PE20181534A1 - Metodos escalables para producir un vector viral adenoasociado (aav) recombinante en un sistema de cultivo celular en suspension sin suero adecuado para uso clinico - Google Patents

Metodos escalables para producir un vector viral adenoasociado (aav) recombinante en un sistema de cultivo celular en suspension sin suero adecuado para uso clinico

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Publication number
PE20181534A1
PE20181534A1 PE2018001066A PE2018001066A PE20181534A1 PE 20181534 A1 PE20181534 A1 PE 20181534A1 PE 2018001066 A PE2018001066 A PE 2018001066A PE 2018001066 A PE2018001066 A PE 2018001066A PE 20181534 A1 PE20181534 A1 PE 20181534A1
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Peru
Prior art keywords
adeno
associated viral
recombining
aav
produce
Prior art date
Application number
PE2018001066A
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English (en)
Inventor
Guang Qu
Lin Lu
John Fraser Wright
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Spark Therapeutics Inc
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Publication date
Application filed by Spark Therapeutics Inc filed Critical Spark Therapeutics Inc
Publication of PE20181534A1 publication Critical patent/PE20181534A1/es

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Abstract

Se describen metodos y composiciones para transfectar celulas con plasmidos. En ciertas modalidades, se divulgan metodos y composiciones en las que la eficacia de la transfeccion aumenta significativamente al poner en contacto las celulas que se transducen con polietilenimina (PEI) que esta libre de acido nucleico durante el proceso de transfeccion. Tambien se describen vectores virales adenoasociados terapeuticamente utiles generados de acuerdo con los metodos y composiciones divulgados
PE2018001066A 2015-12-01 2016-12-01 Metodos escalables para producir un vector viral adenoasociado (aav) recombinante en un sistema de cultivo celular en suspension sin suero adecuado para uso clinico PE20181534A1 (es)

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US201562261815P 2015-12-01 2015-12-01

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PE20181534A1 true PE20181534A1 (es) 2018-09-26

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PE2018001066A PE20181534A1 (es) 2015-12-01 2016-12-01 Metodos escalables para producir un vector viral adenoasociado (aav) recombinante en un sistema de cultivo celular en suspension sin suero adecuado para uso clinico
PE2023002040A PE20240371A1 (es) 2015-12-01 2016-12-01 Metodos escalables para producir un vector viral adenoasociado (aav) recombinante en un sistema de cultivo celular en suspension sin suero adecuado para uso clinico

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US (1) US20190292561A1 (es)
EP (1) EP3384015A4 (es)
JP (2) JP7444521B2 (es)
KR (1) KR20180091863A (es)
CN (1) CN108603174A (es)
AU (2) AU2016362317B2 (es)
BR (1) BR112018011193A2 (es)
CA (1) CA3006309A1 (es)
CO (1) CO2018006699A2 (es)
IL (1) IL259595B2 (es)
MX (2) MX2018006682A (es)
PE (2) PE20181534A1 (es)
PH (1) PH12018501168A1 (es)
RU (1) RU2766583C2 (es)
SG (2) SG10202106287YA (es)
WO (1) WO2017096039A1 (es)

