[go: up one dir, main page]

PL2657248T3 - Wiriony AAV o zmniejszonej immunoreaktywności i ich zastosowanie - Google Patents

Wiriony AAV o zmniejszonej immunoreaktywności i ich zastosowanie

Info

Publication number
PL2657248T3
PL2657248T3 PL13163182T PL13163182T PL2657248T3 PL 2657248 T3 PL2657248 T3 PL 2657248T3 PL 13163182 T PL13163182 T PL 13163182T PL 13163182 T PL13163182 T PL 13163182T PL 2657248 T3 PL2657248 T3 PL 2657248T3
Authority
PL
Poland
Prior art keywords
uses therefor
aav virions
decreased immunoreactivity
immunoreactivity
decreased
Prior art date
Application number
PL13163182T
Other languages
English (en)
Inventor
Alejandra Elena Arbetman
Peter C. Colosi
Michael A. Lochrie
Richard T. 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 PL2657248T3 publication Critical patent/PL2657248T3/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
    • 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
    • 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)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Genetics & Genomics (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (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)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Obesity (AREA)
  • Cardiology (AREA)
  • Neurology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Neurosurgery (AREA)
  • Emergency Medicine (AREA)
  • Endocrinology (AREA)
PL13163182T 2003-06-19 2004-06-21 Wiriony AAV o zmniejszonej immunoreaktywności i ich zastosowanie PL2657248T3 (pl)

Applications Claiming Priority (3)

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

Publications (1)

Publication Number Publication Date
PL2657248T3 true PL2657248T3 (pl) 2017-09-29

Family

ID=33545345

Family Applications (5)

Application Number Title Priority Date Filing Date
PL17162137T PL3235827T3 (pl) 2003-06-19 2004-06-21 Wiriony AAV o zmniejszonej immunoreaktywności i ich zastosowania
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
PL13163182T PL2657248T3 (pl) 2003-06-19 2004-06-21 Wiriony AAV o zmniejszonej immunoreaktywności i ich zastosowanie

Family Applications Before (4)

Application Number Title Priority Date Filing Date
PL17162137T PL3235827T3 (pl) 2003-06-19 2004-06-21 Wiriony AAV o zmniejszonej immunoreaktywności i ich zastosowania
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) EP2657247B1 (pl)
JP (7) JP2007524386A (pl)
CY (5) CY1117575T1 (pl)
DK (5) DK1633772T3 (pl)
ES (5) ES2862206T3 (pl)
HU (5) HUE034546T2 (pl)
PL (5) PL3235827T3 (pl)
PT (5) PT3235827T (pl)
SI (5) SI2657248T1 (pl)
WO (1) WO2004112727A2 (pl)

