SI3133923T1 - Genska terapija za multiplo sklerozo na osnovi AAV - Google Patents

Genska terapija za multiplo sklerozo na osnovi AAV

Info

Publication number
SI3133923T1
SI3133923T1 SI201531336T SI201531336T SI3133923T1 SI 3133923 T1 SI3133923 T1 SI 3133923T1 SI 201531336 T SI201531336 T SI 201531336T SI 201531336 T SI201531336 T SI 201531336T SI 3133923 T1 SI3133923 T1 SI 3133923T1
Authority
SI
Slovenia
Prior art keywords
aav
gene therapy
multiple sclerosis
based gene
sclerosis
Prior art date
Application number
SI201531336T
Other languages
English (en)
Slovenian (sl)
Inventor
Brad E. Hoffman
Original Assignee
University Of Florida Research Foundation, Inc.
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by University Of Florida Research Foundation, Inc. filed Critical University Of Florida Research Foundation, Inc.
Publication of SI3133923T1 publication Critical patent/SI3133923T1/sl

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)
SI201531336T 2014-04-24 2015-04-24 Genska terapija za multiplo sklerozo na osnovi AAV SI3133923T1 (sl)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201461983924P 2014-04-24 2014-04-24
PCT/US2015/027598 WO2015164789A1 (en) 2014-04-24 2015-04-24 Aav-based gene therapy for multiple sclerosis
EP15782332.9A EP3133923B1 (en) 2014-04-24 2015-04-24 Aav-based gene therapy for multiple sclerosis

Publications (1)

Publication Number Publication Date
SI3133923T1 true SI3133923T1 (sl) 2020-11-30

Family

ID=54333299

Family Applications (1)

Application Number Title Priority Date Filing Date
SI201531336T SI3133923T1 (sl) 2014-04-24 2015-04-24 Genska terapija za multiplo sklerozo na osnovi AAV

Country Status (17)

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

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
US12290574B2 (en) * 2018-08-24 2025-05-06 Spark Therapeutics, Inc. Optimized promoter sequences, intron-free expression constructs and methods of use
CN114555063A (zh) * 2019-06-04 2022-05-27 托马斯杰斐逊大学 用于治疗多发性硬化症的少突胶质细胞来源的细胞外囊泡
TW202204625A (zh) * 2020-04-14 2022-02-01 美商佛羅里達大學研究基金會公司 多發性硬化之基因免疫治療與免疫抑制劑之增強功效
WO2021211614A1 (en) * 2020-04-14 2021-10-21 University Of Florida Research Foundation, Incorporated Aav-based gene therapies for treatment of autoimmune diseases
KR20230031329A (ko) * 2020-06-30 2023-03-07 칠드런스 내셔널 메디컬 센터 골격 근섬유 복구를 개선하기 위한 재조합 인간 산성 스핑고미엘리나제의 용도
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
KR960705038A (ko) * 1993-09-03 1996-10-09 스테시 엘. 채닝 자기 면역 질환에 관련된 프로토콜에서 미엘린 올리고덴드로시트 당 단백질 및 그의 펩타이드 부분들의 용도(users of myelin oligodendrocyte glycoprotein and peptide portions thereof in protocols related to autoimmune desease)
AU5588298A (en) * 1996-12-02 1998-06-29 Cell Genesys, Inc. Adeno-associated viral vector-mediated delivery of dna to cells of the liver
KR100449454B1 (ko) * 2000-10-02 2004-09-21 이현철 단일사슬 인슐린 유도체의 유전자를 포함하는 당뇨병치료용 벡터
EP1193272B1 (en) * 2000-10-02 2004-06-30 Yonsei University Single-chain insulin analogs
CA2361462A1 (en) * 2001-11-07 2003-05-08 Katherine A. High Induction of tolerance to a therapeutic polypeptide
AU2003274397A1 (en) 2002-06-05 2003-12-22 University Of Florida Production of pseudotyped recombinant aav virions
US20090304726A1 (en) * 2006-02-15 2009-12-10 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
WO2010055413A1 (en) * 2008-11-12 2010-05-20 Fondazione Centro San Raffaele Del Monte Tabor Gene vector for inducing transgene-specific immune tolerance
SG10201600912SA (en) * 2011-03-04 2016-03-30 Intrexon Corp Vectors conditionally expressing protein
US9592259B2 (en) * 2011-09-26 2017-03-14 University Of Zurich APC-mediated tolerance induction for therapy of multiple sclerosis
CA2929273C (en) * 2013-11-22 2022-05-31 Amarna Holding B.V. Method for restoring immune tolerance in vivo
WO2021211614A1 (en) * 2020-04-14 2021-10-21 University Of Florida Research Foundation, Incorporated Aav-based gene therapies for treatment of autoimmune diseases

Also Published As

Publication number Publication date
EP3744177A1 (en) 2020-12-02
EP3133923B1 (en) 2020-06-03
EP3133923A4 (en) 2017-12-27
RS60816B1 (sr) 2020-10-30
PL3133923T3 (pl) 2021-02-22
DK3133923T3 (da) 2020-08-10
CA2945984C (en) 2023-09-26
HUE050681T2 (hu) 2020-12-28
HRP20201342T1 (hr) 2020-11-27
EP3133923A1 (en) 2017-03-01
JP7430898B2 (ja) 2024-02-14
JP2020062038A (ja) 2020-04-23
JP2017513500A (ja) 2017-06-01
AU2019202958A1 (en) 2019-05-16
LT3133923T (lt) 2020-08-25
CY1123312T1 (el) 2021-12-31
US20200138978A1 (en) 2020-05-07
SMT202000453T1 (it) 2020-09-10
US20170043036A1 (en) 2017-02-16
CA2945984A1 (en) 2015-10-29
JP6646282B2 (ja) 2020-02-14
WO2015164789A1 (en) 2015-10-29
AU2015249328B2 (en) 2019-01-17
US20230381342A1 (en) 2023-11-30
AU2015249328A1 (en) 2016-10-27
JP2022081549A (ja) 2022-05-31
AU2019202958B2 (en) 2021-08-05
PT3133923T (pt) 2020-09-04
ES2814280T3 (es) 2021-03-26

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