IN2012DN06629A - - Google Patents

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Publication number
IN2012DN06629A
IN2012DN06629A IN6629DEN2012A IN2012DN06629A IN 2012DN06629 A IN2012DN06629 A IN 2012DN06629A IN 6629DEN2012 A IN6629DEN2012 A IN 6629DEN2012A IN 2012DN06629 A IN2012DN06629 A IN 2012DN06629A
Authority
IN
India
Prior art keywords
formulations
aav
superior
methods
clinical use
Prior art date
Application number
Other languages
English (en)
Inventor
John Fraser Wright
Guang Qu
Bernd Hauck
Katherine High
Original Assignee
Philadelphia Children Hospital
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 Philadelphia Children Hospital filed Critical Philadelphia Children Hospital
Publication of IN2012DN06629A publication Critical patent/IN2012DN06629A/en

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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
    • 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)
  • General Health & Medical Sciences (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Medicinal Chemistry (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Virology (AREA)
  • Biophysics (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Epidemiology (AREA)
  • Immunology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Cell Biology (AREA)
  • Manufacturing & Machinery (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Mycology (AREA)
  • Hematology (AREA)
  • Diabetes (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)
IN6629DEN2012 2010-01-28 2011-01-25 IN2012DN06629A (fr)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US29918410P 2010-01-28 2010-01-28
PCT/US2011/022371 WO2011094198A1 (fr) 2010-01-28 2011-01-25 Plateforme de fabrication évolutive pour purification de vecteurs viraux et vecteurs viraux ainsi purifiés utilisables en thérapie génique

Publications (1)

Publication Number Publication Date
IN2012DN06629A true IN2012DN06629A (fr) 2015-10-23

Family

ID=44319714

Family Applications (1)

Application Number Title Priority Date Filing Date
IN6629DEN2012 IN2012DN06629A (fr) 2010-01-28 2011-01-25

Country Status (13)