Families Citing this family (192)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20030215422A1 (en) 1996-09-11 2003-11-20 John A. Chiorini Aav4 vector and uses thereof
CA2745131C (en) 1998-05-28 2016-08-09 John A. Chiorini Aav5 vector and uses thereof
EP1190249A2 (en) 1999-06-08 2002-03-27 University Of Iowa Research Foundation Compounds and methods to enhance raav transduction
BRPI0214119B8 (pt) 2001-11-13 2021-05-25 Univ Pennsylvania vírus adenoassociado recombinante, método de geração do referido vírus e composição compreendendo o referido vírus
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
EP1608410A2 (en) 2003-03-31 2005-12-28 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
ES2862206T3 (es) 2003-06-19 2021-10-07 Genzyme Corp Viriones de AAV con inmunorreactividad disminuida y sus usos
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
WO2006029196A1 (en) * 2004-09-08 2006-03-16 The Government Of The United States Of America, As Represented By The Secretary, Department Of Health And Human Services, National Institutes Of H Ealth Transcytosis of adeno-associated viruses
US7892809B2 (en) * 2004-12-15 2011-02-22 The University Of North Carolina At Chapel Hill Chimeric vectors
EP2359867B1 (en) * 2005-04-07 2014-10-08 The Trustees of The University of Pennsylvania Method of increasing the function of an AAV vector
WO2006119432A2 (en) 2005-04-29 2006-11-09 The Government Of The U.S.A., As Rep. By The Sec., Dept. Of Health & Human Services Isolation, cloning and characterization of new adeno-associated virus (aav) serotypes
CA2651180A1 (en) * 2006-04-28 2007-11-08 University Of Iowa Research Foundation Methods and compounds to alter virus infection
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
WO2010093784A2 (en) 2009-02-11 2010-08-19 The University Of North Carolina At Chapel Hill Modified virus vectors and methods of making and using the same
CA2756596A1 (en) 2009-03-27 2010-09-30 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
CN102712933A (zh) 2009-11-05 2012-10-03 西马生物医学计划公司 调控的表达系统
DK2384761T3 (da) * 2010-05-07 2013-12-09 Deutsches Krebsforsch Modificeret gnaverparvovirus, som er i stand til formering og spredning i humane gliomer
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
CN103189507A (zh) * 2010-10-27 2013-07-03 学校法人自治医科大学 用于向神经系统细胞导入基因的腺相关病毒粒子
KR102391725B1 (ko) 2011-04-22 2022-04-28 더 리젠츠 오브 더 유니버시티 오브 캘리포니아 변이체 캡시드를 지니는 아데노-관련 바이러스 및 이의 사용 방법
US9677088B2 (en) 2012-05-09 2017-06-13 Oregon Health & Science University Adeno associated virus plasmids and vectors
US10294281B2 (en) * 2012-05-15 2019-05-21 University Of Florida Research Foundation, Incorporated High-transduction-efficiency rAAV vectors, compositions, and methods of use
AU2013279284A1 (en) 2012-06-21 2015-01-29 Association Institut De Myologie Widespread gene delivery of gene therapy vectors
ES2897508T3 (es) 2013-05-31 2022-03-01 Univ California Variantes de virus adenoasociados y métodos de uso de las mismas
HUE047996T2 (hu) * 2013-07-22 2020-05-28 Childrens Hospital Philadelphia AAV variáns és készítmények, eljárások és alkalmazások sejtekbe, szervekbe és szövetekbe történõ géntranszferhez
US20160369298A1 (en) 2013-09-26 2016-12-22 University Of Florida Research Foundation, Inc. Synthetic combinatorial aav capsid library for targeted gene therapy
CA2927366A1 (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
EP3800191A1 (en) 2014-03-17 2021-04-07 Adverum Biotechnologies, Inc. Compositions and methods for enhanced gene expression in cone cells
IL292951B2 (en) 2014-05-02 2024-10-01 Genzyme Corp Aav vectors for retinal and cns gene therapy
