CY1123312T1 - Aav-βασιζομενη γονιδιακη θεραπεια για σκληρυνση κατα πλακας - Google Patents

Aav-βασιζομενη γονιδιακη θεραπεια για σκληρυνση κατα πλακας

Info

Publication number
CY1123312T1
CY1123312T1 CY20201100820T CY201100820T CY1123312T1 CY 1123312 T1 CY1123312 T1 CY 1123312T1 CY 20201100820 T CY20201100820 T CY 20201100820T CY 201100820 T CY201100820 T CY 201100820T CY 1123312 T1 CY1123312 T1 CY 1123312T1
Authority
CY
Cyprus
Prior art keywords
aav
gene therapy
based gene
stickering
plaque
Prior art date
Application number
CY20201100820T
Other languages
Greek (el)
English (en)
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 CY1123312T1 publication Critical patent/CY1123312T1/el

Links

Classifications

    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005Medicinal 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
    • A61K48/0058Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00Medicinal preparations containing antigens or antibodies
    • A61K39/0005Vertebrate antigens
    • A61K39/0008Antigens related to auto-immune diseases; Preparations to induce self-tolerance
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P29/00Non-central analgesic, antipyretic or antiinflammatory agents, e.g. antirheumatic agents; Non-steroidal antiinflammatory drugs [NSAID]
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00Drugs for immunological or allergic disorders
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00Medicinal preparations containing antigens or antibodies
    • A61K2039/51Medicinal preparations containing antigens or antibodies comprising whole cells, viruses or DNA/RNA
    • A61K2039/53DNA (RNA) vaccination
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00Medicinal preparations containing antigens or antibodies
    • A61K2039/57Medicinal preparations containing antigens or antibodies characterised by the type of response, e.g. Th1, Th2
    • A61K2039/577Medicinal preparations containing antigens or antibodies characterised by the type of response, e.g. Th1, Th2 tolerising response
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143Use 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)
  • Bioinformatics & Cheminformatics (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Microbiology (AREA)
  • Epidemiology (AREA)
  • Virology (AREA)
  • Rheumatology (AREA)
  • Immunology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (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)
CY20201100820T 2014-04-24 2020-09-02 Aav-βασιζομενη γονιδιακη θεραπεια για σκληρυνση κατα πλακας CY1123312T1 (el)

Applications Claiming Priority (2)

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

Publications (1)

Publication Number Publication Date
CY1123312T1 true CY1123312T1 (el) 2021-12-31

Family

ID=54333299

Family Applications (1)

Application Number Title Priority Date Filing Date
CY20201100820T CY1123312T1 (el) 2014-04-24 2020-09-02 Aav-βασιζομενη γονιδιακη θεραπεια για σκληρυνση κατα πλακας

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
BR112021003399A2 (pt) * 2018-08-24 2021-05-18 Spark Therapeutics, Inc. sequências de promotores otimizadas, construtos de expressão livres de íntron e métodos de uso
CA3142487A1 (en) * 2019-06-04 2020-12-10 Thomas Jefferson University Oligodendrocyte-derived extracellular vesicles for therapy of multiple sclerosis
WO2021211614A1 (en) * 2020-04-14 2021-10-21 University Of Florida Research Foundation, Incorporated Aav-based gene therapies for treatment of autoimmune diseases
WO2021211640A1 (en) * 2020-04-14 2021-10-21 University Of Florida Research Foundation, Incorporated Enhanced effects of gene-immunotherapy and immunosuppressants in multiple sclerosis
JP2023532923A (ja) * 2020-06-30 2023-08-01 チルドレンズ ナショナル メディカル センター 骨格筋線維の修復を改善するためのリコンビナントヒト酸性スフィンゴミエリナーゼの使用
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
EP2364362B1 (en) * 2008-11-12 2015-10-21 Ospedale San Raffaele S.r.l. Gene vector for inducing transgene-specific immune tolerance
JP6189754B2 (ja) * 2011-03-04 2017-08-30 イントレキソン コーポレーション タンパク質を条件的に発現するベクター
JP2015502135A (ja) * 2011-09-26 2015-01-22 ウニヴェルジテート チューリッヒ プロレクトラート エムエヌヴェーUniversitaet Zuerich Prorektorat Mnw 多発性硬化症の治療のためのapc媒介寛容誘導
US20160287685A1 (en) * 2013-11-22 2016-10-06 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
PT3133923T (pt) 2020-09-04
AU2019202958B2 (en) 2021-08-05
US20170043036A1 (en) 2017-02-16
AU2015249328B2 (en) 2019-01-17
PL3133923T3 (pl) 2021-02-22
LT3133923T (lt) 2020-08-25
JP7430898B2 (ja) 2024-02-14
SMT202000453T1 (it) 2020-09-10
CA2945984A1 (en) 2015-10-29
AU2015249328A1 (en) 2016-10-27
US20200138978A1 (en) 2020-05-07
US20230381342A1 (en) 2023-11-30
JP2017513500A (ja) 2017-06-01
JP2022081549A (ja) 2022-05-31
JP6646282B2 (ja) 2020-02-14
JP2020062038A (ja) 2020-04-23
HRP20201342T1 (hr) 2020-11-27
EP3133923A1 (en) 2017-03-01
AU2019202958A1 (en) 2019-05-16
ES2814280T3 (es) 2021-03-26
EP3744177A1 (en) 2020-12-02
RS60816B1 (sr) 2020-10-30
DK3133923T3 (da) 2020-08-10
HUE050681T2 (hu) 2020-12-28
SI3133923T1 (sl) 2020-11-30
EP3133923B1 (en) 2020-06-03
WO2015164789A1 (en) 2015-10-29
EP3133923A4 (en) 2017-12-27
CA2945984C (en) 2023-09-26

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