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

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
US8734809B2 (en) 2009-05-28 2014-05-27 University Of Massachusetts AAV's and uses thereof
WO2011094198A1 (fr) * 2010-01-28 2011-08-04 The Children's Hospital Of Philadelphia Research Institute, Abramson Research Center Plateforme de fabrication évolutive pour purification de vecteurs viraux et vecteurs viraux ainsi purifiés utilisables en thérapie génique
EP2561073B1 (fr) 2010-04-23 2016-08-24 University of Massachusetts Vecteurs aav ciblant le système nerveux central et leurs procédés d'utilisation
US9546369B2 (en) 2010-04-23 2017-01-17 University Of Massachusetts Multicistronic expression constructs
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
EP4282419A1 (fr) 2012-12-20 2023-11-29 Purdue Research Foundation Cellules t exprimant un récepteur d'antigène chimérique et produits thérapeutiques anticancéreux
FR3002237B1 (fr) * 2013-02-15 2017-12-15 Genethon Methodes pour la production de particules virales aav double brin
AU2014354839B2 (en) 2013-11-26 2020-06-04 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
CA2942515C (fr) 2014-03-18 2025-12-09 University Of Massachusetts Compositions a base de virus adeno-associe recombinant raav et methodes de traitement de la sclerose laterale amyotrophique
PL3628334T3 (pl) * 2014-03-21 2023-12-18 Genzyme Corporation Terapia genowa w retinopatii barwnikowej
WO2015164786A1 (fr) 2014-04-25 2015-10-29 University Of Massachusetts Vecteurs de virus adéno-associés recombinants utiles pour réduire une immunité contre des produits transgéniques
EP3134431B1 (fr) 2014-04-25 2021-04-07 The Trustees Of The University Of Pennsylvania Variants de ldlr et leur utilisation dans des compositions permettant de réduire les taux de cholestérol
WO2015187825A2 (fr) 2014-06-03 2015-12-10 University Of Massachusetts Compositions et méthodes de modulation de l'expression de la dysferline
US10370432B2 (en) 2014-10-03 2019-08-06 University Of Massachusetts Heterologous targeting peptide grafted AAVS
EP3200830B1 (fr) 2014-10-03 2020-09-09 University of Massachusetts Vecteurs aav identifiés au moyen de banques à efficacité élevée
CN107073051B (zh) 2014-10-21 2021-08-24 马萨诸塞大学 重组aav变体及其用途
HUE046643T2 (hu) * 2014-11-28 2021-11-29 Uniqure Ip Bv DNS szennyezõdések parvovirális virionokat tartalmazó készítményben
WO2016106303A1 (fr) 2014-12-23 2016-06-30 THE UNITED STATES OF AMERICA, as representd by THE SECRETARY, DEPARTMENT OF HEALTH AND HUMAN SERVICES Vecteurs à base de virus adéno-associé codant pour une g6pc modifiée, et utilisations de ces derniers
US9862936B2 (en) 2015-01-13 2018-01-09 Alfa Wassermann, Inc. Methods of purifying adeno-associated virus (AAV) and/or recombinant adeno-associated virus (rAAV) and gradients and flow-through buffers therefore
EP3054007A1 (fr) 2015-02-09 2016-08-10 Institut National De La Sante Et De La Recherche Medicale (Inserm) Purification de particules de virus adéno-associés de recombinaison comprenant une étape de purification par affinité immunologique
EP3054006A1 (fr) 2015-02-09 2016-08-10 Institut National De La Sante Et De La Recherche Medicale (Inserm) Purification de particule de virus adéno-associé recombinant avec une chromatographie à échange d'anions en plusieurs étapes
EP3256170B1 (fr) 2015-02-13 2020-09-23 University of Massachusetts Compositions et procédés pour l'administration transitoire de nucléases
WO2016137949A1 (fr) * 2015-02-23 2016-09-01 Voyager Therapeutics, Inc. Expression régulable au moyen du virus adéno-associé (vaa)
EP3283126B1 (fr) 2015-04-16 2019-11-06 Emory University Promoteurs et vecteurs recombinés pour l'expression de protéines dans le foie et utilisation associée
ES2856936T3 (es) 2015-04-23 2021-09-28 Univ Washington State Suministro del gen Smad7 como un agente terapéutico
US11046955B2 (en) 2015-04-24 2021-06-29 University Of Massachusetts Modified AAV constructs and uses thereof
WO2017070516A1 (fr) 2015-10-22 2017-04-27 University Of Massachusetts Vecteurs du sérotype de virus adéno-associés ciblant la prostate
EP3364997B1 (fr) 2015-10-22 2024-01-17 University of Massachusetts Aspartoacylase en thérapie génique dans le traitement de la maladie de canavan
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
AU2016366560A1 (en) * 2015-12-11 2018-06-28 The Trustees Of The University Of Pennsylvania Gene therapy for treating familial hypercholesterolemia
EP3387138B1 (fr) 2015-12-11 2022-01-26 The Trustees Of The University Of Pennsylvania Méthode de purification évolutive pour virus adéno-associé 9 (aav9)
WO2017100674A1 (fr) 2015-12-11 2017-06-15 The Trustees Of The University Of Pennsylvania Procédé de purification évolutif pour virus adéno-associé (aav) 1
ES2934848T3 (es) * 2015-12-11 2023-02-27 Univ Pennsylvania Método de purificación escalable para AAV8
US11028372B2 (en) * 2015-12-11 2021-06-08 The Trustees Of The University Of Pennsylvania Scalable purification method for AAVRH10
