PT2529020T - Plataforma de fabrico escalável para purificação de vetor viral e vetores virais assim purificados para utilização na terapia génica - Google Patents

Plataforma de fabrico escalável para purificação de vetor viral e vetores virais assim purificados para utilização na terapia génica

Info

Publication number
PT2529020T
PT2529020T PT117375089T PT11737508T PT2529020T PT 2529020 T PT2529020 T PT 2529020T PT 117375089 T PT117375089 T PT 117375089T PT 11737508 T PT11737508 T PT 11737508T PT 2529020 T PT2529020 T PT 2529020T
Authority
PT
Portugal
Prior art keywords
purified
gene therapy
viral
manufacturing platform
scalable manufacturing
Prior art date
Application number
PT117375089T
Other languages
English (en)
Portuguese (pt)
Inventor
Fraser Wright John
Qu Guang
Hauck Bernd
High Katherine
Original Assignee
Childrens Hospital Philadelphia
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 Childrens Hospital Philadelphia filed Critical Childrens Hospital Philadelphia
Publication of PT2529020T publication Critical patent/PT2529020T/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
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00—Medicinal preparations containing antigens or antibodies
    • A61K39/12—Viral antigens
    • A61K39/23—Parvoviridae, e.g. feline panleukopenia virus
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
    • A61K38/465—Hydrolases (3) acting on ester bonds (3.1), e.g. lipases, ribonucleases
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0091—Purification or manufacturing processes for gene therapy compositions
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • A61P7/04—Antihaemorrhagics; Procoagulants; Haemostatic agents; Antifibrinolytic agents
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2/00—Peptides of undefined number of amino acids; Derivatives 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
    • 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
    • C12N15/64—General methods for preparing the vector, for introducing it into the cell or for selecting the vector-containing host
    • 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
    • C12N15/864—Parvoviral vectors, e.g. parvovirus, densovirus
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • 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
    • C12N15/864—Parvoviral vectors, e.g. parvovirus, densovirus
    • C12N15/8645—Adeno-associated virus
    • 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
    • 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
    • C12N2750/14051—Methods of production or purification of viral material
    • 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
    • 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
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y301/00—Hydrolases acting on ester bonds (3.1)
    • C12Y301/01—Carboxylic ester hydrolases (3.1.1)
    • C12Y301/01064—Retinoid isomerohydrolase (3.1.1.64)

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biotechnology (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Medicinal Chemistry (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Virology (AREA)
  • Biophysics (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Epidemiology (AREA)
  • Immunology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Manufacturing & Machinery (AREA)
  • Cell Biology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Mycology (AREA)
  • Diabetes (AREA)
  • Hematology (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)
PT117375089T 2010-01-28 2011-01-25 Plataforma de fabrico escalável para purificação de vetor viral e vetores virais assim purificados para utilização na terapia génica PT2529020T (pt)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US29918410P 2010-01-28 2010-01-28

Publications (1)

Publication Number Publication Date
PT2529020T true PT2529020T (pt) 2018-07-30

Family

ID=44319714

Family Applications (1)

Application Number Title Priority Date Filing Date
PT117375089T PT2529020T (pt) 2010-01-28 2011-01-25 Plataforma de fabrico escalável para purificação de vetor viral e vetores virais assim purificados para utilização na terapia génica

Country Status (13)

Country Link
US (4) US9408904B2 (enExample)
EP (1) EP2529020B1 (enExample)
JP (4) JP2013517798A (enExample)
CN (2) CN102947453A (enExample)
AU (1) AU2011209743B2 (enExample)
CA (1) CA2787827C (enExample)
DK (1) DK2529020T3 (enExample)
ES (1) ES2680915T3 (enExample)
IL (1) IL221158B (enExample)
IN (1) IN2012DN06629A (enExample)
MX (1) MX340102B (enExample)
PT (1) PT2529020T (enExample)
WO (1) WO2011094198A1 (enExample)

