PL3235827T3 - Wiriony AAV o zmniejszonej immunoreaktywności i ich zastosowania - Google Patents

Wiriony AAV o zmniejszonej immunoreaktywności i ich zastosowania

Info

Publication number
PL3235827T3
PL3235827T3 PL17162137T PL17162137T PL3235827T3 PL 3235827 T3 PL3235827 T3 PL 3235827T3 PL 17162137 T PL17162137 T PL 17162137T PL 17162137 T PL17162137 T PL 17162137T PL 3235827 T3 PL3235827 T3 PL 3235827T3
Authority
PL
Poland
Prior art keywords
uses therefor
aav virions
decreased immunoreactivity
immunoreactivity
decreased
Prior art date
Application number
PL17162137T
Other languages
English (en)
Inventor
Alejandra Elena Arbetman
Peter Colosi
Michael A. Lochrie
Richard SUROSKY
Original Assignee
Genzyme Corporation
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Genzyme Corporation filed Critical Genzyme Corporation
Publication of PL3235827T3 publication Critical patent/PL3235827T3/pl

Links

Classifications

    • 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
    • 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
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
    • 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)
    • A61K38/48—Hydrolases (3) acting on peptide bonds (3.4)
    • A61K38/482—Serine endopeptidases (3.4.21)
    • A61K38/4846—Factor VII (3.4.21.21); Factor IX (3.4.21.22); Factor Xa (3.4.21.6); Factor XI (3.4.21.27); Factor XII (3.4.21.38)
    • 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
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • 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
    • A61K48/0075—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the delivery route, e.g. oral, subcutaneous
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P11/00—Drugs for disorders of the respiratory system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P13/00—Drugs for disorders of the urinary system
    • A61P13/12—Drugs for disorders of the urinary system of the kidneys
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P19/00—Drugs for skeletal disorders
    • A61P19/06—Antigout agents, e.g. antihyperuricemic or uricosuric agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A61P21/04—Drugs for disorders of the muscular or neuromuscular system for myasthenia gravis
    • 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
    • A61P25/16—Anti-Parkinson drugs
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P29/00—Non-central analgesic, antipyretic or antiinflammatory agents, e.g. antirheumatic agents; Non-steroidal antiinflammatory drugs [NSAID]
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A61P3/06—Antihyperlipidemics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A61P3/08—Drugs for disorders of the metabolism for glucose homeostasis
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A61P3/08—Drugs for disorders of the metabolism for glucose homeostasis
    • A61P3/10—Drugs for disorders of the metabolism for glucose homeostasis for hyperglycaemia, e.g. antidiabetics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00—Drugs for immunological or allergic disorders
    • A61P37/02—Immunomodulators
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00—Drugs for immunological or allergic disorders
    • A61P37/02—Immunomodulators
    • A61P37/04—Immunostimulants
    • 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
    • A61P7/02—Antithrombotic agents; Anticoagulants; Platelet aggregation inhibitors
    • 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
    • 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
    • A61P7/06—Antianaemics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • A61P9/04—Inotropic agents, i.e. stimulants of cardiac contraction; Drugs for heart failure
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • A61P9/10—Drugs for disorders of the cardiovascular system for treating ischaemic or atherosclerotic diseases, e.g. antianginal drugs, coronary vasodilators, drugs for myocardial infarction, retinopathy, cerebrovascula insufficiency, renal arteriosclerosis
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y304/00—Hydrolases acting on peptide bonds, i.e. peptidases (3.4)
    • C12Y304/21—Serine endopeptidases (3.4.21)
    • C12Y304/21022—Coagulation factor IXa (3.4.21.22)
    • 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
    • C12N2750/14121—Viruses as such, e.g. new isolates, mutants or their genomic sequences
    • 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
    • C12N2750/14122—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
    • 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
    • C12N2750/14161—Methods of inactivation or attenuation
    • C12N2750/14162—Methods of inactivation or attenuation by genetic engineering
    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination
    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/80—Vector systems having a special element relevant for transcription from vertebrates
    • C12N2830/85—Vector systems having a special element relevant for transcription from vertebrates mammalian

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Genetics & Genomics (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Diabetes (AREA)
  • Molecular Biology (AREA)
  • Biomedical Technology (AREA)
  • Immunology (AREA)
  • General Engineering & Computer Science (AREA)
  • Epidemiology (AREA)
  • Hematology (AREA)
  • Virology (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Obesity (AREA)
  • Neurology (AREA)
  • Cardiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Emergency Medicine (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Physical Education & Sports Medicine (AREA)
PL17162137T 2003-06-19 2004-06-21 Wiriony AAV o zmniejszonej immunoreaktywności i ich zastosowania PL3235827T3 (pl)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US48039503P 2003-06-19 2003-06-19
US56731004P 2004-04-30 2004-04-30
US57650104P 2004-06-03 2004-06-03
EP17162137.8A EP3235827B1 (en) 2003-06-19 2004-06-21 Aav virions with decreased immunoreactivity and uses therefor
PCT/US2004/019884 WO2004112727A2 (en) 2003-06-19 2004-06-21 Aav virions with decreased immunoreactivity and uses therefor
EP04755800.2A EP1633772B1 (en) 2003-06-19 2004-06-21 Aav virions with decreased immunoreactivity and uses therefor
EP13163182.2A EP2657248B1 (en) 2003-06-19 2004-06-21 AAV virions with decreased immunoreactivity and uses therefor

Publications (1)

Publication Number Publication Date
PL3235827T3 true PL3235827T3 (pl) 2021-07-05

Family

ID=33545345

Family Applications (5)

Application Number Title Priority Date Filing Date
PL13163182T PL2657248T3 (pl) 2003-06-19 2004-06-21 Wiriony AAV o zmniejszonej immunoreaktywności i ich zastosowanie
PL13163168T PL2657247T3 (pl) 2003-06-19 2004-06-21 Wiriony AAV o zmniejszonej immunoreaktywności i ich zastosowanie
PL11158742T PL2357189T3 (pl) 2003-06-19 2004-06-21 Wiriony AAV o zmniejszonej immunoreaktywności i ich zastosowanie
PL04755800.2T PL1633772T3 (pl) 2003-06-19 2004-06-21 Wiriony AAV o zmniejszonej immunoreaktywności i ich zastosowanie
PL17162137T PL3235827T3 (pl) 2003-06-19 2004-06-21 Wiriony AAV o zmniejszonej immunoreaktywności i ich zastosowania