EP3389677B1 (fr) 2015-12-18 2024-06-26 Sangamo Therapeutics, Inc. Disruption ciblée du récepteur des lymphocytes t
BR112018012235A2 (pt) 2015-12-18 2018-12-04 Sangamo Therapeutics Inc rompimento alvejado do receptor de células de mhc
CA3011939A1 (fr) 2016-02-02 2017-08-10 University Of Massachusetts Procede pour ameliorer l'efficacite de l'administration au systeme nerveux central d'un gene aav par voie systemique
KR20180101442A (ko) 2016-02-02 2018-09-12 상가모 테라퓨틱스, 인코포레이티드 Dna-결합 도메인 및 절단 도메인을 연결시키기 위한 조성물
EP3411484B1 (fr) 2016-02-05 2023-10-04 Emory University Injection de virus adéno-associé simple brin ou auto-complémentaire 9 dans le liquide cérébrospinal
CA3012344A1 (fr) 2016-02-12 2017-08-17 University Of Massachusetts Agents therapeutiques a base de micro-arn anti-angiogeniques pour l'inhibition de la neovascularisation corneenne
LT3436051T (lt) * 2016-03-31 2021-11-25 Spark Therapeutics, Inc. Kolonėlėmis pagrįstas visiškai keičiamo masto raav gavimo būdas
US11207426B2 (en) 2016-04-05 2021-12-28 University Of Massachusetts Compositions and methods for selective inhibition of grainyhead-like protein expression
EP3439675A4 (fr) 2016-04-08 2019-12-18 Purdue Research Foundation Méthodes et compositions pour thérapie par lymphocytes t car
US11413356B2 (en) 2016-04-15 2022-08-16 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
IL301963A (en) * 2016-07-21 2023-06-01 Spark Therapeutics Inc Scalable High Recovery Methods for High-Throughput Recombinant Adeno-Associated Viral Vector Production and Recombinant Adeno-Associated Viral Vectors Made So
WO2018022511A1 (fr) 2016-07-25 2018-02-01 The Trustees Of The University Of Pennsylvania Compositions comprenant un variant de la lécithine-cholestérol-acyl-transférase et leurs utilisations
EP3510161A4 (fr) 2016-08-23 2020-04-22 Akouos, Inc. Compositions et méthodes pour traiter une déficience auditive non associée à l'âge chez un sujet humain
EP3995574A1 (fr) 2016-08-24 2022-05-11 Sangamo Therapeutics, Inc. Régulation de l'expression génique à l'aide de nucléases modifiées
KR102455249B1 (ko) 2016-08-24 2022-10-17 상가모 테라퓨틱스, 인코포레이티드 가공된 표적 특이적 뉴클레아제
US10457940B2 (en) 2016-09-22 2019-10-29 University Of Massachusetts AAV treatment of Huntington's disease
AU2017341849B2 (en) 2016-10-13 2024-03-21 University Of Massachusetts AAV capsid designs
CA3040288A1 (fr) * 2016-11-04 2018-07-12 Baxalta Incorporated Procedes de purification de virus adeno-associe
EP3321357A1 (fr) 2016-11-09 2018-05-16 Deutsches Krebsforschungszentrum Procédé évolutif de production et de purification de parvovirus oncolytique h-1 du rat à base d'élimination de particules vides par points isoélectriques
CA3050917A1 (fr) 2017-01-30 2018-08-02 The Government Of The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Vecteurs viraux recombinants pour le traitement de la maladie du stockage du glycogene
US11649288B2 (en) 2017-02-07 2023-05-16 Seattle Children's Hospital Phospholipid ether (PLE) CAR T cell tumor targeting (CTCT) agents
JP2020510648A (ja) 2017-02-20 2020-04-09 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア 家族性高コレステロール血症を処置するための遺伝子治療
WO2018160622A1 (fr) 2017-02-28 2018-09-07 Endocyte, Inc. Compositions et méthodes de thérapie par lymphocytes t car
JP2020519272A (ja) 2017-05-09 2020-07-02 エモリー ユニバーシティー 凝固因子変異体及びその使用
US11859179B2 (en) 2017-05-09 2024-01-02 University Of Massachusetts Methods of treating amyotrophic lateral sclerosis (ALS)
WO2018226887A1 (fr) 2017-06-07 2018-12-13 Spark Therapeutics, Inc. Agents d'activation destinés à la transfection de cellules et/ou la production de vecteur raav améliorées
PE20200737A1 (es) * 2017-06-30 2020-07-23 Spark Therapeutics Inc Metodos de purificacion de columna de vector aav
AU2018313167A1 (en) 2017-08-08 2020-02-27 Sangamo Therapeutics, Inc. Chimeric antigen receptor mediated cell targeting
CN111448321A (zh) 2017-09-22 2020-07-24 马萨诸塞大学 Sod1双表达载体及其用途
US11926653B2 (en) 2017-10-20 2024-03-12 Research Institute At Nationwide Children's Hospital Methods and materials for NT-3 gene therapy
WO2019094253A1 (fr) * 2017-11-08 2019-05-16 Avexis Inc. Moyens et procédé de préparation de vecteurs viraux et leurs utilisations
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
US20190256867A1 (en) 2018-02-01 2019-08-22 Homology Medicines, Inc. Adeno-associated virus compositions for restoring pah gene function and methods of use thereof
US12312416B2 (en) 2018-02-06 2025-05-27 Seattle Children's Hospital 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
AU2019225174B2 (en) 2018-02-23 2025-11-20 Endocyte, Inc. Sequencing method for CAR T cell therapy
EP3758736B1 (fr) 2018-03-02 2024-05-01 The United States of America, as represented by The Secretary, Department of Health and Human Services L'il-34 pour utilisation dans une méthode de traitement l'inflammation rétinienne et la neurodégénérescence
WO2019195729A1 (fr) * 2018-04-05 2019-10-10 Nightstarx Ltd. Compositions de vaa, procédés de préparation et méthodes d'utilisation