Families Citing this family (124)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US9217155B2 (en) 2008-05-28 2015-12-22 University Of Massachusetts Isolation of novel AAV'S and uses thereof
WO2010138263A2 (en) 2009-05-28 2010-12-02 University Of Massachusetts Novel aav 's and uses thereof
ES2680915T3 (es) * 2010-01-28 2018-09-11 The Children's Hospital Of Philadelphia Plataforma de fabricación escalable para la purificación de vectores virales y vectores virales purificados de este modo para su utilización en terapia génica
CA2833905C (en) 2010-04-23 2019-09-10 University Of Massachusetts Multicistronic expression constructs
EP3567106A1 (en) 2010-04-23 2019-11-13 University of Massachusetts Cns targeting aav vectors and methods of use thereof
AU2012304993B2 (en) * 2011-09-08 2017-09-14 Uniqure Ip B.V. Removal of contaminating viruses from AAV preparations
EP4282419A1 (en) 2012-12-20 2023-11-29 Purdue Research Foundation Chimeric antigen receptor-expressing t cells as anti-cancer therapeutics
FR3002237B1 (fr) * 2013-02-15 2017-12-15 Genethon Methodes pour la production de particules virales aav double brin
US9644216B2 (en) 2013-11-26 2017-05-09 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Adeno-associated virus vectors for treatment of glycogen storage disease
GB201401707D0 (en) 2014-01-31 2014-03-19 Sec Dep For Health The Adeno-associated viral vectors
US10072251B2 (en) 2014-02-19 2018-09-11 University Of Massachusetts Recombinant AAVS having useful transcytosis properties
CA3276269A1 (en) 2014-03-18 2026-03-02 University Of Massachusetts Raav-based compositions and methods for treating amyotrophic lateral sclerosis
DK3628334T5 (da) 2014-03-21 2024-09-02 Genzyme Corp Genterapi til behandling af retinitis pigmentosa
US10975391B2 (en) 2014-04-25 2021-04-13 University Of Massachusetts Recombinant AAV vectors useful for reducing immunity against transgene products
CA2946392A1 (en) 2014-04-25 2015-10-29 James M. Wilson Ldlr variants and their use in compositions for reducing cholesterol levels
WO2015187825A2 (en) 2014-06-03 2015-12-10 University Of Massachusetts Compositions and methods for modulating dysferlin expression
EP3200830B1 (en) 2014-10-03 2020-09-09 University of Massachusetts High efficiency library-identified aav vectors
US10370432B2 (en) 2014-10-03 2019-08-06 University Of Massachusetts Heterologous targeting peptide grafted AAVS
RU2020140209A (ru) 2014-10-21 2021-01-25 Юниверсити Оф Массачусетс Варианты рекомбинантных aav и их применения
BR112017011217A2 (pt) * 2014-11-28 2018-02-06 Uniqure Ip B.V. impurezas de dna em uma composição compreendendo um vírion parvoviral
ES2824829T3 (es) 2014-12-23 2021-05-13 Us Health Vectores de virus adenoasociado que codifican G6PC modificada y usos de estos
CN107428799A (zh) 2015-01-13 2017-12-01 阿尔法韦士曼公司 纯化腺相关病毒(AAV)及/或重组腺相关病毒(rAAV)之方法及其梯度和流通式缓冲液
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
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
WO2016131009A1 (en) 2015-02-13 2016-08-18 University Of Massachusetts Compositions and methods for transient delivery of nucleases
EP3262162A4 (en) * 2015-02-23 2018-08-08 Voyager Therapeutics, Inc. Regulatable expression using adeno-associated virus (aav)
EP3283126B1 (en) 2015-04-16 2019-11-06 Emory University Recombinant promoters and vectors for protein expression in liver and use thereof
CA2983808C (en) 2015-04-23 2022-03-22 Washington State University Smad7 gene delivery as a therapeutic
US11046955B2 (en) 2015-04-24 2021-06-29 University Of Massachusetts Modified AAV constructs and uses thereof
EP3364996B1 (en) 2015-10-22 2021-08-25 University of Massachusetts Prostate-targeting adeno-associated virus serotype vectors