Family Applications Before (4)

Application Number Title Priority Date Filing Date
PL13163182T PL2657248T3 (pl) 2003-06-19 2004-06-21 Wiriony AAV o zmniejszonej immunoreaktywności i ich zastosowanie
PL13163168T PL2657247T3 (pl) 2003-06-19 2004-06-21 Wiriony AAV o zmniejszonej immunoreaktywności i ich zastosowanie
PL11158742T PL2357189T3 (pl) 2003-06-19 2004-06-21 Wiriony AAV o zmniejszonej immunoreaktywności i ich zastosowanie
PL04755800.2T PL1633772T3 (pl) 2003-06-19 2004-06-21 Wiriony AAV o zmniejszonej immunoreaktywności i ich zastosowanie

Country Status (11)

Country Link
US (6) US7259151B2 (pl)
EP (5) EP1633772B1 (pl)
JP (7) JP2007524386A (pl)
CY (5) CY1117575T1 (pl)
DK (5) DK2357189T3 (pl)
ES (5) ES2629087T3 (pl)
HU (5) HUE034597T2 (pl)
PL (5) PL2657248T3 (pl)
PT (5) PT2657248T (pl)
SI (5) SI2657248T1 (pl)
WO (1) WO2004112727A2 (pl)

Families Citing this family (225)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU4645697A (en) 1996-09-11 1998-04-02 Government Of The United States Of America, As Represented By The Secretary Of The Department Of Health And Human Services, The Aav4 vector and uses thereof
DE69939169D1 (de) 1998-05-28 2008-09-04 Us Gov Health & Human Serv Aav5 vektoren und deren verwendung
JP4969002B2 (ja) 1999-06-08 2012-07-04 ユニバーシテイ・オブ・アイオワ・リサーチ・フアウンデーシヨン ｒＡＡＶ形質導入を増加するための化合物および方法
CN103589692B (zh) 2001-11-13 2018-07-24 宾夕法尼亚大学托管会 检测和/或鉴定腺伴随病毒(aav)序列以及分离所鉴定的新型序列的方法
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
WO2004089423A2 (en) 2003-03-31 2004-10-21 University Of Iowa Research Foundation Pharmico-gene theraphy of epithelial sodium channel associated disoders
WO2005017101A2 (en) 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 Avian adenoassociated virus (aaav) and uses thereof
PL2657248T3 (pl) 2003-06-19 2017-09-29 Genzyme Corporation Wiriony AAV o zmniejszonej immunoreaktywności i ich zastosowanie
US9233131B2 (en) * 2003-06-30 2016-01-12 The Regents Of The University Of California Mutant adeno-associated virus virions and methods of use thereof
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
WO2005056807A2 (en) 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 Bovine adeno-associated viral (baav) vector and uses thereof
US20080188431A1 (en) * 2004-09-08 2008-08-07 Chiorini John A Transcytosis of Adeno-Associated Viruses
EP1828390B1 (en) * 2004-12-15 2012-06-13 The University Of North Carolina At Chapel Hill Chimeric vectors
DE23193606T1 (de) * 2005-04-07 2024-02-15 The Trustees Of The University Of Pennsylvania Verfahren zur erhöhung der funktion eines aav-vektors
US8283151B2 (en) 2005-04-29 2012-10-09 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Isolation, cloning and characterization of new adeno-associated virus (AAV) serotypes
EP2019964A2 (en) * 2006-04-28 2009-02-04 University of Iowa Research Foundation Methods and compounds to modulate parvovirus transduction of mammalian cells or to alter virus infection. method to identify a viral receptor or co-receptor
DK2623605T3 (en) 2007-04-09 2019-03-25 Univ Florida RAAV VECTOR COMPOSITIONS WITH TYROSIN MODIFIED CAPSIDE PROTEINS AND PROCEDURES FOR USE THEREOF
EP2019143A1 (en) 2007-07-23 2009-01-28 Genethon CNS gene delivery using peripheral administration of AAV vectors
EP2058401A1 (en) 2007-10-05 2009-05-13 Genethon Widespread gene delivery to motor neurons using peripheral injection of AAV vectors
EP2384200B1 (en) 2009-01-30 2013-07-10 UniQure IP B.V. Alanine-glyoxylate aminotransferase therapeutics
EP2396343B1 (en) 2009-02-11 2017-05-17 The University of North Carolina At Chapel Hill Modified virus vectors and methods of making and using the same
AU2010227419A1 (en) 2009-03-27 2011-10-20 Proyecto De Biomedicina Cima, S.L. Methods and compositions for the treatment of cirrhosis and liver fibrosis
EP2287323A1 (en) 2009-07-31 2011-02-23 Association Institut de Myologie Widespread gene delivery to the retina using systemic administration of AAV vectors
WO2011032100A1 (en) 2009-09-11 2011-03-17 Government Of The U.S.A., As Represented By The Secretary, Department Of Health And Human Services Inhibitors of kshv vil6 and human il6
JP2014503173A (ja) 2009-11-05 2014-02-13 プロイェクト、デ、ビオメディシナ、シーマ、ソシエダッド、リミターダ 制御された発現系
ES2436317T3 (es) * 2010-05-07 2013-12-30 Deutsches Krebsforschungszentrum Parvovirus de roedor modificado capaz de propagarse y difundirse en gliomas humanos
US8663624B2 (en) 2010-10-06 2014-03-04 The Regents Of The University Of California Adeno-associated virus virions with variant capsid and methods of use thereof
US10738326B2 (en) 2010-10-27 2020-08-11 Jichi Medical University Adeno-associated virus vector for gene transfer to nervous system cells
DK3693025T3 (da) 2011-04-22 2022-01-10 Univ California Adenoassocierede virus-virioner med variant capsid og fremgangsmåder til anvendelse heraf
EP3470523A1 (en) 2012-05-09 2019-04-17 Oregon Health & Science University Adeno associated virus plasmids and vectors
US12358954B2 (en) 2012-05-15 2025-07-15 University Of Florida Research Foundation, Incorporated Capsid-modified rAAV vector compositions and methods therefor
US10294281B2 (en) * 2012-05-15 2019-05-21 University Of Florida Research Foundation, Incorporated High-transduction-efficiency rAAV vectors, compositions, and methods of use
CN104704123A (zh) 2012-06-21 2015-06-10 肌肉学研究协会 基因治疗载体的广泛的基因递送
US11136557B2 (en) 2013-05-31 2021-10-05 The Regents Of The University Of California Adeno-associated virus variants and methods of use thereof
NZ716102A (en) * 2013-07-22 2021-12-24 The Children’S Hospital Of Philadelphia Variant aav and compositions, methods and uses for gene transfer to cells, organs and tissues
EP3049527A4 (en) 2013-09-26 2017-08-16 University of Florida Research Foundation, Inc. Synthetic combinatorial aav capsid library for targeted gene therapy
