HUE050681T2 - AAV-alapú génterápia szklerózis multiplexre - Google Patents

AAV-alapú génterápia szklerózis multiplexre

Info

Publication number
HUE050681T2
HUE050681T2 HUE15782332A HUE15782332A HUE050681T2 HU E050681 T2 HUE050681 T2 HU E050681T2 HU E15782332 A HUE15782332 A HU E15782332A HU E15782332 A HUE15782332 A HU E15782332A HU E050681 T2 HUE050681 T2 HU E050681T2
Authority
HU
Hungary
Prior art keywords
aav
gene therapy
multiple sclerosis
based gene
sclerosis
Prior art date
Application number
HUE15782332A
Other languages
English (en)
Hungarian (hu)
Inventor
Brad Hoffman
Original Assignee
Univ Florida
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 Univ Florida filed Critical Univ Florida
Publication of HUE050681T2 publication Critical patent/HUE050681T2/hu

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
    • A61K48/0058—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
    • A61K39/00—Medicinal preparations containing antigens or antibodies
    • A61K39/0005—Vertebrate antigens
    • A61K39/0008—Antigens related to auto-immune diseases; Preparations to induce self-tolerance
    • 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
    • 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
    • A61P37/00—Drugs for immunological or allergic disorders
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00—Medicinal preparations containing antigens or antibodies
    • A61K2039/51—Medicinal preparations containing antigens or antibodies comprising whole cells, viruses or DNA/RNA
    • A61K2039/53—DNA (RNA) vaccination
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00—Medicinal preparations containing antigens or antibodies
    • A61K2039/57—Medicinal preparations containing antigens or antibodies characterised by the type of response, e.g. Th1, Th2
    • A61K2039/577—Medicinal preparations containing antigens or antibodies characterised by the type of response, e.g. Th1, Th2 tolerising response
    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Biochemistry (AREA)
  • Zoology (AREA)
  • Molecular Biology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Microbiology (AREA)
  • Epidemiology (AREA)
  • Plant Pathology (AREA)
  • Virology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Immunology (AREA)
  • Rheumatology (AREA)
  • Mycology (AREA)
  • Pain & Pain Management (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Peptides Or Proteins (AREA)
HUE15782332A 2014-04-24 2015-04-24 AAV-alapú génterápia szklerózis multiplexre HUE050681T2 (hu)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US201461983924P 2014-04-24 2014-04-24

Publications (1)

Publication Number Publication Date
HUE050681T2 true HUE050681T2 (hu) 2020-12-28

Family

ID=54333299

Family Applications (1)

Application Number Title Priority Date Filing Date
HUE15782332A HUE050681T2 (hu) 2014-04-24 2015-04-24 AAV-alapú génterápia szklerózis multiplexre

Country Status (17)

Country Link
US (3) US20170043036A1 (cg-RX-API-DMAC7.html)
EP (2) EP3133923B1 (cg-RX-API-DMAC7.html)
JP (3) JP6646282B2 (cg-RX-API-DMAC7.html)
AU (2) AU2015249328B2 (cg-RX-API-DMAC7.html)
CA (1) CA2945984C (cg-RX-API-DMAC7.html)
CY (1) CY1123312T1 (cg-RX-API-DMAC7.html)
DK (1) DK3133923T3 (cg-RX-API-DMAC7.html)
ES (1) ES2814280T3 (cg-RX-API-DMAC7.html)
HR (1) HRP20201342T1 (cg-RX-API-DMAC7.html)
HU (1) HUE050681T2 (cg-RX-API-DMAC7.html)
LT (1) LT3133923T (cg-RX-API-DMAC7.html)
PL (1) PL3133923T3 (cg-RX-API-DMAC7.html)
PT (1) PT3133923T (cg-RX-API-DMAC7.html)
RS (1) RS60816B1 (cg-RX-API-DMAC7.html)
SI (1) SI3133923T1 (cg-RX-API-DMAC7.html)
SM (1) SMT202000453T1 (cg-RX-API-DMAC7.html)
WO (1) WO2015164789A1 (cg-RX-API-DMAC7.html)