WO2015191508A1 (en) 2014-06-09 2015-12-17 Voyager Therapeutics, Inc. Chimeric capsids
US10335466B2 (en) * 2014-11-05 2019-07-02 Voyager Therapeutics, Inc. AADC polynucleotides for the treatment of parkinson's disease
SG10202001102XA (en) 2014-11-14 2020-03-30 Voyager Therapeutics Inc Modulatory polynucleotides
IL292999A (en) 2014-11-14 2022-07-01 Voyager Therapeutics Inc Compositions and methods of treating amyotrophic lateral sclerosis (als)
DK3224376T4 (da) 2014-11-28 2023-05-30 Uniqure Ip Bv DNA-forureninger i en sammensætning omfattende en parvoviral virion
US11697825B2 (en) 2014-12-12 2023-07-11 Voyager Therapeutics, Inc. Compositions and methods for the production of scAAV
KR20170137730A (ko) 2015-03-02 2017-12-13 애드베룸 바이오테크놀로지스, 인코포레이티드 망막 추상체에 폴리뉴클레오타이드의 유리체 내 전달을 위한 조성물 및 방법
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
CN114606267A (zh) 2015-09-28 2022-06-10 北卡罗来纳-查佩尔山大学 逃避抗体的病毒载体的方法和组合物
WO2017070476A2 (en) 2015-10-22 2017-04-27 University Of Florida Research Foundation, Inc. Synthetic combinatorial aav3 capsid library
CA3006569A1 (en) 2015-12-02 2017-06-08 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
AU2017229347A1 (en) 2016-03-07 2018-11-01 University Of Iowa Research Foundation AAV-mediated expression using a synthetic promoter and enhancer
US11326182B2 (en) 2016-04-29 2022-05-10 Voyager Therapeutics, Inc. Compositions for the treatment of disease
EP3448987A4 (en) 2016-04-29 2020-05-27 Voyager Therapeutics, Inc. COMPOSITIONS FOR THE TREATMENT OF DISEASES
WO2017192699A1 (en) * 2016-05-03 2017-11-09 Children's Medical Research Institute Adeno-associated virus polynucleotides, polypeptides and virions
CR20180589A (es) 2016-05-13 2019-02-27 4D Molecular Therapeutics Inc Cápsulas variantes de virus adenoasociados y métodos de uso de estas
SG11201809699XA (en) 2016-05-18 2018-12-28 Voyager Therapeutics Inc Modulatory polynucleotides
RU2764587C2 (ru) 2016-05-18 2022-01-18 Вояджер Терапьютикс, Инк. Способы и композиции для лечения хореи гентингтона
WO2017205739A1 (en) 2016-05-26 2017-11-30 University Of Iowa Research Foundation cis AND trans REQUIREMENTS FOR TERMINAL RESOLUTION OF HUMAN BOCAVIRUS 1
KR102511979B1 (ko) 2016-07-29 2023-03-20 더 리젠츠 오브 더 유니버시티 오브 캘리포니아 변이체 캡시드를 갖는 아데노-관련된 바이러스 비리온 및 이의 사용 방법
EP3831281A1 (en) 2016-08-30 2021-06-09 The Regents of The University of California Methods for biomedical targeting and delivery and devices and systems for practicing the same
AU2017345470B2 (en) 2016-10-19 2023-08-03 Adverum Biotechnologies, Inc. Modified AAV capsids and uses thereof
WO2018132747A1 (en) 2017-01-13 2018-07-19 University Of Iowa Research Foundation Bocaparvovirus small noncoding rna and uses thereof
WO2018139634A1 (ja) * 2017-01-30 2018-08-02 学校法人日本医科大学 アデノ随伴ウイルス（ａａｖ）キャプシドタンパク質の変異体
EP3576760A2 (en) * 2017-02-01 2019-12-11 The Trustees Of The University Of Pennsylvania Gene therapy for treating citrullenemia
JP7237843B2 (ja) 2017-02-21 2023-03-13 ユニバーシティー オブ フロリダ リサーチ ファンデーション， インク． 修飾されたａａｖキャプシドタンパク質およびその使用
EP3619308A4 (en) 2017-05-05 2021-01-27 Voyager Therapeutics, Inc. COMPOSITIONS AND METHODS OF TREATMENT FOR HUNTINGTON'S MORBUS
SG11201909870SA (en) 2017-05-05 2019-11-28 Voyager Therapeutics Inc Compositions and methods of treating amyotrophic lateral sclerosis (als)
JOP20190269A1 (ar) 2017-06-15 2019-11-20 Voyager Therapeutics Inc بولي نوكليوتيدات aadc لعلاج مرض باركنسون
US11497576B2 (en) 2017-07-17 2022-11-15 Voyager Therapeutics, Inc. Trajectory array guide system
WO2019028306A2 (en) 2017-08-03 2019-02-07 Voyager Therapeutics, Inc. COMPOSITIONS AND METHODS FOR ADMINISTRATION OF ADENO-ASSOCIATED VIRUSES