WO2015060722A1 (en) 2013-10-24 2015-04-30 Uniqure Ip B.V. Aav-5 pseudotyped vector for gene therapy for neurological diseases
GB201403684D0 (en) 2014-03-03 2014-04-16 King S College London Vector
ES2822551T3 (es) 2014-03-17 2021-05-04 Adverum Biotechnologies Inc Composiciones y métodos para la expresión génica mejorada en células de los conos
LT3137497T (lt) 2014-05-02 2021-07-26 Genzyme Corporation Aav vektoriai, skirti tinklainės ir cns genų terapijai
US10577627B2 (en) 2014-06-09 2020-03-03 Voyager Therapeutics, Inc. Chimeric capsids
EP3215191B1 (en) 2014-11-05 2024-08-14 Voyager Therapeutics, Inc. Aadc polynucleotides for the treatment of parkinson's disease
CN114717264A (zh) 2014-11-14 2022-07-08 沃雅戈治疗公司 治疗肌萎缩性侧索硬化(als)的组合物和方法
KR20230145206A (ko) 2014-11-14 2023-10-17 보이저 테라퓨틱스, 인크. 조절성 폴리뉴클레오티드
HUE046643T2 (hu) 2014-11-28 2021-11-29 Uniqure Ip Bv DNS szennyezõdések parvovirális virionokat tartalmazó készítményben
US11697825B2 (en) 2014-12-12 2023-07-11 Voyager Therapeutics, Inc. Compositions and methods for the production of scAAV
FI3265178T3 (fi) 2015-03-02 2026-08-03 Adverum Biotechnologies Inc Koostumuksia ja menetelmiä polynukleotidien viemiseksi intravitreaalisesti retinaalisiin tappisoluihin
JP6836999B2 (ja) 2015-03-24 2021-03-03 ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニアＴｈｅ Ｒｅｇｅｎｔｓ Ｏｆ Ｔｈｅ Ｕｎｉｖｅｒｓｉｔｙ Ｏｆ Ｃａｌｉｆｏｒｎｉａ アデノ随伴ウイルス変異体及びその使用方法
US10081659B2 (en) 2015-04-06 2018-09-25 The United States Of America, As Represented By The Secretary, Dept. Of Health And Human Services Adeno-associated vectors for enhanced transduction and reduced immunogenicity
GB201508026D0 (en) 2015-05-11 2015-06-24 Ucl Business Plc Capsid
IL314171A (en) 2015-09-28 2024-09-01 Univ Florida Methods and compositions for antibody-evading virus vectors
WO2017070476A2 (en) 2015-10-22 2017-04-27 University Of Florida Research Foundation, Inc. Synthetic combinatorial aav3 capsid library
AU2016362477A1 (en) 2015-12-02 2018-06-14 Voyager Therapeutics, Inc. Assays for the detection of AAV neutralizing antibodies
WO2017139381A1 (en) 2016-02-08 2017-08-17 University Of Iowa Research Foundation Methods to produce chimeric adeno-associated virus/bocavirus parvovirus
WO2017155973A1 (en) 2016-03-07 2017-09-14 University Of Iowa Research Foundation Aav-mediated expression using a synthetic promoter and enhancer
EP3448987A4 (en) 2016-04-29 2020-05-27 Voyager Therapeutics, Inc. COMPOSITIONS FOR TREATING A DISEASE
EP3448874A4 (en) 2016-04-29 2020-04-22 Voyager Therapeutics, Inc. COMPOSITIONS FOR TREATING A DISEASE
AU2017261249B2 (en) 2016-05-03 2021-05-06 Children's Medical Research Institute Adeno-associated virus polynucleotides, polypeptides and virions
WO2017197355A2 (en) 2016-05-13 2017-11-16 4D Molecular Therapeutics Inc. Adeno-associated virus variant capsids and methods of use thereof
US10584337B2 (en) 2016-05-18 2020-03-10 Voyager Therapeutics, Inc. Modulatory polynucleotides
IL297576B2 (en) 2016-05-18 2024-02-01 Voyager Therapeutics Inc Compositions and methods of treating huntington's disease
US12173305B2 (en) 2016-05-26 2024-12-24 University Of Iowa Research Foundation cis and trans requirements for terminal resolution of human bocavirus 1
CN109640949B (zh) 2016-07-29 2022-11-25 加利福尼亚大学董事会 具有变异衣壳的腺相关病毒病毒体和其使用方法
EP3831281B1 (en) 2016-08-30 2026-02-25 The Regents of The University of California Devices and systems for biomedical targeting and delivery
AU2017345470B2 (en) 2016-10-19 2023-08-03 Adverum Biotechnologies, Inc. Modified AAV capsids and uses thereof
US11142775B2 (en) 2017-01-13 2021-10-12 University Of Iowa Research Foundation Bocaparvovirus small noncoding RNA and uses thereof
EP3575398A4 (en) * 2017-01-30 2020-11-11 Nippon Medical School Foundation ADENO-ASSOCIATED VIRUS (AAV) CAPSIDE PROTEIN MUTANT
WO2018144709A2 (en) * 2017-02-01 2018-08-09 The Trustees Of The University Of Pennsylvania Gene therapy for treating citrullenemia
EP3585883A4 (en) * 2017-02-21 2021-04-14 University of Florida Research Foundation, Incorporated MODIFIED AAV CAPSID PROTEINS AND USES THEREOF
MX2019013172A (es) 2017-05-05 2020-09-07 Voyager Therapeutics Inc Composiciones y metodos para tratar la enfermedad de huntington.
EP3618839A4 (en) 2017-05-05 2021-06-09 Voyager Therapeutics, Inc. COMPOSITIONS AND METHODS OF TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS)
JOP20190269A1 (ar) 2017-06-15 2019-11-20 Voyager Therapeutics Inc بولي نوكليوتيدات aadc لعلاج مرض باركنسون
KR20250023600A (ko) 2017-06-30 2025-02-18 더 리젠츠 오브 더 유니버시티 오브 캘리포니아 변이체 캡시드를 보유한 아데노-부속 바이러스 비리온 및 이의 사용 방법
CA3070087A1 (en) 2017-07-17 2019-01-24 Voyager Therapeutics, Inc. Trajectory array guide system
US11512327B2 (en) 2017-08-03 2022-11-29 Voyager Therapeutics, Inc. Compositions and methods for delivery of AAV
MX2020001997A (es) 2017-08-28 2020-07-20 Univ California Variantes de capside de virus adenoasociado y metodos de uso de estas.
DK3684423T5 (da) 2017-09-20 2024-08-26 4D Molecular Therapeutics Inc Adeno-associerede virusvariantcapsider og fremgangsmåder til anvendelse deraf
MX2020003042A (es) 2017-09-29 2020-11-18 Voyager Therapeutics Inc Rescate del fenotipo neurológico central y periférico de la ataxia de friedreich mediante administración intravenosa.
TW202413649A (zh) 2017-10-16 2024-04-01 美商航海家醫療公司 肌萎縮性脊髓側索硬化症(als)之治療
WO2019079242A1 (en) 2017-10-16 2019-04-25 Voyager Therapeutics, Inc. TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS)
IL274702B2 (en) 2017-11-27 2026-04-01 Coda Biotherapeutics Inc Compounds and methods for neurological diseases
US11766489B2 (en) 2017-11-27 2023-09-26 4D Molecular Therapeutics, Inc. Adeno-associated virus variant capsids and use for inhibiting angiogenesis
