BRPI1015176A2 - métodos aperfeiçoados para purificação de vetores aav recombinantes - Google Patents

métodos aperfeiçoados para purificação de vetores aav recombinantes

Info

Publication number
BRPI1015176A2
BRPI1015176A2 BRPI1015176A BRPI1015176A BRPI1015176A2 BR PI1015176 A2 BRPI1015176 A2 BR PI1015176A2 BR PI1015176 A BRPI1015176 A BR PI1015176A BR PI1015176 A BRPI1015176 A BR PI1015176A BR PI1015176 A2 BRPI1015176 A2 BR PI1015176A2
Authority
BR
Brazil
Prior art keywords
purification
improved methods
aav vectors
recombinant aav
recombinant
Prior art date
Application number
BRPI1015176A
Other languages
English (en)
Inventor
A Thorne Barbara
Nichols Gina
Mclean Quigley Sheldon Paulene
S Gagnon Peter
Original Assignee
Genzyme Corp
Priority date (The priority date 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 date listed.)
Filing date
Publication date
Application filed by Genzyme Corp filed Critical Genzyme Corp
Publication of BRPI1015176A2 publication Critical patent/BRPI1015176A2/pt
Publication of BRPI1015176B1 publication Critical patent/BRPI1015176B1/pt

Links

Classifications

    • 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
    • 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
    • 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

Landscapes

  • 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)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Biotechnology (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)
  • General Physics & Mathematics (AREA)
  • Analytical Chemistry (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)
BRPI1015176-1A 2009-06-16 2010-06-16 método para isolar uma população de partículas de vírus adenoassociado recombinante (raav) de impurezas de processo em uma corrente de alimentação BRPI1015176B1 (pt)

Applications Claiming Priority (2)

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

Publications (2)

Publication Number Publication Date
BRPI1015176A2 true BRPI1015176A2 (pt) 2017-09-26
BRPI1015176B1 BRPI1015176B1 (pt) 2019-10-29

Family

ID=43356746

Family Applications (1)

Application Number Title Priority Date Filing Date
BRPI1015176-1A BRPI1015176B1 (pt) 2009-06-16 2010-06-16 método para isolar uma população de partículas de vírus adenoassociado recombinante (raav) de impurezas de processo em uma corrente de alimentação

Country Status (24)

Country Link
US (4) US10017746B2 (pt)
EP (4) EP3067417B1 (pt)
JP (3) JP5956331B2 (pt)
KR (2) KR101812813B1 (pt)
CN (2) CN102803478B (pt)
BR (1) BRPI1015176B1 (pt)
CA (2) CA3077531C (pt)
CY (3) CY1117936T1 (pt)
DK (2) DK3444335T3 (pt)
ES (3) ES2693194T3 (pt)
HR (2) HRP20181743T1 (pt)
HU (3) HUE040636T2 (pt)
IL (3) IL216960A (pt)
LT (2) LT3067417T (pt)
MX (3) MX2011013613A (pt)
PL (2) PL2443233T3 (pt)
PT (3) PT3067417T (pt)
RS (2) RS62086B1 (pt)
RU (1) RU2016119739A (pt)
SG (2) SG10201403290SA (pt)
SI (2) SI3444335T1 (pt)
SM (1) SMT201800554T1 (pt)
TR (1) TR201815937T4 (pt)
WO (1) WO2010148143A1 (pt)