WO2017070525A1 (en) 2015-10-22 2017-04-27 University Of Massachusetts Methods and compositions for treating metabolic imbalance in neurodegenerative disease
CN105420275A (zh) * 2015-11-27 2016-03-23 中国科学院苏州生物医学工程技术研究所 制备外源功能基因定点整合的人神经干细胞的方法
WO2017100676A1 (en) * 2015-12-11 2017-06-15 The Trustees Of The University Of Pennsylvania Scalable purification method for aav8
US11098286B2 (en) 2015-12-11 2021-08-24 The Trustees Of The University Of Pennsylvania Scalable purification method for AAV9
US11015173B2 (en) 2015-12-11 2021-05-25 The Trustees Of The University Of Pennsylvania Scalable purification method for AAV1
CA3008142A1 (en) * 2015-12-11 2017-06-15 The Trustees Of The University Of Pennsylvania Gene therapy for treating familial hypercholesterolemia
ES2918998T3 (es) 2015-12-11 2022-07-21 Univ Pennsylvania Método de purificación escalable para AAVrh10
ES2983043T3 (es) 2015-12-18 2024-10-21 Sangamo Therapeutics Inc Alteración dirigida del receptor de células T
AU2016374253B2 (en) 2015-12-18 2021-10-21 Sangamo Therapeutics, Inc. Targeted disruption of the MHC cell receptor
CN109069568B (zh) 2016-02-02 2023-07-07 桑格摩生物治疗股份有限公司 用于连接dna结合结构域和切割结构域的组合物
WO2017136536A1 (en) 2016-02-02 2017-08-10 University Of Massachusetts Method to enhance the efficiency of systemic aav gene delivery to the central nervous system
US12070510B2 (en) 2016-02-05 2024-08-27 Emory University Injection of single-stranded or self-complementary adeno-associated virus 9 into the cerebrospinal fluid
WO2017139643A1 (en) 2016-02-12 2017-08-17 University Of Massachusetts Anti-angiogenic mirna therapeutics for inhibiting corneal neovascularization
ES2896080T3 (es) * 2016-03-31 2022-02-23 Spark Therapeutics Inc Procedimiento en columna totalmente escalable de preparación de VAAr
WO2017176929A1 (en) 2016-04-05 2017-10-12 University Of Massachusetts Compositions and methods for selective inhibition of grainyhead-like protein expression
US12144850B2 (en) 2016-04-08 2024-11-19 Purdue Research Foundation Methods and compositions for car T cell therapy
WO2017181105A1 (en) 2016-04-15 2017-10-19 University Of Massachusetts Methods and compositions for treating metabolic imbalance
US11882815B2 (en) 2016-06-15 2024-01-30 University Of Massachusetts Recombinant adeno-associated viruses for delivering gene editing molecules to embryonic cells
GB201612248D0 (en) 2016-07-14 2016-08-31 Puridify Ltd New process
KR20190045174A (ko) * 2016-07-21 2019-05-02 스파크 테라퓨틱스, 인코포레이티드 재조합 아데노신 관련 바이러스(rAAV) 벡터를 고수율로 생산하기 위한 확장 가능한 고회수 방법 및 이에 의해 생산된 재조합 아데노-관련 바이러스(rAAV) 벡터
US11883470B2 (en) 2016-07-25 2024-01-30 The Trustees Of The University Of Pennsylvania Compositions comprising a lecithin cholesterol acyltransferase variant and uses thereof
EP3510161A4 (en) 2016-08-23 2020-04-22 Akouos, Inc. COMPOSITIONS AND METHODS FOR TREATING PERSONAL HEARING LOSS IN A PERSON
IL300783B2 (en) 2016-08-24 2026-03-01 Sangamo Therapeutics Inc Engineered nucleases with target-specific zinc fingers
BR112019003100A2 (pt) 2016-08-24 2019-07-09 Sangamo Therapeutics, Inc. regulação da expressão gênica usando nucleases manipuladas
US10457940B2 (en) 2016-09-22 2019-10-29 University Of Massachusetts AAV treatment of Huntington's disease
CA3040483A1 (en) 2016-10-13 2018-04-19 University Of Massachusetts Aav capsid designs
CN110168081B (zh) * 2016-11-04 2024-08-16 武田药品工业株式会社 腺相关病毒纯化方法
EP3321357A1 (en) 2016-11-09 2018-05-16 Deutsches Krebsforschungszentrum Scalable process for oncolytic rat parvovirus h-1 production and purification based on isoelectric point-based elimination of empty particles
JP2020506695A (ja) 2017-01-30 2020-03-05 ザ ユナイテッド ステイツ オブ アメリカ， アズ リプレゼンテッド バイ ザ セクレタリー， デパートメント オブ ヘルス アンド ヒューマン サービシーズ 糖原病を処置するための組換えウイルスベクター