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
WO2019152841A1 (en) 2018-02-01 2019-08-08 Homology Medicines, Inc. Adeno-associated virus compositions for pah gene transfer and methods of use thereof
US11306329B2 (en) 2018-02-19 2022-04-19 City Of Hope Adeno-associated virus compositions for restoring F8 gene function and methods of use thereof
JP7098375B2 (ja) * 2018-03-26 2022-07-11 株式会社三共 スロットマシン
WO2019191716A1 (en) * 2018-03-29 2019-10-03 University Of Florida Research Foundation, Incorporated Aav6 variants
EP3774853A1 (en) * 2018-03-30 2021-02-17 The Board of Trustees of the Leland Stanford Junior University Novel recombinant adeno-associated virus capsids with enhanced human pancreatic tropism
MX2020010465A (es) 2018-04-03 2021-01-08 Vectores de virus para direccionamiento a tejidos oftalmicos.
CA3095179A1 (en) 2018-04-03 2019-10-10 Stridebio, Inc. Antibody-evading virus vectors
BR112020020266A2 (pt) 2018-04-03 2021-01-19 Stridebio, Inc. Vetores de vírus com evasão de anticorpos
CA3097192A1 (en) 2018-04-27 2019-10-31 Voyager Therapeutics, Inc. Methods for measuring the potency of aadc viral vectors
CA3098807A1 (en) 2018-05-08 2019-11-14 Neuracle Science Co., Ltd. Adeno-associated virus (aav) delivery of anti-fam19a5 antibodies
WO2019222329A1 (en) 2018-05-15 2019-11-21 Voyager Therapeutics, Inc. Compositions and methods for delivery of aav
CN112424359A (zh) 2018-05-15 2021-02-26 沃雅戈治疗公司 用于治疗帕金森氏病的组合物和方法
WO2019222444A2 (en) 2018-05-16 2019-11-21 Voyager Therapeutics, Inc. Directed evolution
US20210207167A1 (en) 2018-05-16 2021-07-08 Voyager Therapeutics, Inc. Aav serotypes for brain specific payload delivery
WO2019241486A1 (en) 2018-06-13 2019-12-19 Voyager Therapeutics, Inc. Engineered 5' untranslated regions (5' utr) for aav production
JP7152734B2 (ja) * 2018-06-28 2022-10-13 ネット株式会社 遊技機
US20210254103A1 (en) 2018-07-02 2021-08-19 Voyager Therapeutics, Inc. Treatment of amyotrophic lateral sclerosis and disorders associated with the spinal cord
WO2020010035A1 (en) 2018-07-02 2020-01-09 Voyager Therapeutics, Inc. Cannula system
WO2020023612A1 (en) 2018-07-24 2020-01-30 Voyager Therapeutics, Inc. Systems and methods for producing gene therapy formulations
EP3830107A2 (en) 2018-08-03 2021-06-09 Voyager Therapeutics, Inc. Aav variants with enhanced tropism
KR20210102870A (ko) 2018-08-30 2021-08-20 테나야 테라퓨틱스, 인코포레이티드 미오카르딘 및 ascl1을 사용한 심장 세포 재프로그래밍
SG11202101819QA (en) * 2018-08-30 2021-03-30 Res Inst Nationwide Childrens Hospital Gene therapy for the treatment of galactosemia
US12281321B2 (en) 2018-09-28 2025-04-22 Voyager Therapeutics, Inc. Frataxin expression constructs having engineered promoters and methods of use thereof
AU2019354296A1 (en) 2018-10-01 2021-04-29 Ultragenyx Pharmaceutical Inc. Gene therapy for treating propionic acidemia
CN113166208B (zh) 2018-10-02 2024-11-22 沃雅戈治疗公司 重新定向aav衣壳的嗜性
WO2020072849A1 (en) 2018-10-04 2020-04-09 Voyager Therapeutics, Inc. Methods for measuring the titer and potency of viral vector particles
AU2019354793A1 (en) 2018-10-05 2021-05-13 Voyager Therapeutics, Inc. Engineered nucleic acid constructs encoding AAV production proteins
US20210371470A1 (en) 2018-10-12 2021-12-02 Voyager Therapeutics, Inc. Compositions and methods for delivery of aav
WO2020081490A1 (en) 2018-10-15 2020-04-23 Voyager Therapeutics, Inc. EXPRESSION VECTORS FOR LARGE-SCALE PRODUCTION OF rAAV IN THE BACULOVIRUS/Sf9 SYSTEM
CA3125770A1 (en) 2019-01-18 2020-07-23 Voyager Therapeutics, Inc. Methods and systems for producing aav particles
CN119390787A (zh) 2019-01-31 2025-02-07 俄勒冈健康与科学大学 用于aav衣壳的使用转录依赖性定向进化的方法
EP3931335A2 (en) 2019-02-25 2022-01-05 Novartis AG Compositions and methods to treat bietti crystalline dystrophy
US20220154210A1 (en) 2019-02-25 2022-05-19 Novartis Ag Compositions and methods to treat bietti crystalline dystrophy
CN113727992A (zh) 2019-03-21 2021-11-30 斯特里迪比奥公司 重组腺相关病毒载体
CN114450411A (zh) 2019-04-01 2022-05-06 特纳亚治疗股份有限公司 具有工程化衣壳的腺相关病毒
US20220243225A1 (en) 2019-04-29 2022-08-04 Voyager Therapeutics, Inc. SYSTEMS AND METHODS FOR PRODUCING BACULOVIRAL INFECTED INSECT CELLS (BIICs) IN BIOREACTORS
WO2020223280A1 (en) 2019-04-29 2020-11-05 Voyager Therapeutics, Inc. Aav variants with enhanced tropism
EP3966227A1 (en) 2019-05-07 2022-03-16 Voyager Therapeutics, Inc. Compositions and methods for the vectored augmentation of protein destruction, expression and/or regulation
EP3997226A1 (en) 2019-07-11 2022-05-18 Tenaya Therapeutics, Inc. Cardiac cell reprogramming with micrornas and other factors
WO2021025995A1 (en) 2019-08-02 2021-02-11 Voyager Therapeutics, Inc. Aav variants with enhanced tropism
EP4010465A1 (en) 2019-08-09 2022-06-15 Voyager Therapeutics, Inc. Cell culture medium for use in producing gene therapy products in bioreactors
EP4010357A2 (en) 2019-08-09 2022-06-15 University of Florida Research Foundation, Incorporated Aav capsid variants for targeting human glioblastoma cells
EP4022070A1 (en) 2019-08-26 2022-07-06 Voyager Therapeutics, Inc. Controlled expression of viral proteins
WO2021046155A1 (en) 2019-09-03 2021-03-11 Voyager Therapeutics, Inc. Vectorized editing of nucleic acids to correct overt mutations
WO2021067598A1 (en) 2019-10-04 2021-04-08 Ultragenyx Pharmaceutical Inc. Methods for improved therapeutic use of recombinant aav
MX2022004353A (es) 2019-10-17 2022-07-19 Casete de transferencia de aav.
CN114787180A (zh) 2019-10-17 2022-07-22 斯特里迪比奥公司 用于治疗c型尼曼-匹克病的腺相关病毒载体
WO2021108467A1 (en) 2019-11-25 2021-06-03 University Of Florida Research Foundation, Incorporated Novel aav3b variants that target human hepatocytes and evade the humoral immune response
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
