015-09-24 株式会社日立国際電気 基板処理装置、半導体装置の製造方法及び記録媒体
EP2933335A1 (en) 2014-04-18 2015-10-21 Genethon A method of treating peripheral neuropathies and motor neuron diseases
WO2016004319A1 (en) * 2014-07-02 2016-01-07 University Of Florida Research Foundation, Inc. Compositions and methods for purifying recombinant adeno-associated virus
JP6197169B2 (ja) 2014-09-29 2017-09-20 東芝メモリ株式会社 半導体装置の製造方法
WO2016100575A1 (en) 2014-12-16 2016-06-23 Board Of Regents Of The University Of Nebraska Gene therapy for juvenile batten disease
EP3054006A1 (en) * 2015-02-09 2016-08-10 Institut National De La Sante Et De La Recherche Medicale (Inserm) Recombinant adeno-associated virus particle purification with multiple-step anion exchange chromatography
EP3054007A1 (en) 2015-02-09 2016-08-10 Institut National De La Sante Et De La Recherche Medicale (Inserm) Recombinant adeno-associated virus particle purification comprising an immuno-affinity purification step
EP3387138B1 (en) * 2015-12-11 2022-01-26 The Trustees Of The University Of Pennsylvania Scalable purification method for aav9
LT3436051T (lt) 2016-03-31 2021-11-25 Spark Therapeutics, Inc. Kolonėlėmis pagrįstas visiškai keičiamo masto raav gavimo būdas
SG10201912761UA (en) * 2016-04-15 2020-02-27 The Trustees Of The Univ Of Pennsyvania Gene therapy for treating mucopolysaccharidosis type ii
EP3299456A1 (en) 2016-09-26 2018-03-28 Université de Strasbourg Recombinant dgkk gene for fragile x syndrome gene therapy
JP7106534B2 (ja) 2016-11-17 2022-07-26 ネイションワイド チルドレンズ ホスピタル， インコーポレイテッド メチル－ｃｐｇ結合タンパク質２をコードする組換えアデノ随伴ウイルスの髄腔内送達
WO2019011817A1 (en) 2017-07-08 2019-01-17 Genethon TREATMENT OF SPINAL AMYOTROPHY
WO2019094253A1 (en) 2017-11-08 2019-05-16 Avexis Inc. Means and method for preparing viral vectors and uses of same
EP3801638A1 (en) 2018-06-08 2021-04-14 Novartis AG Cell-based assay for measuring drug product potency

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN105087650A (zh) * 2007-10-05 2015-11-25 吉尼松公司 使用外周注射aav载体向运动神经元进行广泛基因投送
WO2011094198A1 (en) * 2010-01-28 2011-08-04 The Children's Hospital Of Philadelphia Research Institute, Abramson Research Center A scalable manufacturing platform for viral vector purification and viral vectors so purified for use in gene therapy

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