CA2945984C - Aav-based gene therapy for multiple sclerosis - Google Patents

Aav-based gene therapy for multiple sclerosis Download PDF

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Publication number
CA2945984C
CA2945984C CA2945984A CA2945984A CA2945984C CA 2945984 C CA2945984 C CA 2945984C CA 2945984 A CA2945984 A CA 2945984A CA 2945984 A CA2945984 A CA 2945984A CA 2945984 C CA2945984 C CA 2945984C
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Prior art keywords
nucleic acid
raav
vector
protein
raav vector
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CA2945984A
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English (en)
French (fr)
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CA2945984A1 (en
Inventor
Brad E. HOFFMAN
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University of Florida Research Foundation Inc
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University of Florida Research Foundation Inc
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    • 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

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Public Health (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (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)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Immunology (AREA)
  • Virology (AREA)
  • Physics & Mathematics (AREA)
  • Rheumatology (AREA)
  • Mycology (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Pain & Pain Management (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)
CA2945984A 2014-04-24 2015-04-24 Aav-based gene therapy for multiple sclerosis Active CA2945984C (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201461983924P 2014-04-24 2014-04-24
US61/983,924 2014-04-24
PCT/US2015/027598 WO2015164789A1 (en) 2014-04-24 2015-04-24 Aav-based gene therapy for multiple sclerosis

Publications (2)

Publication Number Publication Date
CA2945984A1 CA2945984A1 (en) 2015-10-29
CA2945984C true CA2945984C (en) 2023-09-26

Family

ID=54333299

Family Applications (1)

Application Number Title Priority Date Filing Date
CA2945984A Active CA2945984C (en) 2014-04-24 2015-04-24 Aav-based gene therapy for multiple sclerosis

Country Status (17)

Country Link
US (3) US20170043036A1 (enExample)
EP (2) EP3744177A1 (enExample)
JP (3) JP6646282B2 (enExample)
AU (2) AU2015249328B2 (enExample)
CA (1) CA2945984C (enExample)
CY (1) CY1123312T1 (enExample)
DK (1) DK3133923T3 (enExample)
ES (1) ES2814280T3 (enExample)
HR (1) HRP20201342T1 (enExample)
HU (1) HUE050681T2 (enExample)
LT (1) LT3133923T (enExample)
PL (1) PL3133923T3 (enExample)
PT (1) PT3133923T (enExample)
RS (1) RS60816B1 (enExample)
SI (1) SI3133923T1 (enExample)
SM (1) SMT202000453T1 (enExample)
WO (1) WO2015164789A1 (enExample)

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
JP2021533805A (ja) * 2018-08-24 2021-12-09 スパーク セラピューティクス インコーポレイテッドSpark Therapeutics, Inc. 最適化されたプロモーター配列、イントロンを含まない発現構築物及び使用方法
AU2020289326A1 (en) * 2019-06-04 2022-01-27 Thomas Jefferson University Oligodendrocyte-derived extracellular vesicles for therapy of multiple sclerosis
US20230263909A1 (en) * 2020-04-14 2023-08-24 University Of Florida Research Foundation, Incorporated Enhanced effects of gene-immunotherapy and immunosuppressants in multiple sclerosis
EP4135777A4 (en) * 2020-04-14 2025-03-12 University Of Florida Research Foundation, Incorporated AAV-BASED GENE THERAPIES FOR THE TREATMENT OF AUTOIMMUNE DISEASES
CN116209477A (zh) * 2020-06-30 2023-06-02 儿童国家医疗中心 使用重组人酸性鞘氨醇酶改善骨骼肌纤维的修复
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
AU7725894A (en) * 1993-09-03 1995-03-22 Immulogic Pharmaceutical Corporation Uses of myelin oligodendrocyte glycoprotein and peptide portions thereof in protocols related to autoimmune disease
WO1998024479A1 (en) * 1996-12-02 1998-06-11 Cell Genesys, Inc. Adeno-associated viral vector-mediated delivery of dna to cells of the liver
ATE270306T1 (de) * 2000-10-02 2004-07-15 Univ Yonsei Seoul 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
WO2007094003A2 (en) * 2006-02-15 2007-08-23 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
US9938540B2 (en) * 2008-11-12 2018-04-10 Ospedale San Raffaele S.R.L. Gene vector for inducing transgene-specific immune tolerance
US9402919B2 (en) * 2011-03-04 2016-08-02 Intrexon Corporation Vectors conditionally expressing protein
EP2760478A1 (en) * 2011-09-26 2014-08-06 Universität Zürich Prorektorat MNW Apc-mediated tolerance induction for therapy of multiple sclerosis
ES2719691T3 (es) * 2013-11-22 2019-07-12 Amarna Holding B V Procedimiento de restauración de tolerancia inmunitaria in vivo
EP4135777A4 (en) * 2020-04-14 2025-03-12 University Of Florida Research Foundation, Incorporated AAV-BASED GENE THERAPIES FOR THE TREATMENT OF AUTOIMMUNE DISEASES

Also Published As

Publication number Publication date
SMT202000453T1 (it) 2020-09-10
EP3133923A4 (en) 2017-12-27
EP3133923B1 (en) 2020-06-03
AU2015249328A1 (en) 2016-10-27
LT3133923T (lt) 2020-08-25
AU2019202958A1 (en) 2019-05-16
PT3133923T (pt) 2020-09-04
CA2945984A1 (en) 2015-10-29
JP2017513500A (ja) 2017-06-01
PL3133923T3 (pl) 2021-02-22
HUE050681T2 (hu) 2020-12-28
AU2015249328B2 (en) 2019-01-17
DK3133923T3 (da) 2020-08-10
HRP20201342T1 (hr) 2020-11-27
JP6646282B2 (ja) 2020-02-14
US20230381342A1 (en) 2023-11-30
EP3133923A1 (en) 2017-03-01
JP2020062038A (ja) 2020-04-23
CY1123312T1 (el) 2021-12-31
WO2015164789A1 (en) 2015-10-29
ES2814280T3 (es) 2021-03-26
SI3133923T1 (sl) 2020-11-30
JP7430898B2 (ja) 2024-02-14
US20170043036A1 (en) 2017-02-16
RS60816B1 (sr) 2020-10-30
EP3744177A1 (en) 2020-12-02
US20200138978A1 (en) 2020-05-07
JP2022081549A (ja) 2022-05-31
AU2019202958B2 (en) 2021-08-05

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