SG11202100704PA - Systems and methods for producing gene therapy formulations - Google Patents

Systems and methods for producing gene therapy formulations

Info

Publication number
SG11202100704PA
SG11202100704PA SG11202100704PA SG11202100704PA SG11202100704PA SG 11202100704P A SG11202100704P A SG 11202100704PA SG 11202100704P A SG11202100704P A SG 11202100704PA SG 11202100704P A SG11202100704P A SG 11202100704PA SG 11202100704P A SG11202100704P A SG 11202100704PA
Authority
SG
Singapore
Prior art keywords
systems
methods
gene therapy
producing gene
therapy formulations
Prior art date
Application number
SG11202100704PA
Other languages
English (en)
Inventor
Jacob J Cardinal
Robert Steininger
Lori B Karpes
Christopher J Morrison
Daniel S Hurwit
Matthew Luther
Andrew M Wood
Dinah Wen-Yee Sah
Pengcheng Zhou
Jeffrey S Thompson
Christina Gamba-Vitalo
Soper Jenna Carroll
Steven M Hersch
Todd Carter
Original Assignee
Voyager Therapeutics Inc
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 Voyager Therapeutics Inc filed Critical Voyager Therapeutics Inc
Publication of SG11202100704PA publication Critical patent/SG11202100704PA/en

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
    • C12N7/02—Recovery or purification
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/66—Microorganisms or materials therefrom
    • A61K35/76—Viruses; Subviral particles; Bacteriophages
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering N.A.
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering N.A.
    • C12N2310/141—MicroRNAs, miRNAs
    • 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
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/32—Special delivery means, e.g. tissue-specific
    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Zoology (AREA)
  • General Health & Medical Sciences (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Medicinal Chemistry (AREA)
  • Microbiology (AREA)
  • Virology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Biochemistry (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Plant Pathology (AREA)
  • Neurosurgery (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Molecular Biology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Psychology (AREA)
  • Neurology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Epidemiology (AREA)
  • Immunology (AREA)
  • Mycology (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicinal Preparation (AREA)
SG11202100704PA 2018-07-24 2019-07-24 Systems and methods for producing gene therapy formulations SG11202100704PA (en)

Applications Claiming Priority (10)

Application Number Priority Date Filing Date Title
US201862702687P 2018-07-24 2018-07-24
US201862702679P 2018-07-24 2018-07-24
US201862725432P 2018-08-31 2018-08-31
US201862741508P 2018-10-04 2018-10-04
US201962794212P 2019-01-18 2019-01-18
US201962794213P 2019-01-18 2019-01-18
US201962794199P 2019-01-18 2019-01-18
US201962826363P 2019-03-29 2019-03-29
US201962839880P 2019-04-29 2019-04-29
PCT/US2019/043196 WO2020023612A1 (fr) 2018-07-24 2019-07-24 Systèmes et méthodes de production de formulations de thérapie génique

Publications (1)

Publication Number Publication Date
SG11202100704PA true SG11202100704PA (en) 2021-02-25

Family

ID=67766241

Family Applications (1)

Application Number Title Priority Date Filing Date
SG11202100704PA SG11202100704PA (en) 2018-07-24 2019-07-24 Systems and methods for producing gene therapy formulations

Country Status (10)

Country Link
US (1) US20210355454A1 (fr)
EP (1) EP3826719A1 (fr)
JP (1) JP2021530548A (fr)
CN (1) CN112770812A (fr)
AU (1) AU2019310459A1 (fr)
CA (1) CA3107462A1 (fr)
IL (1) IL280350A (fr)
MX (1) MX2021000810A (fr)
SG (1) SG11202100704PA (fr)
WO (1) WO2020023612A1 (fr)

