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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Publication of CA2945984A1 publication Critical patent/CA2945984A1/en
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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)
  • 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)
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 (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
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
SI3133923T1 (sl) 2020-11-30
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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