Families Citing this family (53)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2015191508A1 (en) 2014-06-09 2015-12-17 Voyager Therapeutics, Inc. Chimeric capsids
SG11201703148TA (en) 2014-11-05 2017-05-30 Voyager Therapeutics Inc Aadc polynucleotides for the treatment of parkinson's disease
GB201420139D0 (en) 2014-11-12 2014-12-24 Ucl Business Plc Factor IX gene therapy
AU2015346162B2 (en) 2014-11-14 2022-02-10 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (ALS)
ES2878451T3 (es) 2014-11-14 2021-11-18 Voyager Therapeutics Inc Polinucleótidos moduladores
WO2016094783A1 (en) 2014-12-12 2016-06-16 Voyager Therapeutics, Inc. Compositions and methods for the production of scaav
CA2996420A1 (en) 2015-09-28 2017-04-06 The University Of North Carolina At Chapel Hill Methods and compositions for antibody-evading virus vectors
PE20181534A1 (es) * 2015-12-01 2018-09-26 Spark Therapeutics Inc Metodos escalables para producir un vector viral adenoasociado (aav) recombinante en un sistema de cultivo celular en suspension sin suero adecuado para uso clinico
EP3448874A4 (en) 2016-04-29 2020-04-22 Voyager Therapeutics, Inc. COMPOSITIONS FOR TREATING A DISEASE
WO2017189964A2 (en) 2016-04-29 2017-11-02 Voyager Therapeutics, Inc. Compositions for the treatment of disease
AU2017267665C1 (en) 2016-05-18 2023-10-05 Voyager Therapeutics, Inc. Modulatory polynucleotides
MX2018014152A (es) 2016-05-18 2019-03-28 Voyager Therapeutics Inc Composiciones y métodos para tratar la enfermedad de huntington.
CN110225975A (zh) 2016-08-23 2019-09-10 阿库斯股份有限公司 用于治疗人受试者中非年龄相关的听力损害的组合物和方法
CA3035522A1 (en) 2016-08-30 2018-03-08 The Regents Of The University Of California Methods for biomedical targeting and delivery and devices and systems for practicing the same
WO2018204786A1 (en) 2017-05-05 2018-11-08 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (als)
WO2018204803A1 (en) 2017-05-05 2018-11-08 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
KR102669561B1 (ko) * 2017-06-30 2024-05-24 스파크 테라퓨틱스, 인코포레이티드 Aav 벡터 컬럼 정제 방법
JP7229989B2 (ja) 2017-07-17 2023-02-28 ボイジャー セラピューティクス インコーポレイテッド 軌道アレイガイドシステム
TWI722310B (zh) 2017-08-03 2021-03-21 美商航海家醫療公司 用於aav之遞送之組合物及方法
US20200237799A1 (en) 2017-10-16 2020-07-30 Voyager Therapeutics, Inc. Treatment of amyotrophic lateral sclerosis (als)
EP4124658A3 (en) 2017-10-16 2023-04-19 Voyager Therapeutics, Inc. Treatment of amyotrophic lateral sclerosis (als)
SG11202003479TA (en) * 2017-10-18 2020-05-28 Regenxbio Inc Fully-human post-translationally modified antibody therapeutics
EP3720500A4 (en) * 2017-12-05 2021-08-25 Applied Genetic Technologies Corporation FORMULATION OPTIMIZATION FOR VIRAL PARTICLES
MX2020010464A (es) 2018-04-03 2021-01-29 Vectores de virus que evitan anticuerpos.
SG11202009451VA (en) 2018-04-03 2020-10-29 Stridebio Inc Antibody-evading virus vectors
CA3094465A1 (en) 2018-04-03 2019-10-10 Stridebio, Inc. Virus vectors for targeting ophthalmic tissues
JP2021522813A (ja) * 2018-05-07 2021-09-02 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. プラスミドを用いないaavベクター産生細胞株
CN108866011A (zh) * 2018-07-31 2018-11-23 深圳生生凡非基因技术有限公司 一种同步包装rAAV的方法
US10842885B2 (en) 2018-08-20 2020-11-24 Ucl Business Ltd Factor IX encoding nucleotides
CN110437317B (zh) * 2019-01-30 2023-05-02 上海科技大学 具有变异衣壳蛋白的腺相关病毒及其用途
US11746360B2 (en) * 2019-02-11 2023-09-05 Florian M. Wurm Eukaryotic cell transfection systems and related methods
AR118465A1 (es) 2019-03-21 2021-10-06 Stridebio Inc Vectores de virus adenoasociados recombinantes
WO2021028837A1 (en) 2019-08-13 2021-02-18 Waters Technologies Corporation Affinity resins and sample preparation devices based on cartilaginous fish ignar derived binding domains
CA3157700A1 (en) 2019-10-17 2021-04-22 Stridebio, Inc. Adeno-associated viral vectors for treatment of niemann-pick disease type c
CN110894494B (zh) * 2019-11-22 2022-09-27 广西梧州制药(集团)股份有限公司 一种大规模高密度悬浮培养293细胞高产腺病毒的方法
CN110938654A (zh) * 2019-12-11 2020-03-31 和元生物技术(上海)股份有限公司 一种细胞转染试剂及其应用
CN115315518A (zh) * 2020-01-17 2022-11-08 阿斯克肋匹奥生物制药公司 重组aav的生产
AU2021225035A1 (en) 2020-02-21 2022-10-13 Akouos, Inc. Compositions and methods for treating non-age-associated hearing impairment in a human subject
EP4200408A1 (en) 2020-08-19 2023-06-28 Sarepta Therapeutics, Inc. Adeno-associated virus vectors for treatment of rett syndrome
WO2022043926A1 (en) * 2020-08-31 2022-03-03 Intas Pharmaceuticals Ltd. Process for preparation of recombinant adeno-associated virus particle
AR123776A1 (es) 2020-10-15 2023-01-11 Hoffmann La Roche Construcciones de ácido nucleico para la activación simultánea de genes
MX2023004052A (es) 2020-10-15 2023-05-03 Hoffmann La Roche Constructos de acido nucleico para transcripcion de arn asociado a un virus (arn va).
CA3218362A1 (en) * 2021-04-27 2022-11-03 Novartis Ag Viral vector production system
WO2023198685A1 (en) 2022-04-13 2023-10-19 F. Hoffmann-La Roche Ag Method for determining aav genomes
EP4279599A1 (en) * 2022-05-18 2023-11-22 Branca Bunus Limited Ordered formulation method to construct polyplex with core-aggregate structure
WO2023227438A1 (en) 2022-05-23 2023-11-30 F. Hoffmann-La Roche Ag Raman-based method for the differentiation of aav particle serotype and aav particle loading status
AR129500A1 (es) 2022-06-03 2024-09-04 Hoffmann La Roche Método para producir partículas de aav recombinante
WO2024013239A1 (en) 2022-07-14 2024-01-18 F. Hoffmann-La Roche Ag Method for producing recombinant aav particles
WO2024056561A1 (en) 2022-09-12 2024-03-21 F. Hoffmann-La Roche Ag Method for separating full and empty aav particles
WO2024129882A1 (en) * 2022-12-14 2024-06-20 Astellas Gene Therapies, Inc. Compositions and methods for improved production of adeno-associated viral particles
WO2024165456A1 (en) 2023-02-07 2024-08-15 F. Hoffmann-La Roche Ag Method for the detection of anti-aav particle antibodies
WO2024194280A1 (en) 2023-03-21 2024-09-26 F. Hoffmann-La Roche Ag Method for the production of recombinant aav particle preparations