Families Citing this family (26)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
RU2764587C2 (ru) 2016-05-18 2022-01-18 Вояджер Терапьютикс, Инк. Способы и композиции для лечения хореи гентингтона
JP6994018B2 (ja) 2016-07-26 2022-01-14 バイオマリン ファーマシューティカル インコーポレイテッド 新規アデノ随伴ウイルスキャプシドタンパク質
JP2019530737A (ja) 2016-08-23 2019-10-24 アコーオス インコーポレイテッド ヒト対象において非加齢性聴力障害を治療するための組成物および方法
SG11201909868YA (en) 2017-05-05 2019-11-28 Voyager Therapeutics Inc Compositions and methods of treating huntington's disease
SG11202010830WA (en) 2018-05-09 2020-11-27 Biomarin Pharm Inc Methods of treating phenylketonuria
TW202005978A (zh) 2018-05-14 2020-02-01 美商拜奧馬林製藥公司 新穎肝靶向腺相關病毒載體
TW202106879A (zh) * 2019-04-29 2021-02-16 美商航海家醫療公司 於生物反應器中生產經桿狀病毒感染之昆蟲細胞(biic)之系統及方法
WO2021016505A1 (fr) * 2019-07-24 2021-01-28 Voyager Therapeutics, Inc. Compositions et méthodes de traitement de la maladie de huntington
EP4010465A1 (fr) * 2019-08-09 2022-06-15 Voyager Therapeutics, Inc. Milieu de culture cellulaire destiné à être utilisé dans la production de produits de thérapie génique dans des bioréacteurs
US20230242939A1 (en) * 2020-01-29 2023-08-03 Voyager Therapeutics, Inc. Methods and systems for producing aav particles
AU2021225035A1 (en) 2020-02-21 2022-10-13 Akouos, Inc. Compositions and methods for treating non-age-associated hearing impairment in a human subject
AR122409A1 (es) * 2020-04-03 2022-09-07 Biomarin Pharm Inc Tratamiento de la fenilcetonuria con aav y formulaciones terapéuticas
WO2021228930A2 (fr) * 2020-05-14 2021-11-18 Merck Patent Gmbh Procédés et compositions pour purifier des particules de virus adéno-associés ou des adénovirus
EP4192514A1 (fr) * 2020-08-06 2023-06-14 Voyager Therapeutics, Inc. Milieu de culture cellulaire destiné à être utilisé dans la production de produits de thérapie génique dans des bioréacteurs
JP2023536883A (ja) * 2020-08-07 2023-08-30 ヤンセン バイオテツク，インコーポレーテツド 高度に精製されたウイルス粒子のための製剤
WO2022076591A1 (fr) * 2020-10-07 2022-04-14 Regenxbio Inc. Formulations pour administration suprachoroïdienne, telles que formulations avec formation d'agrégats
WO2022174000A2 (fr) 2021-02-12 2022-08-18 Alnylam Pharmaceuticals, Inc. Compositions d'arni de superoxyde dismutase 1 (sod1) et procédés d'utilisation correspondants pour traiter ou prévenir des maladies neurodégénératives associées à la superoxyde dismutase 1 (sod1)
WO2022206819A1 (fr) * 2021-03-30 2022-10-06 南京大学 Système d'administration d'arn pour le traitement de la maladie de huntington
CN117836010A (zh) * 2021-06-21 2024-04-05 优尼科生物制药有限公司 改进的裂解程序
EP4365284A1 (fr) 2021-06-30 2024-05-08 Asahi Kasei Medical Co., Ltd. Procédé de récupération de virus
WO2023282796A2 (fr) * 2021-07-08 2023-01-12 Limited Liability Company «ANABION» Composition pharmaceutique de virus non enveloppé
WO2023023590A1 (fr) * 2021-08-18 2023-02-23 Ultragenyx Pharmaceutical Inc. Formulations d'aav recombinants
WO2023056436A2 (fr) * 2021-10-01 2023-04-06 Biomarin Pharmaceutical Inc. Traitement de l'oedème de quincke héréditaire avec des vecteurs de thérapie génique aav et des formulations thérapeutiques
WO2024006548A1 (fr) * 2022-07-01 2024-01-04 Sherlock Biosciences, Inc. Préparation d'acide nucléique
WO2024015369A1 (fr) * 2022-07-12 2024-01-18 Brammer Bio, Llc Procédés et compositions pour l'enrichissement d'acide nucléique contenant des capsides de vaa
CN117138121A (zh) * 2023-08-25 2023-12-01 苏州邦伊医疗科技有限公司 一种减少破坏提高纯度的自体脂肪移植方法