AU2021218703A1 (en) 2020-02-14 2022-07-28 Ultragenyx Pharmaceutical Inc. Gene therapy for treating CDKL5 deficiency disorder
CN115552022A (zh) 2020-03-02 2022-12-30 特纳亚治疗股份有限公司 由心肌细胞表达的微rna控制的基因载体
US20230235353A1 (en) 2020-03-19 2023-07-27 Ultragenyx Pharmaceutical Inc. Compositions and methods for reducing reverse packaging of cap and rep sequences in recombinant aav
US12686874B2 (en) 2020-03-31 2026-07-21 Ultragenyx Pharmaceutical Inc. Gene therapy for treating propionic acidemia
WO2021202651A1 (en) 2020-04-01 2021-10-07 Voyager Therapeutics, Inc. Redirection of tropism of aav capsids
CA3180222A1 (en) 2020-04-15 2021-10-21 Voyager Therapeutics, Inc. Tau binding compounds
AU2021259422A1 (en) 2020-04-20 2022-12-08 Tenaya Therapeutics, Inc. Adeno-associated virus with engineered capsid
GB202005732D0 (en) 2020-04-20 2020-06-03 Synpromics Ltd Regulatory nucleic acid sequences
KR20230005941A (ko) * 2020-05-01 2023-01-10 트러스티스 오브 보스톤 칼리지 향상된 유전자 요법을 위한 아데노-관련 바이러스(aav)의 제어된 변형
US12630842B2 (en) 2020-05-13 2026-05-19 Voyager Therapeutics, Inc. Redirection of tropism of AAV capsids
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
WO2021247995A2 (en) 2020-06-04 2021-12-09 Voyager Therapeutics, Inc. Compositions and methods of treating neuropathic pain
US20230285596A1 (en) 2020-07-27 2023-09-14 Voyager Therapeutics, Inc Compositions and methods for the treatment of niemann-pick type c1 disease
BR112023001456A2 (pt) 2020-07-27 2023-04-11 Voyager Therapeutics Inc Composições e métodos para o tratamento de distúrbios neurológicos relacionados à deficiência de beta glicosilceramidase
EP4192514A1 (en) 2020-08-06 2023-06-14 Voyager Therapeutics, Inc. Cell culture medium for use in producing gene therapy products in bioreactors
CN113855794A (zh) * 2020-08-18 2021-12-31 深圳市俊元生物科技有限公司 一种基于棘突蛋白基因修饰干细胞的新型冠状病毒疫苗
JP2023542614A (ja) 2020-08-19 2023-10-11 サレプタ セラピューティクス， インコーポレイテッド レット症候群の治療のためのアデノ随伴ウイルスベクター
GB202013940D0 (en) 2020-09-04 2020-10-21 Synpromics Ltd Regulatory nucleic acid sequences
US11781156B2 (en) 2020-10-09 2023-10-10 Tenaya Therapeutics, Inc. Plakophillin-2 gene therapy methods and compositions
JP7415042B2 (ja) 2020-10-09 2024-01-16 テナヤ セラピューティクス， インコーポレイテッド プラコフィリン２遺伝子治療の方法および組成物
WO2022187548A1 (en) 2021-03-03 2022-09-09 Voyager Therapeutics, Inc. Controlled expression of viral proteins
WO2022187473A2 (en) 2021-03-03 2022-09-09 Voyager Therapeutics, Inc. Controlled expression of viral proteins
AU2022266615A1 (en) * 2021-04-27 2023-11-09 The General Hospital Corporation Aav capsids and uses thereof
EP4349852A4 (en) 2021-05-28 2025-09-03 Shanghai Regenelead Therapies Co Ltd RECOMBINANT ADENO-ASSOCIATED VIRUS HAVING A VARIANT CAPSID, AND ITS APPLICATION
TW202317768A (zh) 2021-07-08 2023-05-01 美商特納亞治療股份有限公司 用於基因療法的優化表現卡匣
JP2024531138A (ja) 2021-08-11 2024-08-29 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 筋ジストロフィーを処置するための組成物および方法
WO2023044483A2 (en) 2021-09-20 2023-03-23 Voyager Therapeutics, Inc. Compositions and methods for the treatment of her2 positive cancer
EP4426716A1 (en) 2021-11-02 2024-09-11 Voyager Therapeutics, Inc. Aav capsid variants and uses thereof
EP4433169A1 (en) 2021-11-17 2024-09-25 Voyager Therapeutics, Inc. Aav capsid variants and uses thereof
US20250049955A1 (en) 2021-11-17 2025-02-13 Voyager Therapeutics, Inc. Compositons and methods for the treatment of neurological disorders related to glucosylceramidase beta deficiency
CA3238867A1 (en) 2021-11-29 2023-06-01 Shanghai Regenelead Therapies Co., Ltd. Aadc/gdnf polynucleotide, and use thereof in treating parkinson's disease
CA3240342A1 (en) 2021-12-16 2023-06-22 Gopal SAPKOTA Targeted degradation of alpha-synuclein
US20250084129A1 (en) * 2022-01-10 2025-03-13 University Of Florida Research Foundation, Incorporated Aav5 capsid variants
US20250346920A1 (en) 2022-01-25 2025-11-13 Voyager Therapeutics, Inc. Baculovirus expression system
JP2025507338A (ja) 2022-02-08 2025-03-18 ボイジャー セラピューティクス インコーポレイテッド Ａａｖカプシドバリアント及びその使用
GB202201713D0 (en) 2022-02-10 2022-03-30 Univ Dundee An affinity directed phosphatase system for targeted protein dephosphorylation
WO2023155918A1 (zh) 2022-02-21 2023-08-24 上海瑞宏迪医药有限公司 Vegf结合分子及其医药用途
CA3245327A1 (en) 2022-03-07 2023-09-14 Ultragenyx Pharmaceutical Inc. MODIFIED BATCH-BASED AAV PRODUCTION SYSTEMS AND PROCESSES
WO2023201207A1 (en) 2022-04-11 2023-10-19 Tenaya Therapeutics, Inc. Adeno-associated virus with engineered capsid
EP4514844A1 (en) 2022-04-29 2025-03-05 Broadwing Bio LLC Complement factor h related 4-specific antibodies and uses thereof
CN119487063A (zh) 2022-04-29 2025-02-18 博闻生物有限责任公司 双特异性抗体以及治疗眼部疾病的方法
AU2023259251A1 (en) 2022-04-29 2024-11-14 Broadwing Bio, Inc. Angiopoietin-related protein 7-specific antibodies and uses thereof
WO2023220695A2 (en) 2022-05-13 2023-11-16 Voyager Therapeutics, Inc. Compositions and methods for the treatment of her2 positive cancer
EP4532515A1 (en) 2022-06-02 2025-04-09 Voyager Therapeutics, Inc. Aav capsid variants and uses thereof
WO2023240236A1 (en) 2022-06-10 2023-12-14 Voyager Therapeutics, Inc. Compositions and methods for the treatment of spinal muscular atrophy related disorders
US20250243263A1 (en) 2022-06-22 2025-07-31 Voyager Therapeutics, Inc. Tau binding compounds
WO2024006741A1 (en) 2022-06-28 2024-01-04 Voyager Therapeutics, Inc. Aav capsid variants and uses thereof
WO2024011112A1 (en) 2022-07-06 2024-01-11 Voyager Therapeutics, Inc. Aav capsid variants and uses thereof
CA3263767A1 (en) 2022-08-03 2024-02-08 Voyager Therapeutics, Inc. COMPOSITIONS AND PROCESSES ALLOWING CROSSING THE BLOOD-BRAIN BARRIER
WO2024054983A1 (en) 2022-09-08 2024-03-14 Voyager Therapeutics, Inc. Controlled expression of viral proteins
JP2025535203A (ja) 2022-09-15 2025-10-23 ボイジャー セラピューティクス インコーポレイテッド タウ結合化合物