AU2018219226B2 (en) 2017-02-07 2024-12-19 Seattle Children's Hospital (dba Seattle Children's Research Institute) Phospholipid ether (PLE) CAR T cell tumor targeting (CTCT) agents
KR20190118163A (ko) 2017-02-20 2019-10-17 더 트러스티스 오브 더 유니버시티 오브 펜실바니아 가족성 고콜레스테롤혈증을 치료하기 위한 유전자 요법
ES3010559T3 (en) 2017-02-28 2025-04-03 Endocyte Inc Compositions and methods for car t cell therapy
EP3622065A1 (en) 2017-05-09 2020-03-18 Emory University Clotting factor variants and their use
EP3622073A4 (en) 2017-05-09 2021-01-06 University of Massachusetts METHOD FOR TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS)
KR20250030036A (ko) 2017-06-07 2025-03-05 스파크 테라퓨틱스, 인코포레이티드 개선된 세포 트랜스펙션 및/또는 rAAV 벡터 생산을 위한 증진제
SG11201913157RA (en) * 2017-06-30 2020-01-30 Spark Therapeutics Inc Aav vector column purification methods
CA3071683A1 (en) 2017-08-08 2019-02-14 Sangamo Therapeutics, Inc. Chimeric antigen receptor mediated cell targeting
US11739330B2 (en) 2017-09-22 2023-08-29 University Of Massachusetts SOD1 dual expression vectors and uses thereof
IL321310A (en) 2017-10-20 2025-08-01 Res Inst Nationwide Childrens Hospital Methods and materials for NT-3 gene therapy
CN120505370A (zh) * 2017-11-08 2025-08-19 诺华股份有限公司 制备病毒载体的手段和方法及其用途
US11311576B2 (en) 2018-01-22 2022-04-26 Seattle Children's Hospital Methods of use for CAR T cells
US10610606B2 (en) 2018-02-01 2020-04-07 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
WO2019156795A1 (en) 2018-02-06 2019-08-15 Seattle Children's Hospital (dba Seattle Children's Research Institute) Fluorescein-specific cars exhibiting optimal t cell function against fl-ple labelled tumors
JP7244547B2 (ja) 2018-02-19 2023-03-22 ホモロジー・メディシンズ・インコーポレイテッド Ｆ８遺伝子機能を回復させるためのアデノ随伴ウイルス組成物及びその使用の方法
CA3091674A1 (en) 2018-02-23 2019-08-29 Endocyte, Inc. Sequencing method for car t cell therapy
WO2019169291A1 (en) 2018-03-02 2019-09-06 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Use of il-34 to treat retinal inflammation and neurodegeneration
MA52200A (fr) * 2018-04-05 2021-02-17 Nightstarx Ltd Compositions de vaa, procédés de préparation et méthodes d'utilisation
US12163129B2 (en) 2018-06-08 2024-12-10 University Of Massachusetts Antisense oligonucleotides to restore dysferlin protein expression in dysferlinopathy patient cells
US12228571B2 (en) 2018-06-08 2025-02-18 Novartis Ag Cell-based assay for measuring drug product potency
CN113528510A (zh) 2018-06-29 2021-10-22 武汉纽福斯生物科技有限公司 治疗遗传性视神经病变的组合物和方法
CA3109432A1 (en) 2018-08-20 2020-02-27 Wuhan Neurophth Biotechnology Limited Company Compositions and methods for treating leber's hereditary optic neuropathy
IL281615B2 (en) 2018-09-21 2026-01-01 Acuitas Therapeutics Inc Systems and methods for producing lipid nanoparticles and liposomes
US12583816B2 (en) 2018-10-18 2026-03-24 Acuitas Therapeutics, Inc. Lipids for lipid nanoparticle delivery of active agents
AU2019389047A1 (en) * 2018-11-30 2021-05-20 Novartis Ag AAV viral vectors and uses thereof
DK3898981T5 (da) 2018-12-18 2024-08-05 Ultragenyx Pharmaceutical Inc Fremgangsmåder og sammensætninger til behandling af glykogenlagringssygdomme
SG11202106855YA (en) 2019-01-04 2021-07-29 Ultragenyx Pharmaceutical Inc Gene therapy constructs for treating wilson disease
JP7523449B2 (ja) 2019-01-11 2024-07-26 アクイタス セラピューティクス インコーポレイテッド 活性剤の脂質ナノ粒子送達のための脂質
MX2021008542A (es) * 2019-01-18 2021-11-12 Voyager Therapeutics Inc Métodos y sistemas para la producción de partículas de aav.
MA56393A (fr) 2019-06-28 2022-05-04 Bayer Healthcare Llc Matériels et méthodes de régulation de l'édition de gènes
AU2020319168B2 (en) 2019-07-25 2024-02-29 Novartis Ag Regulatable expression systems
