CY1121442T1 - Βελτιωμενες μεθοδοι για τον καθαρισμο ανασυνδυασμενων φορεων aav - Google Patents

Βελτιωμενες μεθοδοι για τον καθαρισμο ανασυνδυασμενων φορεων aav

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Publication number
CY1121442T1
CY1121442T1 CY20181101103T CY181101103T CY1121442T1 CY 1121442 T1 CY1121442 T1 CY 1121442T1 CY 20181101103 T CY20181101103 T CY 20181101103T CY 181101103 T CY181101103 T CY 181101103T CY 1121442 T1 CY1121442 T1 CY 1121442T1
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Cyprus
Prior art keywords
recombinant aav
improved methods
aav carriers
cleaning
cleaning recombinant
Prior art date
Application number
CY20181101103T
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English (en)
Inventor
Paulene Mclean Quigley Sheldon
Peter S. Gagnon
Gina Nichols
Barbara A. Thorne
U.S.A.
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Genzyme Corporation
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Filing date
Publication date
Application filed by Genzyme Corporation filed Critical Genzyme Corporation
Publication of CY1121442T1 publication Critical patent/CY1121442T1/el

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    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • C12N7/02—Recovery or purification
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • C12N15/861—Adenoviral vectors
    • G—PHYSICS
    • G01—MEASURING; TESTING
    • G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N30/00—Investigating or analysing materials by separation into components using adsorption, absorption or similar phenomena or using ion-exchange, e.g. chromatography or field flow fractionation
    • G01N30/02—Column chromatography
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14151—Methods of production or purification of viral material
    • G—PHYSICS
    • G01—MEASURING; TESTING
    • G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N30/00—Investigating or analysing materials by separation into components using adsorption, absorption or similar phenomena or using ion-exchange, e.g. chromatography or field flow fractionation
    • G01N30/02—Column chromatography
    • G01N2030/022—Column chromatography characterised by the kind of separation mechanism
    • G01N2030/027—Liquid chromatography

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Virology (AREA)
  • Microbiology (AREA)
  • Medicinal Chemistry (AREA)
  • Immunology (AREA)
  • Biophysics (AREA)
  • Molecular Biology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Analytical Chemistry (AREA)
  • General Physics & Mathematics (AREA)
  • Pathology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Peptides Or Proteins (AREA)
  • Treatment Of Liquids With Adsorbents In General (AREA)
  • Medicinal Preparation (AREA)

Abstract

Στο παρόν παρέχονται μέθοδοι για τον καθαρισμό ανασυνδυασμένων αδενοσχετιζόμενων ιικών φορέων (rAAV) που μπορεί να χρησιμοποιηθούν για γονιδιακή μεταφορά και συγκεκριμένα για γονιδιακή θεραπεία ή εμβολιασμό. Ανασυνδυασμένοι AAV φορείς της εφεύρεσης είναι ουσιαστικά ελεύθεροι από προσμίξεις κατά τη διεργασία, συμπεριλαμβανομένων συστατικών παραγωγής, όπως κυτταρικών νουκλεϊκών οξέων, κυτταρικών πρωτεϊνών, βοηθητικού ιού, και συστατικών του μέσου.
CY20181101103T 2009-06-16 2018-10-25 Βελτιωμενες μεθοδοι για τον καθαρισμο ανασυνδυασμενων φορεων aav CY1121442T1 (el)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US18760109P 2009-06-16 2009-06-16
PCT/US2010/038897 WO2010148143A1 (en) 2009-06-16 2010-06-16 Improved methods for purification of recombinant aav vectors
EP10790155.5A EP2443233B1 (en) 2009-06-16 2010-06-16 Improved methods for purification of recombinant aav vectors

Publications (1)

Publication Number Publication Date
CY1121442T1 true CY1121442T1 (el) 2020-05-29

Family

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Family Applications (3)

Application Number Title Priority Date Filing Date
CY20161100843T CY1117936T1 (el) 2009-06-16 2016-08-29 Βελτιωμενες μεθοδοι για τον καθαρισμο ανασυνδυασμενων φορεων aav
CY20181101103T CY1121442T1 (el) 2009-06-16 2018-10-25 Βελτιωμενες μεθοδοι για τον καθαρισμο ανασυνδυασμενων φορεων aav
CY20211100639T CY1124323T1 (el) 2009-06-16 2021-07-14 Βελτιωμενες μεθοδοι για τον καθαρισμο ανασυνδυασμενων φορεων aav