Families Citing this family (118)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN101528916B (zh) 2006-04-28 2013-09-04 宾夕法尼亚大学托管会 规模可调的aav生产方法
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
CA2787009C (en) 2010-01-15 2018-04-03 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
JP5881744B2 (ja) 2011-02-02 2016-03-09 バイオ−ラッド ラボラトリーズ インコーポレーティッド アルカリ溶液でのアパタイトの表面中和方法
HRP20180028T4 (hr) 2011-03-16 2020-10-02 F. Hoffmann - La Roche Ag Kromatografija izmjene iona s poboljšanom selektivnošću za odvajanje polipeptidnih monomera, agregata i fragmenata modulacijom pokretne faze
EP3219313B1 (en) * 2011-04-18 2020-10-28 National Center of Neurology and Psychiatry Drug delivery particle and method for producing the same
JP6198278B2 (ja) * 2011-09-08 2017-09-20 ユニキュアー アイピー ビー.ブイ. Aav調製物からの混入ウイルスの除去
US9815695B2 (en) 2012-05-30 2017-11-14 Bio-Rad Laboratories, Inc. In situ restoration of apatite-based chromatography resins
JP6200949B2 (ja) * 2012-05-31 2017-09-20 エイジェンシー フォー サイエンス,テクノロジー アンド リサーチ 固相ウレイドに対する生物学的標的物の選択的結合
GB2525562B (en) * 2013-02-15 2017-10-11 T Gjerde Douglas Methods for purification of biological cells
US11332719B2 (en) 2013-03-15 2022-05-17 The Broad Institute, Inc. Recombinant virus and preparations thereof
PT2970920T (pt) * 2013-03-15 2018-07-30 Childrens Hospital Philadelphia Processo de fabrico escalável para produzir vetores lentivirais recombinantes em sistema de cultura de células de suspensão livres de soro
US10640771B2 (en) 2013-04-17 2020-05-05 Genzyme Corporation Compositions and methods for treating and preventing macular degeneration
KR102069561B1 (ko) 2013-07-11 2020-01-23 다카라 바이오 가부시키가이샤 외피 비보유 바이러스의 제조 방법
KR102234695B1 (ko) 2014-02-06 2021-04-02 젠자임 코포레이션 황반 변성을 치료하고 예방하기 위한 조성물 및 방법
LT3137497T (lt) 2014-05-02 2021-07-26 Genzyme Corporation Aav vektoriai, skirti tinklainės ir cns genų terapijai
US10577627B2 (en) 2014-06-09 2020-03-03 Voyager Therapeutics, Inc. Chimeric capsids
EP3157863B1 (en) 2014-06-23 2023-06-21 Bio-Rad Laboratories, Inc. Apatite in-situ restoration
CN105392569B (zh) 2014-06-23 2019-08-20 生物辐射实验室股份有限公司 磷灰石预处理
US10023846B2 (en) 2014-07-10 2018-07-17 Takara Bio Inc. Production method for non-enveloped virus particles
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
AR102198A1 (es) 2014-10-09 2017-02-08 Regeneron Pharma Proceso para reducir partículas subvisibles en una formulación farmacéutica
EP3215191B1 (en) 2014-11-05 2024-08-14 Voyager Therapeutics, Inc. Aadc polynucleotides for the treatment of parkinson's disease
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
KR20230145206A (ko) 2014-11-14 2023-10-17 보이저 테라퓨틱스, 인크. 조절성 폴리뉴클레오티드
US11697825B2 (en) 2014-12-12 2023-07-11 Voyager Therapeutics, Inc. Compositions and methods for the production of scAAV
CN107109374B (zh) 2015-01-09 2021-04-06 宝生物工程株式会社 用于生产无包膜病毒颗粒的方法
IL310375B2 (en) 2015-01-20 2025-05-01 Genzyme Corp Analytical ultracentrifugation for characterization of recombinant viral particles
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
EA201791803A1 (ru) 2015-02-10 2017-11-30 Джензим Корпорейшн Улучшенная доставка вирусных частиц в полосатое тело и кору больших полушарий
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
