JP2022541238A5 - - Google Patents

Info

Publication number
JP2022541238A5
JP2022541238A5 JP2022502843A JP2022502843A JP2022541238A5 JP 2022541238 A5 JP2022541238 A5 JP 2022541238A5 JP 2022502843 A JP2022502843 A JP 2022502843A JP 2022502843 A JP2022502843 A JP 2022502843A JP 2022541238 A5 JP2022541238 A5 JP 2022541238A5
Authority
JP
Japan
Prior art keywords
cells
raav
culture medium
protein
nucleic acid
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.)
Pending
Application number
JP2022502843A
Other languages
English (en)
Japanese (ja)
Other versions
JP2022541238A (ja
Filing date
Publication date
Priority claimed from US16/511,612 external-priority patent/US10801042B1/en
Application filed filed Critical
Publication of JP2022541238A publication Critical patent/JP2022541238A/ja
Publication of JP2022541238A5 publication Critical patent/JP2022541238A5/ja
Pending legal-status Critical Current

Links

JP2022502843A 2019-07-15 2020-08-12 組換えアデノ随伴ウイルスのパッケージング効率を増加させるためのイオン濃度の使用 Pending JP2022541238A (ja)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US16/511,612 US10801042B1 (en) 2019-07-15 2019-07-15 Use of ion concentrations to increase the packaging efficiency of recombinant adeno-associated virus
PCT/US2020/045906 WO2021011941A1 (en) 2019-07-15 2020-08-12 Use of ion concentrations to increase the packaging efficiency of recombinant adeno-associated virus

Publications (2)

Publication Number Publication Date
JP2022541238A JP2022541238A (ja) 2022-09-22
JP2022541238A5 true JP2022541238A5 (https=) 2023-08-17

Family

ID=72750230

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2022502843A Pending JP2022541238A (ja) 2019-07-15 2020-08-12 組換えアデノ随伴ウイルスのパッケージング効率を増加させるためのイオン濃度の使用

Country Status (7)

Country Link
US (2) US10801042B1 (https=)
EP (1) EP3999633A4 (https=)
JP (1) JP2022541238A (https=)
KR (1) KR20230039592A (https=)
CN (1) CN114402066A (https=)
AU (1) AU2020315477A1 (https=)
WO (1) WO2021011941A1 (https=)

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
WO2022109241A1 (en) * 2020-11-20 2022-05-27 University Of Florida Research Foundation, Incorporated Improved aav production using potassium chloride
WO2024227006A2 (en) * 2023-04-26 2024-10-31 Genzyme Corporation Methods of improving raav production
CN117683797B (zh) * 2023-12-04 2024-07-05 广州派真生物技术有限公司 一种用于重组腺相关病毒包装的质粒系统及其应用