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CY20161100843T CY1117936T1 (el) 2009-06-16 2016-08-29 Βελτιωμενες μεθοδοι για τον καθαρισμο ανασυνδυασμενων φορεων aav

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Country Status (24)

Country Link
US (3) US10017746B2 (el)
EP (4) EP3067417B1 (el)
JP (3) JP5956331B2 (el)
KR (2) KR20180000341A (el)
CN (2) CN105861454B (el)
BR (1) BRPI1015176B1 (el)
CA (2) CA2764176C (el)
CY (3) CY1117936T1 (el)
DK (2) DK3444335T3 (el)
ES (3) ES2881050T3 (el)
HK (1) HK1165830A1 (el)
HR (2) HRP20181743T1 (el)
HU (3) HUE054940T2 (el)
IL (3) IL216960A (el)
LT (2) LT3444335T (el)
MX (2) MX2011013613A (el)
PL (2) PL3067417T3 (el)
PT (3) PT2443233T (el)
RS (2) RS57784B1 (el)
RU (1) RU2016119739A (el)
SG (2) SG10201403290SA (el)
SI (2) SI3444335T1 (el)
TR (1) TR201815937T4 (el)
WO (1) WO2010148143A1 (el)

Families Citing this family (96)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US9198984B2 (en) 2006-04-28 2015-12-01 The Trustees Of The University Of Pennsylvania Scalable production method for AAV
JP5956331B2 (ja) * 2009-06-16 2016-07-27 ジェンザイム・コーポレーション 組換えａａｖベクターの改良された精製方法
US8951807B2 (en) 2010-01-15 2015-02-10 Bio-Rad Laboratories, Inc. Surface neutralization of apatite
US8895707B2 (en) 2010-08-18 2014-11-25 Bio-Rad Laboratories, Inc. Elution of proteins from hydroxyapatite resins without resin deterioration
CA2825651C (en) 2011-02-02 2020-03-24 Bio-Rad Laboratories, Inc. Apatite surface neutralization with alkali solutions
SI3318571T1 (sl) 2011-03-16 2021-08-31 F. Hoffmann-La Roche Ag Ionska izmenjevalna kromatografija z izboljšano selektivnostjo za ločevanje polipeptidnih monomerov, agregatov in fragmentov s spremembo mobilne faze
JP6007463B2 (ja) * 2011-04-18 2016-10-12 国立研究開発法人国立精神・神経医療研究センター 薬剤送達粒子及びその製造方法
WO2013036118A1 (en) * 2011-09-08 2013-03-14 Uniqure Ip B.V. Removal of contaminating viruses from aav preparations
CN104507952B (zh) 2012-05-30 2018-08-10 生物辐射实验室股份有限公司 基于磷灰石的色谱树脂的原位恢复
KR20150023297A (ko) * 2012-05-31 2015-03-05 에이전시 포 사이언스, 테크놀로지 앤드 리서치 고상 우레이드에 대한 생물학적 표적의 선택적 결합
GB2525562B (en) * 2013-02-15 2017-10-11 T Gjerde Douglas Methods for purification of biological cells
BR112015022292A2 (pt) * 2013-03-15 2017-07-18 Childrens Hospital Philadelphia processo de fabricação escalonável para produzir vetores lentivirais recombinantes em um sistema de cultura celular em suspensão sem soro
US11332719B2 (en) 2013-03-15 2022-05-17 The Broad Institute, Inc. Recombinant virus and preparations thereof
MX2015014568A (es) 2013-04-17 2016-12-02 Genzyme Corp Composiciones y metodos para tratar y prevenir la degeneracion macular.
KR102069561B1 (ko) * 2013-07-11 2020-01-23 다카라 바이오 가부시키가이샤 외피 비보유 바이러스의 제조 방법
JP6719381B2 (ja) 2014-02-06 2020-07-08 ジェンザイム・コーポレーション 黄斑変性を処置し、予防するための組成物および方法
ES2879636T3 (es) 2014-05-02 2021-11-22 Genzyme Corp Vectores AAV para la terapia génica de la retina y el SNC
WO2015191508A1 (en) 2014-06-09 2015-12-17 Voyager Therapeutics, Inc. Chimeric capsids
CN105517718B (zh) 2014-06-23 2019-12-31 生物辐射实验室股份有限公司 磷灰石原位恢复
