JP2021502123A5 - - Google Patents

Download PDF

Info

Publication number
JP2021502123A5
JP2021502123A5 JP2020544560A JP2020544560A JP2021502123A5 JP 2021502123 A5 JP2021502123 A5 JP 2021502123A5 JP 2020544560 A JP2020544560 A JP 2020544560A JP 2020544560 A JP2020544560 A JP 2020544560A JP 2021502123 A5 JP2021502123 A5 JP 2021502123A5
Authority
JP
Japan
Prior art keywords
aav
viral vector
less
dose
protein
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Granted
Application number
JP2020544560A
Other languages
English (en)
Japanese (ja)
Other versions
JP2021502123A (ja
JP7587422B2 (ja
Filing date
Publication date
Application filed filed Critical
Priority claimed from PCT/US2018/058744 external-priority patent/WO2019094253A1/en
Publication of JP2021502123A publication Critical patent/JP2021502123A/ja
Publication of JP2021502123A5 publication Critical patent/JP2021502123A5/ja
Priority to JP2024150419A priority Critical patent/JP2025017361A/ja
Application granted granted Critical
Publication of JP7587422B2 publication Critical patent/JP7587422B2/ja
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

Images

JP2020544560A 2017-11-08 2018-11-01 ウイルスベクターの調製手段及び方法並びにその使用 Active JP7587422B2 (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2024150419A JP2025017361A (ja) 2017-11-08 2024-09-02 ウイルスベクターの調製手段及び方法並びにその使用

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201762583035P 2017-11-08 2017-11-08
US62/583,035 2017-11-08
PCT/US2018/058744 WO2019094253A1 (en) 2017-11-08 2018-11-01 Means and method for preparing viral vectors and uses of same

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2024150419A Division JP2025017361A (ja) 2017-11-08 2024-09-02 ウイルスベクターの調製手段及び方法並びにその使用

Publications (3)

Publication Number Publication Date
JP2021502123A JP2021502123A (ja) 2021-01-28
JP2021502123A5 true JP2021502123A5 (https=) 2021-12-09
JP7587422B2 JP7587422B2 (ja) 2024-11-20

Family

ID=64362706

Family Applications (2)

Application Number Title Priority Date Filing Date
JP2020544560A Active JP7587422B2 (ja) 2017-11-08 2018-11-01 ウイルスベクターの調製手段及び方法並びにその使用
JP2024150419A Pending JP2025017361A (ja) 2017-11-08 2024-09-02 ウイルスベクターの調製手段及び方法並びにその使用

Family Applications After (1)

Application Number Title Priority Date Filing Date
JP2024150419A Pending JP2025017361A (ja) 2017-11-08 2024-09-02 ウイルスベクターの調製手段及び方法並びにその使用

Country Status (17)

Country Link
US (2) US12168777B2 (https=)
EP (2) EP3707264A1 (https=)
JP (2) JP7587422B2 (https=)
KR (1) KR20200086292A (https=)
CN (4) CN120505370A (https=)
AU (2) AU2018365677C1 (https=)
BR (1) BR112019013268A2 (https=)
CA (1) CA3082136A1 (https=)
CL (3) CL2020001193A1 (https=)
CO (1) CO2020006900A2 (https=)
IL (2) IL314692A (https=)
MX (3) MX2020004830A (https=)
MY (1) MY208779A (https=)
SA (1) SA520411930B1 (https=)
SG (1) SG11202004406VA (https=)
TW (1) TWI827560B (https=)
WO (1) WO2019094253A1 (https=)

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

Family Cites Families (59)

* 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
EP2058401A1 (en) * 2007-10-05 2009-05-13 Genethon Widespread gene delivery to motor neurons using peripheral injection of AAV vectors
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
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
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

Similar Documents

Publication Publication Date Title
JP2021502123A5 (https=)
US12612647B2 (en) Compositions useful in treatment of spinal muscular atrophy
JP7808674B2 (ja) Aavウイルスベクター及びその使用
Rodrigues et al. Pharmaceutical Development of AAV-Based Gene Therapy Products for the Eye: Rodrigues et al.
EP4349852A1 (en) Recombinant adeno-associated virus having variant capsid, and application thereof
JP2021121638A (ja) 変異体キャプシドを有するアデノ関連ウイルスビリオンおよびその使用方法
US12594348B2 (en) Compositions for treating Friedreich's ataxia
US20170007720A1 (en) Methods and compositions for gene delivery to on bipolar cells
CN117821513A (zh) 治疗退行性眼部疾病的药物组合物及方法
JP2025527658A (ja) 組換えアデノ随伴ウイルス及びその使用
CN113574176A (zh) 腺相关病毒对cln6多核苷酸的递送
WO2024094044A1 (zh) 优化的pah基因和表达盒及其用途
WO2023169115A1 (zh) 一种神经系统高亲和性的aav载体及其应用
TW202508610A (zh) 醫藥組成物及其用途
CN116042719B (zh) 一种重组腺相关病毒载体及其应用
CN119971081B (zh) 用于治疗智力障碍的组合物、其应用以及药物筛选方法
RU2020118557A (ru) Средства и способ получения вирусных векторов и варианты их применения
CN117281923A (zh) 包含aav的药物组合物及其制备方法
CN121175424A (zh) 一种携带smn基因表达框的病毒载体及其用途
Engelhardt et al. Virus-Mediated Transduction of Murine
RU2024110309A (ru) Средства и способ получения вирусных векторов и варианты их применения
Choudhury et al. In vivo selection yields AAV-B1 capsid for CNS and muscle gene therapy
NZ792477A (en) Aav capsid designs