WO2024145474A2 (en) 2022-12-29 2024-07-04 Voyager Therapeutics, Inc. Compositions and methods for regulating mapt
AU2023427408A1 (en) 2023-02-02 2025-09-04 Voyager Therapeutics, Inc. Compositions and methods for the treatment of neurological disorders related to glucosylceramidase beta deficiency
WO2024215655A1 (en) 2023-04-10 2024-10-17 Tenaya Therapeutics, Inc. Cardioprotective bag3 therapies
EP4695396A1 (en) 2023-04-10 2026-02-18 Tenaya Therapeutics, Inc. Guide rnas, vectors, and virions for targeting mutations in the pln gene
WO2024226790A1 (en) 2023-04-26 2024-10-31 Voyager Therapeutics, Inc. Aav capsid variants and uses thereof
WO2024227154A1 (en) 2023-04-28 2024-10-31 Broadwing Bio Llc Complement component 3 (c3)-specific antibodies and uses thereof
EP4705324A1 (en) 2023-05-02 2026-03-11 Voyager Therapeutics, Inc. Compositions and methods for regulating mapt
IL324036A (en) 2023-05-03 2025-12-01 Voyager Therapeutics Inc Compositions and methods for the treatment of disorders related to glucosylceramidase beta 1 deficiency
EP4705486A1 (en) 2023-05-03 2026-03-11 Voyager Therapeutics, Inc. Compositions and methods for the treatment of disorders related to syntaxin-binding protein 1 deficiency
WO2024229125A2 (en) 2023-05-03 2024-11-07 Voyager Therapeutics, Inc. Compositions and methods for the treatment of disorders related to frataxin deficiency
WO2024229164A2 (en) 2023-05-03 2024-11-07 Voyager Therapeutics, Inc. Compositions and methods for the treatment of disorders related to dystrophia myotonica protein kinase
WO2024229173A2 (en) 2023-05-03 2024-11-07 Voyager Therapeutics, Inc. Compositions and methods for the treatment of disorders related to ataxin-2
JP2026515815A (ja) 2023-05-03 2026-05-19 ボイジャー セラピューティクス インコーポレイテッド Ｃｄｋｌ５欠損に関連する障害の治療のための組成物及び方法
JP2026515970A (ja) 2023-05-04 2026-05-19 ボイジャー セラピューティクス インコーポレイテッド Ａａｖカプシドバリアント及びその使用
WO2025038430A1 (en) 2023-08-16 2025-02-20 Voyager Therapeutics, Inc. Aav capsid variants and uses thereof
WO2025038802A1 (en) 2023-08-16 2025-02-20 Voyager Therapeutics, Inc. Compositions and methods for the treatment of disorders related to ataxin-2
WO2025038800A1 (en) 2023-08-16 2025-02-20 Voyager Therapeutics, Inc. Compositions and methods for the treatment of disorders related to frataxin deficiency
WO2025038805A1 (en) 2023-08-16 2025-02-20 Voyager Therapeutics, Inc. Compositions and methods for the treatment of disorders related to glucosylceramidase beta 1 deficiency
WO2025038796A1 (en) 2023-08-16 2025-02-20 Voyager Therapeutics, Inc. Compositions and methods for the treatment of disorders related to cdkl5 deficiency
WO2025038795A1 (en) 2023-08-16 2025-02-20 Voyager Therapeutics, Inc. Compositions and methods for the treatment of disorders related to dystrophia myotonica protein kinase
TW202508610A (zh) 2023-08-23 2025-03-01 大陸商上海瑞宏迪醫藥有限公司 醫藥組成物及其用途
WO2025122548A1 (en) 2023-12-05 2025-06-12 Voyager Therapeutics, Inc. Compositions and methods for the treatment of disorders related to cdkl5 deficiency
AR134555A1 (es) 2023-12-05 2026-01-28 Voyager Therapeutics Inc Composiciones y métodos para el tratamiento de trastornos relacionados con la deficiencia de frataxina
WO2025122644A1 (en) 2023-12-05 2025-06-12 Voyager Therapeutics, Inc. Compositions and methods for regulating mapt
WO2025122532A1 (en) 2023-12-05 2025-06-12 Voyager Therapeutics, Inc. Compositions and methods for the treatment of disorders related to ataxin-2
WO2025122536A1 (en) 2023-12-05 2025-06-12 Voyager Therapeutics, Inc. Compositions and methods for the treatment of disorders related to dystrophia myotonica protein kinase
WO2025122530A1 (en) 2023-12-05 2025-06-12 Voyager Therapeutics, Inc. Compositions and methods for the treatment of disorders related to glucosylceramidase beta 1 deficiency
WO2025122543A1 (en) 2023-12-05 2025-06-12 Voyager Therapeutics, Inc. Compositions and methods for the treatment of disorders related to syntaxin-binding protein 1 deficiency
WO2025122634A1 (en) 2023-12-05 2025-06-12 Voyager Therapeutics, Inc. Compositions and methods for the treatment of tau-related disorders
WO2025137219A1 (en) 2023-12-21 2025-06-26 Voyager Therapeutics, Inc. Compositions and methods for the treatment of disorders related to dystrophia myotonica protein kinase
WO2025147436A1 (en) 2024-01-03 2025-07-10 Voyager Therapeutics, Inc. Aav capsid variants and uses thereof
WO2025235734A2 (en) 2024-05-09 2025-11-13 Voyager Therapeutics, Inc. Aav capsid variants and uses thereof
WO2026010722A1 (en) 2024-07-03 2026-01-08 Voyager Therapeutics, Inc. Compositions and methods for regulating mapt
WO2026015039A1 (en) 2024-07-09 2026-01-15 Bial - Portela & Ca., S.A. Gene therapy for treatment of syngap1-related epileptic encephalopathy
WO2026055342A1 (en) 2024-09-04 2026-03-12 Arsenal Biosciences, Inc. Synthetic pathway activators
WO2026096599A1 (en) 2024-10-30 2026-05-07 Neurocrine Biosciences, Inc. Compositions and methods for delivering syntaxin-binding protein-1
WO2026096611A1 (en) 2024-10-30 2026-05-07 Neurocrine Biosciences, Inc. Compositions and methods for delivering cyclin-dependent kinase-like 5 protein
WO2026096603A1 (en) 2024-10-30 2026-05-07 Neurocrine Biosciences, Inc. Compositions and methods for delivering syntaxin-binding protein-1
WO2026096600A1 (en) 2024-10-30 2026-05-07 Neurocrine Biosciences, Inc. Compositions and methods for delivering cyclin-dependent kinase-like 5 protein
WO2026101977A1 (en) 2024-11-06 2026-05-15 Voyager Therapeutics, Inc. Compositions and methods for regulating mapt
WO2026102093A1 (en) 2024-11-07 2026-05-15 Voyager Therapeutics, Inc. Compositions and methods for the treatment of tau-related disorders
NL2039429B1 (en) 2024-12-20 2026-07-13 Academisch Ziekenhuis Leiden Pabpn1 isoforms for use in therapy