KR20200039617A (ko) 2017-08-28 2020-04-16 더 리젠츠 오브 더 유니버시티 오브 캘리포니아 아데노-연관 바이러스 캡시드 변이체 및 이의 사용 방법
PE20201264A1 (es) 2017-09-20 2020-11-19 4D Molecular Therapeutics Inc Variantes de capsides de virus adenoasociados y metodos de uso de estas
WO2019067840A1 (en) 2017-09-29 2019-04-04 Voyager Therapeutics, Inc. RESTRICTING CENTRAL AND PERIPHERAL NEUROLOGICAL PHENOTYPE FROM FRIEDREICH ATAXIA BY INTRAVENOUS ADMINISTRATION
WO2019079242A1 (en) 2017-10-16 2019-04-25 Voyager Therapeutics, Inc. TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS)
AU2018352236A1 (en) 2017-10-16 2020-04-23 The Curators Of The University Of Missouri Treatment of amyotrophic lateral sclerosis (ALS)
CA3082907A1 (en) 2017-11-27 2019-05-31 Coda Biotherapeutics, Inc. Compositions and methods for neurological diseases
ES2946747T3 (es) 2017-11-27 2023-07-25 4D Molecular Therapeutics Inc Variantes de cápsides de virus adenoasociado y su utilización para inhibir la angiogénesis
MX2020008133A (es) 2018-02-01 2020-10-14 Homology Medicines Inc Composiciones de virus adeno-asociado para restaurar la funcion del gen pah y metodos de uso de las mismas.
US10610606B2 (en) 2018-02-01 2020-04-07 Homology Medicines, Inc. Adeno-associated virus compositions for PAH gene transfer and methods of use thereof
EP3755795A4 (en) 2018-02-19 2022-07-20 Homology Medicines, Inc. 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
CN112543766A (zh) 2018-04-03 2021-03-23 斯特里迪比奥公司 抗体逃避性病毒载体
US20210363191A1 (en) 2018-04-03 2021-11-25 Stridebio, Inc. Antibody-evading virus vectors
AU2019247191A1 (en) 2018-04-03 2020-10-15 Ginkgo Bioworks, Inc. Virus vectors for targeting ophthalmic tissues
JP2021521852A (ja) 2018-04-27 2021-08-30 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． Ａａｄｃウイルスベクターの効力を測定する方法
CN119662732A (zh) 2018-05-08 2025-03-21 纽洛可科学有限公司 抗fam19a5抗体的腺相关病毒（aav）递送
TW202015742A (zh) 2018-05-15 2020-05-01 美商航海家醫療公司 投遞腺相關病毒（ａａｖ）之組成物和方法
BR112020023082A2 (pt) 2018-05-15 2021-02-09 Voyager Therapeutics, Inc. composições e métodos para o tratamento de doença de parkinson
WO2019222444A2 (en) 2018-05-16 2019-11-21 Voyager Therapeutics, Inc. Directed evolution
EP3793686A1 (en) 2018-05-16 2021-03-24 Voyager Therapeutics, Inc. Aav serotypes for brain specific payload delivery
US12060567B2 (en) 2018-06-13 2024-08-13 Voyager Therapeutics, Inc. Engineered untranslated regions (UTR) for AAV production
JP7152734B2 (ja) * 2018-06-28 2022-10-13 ネット株式会社 遊技機
CN112567035A (zh) 2018-07-02 2021-03-26 沃雅戈治疗公司 肌萎缩侧索硬化症及脊髓相关病症的治疗
WO2020010035A1 (en) 2018-07-02 2020-01-09 Voyager Therapeutics, Inc. Cannula system
JP2021530548A (ja) 2018-07-24 2021-11-11 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. 遺伝子治療製剤を生産するための系および方法
EP3830107A2 (en) 2018-08-03 2021-06-09 Voyager Therapeutics, Inc. Aav variants with enhanced tropism
CA3110290A1 (en) * 2018-08-30 2020-03-05 Research Institute At Nationwide Children's Hospital Gene therapy for the treatment of galactosemia
WO2020047467A2 (en) 2018-08-30 2020-03-05 Tenaya Therapeutics, Inc. Cardiac cell reprogramming with myocardin and ascl1
AU2019346655B2 (en) 2018-09-28 2025-03-13 Voyager Therapeutics, Inc. Frataxin expression constructs having engineered promoters and methods of use thereof
CN119684417A (zh) 2018-10-02 2025-03-25 沃雅戈治疗公司 重新定向aav衣壳的嗜性
US20210348242A1 (en) 2018-10-04 2021-11-11 Voyager Therapeutics, Inc. Methods for measuring the titer and potency of viral vector particles
EP3861107A1 (en) 2018-10-05 2021-08-11 Voyager Therapeutics, Inc. Engineered nucleic acid constructs encoding aav production proteins