Family Cites Families (219)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US465302A (en) 1891-12-15 Horse-collar-stuffing machine
US738609A (en) 1903-04-02 1903-09-08 Theodore Ferdinand Gensmer Resilient rest for vehicle-tops.
GB2097696B (en) 1981-05-04 1985-07-10 Purdue Research Foundation Sorbent mixture for use in hemodialysis
US4704791A (en) 1986-03-05 1987-11-10 International Business Machines Corporation Process for providing a landless through-hole connection
FR2640638B1 (fr) 1988-12-20 1991-02-15 Commissariat Energie Atomique Bioreacteur et dispositif pour la culture de cellules animales
US5173414A (en) 1990-10-30 1992-12-22 Applied Immune Sciences, Inc. Production of recombinant adeno-associated virus vectors
US5387484A (en) 1992-07-07 1995-02-07 International Business Machines Corporation Two-sided mask for patterning of materials with electromagnetic radiation
CA2187626C (fr) 1994-04-13 2009-11-03 Michael G. Kaplitt Transmission par virus adenoassocie d'adn a des cellules du systeme nerveux
US6204059B1 (en) 1994-06-30 2001-03-20 University Of Pittsburgh AAV capsid vehicles for molecular transfer
CA2143791A1 (fr) 1994-08-03 1996-02-04 Donald M. Fries Article absorbant dote de panneaux lateraux elastiques relies par une bande
US5856152A (en) 1994-10-28 1999-01-05 The Trustees Of The University Of Pennsylvania Hybrid adenovirus-AAV vector and methods of use therefor
WO1996017947A1 (fr) 1994-12-06 1996-06-13 Targeted Genetics Corporation Lignees cellulaires d'encapsidation utilisees pour la generation de titres hauts de vecteurs aav recombinants
US6281010B1 (en) 1995-06-05 2001-08-28 The Trustees Of The University Of Pennsylvania Adenovirus gene therapy vehicle and cell line
US5756283A (en) 1995-06-05 1998-05-26 The Trustees Of The University Of Pennsylvania Method for improved production of recombinant adeno-associated viruses for gene therapy
US5741683A (en) 1995-06-07 1998-04-21 The Research Foundation Of State University Of New York In vitro packaging of adeno-associated virus DNA
US5688676A (en) 1995-06-07 1997-11-18 Research Foundation Of State University Of New York In vitro packaging of adeno-associated virus DNA
US6197293B1 (en) 1997-03-03 2001-03-06 Calydon, Inc. Adenovirus vectors specific for cells expressing androgen receptor and methods of use thereof
US6676935B2 (en) 1995-06-27 2004-01-13 Cell Genesys, Inc. Tissue specific adenoviral vectors
CA2625279A1 (fr) 1995-08-30 1997-03-06 Genzyme Corporation Purification d'adenovirus et de virus adeno-associe (aav) par voie chromatographique
US6265389B1 (en) 1995-08-31 2001-07-24 Alkermes Controlled Therapeutics, Inc. Microencapsulation and sustained release of oligonucleotides
US7026468B2 (en) 1996-07-19 2006-04-11 Valentis, Inc. Process and equipment for plasmid purification
US6083716A (en) 1996-09-06 2000-07-04 The Trustees Of The University Of Pennsylvania Chimpanzee adenovirus vectors
WO1998010088A1 (fr) 1996-09-06 1998-03-12 Trustees Of The University Of Pennsylvania Procede inductible de production de virus adeno-associes recombines au moyen de la polymerase t7
EP0950111A1 (fr) 1996-09-06 1999-10-20 The Trustees Of The University Of Pennsylvania Procede d'utilisation de cre-lox pour la production de virus adeno-associes de recombinaison
US7732129B1 (en) 1998-12-01 2010-06-08 Crucell Holland B.V. Method for the production and purification of adenoviral vectors
EP0968284B1 (fr) 1996-11-20 2006-12-13 Introgen Therapeutics, Inc. Procede ameliore pour production et purification de vecteurs d'adenovirus
JP2001506133A (ja) 1996-12-18 2001-05-15 ターゲティッド ジェネティクス コーポレイション 組換えａａｖベクターの産生における使用のための、ａａｖスプリット−パッケージング遺伝子およびこのような遺伝子を含む細胞株
US6156303A (en) 1997-06-11 2000-12-05 University Of Washington Adeno-associated virus (AAV) isolates and AAV vectors derived therefrom
US6251677B1 (en) 1997-08-25 2001-06-26 The Trustees Of The University Of Pennsylvania Hybrid adenovirus-AAV virus and methods of use thereof
US6566118B1 (en) 1997-09-05 2003-05-20 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released 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
EP1015619A1 (fr) 1997-09-19 2000-07-05 The Trustees Of The University Of Pennsylvania Procedes et lignee cellulaire utiles pour la production de virus adeno-associes recombines
CA2303768C (fr) 1997-09-19 2009-11-24 The Trustees Of The University Of Pennsylvania Procedes et produits genetiques vectoriels utiles pour obtenir un virus adeno-associe (aav)
US6410300B1 (en) 1998-01-12 2002-06-25 The University Of North Carolina At Chapel Hill Methods and formulations for mediating adeno-associated virus (AAV) attachment and infection and methods for purifying AAV
US6953690B1 (en) 1998-03-20 2005-10-11 The Trustees Of The University Of Pennsylvania Compositions and methods for helper-free production of recombinant adeno-associated viruses
ES2235470T3 (es) 1998-03-20 2005-07-01 The Trustees Of The University Of Pennsylvania Composiciones y metodos para la produccion libre de cooperadores de virus adnoasociados recombinantes.
FR2778413B1 (fr) 1998-05-07 2000-08-04 Immunotech Sa Nouveaux reactifs et methode de lyse des erythrocytes
WO1999058700A1 (fr) 1998-05-11 1999-11-18 Ariad Gene Therapeutics, Inc. Compositions multivirales et utilisations de celles-ci
US6146874A (en) 1998-05-27 2000-11-14 University Of Florida Method of preparing recombinant adeno-associated virus compositions
JP4060531B2 (ja) 1998-05-28 2008-03-12 アメリカ合衆国 Ａａｖ５ベクターおよびその使用
US6984517B1 (en) 1998-05-28 2006-01-10 The United States Of America As Represented By The Department Of Health And Human Services AAV5 vector and uses thereof
GB2338236B (en) 1998-06-13 2003-04-09 Aea Technology Plc Microbiological cell processing
US6900049B2 (en) 1998-09-10 2005-05-31 Cell Genesys, Inc. Adenovirus vectors containing cell status-specific response elements and methods of use thereof
AU764130B2 (en) 1998-10-27 2003-08-14 Crucell Holland B.V. Improved AAV vector production