Family Cites Families (17)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US116A (en) 1837-02-03 William croasdale
US4816567A (en) 1983-04-08 1989-03-28 Genentech, Inc. Recombinant immunoglobin preparations
US5139941A (en) 1985-10-31 1992-08-18 University Of Florida Research Foundation, Inc. AAV transduction vectors
US5225347A (en) 1989-09-25 1993-07-06 Innovir Laboratories, Inc. Therapeutic ribozyme compositions and expression vectors
GB2276169A (en) 1990-07-05 1994-09-21 Celltech Ltd Antibodies specific for carcinoembryonic antigen
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
US6177403B1 (en) 1996-10-21 2001-01-23 The Trustees Of The University Of Pennsylvania Compositions, methods, and apparatus for delivery of a macromolecular assembly to an extravascular tissue of an animal
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.
US6156303A (en) * 1997-06-11 2000-12-05 University Of Washington Adeno-associated virus (AAV) isolates and AAV vectors derived therefrom
EP1135468B1 (en) * 1998-11-10 2010-01-06 University Of North Carolina At Chapel Hill Virus vectors and methods of making and administering the same
US6498244B1 (en) * 1999-05-28 2002-12-24 Cell Genesys, Inc. Adeno-associated virus capsid immunologic determinants
AU2001257611A1 (en) 2000-04-28 2001-11-12 Avigen, Inc. Polynucleotides for use in recombinant adeno-associated virus virion production
WO2002053703A2 (en) * 2001-01-05 2002-07-11 Children's Hospital, Inc. Aav2 vectors and methods
CN103589692B (zh) * 2001-11-13 2018-07-24 宾夕法尼亚大学托管会 检测和/或鉴定腺伴随病毒(aav)序列以及分离所鉴定的新型序列的方法
AU2003221733A1 (en) * 2002-04-17 2003-11-03 University Of Florida Research Foundation, Inc. Improved raav vectors
PL2657248T3 (pl) 2003-06-19 2017-09-29 Genzyme Corporation Wiriony AAV o zmniejszonej immunoreaktywności i ich zastosowanie