WO2019236995A1 (fr) 2018-06-08 2019-12-12 University Of Massachusetts Oligonucléotides antisens pour restaurer l'expression de la protéine dysferline dans des cellules de patients souffrant d'une dysferlinopathie
EP3801638A1 (fr) 2018-06-08 2021-04-14 Novartis AG Dosage basé sur des cellules permettant de mesurer la puissance d'un produit médicamenteux
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
SG11202101032VA (en) 2018-08-20 2021-02-25 Wuhan Neurophth Biotechnology Ltd 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
CA3116576A1 (fr) 2018-10-18 2020-04-23 Acuitas Therapeutics, Inc. Lipides pour l'administration de nanoparticules lipidiques d'agents actifs
CN120860258A (zh) * 2018-11-30 2025-10-31 北京诺华制药有限公司 Aav病毒载体及其用途
BR112021012104A2 (pt) 2018-12-18 2021-09-08 Ultragenyx Pharmaceutical Inc. Métodos e composições para o tratamento de doenças de armazenamento de glicogênio
DK3906066T5 (da) 2019-01-04 2024-08-05 Ultragenyx Pharmaceutical Inc Geneterapikonstruktioner til behandling af wilsons sygdom
PT3908568T (pt) 2019-01-11 2024-09-30 Acuitas Therapeutics Inc Lípidos para a administração de agentes ativos por nanopartículas lipídicas
SG11202107645RA (en) * 2019-01-18 2021-08-30 Voyager Therapeutics Inc Methods and systems for producing aav particles
MA56393A (fr) 2019-06-28 2022-05-04 Bayer Healthcare Llc Matériels et méthodes de régulation de l'édition de gènes
EP4004213A1 (fr) 2019-07-25 2022-06-01 Novartis AG Systèmes d'expression régulables
WO2021062012A1 (fr) 2019-09-25 2021-04-01 Emory University Utilisation de klk10 et dérivations artificielles associées
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
US20230013253A1 (en) 2019-12-19 2023-01-19 Pfizer Inc. Compostions 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
JP7703543B2 (ja) 2020-01-17 2025-07-07 ザ ユナイテッド ステイツ オブ アメリカ アズ リプリゼンテッド 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 Ｃｒｘ常染色体優性網膜症の処置のための遺伝子治療
CN115210252A (zh) 2020-02-04 2022-10-18 西雅图儿童医院（Dba西雅图儿童研究所） 抗二硝基苯酚的嵌合抗原受体
JP2023514204A (ja) 2020-02-14 2023-04-05 ウルトラジェニックス ファーマシューティカル インコーポレイテッド Ｃｄｋｌ５欠損障害を処置するための遺伝子治療
KR20220157944A (ko) 2020-02-21 2022-11-29 아카우오스, 인크. 인간 대상체에서 비-연령-연관 청각 장애를 치료하기 위한 조성물 및 방법
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
ES3054438T3 (en) 2020-07-16 2026-02-03 Acuitas Therapeutics Inc Cationic lipids for use in lipid nanoparticles
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
CA3197730A1 (fr) 2020-10-15 2022-04-21 F. Hoffman-La Roche Ag Constructions d'acide nucleique pour transcription de va-arn
EP4229204A1 (fr) 2020-10-15 2023-08-23 F. Hoffmann-La Roche AG Constructions d'acides nucléiques améliorées pour activation de gènes simultanée
WO2023042104A1 (fr) 2021-09-16 2023-03-23 Novartis Ag Nouveaux facteurs de transcription
CA3242402A1 (fr) 2021-12-16 2023-06-22 Acuitas Therapeutics, Inc. Lipides destines a etre utilises dans des formulations de nanoparticules lipidiques
WO2023196898A1 (fr) 2022-04-07 2023-10-12 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Peptides mimétiques de la bêta globine et leur utilisation
EP4508241A1 (fr) 2022-04-13 2025-02-19 F. Hoffmann-La Roche AG Procédé de détermination de génomes d'aav
US20250353883A1 (en) 2022-05-06 2025-11-20 Novartis Ag Novel recombinant aav vp2 fusion polypeptides
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
CN119301260A (zh) 2022-06-03 2025-01-10 豪夫迈·罗氏有限公司 用于生产重组aav颗粒的方法
KR20250036743A (ko) 2022-07-14 2025-03-14 에프. 호프만-라 로슈 아게 재조합 aav 입자를 생산하는 방법
CA3264505A1 (fr) 2022-09-12 2024-03-21 F. Hoffmann-La Roche Ag Procédé de séparation de particules de vaa pleines et vides
AU2024216670A1 (en) 2023-02-07 2025-07-31 F. Hoffmann-La Roche Ag Method for the detection of anti-aav particle antibodies
EP4662231A1 (fr) 2023-02-10 2025-12-17 Expression Therapeutics, LLC Système lentiviral
EP4669654A1 (fr) 2023-02-22 2025-12-31 The United States of America, as represented by The Secretary, Department of Health and Human Services Vecteurs de virus adéno-associés recombinants dépourvus d'épitope de lymphocyte t immunodominant et leur utilisation
WO2024196814A1 (fr) 2023-03-17 2024-09-26 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Méthodes de traitement de la dégénérescence maculaire liée à l'âge
AU2024239386A1 (en) 2023-03-21 2025-08-14 F. Hoffmann-La Roche Ag Method for the production of recombinant aav particle preparations
WO2025090942A1 (fr) * 2023-10-26 2025-05-01 University Of Notre Dame Du Lac Thérapie génique pour le traitement d'une maladie neurométabolique
WO2025168663A1 (fr) 2024-02-09 2025-08-14 F. Hoffmann-La Roche Ag Procédé de production de particules virales adéno-associées recombinées
WO2025252480A1 (fr) 2024-06-07 2025-12-11 F. Hoffmann-La Roche Ag Procédé de purification d'adn plasmidique
WO2026022064A1 (fr) 2024-07-22 2026-01-29 F. Hoffmann-La Roche Ag Nouveaux polypeptides rep et rep orf de vaa
WO2026037757A1 (fr) 2024-08-13 2026-02-19 F. Hoffmann-La Roche Ag Particules d'aav modifiées