US6759237B1 (en) 1998-11-05 2004-07-06 The Trustees Of The University Of Pennsylvania Adeno-associated virus serotype 1 nucleic acid sequences, vectors and host cells containing same
JP4573437B2 (ja) 1998-11-05 2010-11-04 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 アデノ随伴ウイルス血清型１核酸配列、ベクターおよび同一物を含有する宿主細胞
EP1845163A3 (fr) 1998-11-05 2007-10-31 The Trustees Of The University Of Pennsylvania Séquences d'acides nucléiques de sérotype 1 du virus associé à l'adénovirus
CA2348382C (fr) 1998-11-10 2013-09-17 The University Of North Carolina At Chapel Hill Vecteurs de papirovirus chimeriques et procedes de production et d'administration connexes
US6689600B1 (en) 1998-11-16 2004-02-10 Introgen Therapeutics, Inc. Formulation of adenovirus for gene therapy
US6225113B1 (en) 1998-12-04 2001-05-01 Genvec, Inc. Use of trans-activation and cis-activation to modulate the persistence of expression of a transgene in an at least E4-deficient adenovirus
US6387368B1 (en) 1999-02-08 2002-05-14 The Trustees Of The University Of Pennsylvania Hybrid adenovirus-AAV virus and methods of use thereof
DE19905501B4 (de) 1999-02-10 2005-05-19 MediGene AG, Gesellschaft für molekularbiologische Kardiologie und Onkologie Verfahren zur Herstellung eines rekombinanten Adeno-assoziierten Virus, geeignete Mittel hierzu sowie Verwendung zur Herstellung eines Arzneimittels
US6258595B1 (en) 1999-03-18 2001-07-10 The Trustees Of The University Of Pennsylvania Compositions and methods for helper-free production of recombinant adeno-associated viruses
JP4693244B2 (ja) 1999-03-18 2011-06-01 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 組換えアデノ随伴ウイルスのヘルパー無しの生産のための組成物および方法
WO2000075353A1 (fr) 1999-06-02 2000-12-14 Trustees Of The University Of Pennsylvania Compositions et methodes pour la fabrication de virus recombines necessitant des virus auxiliaires
EP1218035A2 (fr) 1999-09-29 2002-07-03 The Trustees Of The University Of Pennsylvania Procedes de modification rapide du peg de vecteurs viraux, compositions servant a ameliorer la transduction de genes, compositions presentant une stabilite physique augmentee, et leurs utilisations
US6365394B1 (en) 1999-09-29 2002-04-02 The Trustees Of The University Of Pennsylvania Cell lines and constructs useful in production of E1-deleted adenoviruses in absence of replication competent adenovirus
CA2392299A1 (fr) 1999-12-10 2001-06-14 Victor Rivera Procedes d'expression haut niveau de genes chez des primates
US6855314B1 (en) 2000-03-22 2005-02-15 The United States Of America As Represented By The Department Of Health And Human Services AAV5 vector for transducing brain cells and lung cells
US6468524B1 (en) 2000-03-22 2002-10-22 The United States Of America, As Represented By The Secretary Of The Department Of Health And Human Services AAV4 vector and uses thereof
US7048920B2 (en) 2000-03-24 2006-05-23 Cell Genesys, Inc. Recombinant oncolytic adenovirus for human melanoma
US20030013189A1 (en) 2000-04-28 2003-01-16 Wilson James M. Compositions and methods useful for non-invasive delivery of therapeutic molecules to the bloodstream
US7056502B2 (en) 2000-04-28 2006-06-06 The Trustees Of The University Of Pennsylvania Recombinant aav vectors with AAV5 capsids and AAV5 vectors pseudotyped in heterologous capsids
ATE364698T1 (de) 2000-07-18 2007-07-15 Takeda Pharmaceutical Neues, physiologisch aktives peptid und dessen verwendung
US6593123B1 (en) 2000-08-07 2003-07-15 Avigen, Inc. Large-scale recombinant adeno-associated virus (rAAV) production and purification
FR2813891B1 (fr) 2000-09-14 2005-01-14 Immunotech Sa Reactif multifonctionnel pour erythrocytes mettant en jeu des carbamates et applications
US9715691B2 (en) 2001-01-16 2017-07-25 Gtj Ventures, Llc Apparatus and method for providing transaction history information, account history information, and/or charge-back information
ES2375557T3 (es) 2001-06-22 2012-03-02 The Trustees Of The University Of Pennsylvania Adenovirus recombinantes que comprenden prote�?nas de adenovirus de simios y usos de los mismos.
US20040136963A1 (en) 2001-06-22 2004-07-15 The Trustees Of The University Of Pennsylvania Simian adenovirus vectors and methods of use
JP2004538005A (ja) 2001-08-08 2004-12-24 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 シアル酸に結合するタンパク質を有するウイルスベクターの精製法
US20030092161A1 (en) 2001-09-19 2003-05-15 The Trustees Of The University Of Pennsylvania Compositions and methods for production of recombinant viruses, and uses therefor
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
US7271002B2 (en) 2001-11-09 2007-09-18 United States Of America, Represented By The Secretary, Department Of Health And Human Services Production of adeno-associated virus in insect cells
NZ532635A (en) 2001-11-13 2007-05-31 Univ Pennsylvania A method of identifying unknown adeno-associated virus (AAV) sequences and a kit for the method
EP1944043A1 (fr) 2001-11-21 2008-07-16 The Trustees of the University of Pennsylvania Séquences d'acides aminés et d'acides nucléiques d'adénovirus simien, vecteurs les contenant, et procédés d'utilisation.
SG2013034475A (en) 2001-11-21 2016-10-28 Univ Pennsylvania Simian adenovirus nucleic acid and amino acid sequences, vectors containing same, and methods of use
JP2005517394A (ja) 2001-12-12 2005-06-16 エフ エイチ フォールディング アンド カンパニー リミテッド ウイルス保存のための組成物
PT1453547T (pt) 2001-12-17 2016-12-28 Univ Pennsylvania Sequências do vírus adeno-associado (aav) do serotipo 8, vetores contendo as mesmas, e utilizações destas
JP4769417B2 (ja) 2001-12-17 2011-09-07 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 アデノ随伴ウイルス（ａａｖ）血清型９の配列、それを含むベクターおよびその使用
EP1468116A2 (fr) 2002-01-16 2004-10-20 Dynal Biotech ASA Methode permettant d'isoler des acides nucleiques et des proteines contenus dans un echantillon unique
JP3943048B2 (ja) 2002-04-29 2007-07-11 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 組織の細胞ｄｎａからの組込みウイルスの直接レスキュー及び増幅の方法