WO2020077165A1 (en) 2018-10-12 2020-04-16 Voyager Therapeutics, Inc. Compositions and methods for delivery of aav
EP3867389A1 (en) 2018-10-15 2021-08-25 Voyager Therapeutics, Inc. Expression vectors for large-scale production of raav in the baculovirus/sf9 system
JP2022522995A (ja) 2019-01-18 2022-04-21 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粒子を生成するための方法及びシステム
AU2020216480A1 (en) 2019-01-31 2021-09-09 Oregon Health & Science University Methods for using transcription-dependent directed evolution of AAV capsids
CN113677801A (zh) 2019-02-25 2021-11-19 诺华股份有限公司 治疗bietti晶体营养不良的组合物和方法
EP3931335A2 (en) 2019-02-25 2022-01-05 Novartis AG Compositions and methods to treat bietti crystalline dystrophy
TW202102525A (zh) 2019-03-21 2021-01-16 美商史崔德生物公司 重組腺相關病毒載體
WO2020205889A1 (en) 2019-04-01 2020-10-08 Tenaya Therapeutics, Inc. Adeno-associated virus with engineered capsid
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
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
US20230137971A1 (en) 2019-07-11 2023-05-04 Tenaya Therapeutics Inc. Cardiac cell reprogramming with micrornas and other factors
US20220281922A1 (en) 2019-08-02 2022-09-08 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
TW202122582A (zh) 2019-08-26 2021-06-16 美商航海家醫療公司 病毒蛋白之控制表現
US20220333133A1 (en) 2019-09-03 2022-10-20 Voyager Therapeutics, Inc. Vectorized editing of nucleic acids to correct overt mutations
EP4038194A1 (en) 2019-10-04 2022-08-10 Ultragenyx Pharmaceutical Inc. Methods for improved therapeutic use of recombinant aav
US11905523B2 (en) 2019-10-17 2024-02-20 Ginkgo Bioworks, Inc. Adeno-associated viral vectors for treatment of Niemann-Pick Disease type-C
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
WO2023172491A1 (en) 2022-03-07 2023-09-14 Ultragenyx Pharmaceutical Inc. Modified batch aav production systems and methods
EP4103724A1 (en) 2020-02-14 2022-12-21 Ultragenyx Pharmaceutical Inc. Gene therapy for treating cdkl5 deficiency disorder
CA3173567A1 (en) 2020-03-02 2021-09-10 Tenaya Therapeutics, Inc. Gene vector control by cardiomyocyte-expressed micrornas
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
WO2021202532A1 (en) 2020-03-31 2021-10-07 Ultragenyx Pharmaceutical Inc. Gene therapy for treating propionic acidemia
EP4126910A1 (en) 2020-04-01 2023-02-08 Voyager Therapeutics, Inc. Redirection of tropism of aav capsids
EP4135841A1 (en) 2020-04-15 2023-02-22 Voyager Therapeutics, Inc. Tau binding compounds
GB202005732D0 (en) 2020-04-20 2020-06-03 Synpromics Ltd Regulatory nucleic acid sequences
EP4143206A1 (en) * 2020-05-01 2023-03-08 Trustees of Boston College Controlled modification of adeno-associated virus (aav) for enhanced gene therapy
CN116096734A (zh) 2020-05-13 2023-05-09 沃雅戈治疗公司 Aav衣壳的向性的重定向
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
WO2022026410A2 (en) 2020-07-27 2022-02-03 Voyager Therapeutics, Inc Compositions and methods for the treatment of niemann-pick type c1 disease
JP2023536091A (ja) 2020-07-27 2023-08-23 ボイジャー セラピューティクス インコーポレイテッド グルコシルセラミダーゼベータ欠損に関連する神経障害の治療のための組成物及び方法
WO2022032153A1 (en) 2020-08-06 2022-02-10 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
TW202227632A (zh) 2020-08-19 2022-07-16 美商史崔德生物公司 用於治療雷特症候群之腺相關病毒載體
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
TW202246516A (zh) 2021-03-03 2022-12-01 美商航海家醫療公司 病毒蛋白之控制表現
US20240141378A1 (en) 2021-03-03 2024-05-02 Voyager Therapeutics, Inc. Controlled expression of viral proteins
CN117157309A (zh) 2021-05-28 2023-12-01 上海瑞宏迪医药有限公司 衣壳变异的重组腺相关病毒及其应用