EP3157689B1 (en) 2014-06-23 2021-04-28 Bio-Rad Laboratories, Inc. Apatite pretreatment
WO2016006658A1 (ja) 2014-07-10 2016-01-14 タカラバイオ株式会社 非エンベロープウイルス粒子の製造方法
CN104232687A (zh) * 2014-09-17 2014-12-24 许瑞安 一种重组腺相关病毒rAAV载体的分离纯化方法
RU2020109343A (ru) 2014-11-05 2020-03-17 Вояджер Терапьютикс, Инк. Полинуклеотиды aadc для лечения болезни паркинсона
JP6863891B2 (ja) 2014-11-14 2021-04-21 ボイジャー セラピューティクス インコーポレイテッドＶｏｙａｇｅｒ Ｔｈｅｒａｐｅｕｔｉｃｓ，Ｉｎｃ． 調節性ポリヌクレオチド
CN114717264A (zh) 2014-11-14 2022-07-08 沃雅戈治疗公司 治疗肌萎缩性侧索硬化(als)的组合物和方法
US11697825B2 (en) 2014-12-12 2023-07-11 Voyager Therapeutics, Inc. Compositions and methods for the production of scAAV
US10415020B2 (en) 2015-01-09 2019-09-17 Takara Bio Inc. Method for producing non-enveloped viral particles
JP2018505695A (ja) 2015-01-20 2018-03-01 ジェンザイム・コーポレーション 組換えウイルス粒子の特徴付けのための分析超遠心分離法
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
EP4219728A3 (en) 2015-02-10 2023-08-23 Genzyme Corporation Enhanced delivery of viral particles to the striatum and cortex
TWI707951B (zh) 2015-04-08 2020-10-21 美商健臻公司 過大腺相關載體之製造
CN105907729B (zh) * 2015-05-05 2019-08-16 广东东阳光药业有限公司 一种制备人乳头瘤病毒l1蛋白类病毒样颗粒的方法
US20170067028A1 (en) * 2015-05-15 2017-03-09 Douglas J. Ballon Radiolabeling of adeno associated virus
JP6985250B2 (ja) 2015-05-16 2021-12-22 ジェンザイム・コーポレーション 深部イントロン突然変異の遺伝子編集
US9663766B2 (en) 2015-07-24 2017-05-30 Bio-Rad Laboratories, Inc. Methods for purifying adenovirus vectors
WO2017160360A2 (en) 2015-12-11 2017-09-21 The Trustees Of The University Of Pennsylvania Scalable purification method for aav9
ES2934848T3 (es) 2015-12-11 2023-02-27 Univ Pennsylvania Método de purificación escalable para AAV8
WO2017100674A1 (en) * 2015-12-11 2017-06-15 The Trustees Of The University Of Pennsylvania Scalable purification method for aav1
US11028372B2 (en) 2015-12-11 2021-06-08 The Trustees Of The University Of Pennsylvania Scalable purification method for AAVRH10
JP7347932B2 (ja) 2015-12-15 2023-09-20 ジェンザイム・コーポレーション ムコリピドーシスｉｉ型を治療するためのアデノ随伴ウイルスベクター
US11208630B2 (en) 2015-12-24 2021-12-28 University Of Florida Research Foundation, Incorporated AAV production using suspension adapted cells
PL3436593T3 (pl) 2016-03-28 2023-03-27 Ultragenyx Pharmaceutical Inc. Sposoby inaktywacji termicznej adenowirusów
AU2017240721B2 (en) * 2016-03-31 2023-10-12 Spark Therapeutics, Inc. Column-based fully scalable rAAV manufacturing process
EP3448987A4 (en) 2016-04-29 2020-05-27 Voyager Therapeutics, Inc. COMPOSITIONS FOR THE TREATMENT OF DISEASES
WO2017189959A1 (en) 2016-04-29 2017-11-02 Voyager Therapeutics, Inc. Compositions for the treatment of disease
GB201608046D0 (en) 2016-05-09 2016-06-22 Cambridge Entpr Ltd And Syndey Children S Hospitals Network Randwick And Westmead Incorporating The Treatment of complement-mediated disorders
JP7066635B2 (ja) 2016-05-18 2022-05-13 ボイジャー セラピューティクス インコーポレイテッド 調節性ポリヌクレオチド
US11951121B2 (en) 2016-05-18 2024-04-09 Voyager Therapeutics, Inc. Compositions and methods for treating Huntington's disease
GB201612248D0 (en) 2016-07-14 2016-08-31 Puridify Ltd New process
KR102425289B1 (ko) 2016-08-15 2022-07-27 젠자임 코포레이션 Aav의 검출 방법