ES2323454T5 (es) 2002-04-30 2012-11-14 Oncolytics Biotech Inc. Procedimiento mejorado de purificación de virus
AU2003229060A1 (en) 2002-05-14 2003-12-02 Merck And Co., Inc. Methods of adenovirus purification
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
AU2003274397A1 (en) 2002-06-05 2003-12-22 University Of Florida Production of pseudotyped recombinant aav virions
US7605249B2 (en) 2002-11-26 2009-10-20 Medtronic, Inc. Treatment of neurodegenerative disease through intracranial delivery of siRNA
JP4226313B2 (ja) 2002-12-19 2009-02-18 株式会社ソニー・コンピュータエンタテインメント 楽音再生装置及び楽音再生プログラム
WO2005017101A2 (fr) 2003-05-19 2005-02-24 THE GOVERNMENT OF THE UNITED STATES OF AMERICA, as represented by THE SECRETARY, DEPARTMENT OF HEALTH & HUMAN SERVICES, NATIONAL INSTITUTES OF HEALTH Virus aviaire adenoassocie (aaav) et ses utilisations
PT1625210E (pt) 2003-05-21 2011-03-15 Genzyme Corp Métodos para produzir preparações de vírions de aav recombinantes substancialmente livres de capsídeos vazios
DK3235827T3 (da) 2003-06-19 2021-04-19 Genzyme Corp Aav-virioner med reduceret immunreaktivitet og anvendelser deraf
US7491508B2 (en) 2003-06-20 2009-02-17 The Trustees Of The University Of Pennsylvania Methods of generating chimeric adenoviruses and uses for such chimeric adenoviruses
US7291498B2 (en) 2003-06-20 2007-11-06 The Trustees Of The University Of Pennsylvania Methods of generating chimeric adenoviruses and uses for such chimeric adenoviruses
US9441244B2 (en) 2003-06-30 2016-09-13 The Regents Of The University Of California Mutant adeno-associated virus virions and methods of use thereof
ES2648241T3 (es) 2003-09-30 2017-12-29 The Trustees Of The University Of Pennsylvania Clados de virus adenoasociados (AAV), secuencias, vectores que contienen el mismo, y usos de los mismos
WO2005056807A2 (fr) 2003-12-04 2005-06-23 The Government Of The United States Of America, As Represented By The Secretary, Department Of Health And Human Services, National Institutes Of Health Vecteur viral associe aux adenovirus bovins et leurs utilisations
US20060166363A1 (en) 2004-01-27 2006-07-27 Sergei Zolotukhin Modified baculovirus expression system for production of pseudotyped rAAV vector
ES2361000T3 (es) 2004-04-28 2011-06-13 The Trustees Of The University Of Pennsylvania Suministro secuencial de moléculas inmunogénicas mediante administraciones de un adenovirus y de un virus adeno-asociado.
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
US7427396B2 (en) 2004-06-03 2008-09-23 Genzyme Corporation AAV vectors for gene delivery to the lung
US7901921B2 (en) 2004-10-22 2011-03-08 Oncolytics Biotech Inc. Viral purification methods
US8614101B2 (en) 2008-05-20 2013-12-24 Rapid Pathogen Screening, Inc. In situ lysis of cells in lateral flow immunoassays
US7625570B1 (en) 2005-03-10 2009-12-01 The Regents Of The University Of California Methods for purifying adeno-associated virus
ES2525067T3 (es) 2005-04-07 2014-12-17 The Trustees Of The University Of Pennsylvania Método de incremento de la función de un vector de AAV
EP1868651A4 (fr) 2005-04-12 2010-10-06 Univ Duke Methode pour induire des anticorps neutralisant le virus de l'immunodeficience humaine
WO2006119432A2 (fr) 2005-04-29 2006-11-09 The Government Of The U.S.A., As Rep. By The Sec., Dept. Of Health & Human Services Isolation, clonage, et caracterisation de nouveaux serotypes de virus adeno-associes (avv)
US7761843B2 (en) 2005-07-01 2010-07-20 Ronald Ortscheid Computer source code generator
AU2006304997B2 (en) 2005-10-20 2012-03-01 Uniqure Ip B.V. Improved AAV vectors produced in insect cells
EP2062530A3 (fr) 2005-11-29 2009-08-12 Surgi-Vision, Inc. Systèmes de mise en place de dérivation et/ou de localisation guidés par IRM et procédés, dispositifs et programmes informatiques associés
WO2007120542A2 (fr) 2006-03-30 2007-10-25 The Board Of Trustees Of The Leland Stanford Junior University Bibliothèque de capsides aav et protéines de capsides aav
ES2400235T3 (es) 2006-04-28 2013-04-08 The Trustees Of The University Of Pennsylvania Método de producción escalable de AAV
CN101495624A (zh) 2006-04-28 2009-07-29 宾夕法尼亚州立大学托管会 衣壳免疫原性降低的经修饰aav载体及其用途
ES2785223T3 (es) 2006-06-21 2020-10-06 Uniqure Ip Bv Células de insecto para la producción de vectores de AAV
GB0613753D0 (en) 2006-07-11 2006-08-23 Norwegian Radium Hospital Res Method
US20090036395A1 (en) 2007-04-26 2009-02-05 Davidson Beverly L Rna interference suppression of neurodegenerative diseases and methods of use thereof
ES2557482T3 (es) 2007-05-17 2016-01-26 Medgenesis Therapeutix Inc. Catéter de entrega mejorada por convección con un miembro de rigidización eliminable
US20100203083A1 (en) 2007-05-31 2010-08-12 Medigene Ag Mutated structural protein of a parvovirus
US8175677B2 (en) 2007-06-07 2012-05-08 MRI Interventions, Inc. MRI-guided medical interventional systems and methods
EP2019143A1 (fr) 2007-07-23 2009-01-28 Genethon Thérapie génique du CNS utilisant l'administration périphérique de vecteurs AAV
JP5634262B2 (ja) 2007-07-26 2014-12-03 ユニキュアー アイピー ビー．ブイ． 差次的コドンバイアスを有する反復コード配列を含むバキュロウイルスベクター
US8825138B2 (en) 2007-09-17 2014-09-02 Wisconsin Alumni Research Foundation Method for reducing motion artifacts in highly constrained medical images
JP2010538675A (ja) 2007-09-19 2010-12-16 アムステルダム モレキュラー セラピューティクス ビー．ブイ． タンパク質産生の改善のためのａａｖ複製機構の使用
US8099150B2 (en) 2007-09-24 2012-01-17 MRI Interventions, Inc. MRI-compatible head fixation frame with cooperating head coil apparatus
US8548569B2 (en) 2007-09-24 2013-10-01 MRI Interventions, Inc. Head fixation assemblies for medical procedures
EP2194906B8 (fr) 2007-09-24 2015-04-22 Mri Interventions, Inc. Système d'intervention médical guidé par irm
EP2058401A1 (fr) 2007-10-05 2009-05-13 Genethon Fourniture généralisée de gènes à des motoneurones utilisant l'injection périphérique de vecteurs AAV