KR20240032971A (ko) 2021-07-08 2024-03-12 테나야 테라퓨틱스, 인코포레이티드 유전자 요법을 위한 최적화된 발현 카세트
WO2023019168A1 (en) 2021-08-11 2023-02-16 Ultragenyx Pharmaceutical Inc. Compositions and methods for treating a muscular dystrophy
EP4405396A2 (en) 2021-09-20 2024-07-31 Voyager Therapeutics, Inc. Compositions and methods for the treatment of her2 positive cancer
US20250001012A1 (en) 2021-11-02 2025-01-02 Voyager Therapeutics, Inc. Aav capsid variants and uses thereof
WO2023091949A2 (en) 2021-11-17 2023-05-25 Voyager Therapeutics, Inc. Compositions and methods for the treatment of neurological disorders related to glucosylceramidase beta deficiency
WO2023091948A1 (en) 2021-11-17 2023-05-25 Voyager Therapeutics, Inc. Aav capsid variants and uses thereof
AU2022397707A1 (en) 2021-11-29 2024-06-13 Shanghai Regenelead Therapies Co., Ltd. Aadc/gdnf polynucleotide, and use thereof in treating parkinson's disease
CN118786151A (zh) 2021-12-16 2024-10-15 邓迪大学 α-突触核蛋白的靶向降解
WO2023133593A2 (en) * 2022-01-10 2023-07-13 University Of Florida Research Foundation, Incorporated Aav5 capsid variants
WO2023147374A2 (en) 2022-01-25 2023-08-03 Voyager Therapeutics, Inc. Baculovirus expression system
TW202346599A (zh) 2022-02-08 2023-12-01 美商航海家醫療公司 Ａａｖ衣殼變異體及其用途
GB202201713D0 (en) 2022-02-10 2022-03-30 Univ Dundee An affinity directed phosphatase system for targeted protein dephosphorylation
EP4484449A1 (en) 2022-02-21 2025-01-01 Shanghai Regenelead Therapies Co., Ltd. Vegf-binding molecule and pharmaceutical use thereof
EP4508062A1 (en) 2022-04-11 2025-02-19 Tenaya Therapeutics, Inc. Adeno-associated virus with engineered capsid
AU2023260822A1 (en) 2022-04-29 2024-11-14 Broadwing Bio Llc Complement factor h related 4-specific antibodies and uses thereof
WO2023212298A1 (en) 2022-04-29 2023-11-02 Broadwing Bio Llc Bispecific antibodies and methods of treating ocular disease
WO2023212294A1 (en) 2022-04-29 2023-11-02 Broadwing Bio Llc 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
TW202417466A (zh) 2022-06-02 2024-05-01 美商航海家醫療公司 Ａａｖ蛋白殼變異體及其用途
WO2023240236A1 (en) 2022-06-10 2023-12-14 Voyager Therapeutics, Inc. Compositions and methods for the treatment of spinal muscular atrophy related disorders
WO2023250388A1 (en) 2022-06-22 2023-12-28 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
TW202435912A (zh) 2022-08-03 2024-09-16 美商航海家醫療公司 用於穿過血腦屏障之組合物及方法
WO2024054983A1 (en) 2022-09-08 2024-03-14 Voyager Therapeutics, Inc. Controlled expression of viral proteins
WO2024059739A1 (en) 2022-09-15 2024-03-21 Voyager Therapeutics, Inc. Tau binding compounds
WO2024145474A2 (en) 2022-12-29 2024-07-04 Voyager Therapeutics, Inc. Compositions and methods for regulating mapt
WO2024163012A1 (en) 2023-02-02 2024-08-08 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
WO2024215653A1 (en) 2023-04-10 2024-10-17 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
TW202446960A (zh) 2023-05-02 2024-12-01 美商航海家醫療公司 用於調控ｍａｐｔ之組合物及方法
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
WO2024229167A1 (en) 2023-05-03 2024-11-07 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
WO2024229163A1 (en) 2023-05-03 2024-11-07 Voyager Therapeutics, Inc. Compositions and methods for the treatment of disorders related to cdkl5 deficiency
WO2024229425A1 (en) 2023-05-04 2024-11-07 Voyager Therapeutics, Inc. Aav capsid variants and uses thereof
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
WO2025038796A1 (en) 2023-08-16 2025-02-20 Voyager Therapeutics, Inc. Compositions and methods for the treatment of disorders related to cdkl5 deficiency
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