WO2021062012A1 (en) 2019-09-25 2021-04-01 Emory University Use of klk10 and engineered derivatizations thereof
CN113025633B (zh) 2019-12-09 2024-08-27 武汉纽福斯生物科技有限公司 编码人nadh脱氢酶亚单位1蛋白的核酸及其应用
WO2021124152A1 (en) 2019-12-19 2021-06-24 Pfizer Inc. Compositions and methods for nucleic acid transfection using cationic polymers and stabilizers
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
EP4090381A1 (en) 2020-01-17 2022-11-23 The United States of America, as represented by the Secretary, Department of Health and Human Services <smallcaps/>? ? ?crx-? ? ? ? ?gene therapy for treatment ofautosomal dominant retinopathies
KR20220163940A (ko) 2020-02-04 2022-12-12 시애틀 칠드런즈 호스피탈 디/비/에이 시애틀 칠드런즈 리서치 인스티튜트 항-디니트로페놀 키메라 항원 수용체
BR112022015921A2 (pt) 2020-02-14 2022-10-04 Ultragenyx Pharmaceutical Inc Terapia gênica para tratar o transtorno de deficiência de cdkl5
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
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
JP7798855B2 (ja) 2020-07-16 2026-01-14 アクイタス セラピューティクス インコーポレイテッド 脂質ナノ粒子に使用するためのカチオン性脂質
JP7526360B2 (ja) * 2020-08-18 2024-07-31 ザルトリウス ビーアイエー セパレーションズ ディー．オー．オー． マルチモーダル金属アフィニティー処理ａａｖカプシド
US20230383278A1 (en) 2020-09-18 2023-11-30 The United States Of America,As Represented By The Secretary,Department Of Health And Human Services Novel adeno-associated viral (aav) vectors to treat hereditary methylmalonic acidemia (mma) caused by methylmalonyl-coa mutase (mmut) deficiency
EP4229204A1 (en) 2020-10-15 2023-08-23 F. Hoffmann-La Roche AG Nucleic acid constructs for simultaneous gene activation
KR20230085929A (ko) 2020-10-15 2023-06-14 에프. 호프만-라 로슈 아게 Va rna 전사를 위한 핵산 구조체
US20240398985A1 (en) 2021-09-16 2024-12-05 Novartis Ag Novel transcription factors
CA3242402A1 (en) 2021-12-16 2023-06-22 Acuitas Therapeutics, Inc. Lipids for use in lipid nanoparticle formulations
WO2023196898A1 (en) 2022-04-07 2023-10-12 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Beta globin mimetic peptides and their use
WO2023198685A1 (en) 2022-04-13 2023-10-19 F. Hoffmann-La Roche Ag Method for determining aav genomes
JP2025517628A (ja) 2022-05-06 2025-06-10 ノバルティス アーゲー 新規の組換えａａｖ ｖｐ２融合ポリペプチド
CN119256220A (zh) 2022-05-23 2025-01-03 豪夫迈·罗氏有限公司 用于区分aav颗粒血清型和aav颗粒荷载状态的基于拉曼的方法
EP4532737A1 (en) 2022-06-03 2025-04-09 F. Hoffmann-La Roche AG Method for producing recombinant aav particles
KR20250036743A (ko) 2022-07-14 2025-03-14 에프. 호프만-라 로슈 아게 재조합 aav 입자를 생산하는 방법
EP4744752A1 (en) 2022-09-12 2026-05-20 Hoffmann La Roche METHOD FOR SEPARING SOLID AND EMPTY AAV PARTICLES
IL322001A (en) 2023-02-07 2025-09-01 Hoffmann La Roche Method for detecting antibodies to anti-AAV particles
WO2024168358A1 (en) 2023-02-10 2024-08-15 Expression Therapeutics, Llc Lentiviral system
JP2026508221A (ja) 2023-02-22 2026-03-10 ザ ユナイテッド ステイツ オブ アメリカ， アズ リプレゼンテッド バイ ザ セクレタリー， デパートメント オブ ヘルス アンド ヒューマン サービシーズ 免疫優性ｔ細胞エピトープを欠く組換えアデノ随伴ウイルスベクターおよびその使用
EP4680243A1 (en) 2023-03-17 2026-01-21 The United States of America, as represented by the Secretary, Department of Health and Human Services Methods for treatment of age-related macular degeneration
EP4683676A1 (en) 2023-03-21 2026-01-28 F. Hoffmann-La Roche AG Method for the production of recombinant aav particle preparations
WO2025090942A1 (en) * 2023-10-26 2025-05-01 University Of Notre Dame Du Lac Gene therapy for treatment of neurometabolic disease
WO2025168663A1 (en) 2024-02-09 2025-08-14 F. Hoffmann-La Roche Ag Method for producing recombinant adeno-associated viral particles
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
WO2026037757A1 (en) 2024-08-13 2026-02-19 F. Hoffmann-La Roche Ag Modified aav particles