WO2009067205A1 (fr) 2007-11-21 2009-05-28 Surgi-Vision, Inc. Procédés, systèmes et programmes informatiques pour le positionnement d'un appareil de guidage par rapport à un patient
BRPI0822651A2 (pt) 2007-11-28 2014-10-14 Univ Pennsylvania Subfamília b de adenovírus sadv-28, -27, 29, -32, -33 e -35 de símio e seus usos
MX347246B (es) 2007-11-28 2017-04-19 Univ Pennsylvania Adenovirus e simianos sadv-39, sadv-25.2, sadv-26, sadv-30, sadv-37 y sadv-38.
WO2009105084A2 (fr) 2007-11-28 2009-08-27 The Trustees Of The University Of Pennsylvania Adénovirus c de la sous-famille simiesque sadv-40, -31, et -34 et leurs utilisations
ES2751999T3 (es) 2008-01-29 2020-04-02 Applied Genetic Tech Corporation Producción recombinante de virus adeno-asociados usando células BHK en suspensión
US8642314B2 (en) 2008-02-19 2014-02-04 Amsterdam Molecular Therapeutics (Amt) B.V. Optimization of expression of parvoviral rep and cap proteins in insect cells
US8470310B2 (en) 2008-03-04 2013-06-25 The Trustees Of The University Of Pennsylvania Simian adenoviruses SAdV-36, -42.1, -42.2, and -44 and uses thereof
EP2315833B8 (fr) 2008-05-20 2015-05-27 Eos Neuroscience, Inc. Vecteurs pour la délivrance de protéines photosensibles et procédés d'utilisation
US9217155B2 (en) 2008-05-28 2015-12-22 University Of Massachusetts Isolation of novel AAV'S and uses thereof
EP2297185A1 (fr) 2008-06-17 2011-03-23 Amsterdam Molecular Therapeutics (AMT) B.V. Capside parvovirale avec région de répétition gly-ala incorporée
US8940290B2 (en) 2008-10-31 2015-01-27 The Trustees Of The University Of Pennsylvania Simian adenoviruses SAdV-43, -45, -46, -47, -48, -49, and -50 and uses thereof
US9925694B2 (en) 2009-02-24 2018-03-27 Gala Industries, Inc. Continuous bagging processes and systems
PL2425000T3 (pl) 2009-04-30 2019-08-30 The Trustees Of The University Of Pennsylvania Kompozycje do kierowania komórek przewodzących do dróg oddechowych zawierające konstrukty wirusów związanych z adenowirusami
US8331384B2 (en) 2009-05-12 2012-12-11 Cisco Technology, Inc. System and method for femto coverage in a wireless network
US8734809B2 (en) 2009-05-28 2014-05-27 University Of Massachusetts AAV's and uses thereof
EP2435575A2 (fr) 2009-05-28 2012-04-04 Deutsches Krebsforschungszentrum Polypeptides de la capside d'aav modifiés
CN102575232B (zh) 2009-05-29 2015-07-22 宾夕法尼亚大学托管会 猿腺病毒41及其应用
WO2012116265A2 (fr) 2011-02-24 2012-08-30 MRI Interventions, Inc. Sondes guidées par irm
HUE028341T2 (en) * 2009-06-16 2016-12-28 Genzyme Corp Improved methods for purifying recombinant AAV vectors
EP2442718B1 (fr) 2009-06-16 2018-04-25 MRI Interventions, Inc. Dispositifs guidés par irm et systèmes d'intervention guidés par irm qui peuvent suivre et générer des visualisations dynamiques des dispositifs presque en temps réel
JP2012531893A (ja) 2009-06-29 2012-12-13 ボード・オブ・リージエンツ，ザ・ユニバーシテイ・オブ・テキサス・システム アルギナーゼ製剤および方法
BR112012000421A2 (pt) 2009-07-06 2019-09-24 Alnylam Pharmaceuticals Inc composições e métodos para intensificar a produção de um produto biológico.
US8462014B1 (en) 2009-08-10 2013-06-11 Ecologic Analytics, LLC Meter data management systems, methods, and software with outage management capabilities
US8587595B2 (en) 2009-10-01 2013-11-19 Hand Held Products, Inc. Low power multi-core decoder system and method
US9228174B2 (en) 2010-03-11 2016-01-05 Uniqure Ip B.V. Mutated rep encoding sequences for use in AAV production
US9546112B2 (en) 2010-03-22 2017-01-17 Association Institut De Myologie Methods of increasing efficiency of vector penetration of target tissue
US10105485B2 (en) 2010-04-16 2018-10-23 MRI Interventions, Inc. MRI surgical systems including MRI-compatible surgical cannulae for transferring a substance to and/or from a patient
DK2826860T3 (en) 2010-04-23 2018-12-03 Univ Massachusetts CNS targeting AAV vectors and methods for their use
WO2011133901A2 (fr) 2010-04-23 2011-10-27 University Of Massachusetts Traitement à base d'aav pour des troubles liés au cholestérol
WO2011133874A1 (fr) 2010-04-23 2011-10-27 University Of Massachusetts Constructions d'expression à cistrons multiples
WO2012057363A1 (fr) 2010-10-27 2012-05-03 学校法人自治医科大学 Virions de virus adéno-associé pour le transfert de gènes dans des cellules neuronales
JP5964846B2 (ja) 2010-10-27 2016-08-03 ヴァンダービルト ユニヴァーシティ ナノ繊維電極およびそれを形成する方法
AU2011332025B2 (en) 2010-11-23 2015-06-25 The Trustees Of The University Of Pennsylvania Subfamily E simian adenoviruses A1321, A1325, A1295, A1309 and A1322 and uses thereof
EP2673286B1 (fr) 2011-02-12 2019-07-03 University of Iowa Research Foundation Composés thérapeutiques
BR112013020734A2 (pt) 2011-02-17 2017-06-13 Univ Pennsylvania composições e métodos para alterar a especificidade do tecido e aprimorar a transferência gênica mediada por aav9
EP2500434A1 (fr) 2011-03-12 2012-09-19 Association Institut de Myologie Vecteurs AAV sans capside, compositions et procédés pour la production des vecteurs et la thérapie génique
ES2736256T3 (es) 2011-03-14 2019-12-27 Drug Delivery Solutions Ltd Composición oftálmica
US20140031418A1 (en) 2011-04-20 2014-01-30 The Trustees Of The University Of Pennsylvania Regimens and Compositions for AAV-Mediated Passive Immunization of Airborne Pathogens
CA2831969A1 (fr) 2011-06-06 2012-12-30 Biocartis S.A. Lyse selective des cellules par des tensioactifs ioniques selective lysis of cells by ionic surfactants
JP6230996B2 (ja) 2011-08-01 2017-11-15 アルキオーネ・ライフサイエンシズ・インコーポレイテッドＡｌｃｙｏｎｅ Ｌｉｆｅｓｃｉｅｎｃｅｓ， Ｉｎｃ． 微小流体薬剤送達装置
WO2013125906A1 (fr) 2012-02-24 2013-08-29 성균관대학교 산학협력단 Séparateur ayant une résistance thermique améliorée, et dispositif électrochimique en étant pourvu
TWI775096B (zh) 2012-05-15 2022-08-21 澳大利亞商艾佛蘭屈澳洲私營有限公司 使用腺相關病毒(aav)sflt-1治療老年性黃斑部退化(amd)
KR20150014505A (ko) 2012-05-18 2015-02-06 더 트러스티스 오브 더 유니버시티 오브 펜실바니아 아과 e 원숭이 아데노바이러스 a1302, a1320, a1331 및 a1337 및 이것들의 사용
ES2750367T3 (es) 2012-05-22 2020-03-25 Inst Nat Sante Rech Med Nuevo péptido antigénico de melanoma y usos del mismo