PL3298134T3 (pl) 2015-05-16 2023-09-18 Genzyme Corporation Edycja genów mutacji głęboko intronowych
US9663766B2 (en) 2015-07-24 2017-05-30 Bio-Rad Laboratories, Inc. Methods for purifying adenovirus vectors
WO2017100674A1 (en) 2015-12-11 2017-06-15 The Trustees Of The University Of Pennsylvania Scalable purification method for aav1
EP3387138B2 (en) 2015-12-11 2026-05-27 The Trustees Of The University Of Pennsylvania Scalable purification method for aav9
US11015174B2 (en) 2015-12-11 2021-05-25 The Trustees Of The University Of Pennsylvania Scalable purification method for AAV8
EP3992283A1 (en) 2015-12-11 2022-05-04 The Trustees Of The University Of Pennsylvania Scalable purification method for aavrh10
SG10201912935WA (en) 2015-12-15 2020-02-27 Genzyme Corp Adeno-associated viral vectors for treating mucolipidosis type ii
US11208630B2 (en) 2015-12-24 2021-12-28 University Of Florida Research Foundation, Incorporated AAV production using suspension adapted cells
ES2938833T3 (es) * 2016-03-28 2023-04-17 Ultragenyx Pharmaceutical Inc Métodos de inactivación térmica de adenovirus
PH12018502104B1 (en) * 2016-03-31 2024-03-27 Spark Therapeutics Inc Column-based fully scalable raav manufacturing process
EP3448987A4 (en) 2016-04-29 2020-05-27 Voyager Therapeutics, Inc. COMPOSITIONS FOR TREATING A DISEASE
EP3448874A4 (en) 2016-04-29 2020-04-22 Voyager Therapeutics, Inc. COMPOSITIONS FOR TREATING A 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
US10584337B2 (en) 2016-05-18 2020-03-10 Voyager Therapeutics, Inc. Modulatory polynucleotides
IL297576B2 (en) 2016-05-18 2024-02-01 Voyager Therapeutics Inc Compositions and methods of treating huntington's disease
GB201612248D0 (en) 2016-07-14 2016-08-31 Puridify Ltd New process
HUE053747T2 (hu) 2016-08-15 2021-07-28 Genzyme Corp Eljárások AAV kimutatására
AU2017312785B2 (en) 2016-08-16 2024-10-10 Regeneron Pharmaceuticals, Inc. Methods for quantitating individual antibodies from a mixture
WO2018039375A1 (en) 2016-08-23 2018-03-01 Akouos, Inc. Compositions and methods for treating non-age-associated hearing impairment in a human subject
EP3831281B1 (en) 2016-08-30 2026-02-25 The Regents of The University of California Devices and systems for biomedical targeting and delivery
ES3012649T3 (en) 2016-10-14 2025-04-09 Ultragenyx Pharmaceutical Inc Use of tonicifying agents to enhance recombinant adeno-associated virus yield
KR102759512B1 (ko) 2016-10-25 2025-02-03 리제너론 파아마슈티컬스, 인크. 크로마토그래피 데이터 분석을 위한 방법 및 시스템
PL3535388T3 (pl) * 2016-11-04 2023-08-14 Takeda Pharmaceutical Company Limited Sposoby oczyszczania wirusów związanych z adenowirusami
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
EP3601581B1 (en) 2017-03-22 2025-04-30 Ultragenyx Pharmaceutical Inc. CELL CULTURE METHODS INVOLVING HDAC INHIBITORS OR REP PROTEINS
CN117821509A (zh) 2017-04-03 2024-04-05 编码治疗公司 组织选择性转基因表达
MX2019013172A (es) 2017-05-05 2020-09-07 Voyager Therapeutics Inc Composiciones y metodos para tratar la enfermedad de huntington.
EP3618839A4 (en) 2017-05-05 2021-06-09 Voyager Therapeutics, Inc. COMPOSITIONS AND METHODS OF TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS)
EP3621734A4 (en) 2017-05-09 2020-12-30 Ultragenyx Pharmaceutical Inc. MODULAR PROCESS FOR THE PRODUCTION OF TRANSFECTION REAGENTS