Family Cites Families (46)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6342390B1 (en) 1994-11-23 2002-01-29 The United States Of America As Represented By The Secretary Of Health And Human Services Lipid vesicles containing adeno-associated virus rep protein for transgene integration and gene therapy
US6093570A (en) 1995-06-07 2000-07-25 The University Of North Carolina At Chapel Hill Helper virus-free AAV production
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
US6989264B2 (en) 1997-09-05 2006-01-24 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
US6566118B1 (en) 1997-09-05 2003-05-20 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
IT1297074B1 (it) 1997-11-21 1999-08-03 Angeletti P Ist Richerche Bio Forme ormone-dipendenti delle proteine rep del virus adeno-associato (aav-2), sequenze di dna codificanti per esse, vettori che le
WO1999061601A2 (en) 1998-05-28 1999-12-02 The Government Of The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Aav5 vector and uses thereof
US6984517B1 (en) 1998-05-28 2006-01-10 The United States Of America As Represented By The Department Of Health And Human Services AAV5 vector and uses thereof
US6759237B1 (en) 1998-11-05 2004-07-06 The Trustees Of The University Of Pennsylvania Adeno-associated virus serotype 1 nucleic acid sequences, vectors and host cells containing same
US6759050B1 (en) 1998-12-03 2004-07-06 Avigen, Inc. Excipients for use in adeno-associated virus pharmaceutical formulations, and pharmaceutical formulations made therewith
DE19905501B4 (de) 1999-02-10 2005-05-19 MediGene AG, Gesellschaft für molekularbiologische Kardiologie und Onkologie Verfahren zur Herstellung eines rekombinanten Adeno-assoziierten Virus, geeignete Mittel hierzu sowie Verwendung zur Herstellung eines Arzneimittels
US6498244B1 (en) 1999-05-28 2002-12-24 Cell Genesys, Inc. Adeno-associated virus capsid immunologic determinants
US7115391B1 (en) 1999-10-01 2006-10-03 Genovo, Inc. Production of recombinant AAV using adenovirus comprising AAV rep/cap genes
US7208315B2 (en) 2000-01-05 2007-04-24 Fred Hutchinson Cancer Research Center Compositions and methods for efficient AAV vector production
WO2002053703A2 (en) 2001-01-05 2002-07-11 Children's Hospital, Inc. Aav2 vectors and methods
US6841357B1 (en) 2001-03-30 2005-01-11 Stratagene California Method and kits for titering adeno-associated virus vectors
US6723551B2 (en) 2001-11-09 2004-04-20 The United States Of America As Represented By The Department Of Health And Human Services Production of adeno-associated virus in insect cells
WO2003042361A2 (en) 2001-11-09 2003-05-22 Government Of The United States Of America, Department Of Health And Human Services Production of adeno-associated virus in insect cells
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
WO2003093479A1 (en) * 2002-05-01 2003-11-13 University Of Florida Research Foundation, Inc. Improved raav expression systems and methods for enhancing transduction of mammalian neural cells
US7419817B2 (en) 2002-05-17 2008-09-02 The United States Of America As Represented By The Secretary Department Of Health And Human Services, Nih. Scalable purification of AAV2, AAV4 or AAV5 using ion-exchange chromatography
US7122348B2 (en) 2002-12-11 2006-10-17 City Of Hope AAV2 Rep protein fusions
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
EP2345731B1 (en) 2003-09-30 2015-10-21 The Trustees of the University of Pennsylvania Adeno-associated virus (AAV) clades, sequences, vectors containing same, and uses thereof
US7625570B1 (en) 2005-03-10 2009-12-01 The Regents Of The University Of California Methods for purifying adeno-associated virus
EP2311966A3 (en) 2005-10-20 2012-09-05 Amsterdam Molecular Therapeutics (AMT) B.V. Improved AAV vectors produced in insect cells
WO2008024998A2 (en) 2006-08-24 2008-02-28 Virovek, Inc. Expression in insect cells of genes with overlapping open reading frames, methods and compositions therefor
GB2445441B (en) * 2006-09-26 2010-06-30 Ge Healthcare Bio Sciences Nucleic acid purification method
KR20180000341A (ko) 2009-06-16 2018-01-02 젠자임 코포레이션 재조합 ａａｖ 벡터에 대한 개선된 정제 방법
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
TR201908020T4 (tr) 2011-02-17 2019-06-21 Univ Pennsylvania Doku özgüllüğünü değiştirmek ve aav9-aracılı gen transferini geliştirmek için bileşimler ve yöntemler.
EP2500434A1 (en) 2011-03-12 2012-09-19 Association Institut de Myologie Capsid-free AAV vectors, compositions, and methods for vector production and gene delivery
US20140155469A1 (en) 2011-04-19 2014-06-05 The Research Foundation Of State University Of New York Adeno-associated-virus rep sequences, vectors and viruses
CN105755044A (zh) 2011-04-22 2016-07-13 加利福尼亚大学董事会 具有变异衣壳的腺相关病毒病毒体及其使用方法
CA2853482C (en) 2011-10-28 2020-05-19 Joshua Grieger Cell line for production of adeno-associated virus
AU2013266968B2 (en) 2012-05-25 2017-06-29 Emmanuelle CHARPENTIER Methods and compositions for RNA-directed target DNA modification and for RNA-directed modulation of transcription
US8697359B1 (en) 2012-12-12 2014-04-15 The Broad Institute, Inc. CRISPR-Cas systems and methods for altering expression of gene products
CN105765066B (zh) 2013-11-29 2018-12-14 宝生物工程株式会社 用于定量腺伴随病毒的方法
WO2017112948A1 (en) 2015-12-24 2017-06-29 University Of Florida Research Foundation, Inc. Improved aav production using suspension adapted cells
PL3526321T3 (pl) * 2016-10-14 2025-04-28 Ultragenyx Pharmaceutical Inc. Zastosowanie środków regulujących ciśnienie osmotyczne w celu zwiększenia wydajności rekombinacyjnego wirusa związanego z adenowirusami
US10294452B2 (en) 2016-11-22 2019-05-21 Dao-Yao He Lysis, extraction and purification of adeno-associated virus and adenovirus from host cells
WO2019094253A1 (en) * 2017-11-08 2019-05-16 Avexis Inc. Means and method for preparing viral vectors and uses of same
EP3833745A1 (en) * 2018-08-10 2021-06-16 REGENXBIO Inc. Scalable method for recombinant aav production
CN109666697B (zh) * 2019-01-30 2020-03-10 广州派真生物技术有限公司 一种提高腺相关病毒产量的生产工艺
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