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
EP1005376B1 (en) 1997-03-14 2010-11-03 The Children's Hospital of Philadelphia Compositions for use in gene therapy for treatment of hemophilia
US6156303A (en) 1997-06-11 2000-12-05 University Of Washington Adeno-associated virus (AAV) isolates and AAV vectors derived therefrom
WO2000028004A1 (en) 1998-11-10 2000-05-18 The 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
WO2001083797A2 (en) 2000-04-28 2001-11-08 Avigen, Inc. Polynucleotides for use in recombinant adeno-associated virus virion production
US6962815B2 (en) * 2001-01-05 2005-11-08 Children's Hopital Inc. AAV2 vectors and methods
BRPI0214119B8 (pt) * 2001-11-13 2021-05-25 Univ Pennsylvania vírus adenoassociado recombinante, método de geração do referido vírus e composição compreendendo o referido vírus
WO2003089612A2 (en) * 2002-04-17 2003-10-30 University Of Florida Research Foundation, Inc. IMPROVED rAAV VECTORS
ES2862206T3 (es) 2003-06-19 2021-10-07 Genzyme Corp Viriones de AAV con inmunorreactividad disminuida y sus usos

Also Published As

Publication number Publication date
EP3235827A2 (en) 2017-10-25
PL2657247T3 (pl) 2017-09-29
EP2357189B1 (en) 2017-03-08
HUE034785T2 (en) 2018-02-28
US11083800B2 (en) 2021-08-10
JP2014057579A (ja) 2014-04-03
WO2004112727A2 (en) 2004-12-29
ES2627913T3 (es) 2017-08-01
SI1633772T1 (sl) 2016-06-30
US7259151B2 (en) 2007-08-21
EP2657248A1 (en) 2013-10-30
PT2657247T (pt) 2017-06-29
JP2010273690A (ja) 2010-12-09
ES2563064T3 (es) 2016-03-10
CY1119241T1 (el) 2018-02-14
EP2657247B1 (en) 2017-03-22
CY1119222T1 (el) 2018-02-14
SI2657248T1 (sl) 2017-07-31
ES2629380T3 (es) 2017-08-09
EP1633772A4 (en) 2008-08-27
JP6759177B2 (ja) 2020-09-23
PL1633772T3 (pl) 2016-10-31
SI3235827T1 (sl) 2021-05-31
PT1633772E (pt) 2016-03-22
PL2357189T3 (pl) 2017-08-31
US20190314526A1 (en) 2019-10-17
JP2020096642A (ja) 2020-06-25
DK2657248T3 (en) 2017-06-26
US20180280540A1 (en) 2018-10-04
US20080008690A1 (en) 2008-01-10
EP1633772A2 (en) 2006-03-15
HUE034597T2 (en) 2018-02-28
EP3235827B1 (en) 2021-01-13
CY1117575T1 (el) 2017-04-26
CY1124371T1 (el) 2022-07-22
US20170232117A1 (en) 2017-08-17
HUE054046T2 (hu) 2021-08-30
DK2357189T3 (en) 2017-06-19
HUE029556T2 (en) 2017-03-28
EP2657248B1 (en) 2017-03-22
JP2018029614A (ja) 2018-03-01
EP2357189A2 (en) 2011-08-17
PT3235827T (pt) 2021-04-09
SI2657247T1 (sl) 2017-07-31
US9506083B2 (en) 2016-11-29
JP2023106497A (ja) 2023-08-01
JP2016084362A (ja) 2016-05-19
SI2357189T1 (sl) 2017-07-31
HUE034546T2 (en) 2018-02-28
JP5551029B2 (ja) 2014-07-16
DK1633772T3 (en) 2016-02-29
EP1633772B1 (en) 2015-11-18
PL3235827T3 (pl) 2021-07-05
US20060051333A1 (en) 2006-03-09
PT2657248T (pt) 2017-06-26
DK3235827T3 (da) 2021-04-19
EP2657247A1 (en) 2013-10-30
EP3235827A3 (en) 2017-12-20
PT2357189T (pt) 2017-06-20
EP2357189A3 (en) 2011-11-16
WO2004112727A3 (en) 2005-05-06
US20220016264A1 (en) 2022-01-20
DK2657247T3 (en) 2017-07-10
ES2862206T3 (es) 2021-10-07
ES2629087T3 (es) 2017-08-07
CY1119279T1 (el) 2018-02-14
JP2007524386A (ja) 2007-08-30

Similar Documents

Publication Publication Date Title
EP1633772A4 (en) VIRIONS AAV HAVING REDUCED IMMUNOREACTIVITY AND USES
TWI348890B (en) Mixtures comprising strobilurins and ethylene modulators
HK1091132A1 (en) Anti-cd33 antibodies and the use thereof -cd33
DE602004023400D1 (en) Lies
GB0308732D0 (en) Absorption enhancers
SG118214A1 (en) Resistor with reduced leakage
GB0315259D0 (en) Use
GB2401084B (en) Disposable printer
TWI367222B (en) Aminoquinoxaline compounds and polyaminoquinoxaline compounds, and use thereof
GB2390109B (en) Nonstructural buoyancy can
GB2400812B (en) Handle structure
GB0329247D0 (en) Compounds and inks
TW584241U (en) Detachable compact keyboard and the application structure
TW592199U (en) Vessel with improved structure
GB2408484B (en) Mailer
HK1057445A2 (en) Chinese ink-box
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
GB0303064D0 (en) Rtm & cppf
GB0301649D0 (en) Improved closure with functionally dedicated handle