JOP20190269A1 (ar) 2017-06-15 2019-11-20 Voyager Therapeutics Inc بولي نوكليوتيدات aadc لعلاج مرض باركنسون
EP3658250A4 (en) * 2017-06-30 2021-10-27 Spark Therapeutics, Inc. AAV VECTOR COLUMN CLEANING PROCEDURE
CA3070087A1 (en) 2017-07-17 2019-01-24 Voyager Therapeutics, Inc. Trajectory array guide system
US11512327B2 (en) 2017-08-03 2022-11-29 Voyager Therapeutics, Inc. Compositions and methods for delivery of AAV
WO2019060062A1 (en) 2017-09-19 2019-03-28 Regeneron Pharmaceuticals Inc. METHODS OF REDUCING PARTICLE FORMATION AND COMPOSITIONS FORMED THEREFROM
MX2020003042A (es) 2017-09-29 2020-11-18 Voyager Therapeutics Inc Rescate del fenotipo neurológico central y periférico de la ataxia de friedreich mediante administración intravenosa.
TW202413649A (zh) 2017-10-16 2024-04-01 美商航海家醫療公司 肌萎縮性脊髓側索硬化症(als)之治療
WO2019079242A1 (en) 2017-10-16 2019-04-25 Voyager Therapeutics, Inc. TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS)
CN118345115A (zh) 2017-12-01 2024-07-16 编码治疗公司 工程化的dna结合蛋白
IL275533B2 (en) 2017-12-29 2025-03-01 Baxalta Inc Adeno-associated virus purification methods
WO2019152841A1 (en) 2018-02-01 2019-08-08 Homology Medicines, Inc. Adeno-associated virus compositions for pah gene transfer and methods of use thereof
AU2019215152A1 (en) 2018-02-01 2020-08-20 Homology Medicines, Inc. Adeno-associated virus compositions for restoring PAH gene function 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
IL325122A (en) 2018-02-22 2026-02-01 Akouos Inc Compositions and methods for treating non-age-related hearing impairment in humans
CA3098565A1 (en) * 2018-04-29 2019-11-07 Claire G. ZHANG Scalable clarification process for recombinant aav production
CN112424359A (zh) 2018-05-15 2021-02-26 沃雅戈治疗公司 用于治疗帕金森氏病的组合物和方法
TWI919355B (zh) 2018-07-02 2026-03-21 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
WO2020061581A1 (en) * 2018-09-21 2020-03-26 Nightstarx Limited Compositions and methods for manufacturing gene therapy vectors
US12281321B2 (en) 2018-09-28 2025-04-22 Voyager Therapeutics, Inc. Frataxin expression constructs having engineered promoters and methods of use thereof
CN120349985A (zh) 2018-10-12 2025-07-22 建新公司 通过肝导向基因替代疗法治疗严重pku的改良的人pah的产生
CA3118936A1 (en) 2018-11-16 2020-05-22 Encoded Therapeutics, Inc. Compositions and methods for treating wilson's disease
CA3123841A1 (en) 2018-12-18 2020-06-25 Ultragenyx Pharmaceutical Inc. Methods and compositions for treating glycogen storage diseases
EA202192422A1 (ru) 2019-03-05 2021-12-09 Ридженерон Фармасьютикалз, Инк. Человеческий сывороточный альбумин в составах
CN114127088B (zh) 2019-04-23 2024-05-14 国家医疗保健研究所 变体衣壳蛋白和腺相关病毒aav变体及其药物组合物
SG11202113048SA (en) 2019-05-29 2021-12-30 Encoded Therapeutics Inc Compositions and methods for selective gene regulation
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
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
US10653731B1 (en) 2019-07-15 2020-05-19 Vigene Biosciences Inc. Recombinantly-modified adeno-associated virus (rAAV) having improved packaging efficiency
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
WO2021155137A1 (en) 2020-01-29 2021-08-05 Genzyme Corporation Modified adeno-associated viral capsid proteins for ocular gene therapy and methods of use thereof