Family Cites Families (15)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6995006B2 (en) * 1997-09-05 2006-02-07 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
JP2001514845A (ja) * 1997-09-05 2001-09-18 ターゲティッド ジェネティクス コーポレイション 組換えaavベクターの高力価ヘルパーなし調製物を生成するための方法
US6593123B1 (en) * 2000-08-07 2003-07-15 Avigen, Inc. Large-scale recombinant adeno-associated virus (rAAV) production and purification
US7605249B2 (en) * 2002-11-26 2009-10-20 Medtronic, Inc. Treatment of neurodegenerative disease through intracranial delivery of siRNA
US7829694B2 (en) * 2002-11-26 2010-11-09 Medtronic, Inc. Treatment of neurodegenerative disease through intracranial delivery of siRNA
RU2303064C2 (ru) * 2005-04-06 2007-07-20 ГУ НИИ вирусных препаратов им. О.Г. Анджапаридзе РАМН Молекулярный комплекс для трансфекции клеток млекопитающих, содержащий плазмидную днк, модифицированный полиэтиленимин и молекулы-лиганды
WO2013123503A1 (en) * 2012-02-17 2013-08-22 The Children's Hospital Of Philadelphia Aav vector compositions and methods for gene transfer to cells, organs and tissues
EP2970920B1 (en) * 2013-03-15 2018-04-25 The Children's Hospital of Philadelphia Scalable manufacturing process to produce recombinant lentiviral vectors in serum-free suspension cell culture system
EP3008191A2 (en) * 2013-06-13 2016-04-20 Shire Human Genetic Therapies, Inc. Messenger rna based viral production
CN103329852B (zh) * 2013-06-19 2015-10-07 中国医学科学院病原生物学研究所 一种hbv持续性感染及纤维化小鼠模型建立
CA2916800C (en) * 2013-06-28 2022-10-25 Ethris Gmbh Compositions comprising a component with oligo(alkylene amine) moieties
KR102380265B1 (ko) * 2013-07-22 2022-03-29 더 칠드런스 호스피탈 오브 필라델피아 변종 aav 및 조성물, 세포, 기관 및 조직으로의 유전자 전이를 위한 방법 및 용도
CN105873613A (zh) * 2013-08-13 2016-08-17 贝勒医学院 用于核酸和药物递送的新型plga-修饰的聚乙烯亚胺自组装纳米技术
PE20181534A1 (es) 2015-12-01 2018-09-26 Spark Therapeutics Inc Metodos escalables para producir un vector viral adenoasociado (aav) recombinante en un sistema de cultivo celular en suspension sin suero adecuado para uso clinico
CN110997912A (zh) * 2017-06-07 2020-04-10 星火治疗有限公司 用于改进的细胞转染和/或rAAV载体生产的增强剂

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