US9192393B2 (en) 2012-05-31 2015-11-24 MRI Interventions, Inc. MRI compatible surgical drills and related methods
US9031636B2 (en) 2012-07-19 2015-05-12 MRI Interventions, Inc. MRI-compatible head fixation apparatus
US9498290B2 (en) 2012-07-19 2016-11-22 MRI Interventions, Inc. Surgical navigation devices and methods
US10206693B2 (en) 2012-07-19 2019-02-19 MRI Interventions, Inc. MRI-guided medical interventional systems and methods
US9192446B2 (en) 2012-09-05 2015-11-24 MRI Interventions, Inc. Trajectory guide frame for MRI-guided surgeries
US9572928B2 (en) 2013-03-13 2017-02-21 MRI Interventions, Inc. Substance delivery devices, systems and methods
US9498575B2 (en) 2013-03-14 2016-11-22 MRI Interventions, Inc. Substance delivery devices, systems and methods
CA2904396A1 (fr) 2013-03-15 2014-09-18 The University Of North Carolina At Chapel Hill Methodes et compositions de double liaison de glycane de vecteurs avv
US9618590B2 (en) 2013-03-15 2017-04-11 MRI Interventions, Inc. Tip assembly for MRI-compatible medical devices and MRI-compatible medical devices incorporating same
JP6129004B2 (ja) 2013-07-18 2017-05-17 ルネサスエレクトロニクス株式会社 半導体メモリ
WO2015038958A1 (fr) 2013-09-13 2015-03-19 California Institute Of Technology Récupération sélective
EP3049527A4 (fr) 2013-09-26 2017-08-16 University of Florida Research Foundation, Inc. Bibliothèque de capsides aav combinatoire synthétique pour la thérapie génique ciblée
US9715694B2 (en) 2013-10-10 2017-07-25 Go Daddy Operating Company, LLC System and method for website personalization from survey data
EP3046500B1 (fr) 2013-10-16 2020-08-26 ClearPoint Neuro, Inc. Système chirurgical de navigation et dispositif associé
US9514018B2 (en) 2014-01-28 2016-12-06 Software Ag Scaling framework for querying
JP2015166890A (ja) 2014-03-03 2015-09-24 ソニー株式会社 情報処理装置、情報処理システム、情報処理方法及びプログラム
US9523093B2 (en) 2014-05-20 2016-12-20 University Of Iowa Research Foundation Huntington's disease therapeutic compounds
WO2015191508A1 (fr) 2014-06-09 2015-12-17 Voyager Therapeutics, Inc. Capsides chimériques
KR102184884B1 (ko) 2014-06-26 2020-12-01 엘지디스플레이 주식회사 유기발광표시장치의 데이터 처리장치
US10526583B2 (en) * 2014-07-02 2020-01-07 University Of Florida Research Foundation, Incorporated Compositions and methods for purifying recombinant adeno-associated virus
MX2017005834A (es) 2014-11-05 2017-11-17 Voyager Therapeutics Inc Polinucleotidos aad para el tratamiento de la enfermedad de parkinson.
CN112375760A (zh) 2014-11-14 2021-02-19 沃雅戈治疗公司 调节性多核苷酸
AU2015346162B2 (en) 2014-11-14 2022-02-10 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (ALS)
JP6187432B2 (ja) 2014-11-14 2017-08-30 株式会社デンソー 制御装置
WO2016094783A1 (fr) 2014-12-12 2016-06-16 Voyager Therapeutics, Inc. Compositions et méthodes pour la production de scaav
AU2016219396B2 (en) 2015-02-10 2022-03-17 Genzyme Corporation Variant RNAi
BR112017017867A2 (pt) 2015-02-20 2018-04-10 Fond Telethon métodos e composições para tratamento de doenças oculares genéticas
US20180245073A1 (en) 2015-02-23 2018-08-30 Voyager Therapeutics, Inc. Regulatable expression using adeno-associated virus (aav)
WO2017015102A1 (fr) 2015-07-17 2017-01-26 The Trustees Of The University Of Pennsylvania Compositions et procédés pour obtenir des niveaux élevés de transduction dans des cellules hépatiques humaines
US20190000940A1 (en) 2015-07-31 2019-01-03 Voyager Therapeutics, Inc. Compositions and methods for the treatment of aadc deficiency
PL3356390T3 (pl) 2015-09-28 2021-07-05 The University Of North Carolina At Chapel Hill Sposoby i kompozycje dla wektorów wirusowych unikających przeciwciał
US9819479B2 (en) 2015-09-29 2017-11-14 Intel IP Corporation Digitally controlled two-points edge interpolator
WO2017066764A2 (fr) 2015-10-16 2017-04-20 William Marsh Rice University Modification de la région n-terminale des protéines de capsides pour améliorer les propriétés des virus adéno-associés
US20180230489A1 (en) 2015-10-28 2018-08-16 Voyager Therapeutics, Inc. Regulatable expression using adeno-associated virus (aav)
EP3374494A4 (fr) 2015-11-11 2019-05-01 Coda Biotherapeutics, Inc. Compositions crispr et leurs méthodes d'utilisation pour la thérapie génique
US10240145B2 (en) 2015-11-25 2019-03-26 The Board Of Trustees Of The Leland Stanford Junior University CRISPR/Cas-mediated genome editing to treat EGFR-mutant lung cancer
WO2017100671A1 (fr) 2015-12-11 2017-06-15 California Institute Of Technology Peptides de ciblage pour diriger des virus adéno-associés (aav)
US9819463B2 (en) 2016-02-18 2017-11-14 Huawei Technologies Co., Ltd. Method and apparatus for transmitting data in a wireless communication system
US20190224339A1 (en) 2016-04-29 2019-07-25 Voyager Therapeutics, Inc. Compositions for the treatment of disease
WO2017189959A1 (fr) 2016-04-29 2017-11-02 Voyager Therapeutics, Inc. Compositions pour le traitement de maladies
EP3448987A4 (fr) 2016-04-29 2020-05-27 Voyager Therapeutics, Inc. Compositions pour le traitement de maladies
RU2764587C2 (ru) * 2016-05-18 2022-01-18 Вояджер Терапьютикс, Инк. Способы и композиции для лечения хореи гентингтона
SG11201809699XA (en) 2016-05-18 2018-12-28 Voyager Therapeutics Inc Modulatory polynucleotides
JP7051132B2 (ja) * 2016-09-16 2022-04-11 ロイコケア・アクチェンゲゼルシャフト ワクチン接種または遺伝子治療のための効率的なウイルスベクターベースの組成物を得るための新規方法
CA3042689A1 (fr) * 2016-11-04 2018-07-12 Baxalta Incorporated Formulations de virus adeno-associe
EP3619310A4 (fr) 2017-05-05 2021-01-27 Voyager Therapeutics, Inc. Polynucléotides modulateurs
SG11201909868YA (en) 2017-05-05 2019-11-28 Voyager Therapeutics Inc Compositions and methods of treating huntington's disease
JP2020518258A (ja) 2017-05-05 2020-06-25 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． 筋萎縮性側索硬化症（ａｌｓ）治療組成物および方法
JOP20190269A1 (ar) 2017-06-15 2019-11-20 Voyager Therapeutics Inc بولي نوكليوتيدات aadc لعلاج مرض باركنسون
US10420284B2 (en) 2017-10-31 2019-09-24 Deere & Company Slip controller for side conveyors of a draper harvesting head