WO2018034885A1 (en) 2016-08-16 2018-02-22 Regeneron Pharmaceuticals, Inc. Methods for quantitating individual antibodies from a mixture
EP3510161A4 (en) 2016-08-23 2020-04-22 Akouos, Inc. COMPOSITIONS AND METHODS FOR TREATING PERSONAL HEARING LOSS IN A PERSON
WO2018044933A1 (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
US11795473B2 (en) 2016-10-14 2023-10-24 Ultragenyx Pharmaceutical Inc. Use of tonicifying agents to enhance recombinant adeno-associated virus yield
DK3532838T3 (da) 2016-10-25 2022-07-04 Regeneron Pharma Fremgangsmåder og systemer til kromatografidataanalyse
IL266162B2 (en) * 2016-11-04 2023-11-01 Baxalta Inc Adeno-associated virus purification methods
US10626376B2 (en) * 2016-11-14 2020-04-21 St. Jude Children's Research Hospital Method for isolating and purifying adeno-associated virus particles using salt
EP3601581A4 (en) 2017-03-22 2021-05-19 Ultragenyx Pharmaceutical Inc. CELL CULTURE METHODS USING HDAC INHIBITORS OR REP PROTEINS
CN110913866A (zh) 2017-05-05 2020-03-24 沃雅戈治疗公司 治疗肌萎缩性侧索硬化(als)的组合物和方法
MX2019013172A (es) 2017-05-05 2020-09-07 Voyager Therapeutics Inc Composiciones y metodos para tratar la enfermedad de huntington.
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
US11497576B2 (en) 2017-07-17 2022-11-15 Voyager Therapeutics, Inc. Trajectory array guide system
EP3662060A2 (en) 2017-08-03 2020-06-10 Voyager Therapeutics, Inc. Compositions and methods for delivery of aav
EP3697905A1 (en) 2017-10-16 2020-08-26 Voyager Therapeutics, Inc. Treatment of amyotrophic lateral sclerosis (als)
US20200237799A1 (en) 2017-10-16 2020-07-30 Voyager Therapeutics, Inc. Treatment of amyotrophic lateral sclerosis (als)
CN111655285A (zh) 2017-12-29 2020-09-11 百深公司 腺相关病毒的纯化方法
US10610606B2 (en) 2018-02-01 2020-04-07 Homology Medicines, Inc. Adeno-associated virus compositions for PAH gene transfer and methods of use thereof
US11306329B2 (en) 2018-02-19 2022-04-19 City Of Hope Adeno-associated virus compositions for restoring F8 gene function and methods of use thereof
WO2019212921A1 (en) * 2018-04-29 2019-11-07 Regenxbio Inc. Scalable clarification process for recombinant aav production
TW202005694A (zh) 2018-07-02 2020-02-01 美商里珍納龍藥品有限公司 自混合物製備多肽之系統及方法
WO2020023612A1 (en) * 2018-07-24 2020-01-30 Voyager Therapeutics, Inc. Systems and methods for producing gene therapy formulations
JP7070238B2 (ja) * 2018-08-22 2022-05-18 株式会社島津製作所 液体クロマトグラフ分析装置、移動相供給装置、液体クロマトグラフ分析方法および移動相供給方法
CA3112824A1 (en) * 2018-09-21 2020-03-26 Nightstarx Limited Compositions and methods for manufacturing gene therapy vectors
WO2020077250A1 (en) 2018-10-12 2020-04-16 Genzyme Corporation Generation of improved human pah for treatment of severe pku by liver-directed gene replacement therapy
WO2020102723A1 (en) 2018-11-16 2020-05-22 Encoded Therapeutics, Inc. Compositions and methods for treating wilson's disease
EP3959325A2 (en) 2019-04-23 2022-03-02 INSERM (Institut National de la Santé et de la Recherche Médicale) New adeno-associated virus (aav) variants and uses thereof for gene therapy