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
EP1005376B1 (fr) * 1997-03-14 2010-11-03 The Children's Hospital of Philadelphia Compositions utilisees en therapie genique dans le traitement de l'hemophilie
US6989264B2 (en) * 1997-09-05 2006-01-24 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
AU780231B2 (en) * 1998-11-10 2005-03-10 University Of North Carolina At Chapel Hill, The Virus vectors and methods of making and administering the same
EP1190055B1 (fr) 1999-06-30 2008-06-25 The Administrators Of The Tulane Educational Fund Retrovirus endogene humain
AU2001249389A1 (en) * 2000-03-22 2001-10-03 The Children's Hospital Of Philadelphia 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
WO2003088899A2 (fr) * 2002-04-05 2003-10-30 The Children's Hospital Of Philadelphia Techniques de production de vecteurs de virus adeno-associes (aav) chimeriques, de composition de vecteurs aav chimeriques et techniques d'utilisation de ceux-ci
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
EP2018421B1 (fr) * 2006-04-28 2012-12-19 The Trustees of the University of Pennsylvania Procédé évolutif de production d'aav
WO2008128251A1 (fr) * 2007-04-17 2008-10-23 The Children's Hospital Of Philadelphia Vecteur viral humanisé et procédés pour d'utilisation
DK2307551T3 (en) * 2008-06-18 2017-03-20 Oxford Biomedica (Uk) Ltd CLEANING RETROVIRAL VECTORS
WO2011094198A1 (fr) * 2010-01-28 2011-08-04 The Children's Hospital Of Philadelphia Research Institute, Abramson Research Center Plateforme de fabrication évolutive pour purification de vecteurs viraux et vecteurs viraux ainsi purifiés utilisables en thérapie génique

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US20190321463A1 (en) 2019-10-24
AU2011209743B2 (en) 2016-03-10
BR112012018899A2 (pt) 2015-09-15
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PT2529020T (pt) 2018-07-30
CN102947453A (zh) 2013-02-27
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