Also Published As

Publication number Publication date
US20210355454A1 (en) 2021-11-18
CN112770812A (zh) 2021-05-07
MX2021000810A (es) 2021-04-28
CA3107462A1 (fr) 2020-01-30
AU2019310459A8 (en) 2021-02-25
IL280350A (en) 2021-03-01
EP3826719A1 (fr) 2021-06-02
WO2020023612A1 (fr) 2020-01-30
JP2021530548A (ja) 2021-11-11
AU2019310459A1 (en) 2021-02-18

Similar Documents

Publication Publication Date Title
IL280350A (en) Systems and methods for producing gene therapy formulations
EP3720549A4 (fr) Systèmes et procédés de traitement de patient
EP3809967A4 (fr) Systèmes et procédés de physiothérapie
IL259100B1 (en) Therapeutic targets for human dystrophin gene repair through gene editing and methods for use
EP3423828A4 (fr) Procédés et systèmes permettant d'évaluer la charge mutationnelle d'une tumeur
EP3250285A4 (fr) Systèmes et procédés de thérapie de patient
EP3866919A4 (fr) Système et procédé de radiothérapie
EP3364870A4 (fr) Dispositifs, systèmes et méthodes pour thérapie de stimulation
EP3371572A4 (fr) Systèmes et procédés d'imagerie optogénétique
EP3174600A4 (fr) Système et procédé pour thérapie optogénétique
EP3177183A4 (fr) Systèmes et procédés de coussin thérapeutique
EP3274047A4 (fr) Procédé et système de stimulation auditive
IL281586A (en) Preparations and methods for the production of gene therapy vectors
ZA201901504B (en) System and method for controlled medical therapy
EP3405573A4 (fr) Procédés et systèmes de séquençage haute fidélité
EP3256042A4 (fr) Procédés et systèmes de neuromodulation thérapeutique
EP3538075A4 (fr) Structures et procédés de thérapie génique
EP3292494A4 (fr) Systèmes et procédés de fourniture de thérapie de rayonnement personnalisé
EP3600549A4 (fr) Systèmes et procédés pour l'administration de radiothérapie
EP3700617A4 (fr) Systèmes et procédés d'administration de thérapie anti-neurodégénérative
EP3743145A4 (fr) Systèmes et procédés thérapeutiques combinatoires
IL279685A (en) Gene therapy
EP3618922A4 (fr) Système et procédé pour une thérapie par particules sans portique
EP3570795A4 (fr) Systèmes et procédés pour une thérapie par contraste rapide
EP3606423A4 (fr) Système et procédé de thérapie respiratoire