Family Cites Families (16)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6001650A (en) 1995-08-03 1999-12-14 Avigen, Inc. High-efficiency wild-type-free AAV helper functions
US6004797A (en) 1995-11-09 1999-12-21 Avigen, Inc. Adenovirus helper-free recombinant AAV Virion production
ES2355588T3 (es) * 1997-03-14 2011-03-29 The Children's Hospital Of Philadelphia Composiciones para uso en terapia génica para el tratamiento de hemofilia.
US6989264B2 (en) * 1997-09-05 2006-01-24 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
ATE454445T1 (de) * 1998-11-10 2010-01-15 Univ North Carolina Virusvektoren und verfahren für ihre herstellung und verabreichung.
MXPA01013388A (es) 1999-06-30 2002-07-02 Tulane University Medical Ct Retrovirus endogeno humano en cancer de pecho.
US20040102388A1 (en) * 2000-03-22 2004-05-27 High Katherine A. Modified blood clotting factors and methods of use
US6593123B1 (en) 2000-08-07 2003-07-15 Avigen, Inc. Large-scale recombinant adeno-associated virus (rAAV) production and purification
AU2003253595A1 (en) * 2002-04-05 2003-11-03 The Children's Hospital Of Philadelphia Methods for the production of chimeric adeno-associated virus (aav) vectors, compositions of chimeric aav vectors, and methods of use thereof
US7261544B2 (en) * 2003-05-21 2007-08-28 Genzyme Corporation Methods for producing preparations of recombinant AAV virions substantially free of empty capsids
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
CN1873012A (zh) * 2005-06-01 2006-12-06 上海二医新生基因科技有限公司 重组腺相关病毒（rAAV）载体大规模生产新工艺
US9198984B2 (en) * 2006-04-28 2015-12-01 The Trustees Of The University Of Pennsylvania Scalable production method for AAV
WO2008128251A1 (en) * 2007-04-17 2008-10-23 The Children's Hospital Of Philadelphia Humanized viral vectors and methods of use thereof
WO2009153563A1 (en) * 2008-06-18 2009-12-23 Oxford Biomedica (Uk) Limited Virus purification
ES2680915T3 (es) * 2010-01-28 2018-09-11 The Children's Hospital Of Philadelphia Plataforma de fabricación escalable para la purificación de vectores virales y vectores virales purificados de este modo para su utilización en terapia génica