Families Citing this family (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP7270946B2 (ja) * 2017-08-08 2023-05-11 国立大学法人浜松医科大学 自己免疫疾患の治療又は予防剤
CA3111047A1 (en) * 2018-08-24 2020-02-27 Spark Therapeutics, Inc. Optimized promoter sequences, intron-free expression constructs and methods of use
EP3979993A4 (en) * 2019-06-04 2023-07-05 Thomas Jefferson University EXTRACELLULAR VESICLES OF OLIGODENDROCYTES FOR THE THERAPY OF MULTIPLE SCLEROSIS
AR121837A1 (es) 2020-04-14 2022-07-13 Univ Florida Terapias génicas basadas en aav para el tratamiento de enfermedades autoinmunes
EP4135852A4 (en) * 2020-04-14 2024-11-20 University Of Florida Research Foundation, Incorporated ENHANCED EFFECTS OF GENE IMMUNOTHERAPY AND IMMUNOSUPPRESSANTS IN MULTIPLE SCLEROSIS
EP4171660A4 (en) * 2020-06-30 2025-03-26 Children's National Medical Center USE OF RECOMBINANT HUMAN ACID SPHINGOMYELINASE TO IMPROVE SKELETAL MYOFIBER REPAIR
WO2024081570A1 (en) * 2022-10-10 2024-04-18 Eli Lilly And Company Methods and systems for using causal networks to develop models for evaluating biological processes
WO2024258925A1 (en) 2023-06-12 2024-12-19 Children's Hospital Medical Center Aav-cftr vectors and methods of using same

Family Cites Families (13)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
FI961000A7 (fi) * 1993-09-03 1996-03-04 Immulogic Pharma Corp Myeliinioligodendrosyyttiglykoproteiinin ja tämän peptidiosien käyttöm uotoja autoimmuunisairauksiin liittyvissä menetelmissä
WO1998024479A1 (en) * 1996-12-02 1998-06-11 Cell Genesys, Inc. Adeno-associated viral vector-mediated delivery of dna to cells of the liver
DE60104069T2 (de) * 2000-10-02 2005-08-25 Yonsei University Einkettige Insulinanaloge
KR100449454B1 (ko) * 2000-10-02 2004-09-21 이현철 단일사슬 인슐린 유도체의 유전자를 포함하는 당뇨병치료용 벡터
CA2361462A1 (en) * 2001-11-07 2003-05-08 Katherine A. High Induction of tolerance to a therapeutic polypeptide
WO2003104413A2 (en) 2002-06-05 2003-12-18 University Of Florida Production of pseudotyped recombinant aav virions
EP1991259B1 (en) * 2006-02-15 2012-10-10 Ramot at Tel-Aviv University Ltd. Viral display vehicles for treating multiple sclerosis
US20120322861A1 (en) 2007-02-23 2012-12-20 Barry John Byrne Compositions and Methods for Treating Diseases
DK2364362T3 (en) * 2008-11-12 2016-01-25 Ospedale San Raffaele Srl Gene vector FOR INDUCTION OF IMMUNE TOLERANCE transgene
AU2012225749B2 (en) * 2011-03-04 2015-01-22 Intrexon Corporation Vectors conditionally expressing protein
CN104010664A (zh) * 2011-09-26 2014-08-27 苏黎世大学数学和自然科学部 用于治疗多发性硬化的apc介导的耐受诱导
CA2929273C (en) * 2013-11-22 2022-05-31 Amarna Holding B.V. Method for restoring immune tolerance in vivo
AR121837A1 (es) * 2020-04-14 2022-07-13 Univ Florida Terapias génicas basadas en aav para el tratamiento de enfermedades autoinmunes

Also Published As

Publication number Publication date
CA2945984C (en) 2023-09-26
EP3133923A4 (en) 2017-12-27
JP2020062038A (ja) 2020-04-23
EP3133923B1 (en) 2020-06-03
DK3133923T3 (da) 2020-08-10
PT3133923T (pt) 2020-09-04
EP3133923A1 (en) 2017-03-01
JP7430898B2 (ja) 2024-02-14
CA2945984A1 (en) 2015-10-29
WO2015164789A1 (en) 2015-10-29
AU2019202958A1 (en) 2019-05-16
ES2814280T3 (es) 2021-03-26
AU2015249328A1 (en) 2016-10-27
JP6646282B2 (ja) 2020-02-14
CY1123312T1 (el) 2021-12-31
AU2019202958B2 (en) 2021-08-05
US20170043036A1 (en) 2017-02-16
JP2017513500A (ja) 2017-06-01
HRP20201342T1 (hr) 2020-11-27
US20230381342A1 (en) 2023-11-30
US20200138978A1 (en) 2020-05-07
AU2015249328B2 (en) 2019-01-17
PL3133923T3 (pl) 2021-02-22
SI3133923T1 (sl) 2020-11-30
RS60816B1 (sr) 2020-10-30
SMT202000453T1 (it) 2020-09-10
EP3744177A1 (en) 2020-12-02
LT3133923T (lt) 2020-08-25
JP2022081549A (ja) 2022-05-31

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