CA3168055A1 (en) 2020-02-21 2021-08-26 Emmanuel John Simons Compositions and methods for treating non-age-associated hearing impairment in a human subject
TW202208632A (zh) 2020-05-27 2022-03-01 美商同源醫藥公司 用於恢復pah基因功能的腺相關病毒組成物及其使用方法
EP3919613B1 (en) * 2020-06-05 2024-08-07 Sartorius BIA Separations d.o.o. Enhanced purification of adeno-associated virus to more effectively remove contaminating dna
JP6898535B1 (ja) * 2020-06-16 2021-07-07 HOYA Technosurgical株式会社 アパタイトカラムを使用したウイルスの精製方法
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
KR20230079172A (ko) 2020-10-01 2023-06-05 젠자임 코포레이션 간-유도 유전자 대체 요법에 의한 pku의 치료를 위한 인간 pah 발현 카세트
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
CN117460832A (zh) 2021-03-22 2024-01-26 建新公司 空aav衣壳和完整aav衣壳的尺寸排阻色谱分析
JP2023098806A (ja) * 2021-12-29 2023-07-11 東ソー株式会社 アデノ随伴ウイルスの精製方法
CN119630802A (zh) 2022-04-12 2025-03-14 建新公司 Irak4调节剂用于基因疗法的用途
US20230407255A1 (en) 2022-04-12 2023-12-21 Genzyme Corporation Dendritic cell assay for innate immunogenicity to gene therapy agents
JP2025513851A (ja) 2022-04-12 2025-04-30 ジェンザイム・コーポレーション 遺伝子治療のためのirak4モジュレーターの使用
EP4507719A1 (en) 2022-04-13 2025-02-19 Universitat Autònoma de Barcelona Treatment of neuromuscular diseases via gene therapy that expresses klotho protein
CN119213120A (zh) 2022-05-16 2024-12-27 建新公司 治疗异染性脑白质营养不良的方法
CA3252767A1 (en) 2022-06-07 2023-12-14 Adverum Biotechnologies, Inc. VARIANTS OF MELANOPSINE FOR VISION RESTORATION
AR134407A1 (es) 2023-11-15 2026-01-14 Genzyme Corp Métodos de tratamiento de la enfermedad de gaucher y de la enfermedad de parkinson gba
CN122206466A (zh) 2023-11-15 2026-06-12 建新公司 治疗神经变性障碍的方法
WO2025158385A1 (en) 2024-01-25 2025-07-31 Genzyme Corporation Pegylated il-2 for suppressing adaptive immune response to gene therapy
WO2025160452A1 (en) 2024-01-25 2025-07-31 Genzyme Corporation Methods for fus-based delivery of viral particles to the brain
WO2025235643A1 (en) 2024-05-08 2025-11-13 Genzyme Corporation Profiling of gene therapy agents
WO2026142957A1 (en) 2024-12-24 2026-07-02 Genzyme Corporation Aav genomes encoding engineered human frataxin transgene
WO2026165267A1 (en) 2025-01-31 2026-08-06 Genzyme Corporation Methods of treating neurodegenerative disorders

Family Cites Families (11)

* 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
US6566118B1 (en) 1997-09-05 2003-05-20 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
CA2995542A1 (en) 1997-09-05 1999-03-11 Genzyme Corporation Methods for generating high titer helper-free preparations of recombinant aav vectors
US6995006B2 (en) 1997-09-05 2006-02-07 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV 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
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
US7427659B2 (en) * 2003-10-24 2008-09-23 Amgen Inc. Process for purifying proteins in a hydrophobic interaction chromatography flow-through fraction
ES2349112T3 (es) * 2005-01-14 2010-12-28 Bayer Healthcare Llc Método de purificación de factor vii.
CA2683977C (en) * 2007-03-14 2017-04-25 Ligocyte Pharmaceuticals, Inc. A method of norovirus virus-like particle purification comprising ion exchange chromatography
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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