Also Published As

Publication number Publication date
EP2529020B1 (en) 2018-04-25
US20190321463A1 (en) 2019-10-24
JP2016185153A (ja) 2016-10-27
AU2011209743B2 (en) 2016-03-10
ES2680915T3 (es) 2018-09-11
CN105838737A (zh) 2016-08-10
IL221158B (en) 2018-07-31
CA2787827A1 (en) 2011-08-04
US11878056B2 (en) 2024-01-23
WO2011094198A1 (en) 2011-08-04
US20200405845A1 (en) 2020-12-31
US10328145B2 (en) 2019-06-25
BR112012018899A2 (pt) 2015-09-15
CA2787827C (en) 2020-11-10
JP6991095B2 (ja) 2022-01-12
EP2529020A1 (en) 2012-12-05
CN102947453A (zh) 2013-02-27
US20160206706A1 (en) 2016-07-21
JP2013517798A (ja) 2013-05-20
IN2012DN06629A (enExample) 2015-10-23
US9408904B2 (en) 2016-08-09
JP2018121653A (ja) 2018-08-09
AU2011209743A1 (en) 2012-08-23
DK2529020T3 (en) 2018-08-06
US20130072548A1 (en) 2013-03-21
EP2529020A4 (en) 2013-10-16
MX2012008757A (es) 2012-11-06
JP2018046820A (ja) 2018-03-29
MX340102B (es) 2016-06-24

Similar Documents

Publication Publication Date Title
EP2529020A4 (en) ADVANCED MANUFACTURING PLATFORM FOR PURIFYING VIRAL VECTORS AND VIRAL VECTORS THUS PURIFIED FOR USE IN GENE THERAPY
ZA201309515B (en) Gene therapy vectors for adrenoleukodystrophy and adrenomyeloneuropathy
EP2486137A4 (en) REPLICATION OF VIRAL VECTORS FOR GENE THERAPY
HUE041799T2 (hu) Onkolitikus Herpes simplex vírus és terápiás alkalmazásai
GB2506989B (en) Multi-Purpose wheeled conveyance
IL231604B (en) Therapeutic peptides
PT3127542T (pt) Terapia antiviral
EP2615109A4 (en) LOCALIZED PEGYLATED AMENDMENTS OF EXENDIN-4 ANALOGUES AND THEIR USE
PL2521562T3 (pl) Ev576 do zastosowania w leczeniu zakażeń wirusowych dróg oddechowych
GB2485630C (en) Improvements in bicycle wheels
SMT201900219T1 (it) Anticorpi anti-s100a4 e relativi usi terapeutici
EP2605786A4 (en) THERAPEUTIC PEPTIDES
EP2711161A4 (en) NANOPRUCTIVE FORM AND CURVED BODY
EP2897564A4 (en) PORTABLE WHEELCHAIR WITH REAR STEERING
IL231876A0 (en) A system for the purification of viral vectors
PL2463117T3 (pl) Konstrukcja obręczy koła z materiału kompozytowego
TWM402806U (en) Caster wheel(I)
AU341452S (en) Alu&#39; sling sunlounger chair
GB201403897D0 (en) Golf trolley wheel and adapter