Similar Documents

Publication Publication Date Title
JP2022541238A5 (https=)
JP7761686B2 (ja) 操作されたプロモータを有するフラタキシン発現コンストラクトおよびその使用方法
JP7818558B2 (ja) 修飾された第ｉｘ因子、並びに、細胞、器官及び組織への遺伝子導入のための組成物、方法及び使用
JP7094236B2 (ja) 変異ａａｖ、及び、細胞、臓器並びに組織への遺伝子導入のための組成物、方法並びに使用法
CN104520428B (zh) 将基因转移到细胞、器官和组织的aav载体组合物和方法
JP6338560B2 (ja) 神経代謝性疾患のための遺伝子治療
JP2020050660A (ja) 脊髄疾患の遺伝子療法
JP2020527333A (ja) パーキンソン病を治療するためのａａｄｃポリヌクレオチド
US20090074723A1 (en) Method of Treating or Retarding the Development of Blindness
US20190000940A1 (en) Compositions and methods for the treatment of aadc deficiency
JP2016517278A (ja) スタッファー／フィラーポリヌクレオチド配列を含むベクターおよびその使用方法
Vance et al. AAV Biology, Infectivity and Therapeutic Use from
BR112020026361A2 (pt) Composições e métodos para tratar neuropatia óptica hereditária de leber
JP2021533801A5 (https=)
KR20250105716A (ko) 간에서의 유전자 발현을 위한 핵산 조절 요소 및 사용 방법
JP2024535121A (ja) 肝臓指向性カプシドタンパク質及び酸性α－グルコシダーゼを含むＡＡＶ粒子並びにポンペ病を治療するための使用
US20240424141A1 (en) Aav particles comprising a liver-tropic capsid protein and alpha-galactosidase and their use to treat fabry disease
WO2026017126A1 (zh) 一种人工设计的新型nrg-1在疾病治疗中的应用
WO2022026410A2 (en) Compositions and methods for the treatment of niemann-pick type c1 disease
US20230321281A1 (en) Compositions and methods for the treatment of eye diseases
WO2024079661A1 (en) Atp7b gene therapy
US20260124322A1 (en) Atp7b gene therapy
TWI813851B (zh) 具有工程化之啟動子之共濟蛋白（ｆｒａｔａｘｉｎ）表現構築體及其使用方法
Zhang et al. AAV-Mediated Gene Therapy for the Inner Ear: Progress, Challenges, and Future Directions
WO2024163012A1 (en) Compositions and methods for the treatment of neurological disorders related to glucosylceramidase beta deficiency