Also Published As

Publication number Publication date
JP6759177B2 (ja) 2020-09-23
PT2357189T (pt) 2017-06-20
CY1124371T1 (el) 2022-07-22
EP2657248A1 (en) 2013-10-30
DK3235827T3 (da) 2021-04-19
SI3235827T1 (sl) 2021-05-31
SI1633772T1 (sl) 2016-06-30
HUE034785T2 (en) 2018-02-28
EP2657247A1 (en) 2013-10-30
EP3235827A3 (en) 2017-12-20
PT2657247T (pt) 2017-06-29
US20190314526A1 (en) 2019-10-17
US20220016264A1 (en) 2022-01-20
ES2862206T3 (es) 2021-10-07
DK2357189T3 (en) 2017-06-19
HUE034597T2 (en) 2018-02-28
PL2657247T3 (pl) 2017-09-29
SI2657247T1 (sl) 2017-07-31
CY1119279T1 (el) 2018-02-14
JP2016084362A (ja) 2016-05-19
JP2007524386A (ja) 2007-08-30
EP2357189B1 (en) 2017-03-08
EP2357189A2 (en) 2011-08-17
ES2563064T3 (es) 2016-03-10
JP2020096642A (ja) 2020-06-25
DK2657247T3 (en) 2017-07-10
EP2357189A3 (en) 2011-11-16
US20080008690A1 (en) 2008-01-10
PT3235827T (pt) 2021-04-09
US20060051333A1 (en) 2006-03-09
EP2657248B1 (en) 2017-03-22
EP1633772B1 (en) 2015-11-18
WO2004112727A2 (en) 2004-12-29
PL2657248T3 (pl) 2017-09-29
CY1117575T1 (el) 2017-04-26
EP1633772A2 (en) 2006-03-15
HUE054046T2 (hu) 2021-08-30
PL1633772T3 (pl) 2016-10-31
DK2657248T3 (en) 2017-06-26
JP2010273690A (ja) 2010-12-09
JP2018029614A (ja) 2018-03-01
HUE034546T2 (en) 2018-02-28
US20170232117A1 (en) 2017-08-17
CY1119241T1 (el) 2018-02-14
EP2657247B1 (en) 2017-03-22
US11083800B2 (en) 2021-08-10
SI2657248T1 (sl) 2017-07-31
JP2023106497A (ja) 2023-08-01
ES2627913T3 (es) 2017-08-01
ES2629087T3 (es) 2017-08-07
US9506083B2 (en) 2016-11-29
US20180280540A1 (en) 2018-10-04
EP1633772A4 (en) 2008-08-27
DK1633772T3 (en) 2016-02-29
EP3235827B1 (en) 2021-01-13
PL2357189T3 (pl) 2017-08-31
ES2629380T3 (es) 2017-08-09
HUE029556T2 (en) 2017-03-28
PT1633772E (pt) 2016-03-22
SI2357189T1 (sl) 2017-07-31
CY1119222T1 (el) 2018-02-14
JP5551029B2 (ja) 2014-07-16
EP3235827A2 (en) 2017-10-25
JP2014057579A (ja) 2014-04-03
WO2004112727A3 (en) 2005-05-06
PT2657248T (pt) 2017-06-26
US7259151B2 (en) 2007-08-21

Similar Documents

Publication Publication Date Title
SI2657248T1 (sl) AAV virioni z zmanjšano imunoreaktivnostjo in njihova uporaba
TWI348890B (en) Mixtures comprising strobilurins and ethylene modulators
GB0308732D0 (en) Absorption enhancers
SG118214A1 (en) Resistor with reduced leakage
EP1596756A4 (en) MEANS TO INCREASE ABSORPTION
GB0305461D0 (en) Improvements in the construction of saw devices
TWI367222B (en) Aminoquinoxaline compounds and polyaminoquinoxaline compounds, and use thereof
GB0302741D0 (en) Use
GB2401084B (en) Disposable printer
GB2390109B (en) Nonstructural buoyancy can
GB2400812B (en) Handle structure
GB0329247D0 (en) Compounds and inks
GB0301879D0 (en) HCV combination therapy
GB0312340D0 (en) Improved game and aricle for use with the game
GB0124430D0 (en) Umbrellan with wooden ribs
GB0316757D0 (en) Use
TW584241U (en) Detachable compact keyboard and the application structure
TW592199U (en) Vessel with improved structure
GB0314212D0 (en) Eneuresis bleep
GB0301602D0 (en) Multi-plug
AU153396S (en) Childroof stopper
GB0300277D0 (en) Conventional hand unit-keyboard
GB0316484D0 (en) Sailing
AU153034S (en) Hatbox with lid
GB0218637D0 (en) Improved dessiccant stopper