US10653731B1 (en) 2019-07-15 2020-05-19 Vigene Biosciences Inc. Recombinantly-modified adeno-associated virus (rAAV) having improved packaging efficiency
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
US10557149B1 (en) 2019-07-15 2020-02-11 Vigene Biosciences, Inc. Recombinantly-modified adeno-associated virus helper vectors and their use to improve the packaging efficiency of recombinantly-modified adeno-associated virus
TW202140791A (zh) 2020-01-13 2021-11-01 美商霍蒙拉奇醫藥公司 治療苯酮尿症之方法
US20210261625A1 (en) 2020-01-29 2021-08-26 Genzyme Corporation Modified adeno-associated viral capsid proteins for ocular gene therapy and methods of use thereof
CN116096905A (zh) 2020-02-21 2023-05-09 阿库斯股份有限公司 用于治疗人受试者的非年龄相关性听力损伤的组合物和方法
EP3919613A1 (en) * 2020-06-05 2021-12-08 Bia Separations D.O.O. Enhanced purification of adeno-associated virus to more effectively remove contaminating dna
US20230212530A1 (en) * 2020-06-16 2023-07-06 Hoya Technosurgical Corporation Virus purification method using apatite column
CN111808828B (zh) * 2020-09-14 2020-11-17 和元生物技术（上海）股份有限公司 腺相关病毒的分离纯化方法
US20220112520A1 (en) 2020-10-01 2022-04-14 Genzyme Corporation Human pah expression cassette for treatment of pku by liver-directed gene replacement therapy
JP2022138144A (ja) 2021-03-09 2022-09-22 Ｊｃｒファーマ株式会社 組換えａａｖ９ビリオンの製造方法
US20240159718A1 (en) 2021-03-22 2024-05-16 Genzyme Corporation Size exclusion chromatography analysis of empty and full aav capsids
TW202402329A (zh) 2022-04-12 2024-01-16 美商健臻公司 Irak4調節劑於基因療法之用途(二)
US20230407255A1 (en) 2022-04-12 2023-12-21 Genzyme Corporation Dendritic cell assay for innate immunogenicity to gene therapy agents
WO2023201272A1 (en) 2022-04-12 2023-10-19 Genzyme Corporation Use of irak4 modulators for gene therapy
WO2023198828A1 (en) 2022-04-13 2023-10-19 Universitat Autònoma De Barcelona Treatment of neuromuscular diseases via gene therapy that expresses klotho protein
US20230398192A1 (en) 2022-05-16 2023-12-14 Genzyme Corporation Methods of treating metachromatic leukodystrophy
WO2023240062A1 (en) 2022-06-07 2023-12-14 Adverum Biotechnologies, Inc. Melanopsin variants for vision restoration

Family Cites Families (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JPH11511326A (ja) * 1995-08-30 1999-10-05 ジエンザイム コーポレイション アデノウィルスおよびａａｖの精製
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
ES2557997T3 (es) 1997-09-05 2016-02-01 Genzyme Corporation Métodos para generar preparados de vectores AAV de título elevado sin virus auxiliar
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
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
US20090047723A1 (en) * 2005-01-14 2009-02-19 Bayer Healthcare Llc Method for purification of factor vii
SG179488A1 (en) * 2007-03-14 2012-04-27 Ligocyte Pharmaceuticals Inc Virus like particle purification
GB2467320A (en) 2009-01-28 2010-08-04 Surrey Nanosystems Ltd Two methods of forming carbon nano-materials using filtered acetylene gas
JP5956331B2 (ja) * 2009-06-16 2016-07-27 ジェンザイム・コーポレーション 組換えａａｖベクターの改良された精製方法

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