PL3265571T3 - System wielowektorowy i jego zastosowania - Google Patents

System wielowektorowy i jego zastosowania

Info

Publication number
PL3265571T3
PL3265571T3 PL16710113.8T PL16710113T PL3265571T3 PL 3265571 T3 PL3265571 T3 PL 3265571T3 PL 16710113 T PL16710113 T PL 16710113T PL 3265571 T3 PL3265571 T3 PL 3265571T3
Authority
PL
Poland
Prior art keywords
vector system
multiple vector
vector
Prior art date
Application number
PL16710113.8T
Other languages
English (en)
Inventor
Pasqualina COLELLA
Alberto Auricchio
Ivana TRAPANI
Original Assignee
Fondazione Telethon
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 Fondazione Telethon filed Critical Fondazione Telethon
Publication of PL3265571T3 publication Critical patent/PL3265571T3/pl

Links

Classifications

    • 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
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A61P21/04—Drugs for disorders of the muscular or neuromuscular system for myasthenia gravis
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • A61P7/04—Antihaemorrhagics; Procoagulants; Haemostatic agents; Antifibrinolytic agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • A61P9/10—Drugs for disorders of the cardiovascular system for treating ischaemic or atherosclerotic diseases, e.g. antianginal drugs, coronary vasodilators, drugs for myocardial infarction, retinopathy, cerebrovascula insufficiency, renal arteriosclerosis
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
    • 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
    • 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
    • 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
    • C12N2800/00—Nucleic acids vectors
    • C12N2800/40—Systems of functionally co-operating vectors
    • 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
    • C12N2840/00—Vectors comprising a special translation-regulating system
    • C12N2840/20—Vectors comprising a special translation-regulating system translation of more than one cistron
    • 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
    • C12N2840/00—Vectors comprising a special translation-regulating system
    • C12N2840/44—Vectors comprising a special translation-regulating system being a specific part of the splice mechanism, e.g. donor, acceptor
    • 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
    • C12N2840/00—Vectors comprising a special translation-regulating system
    • C12N2840/44—Vectors comprising a special translation-regulating system being a specific part of the splice mechanism, e.g. donor, acceptor
    • C12N2840/445—Vectors comprising a special translation-regulating system being a specific part of the splice mechanism, e.g. donor, acceptor for trans-splicing, e.g. polypyrimidine tract, branch point splicing

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Medicinal Chemistry (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Physics & Mathematics (AREA)
  • Virology (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Toxicology (AREA)
  • Immunology (AREA)
  • Cell Biology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Neurology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Epidemiology (AREA)
  • Urology & Nephrology (AREA)
  • Vascular Medicine (AREA)
  • Cardiology (AREA)
PL16710113.8T 2015-03-03 2016-03-03 System wielowektorowy i jego zastosowania PL3265571T3 (pl)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201562127463P 2015-03-03 2015-03-03
PCT/EP2016/054602 WO2016139321A1 (en) 2015-03-03 2016-03-03 Multiple vector system and uses thereof

Publications (1)

Publication Number Publication Date
PL3265571T3 true PL3265571T3 (pl) 2022-09-05

Family

ID=56849204

Family Applications (1)

Application Number Title Priority Date Filing Date
PL16710113.8T PL3265571T3 (pl) 2015-03-03 2016-03-03 System wielowektorowy i jego zastosowania

Country Status (17)

Country Link
US (2) US20180327779A1 (pl)
EP (2) EP4089172A3 (pl)
JP (2) JP7182873B2 (pl)
KR (1) KR102240180B1 (pl)
CN (1) CN107466325A (pl)
AU (1) AU2016227668B2 (pl)
BR (1) BR112017018728A2 (pl)
CA (1) CA2979120A1 (pl)
DK (1) DK3265571T3 (pl)
ES (1) ES2919880T3 (pl)
HR (1) HRP20220776T1 (pl)
MX (2) MX2017011255A (pl)
PL (1) PL3265571T3 (pl)
PT (1) PT3265571T (pl)
RS (1) RS63416B1 (pl)
SI (1) SI3265571T1 (pl)
WO (1) WO2016139321A1 (pl)

Families Citing this family (19)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
RS63416B1 (sr) * 2015-03-03 2022-08-31 Fond Telethon Sistem višestrukih vektora i njegove primene
WO2016145345A1 (en) * 2015-03-11 2016-09-15 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Rp2 and rpgr vectors for treating x-linked retinitis pigmentosa
WO2017108931A1 (en) * 2015-12-22 2017-06-29 INSERM (Institut National de la Santé et de la Recherche Médicale) Improved hybrid dual recombinant aav vector systems for gene therapy
CN110225975A (zh) 2016-08-23 2019-09-10 阿库斯股份有限公司 用于治疗人受试者中非年龄相关的听力损害的组合物和方法
KR20230167138A (ko) * 2017-05-05 2023-12-07 유니버시티 오브 플로리다 리서치 파운데이션, 인코포레이티드 오토펄린을 발현시키기 위한 조성물 및 방법
SG11202009759SA (en) * 2018-04-05 2020-10-29 Univ Oxford Innovation Ltd Compositions and methods for treating macular dystrophy
EP3781213A4 (en) * 2018-04-17 2022-10-19 The Trustees of the University of Pennsylvania TRANS-SPLICE MOLECULES
US11660353B2 (en) 2018-04-27 2023-05-30 Decibel Therapeutics, Inc. Compositions and methods for treating sensorineural hearing loss using otoferlin dual vector systems
AU2019360372A1 (en) * 2018-10-15 2021-06-03 Fondazione Telethon Intein proteins and uses thereof
KR20210086645A (ko) * 2018-10-25 2021-07-08 박스알타 인코퍼레이티드 Aav 삼중-플라스미드 시스템
WO2020214797A1 (en) * 2019-04-19 2020-10-22 University Of Massachusetts Gene therapies for usher syndrome (ush1b)
US20220315948A1 (en) * 2019-04-26 2022-10-06 President And Fellows Of Harvard College Aav vectors encoding mini-pcdh15 and uses thereof
WO2021116138A1 (en) * 2019-12-09 2021-06-17 Ucl Business Ltd Gene therapy composition and treatment for myh7-linked cardiomyopathy
KR20220139924A (ko) * 2020-02-07 2022-10-17 더 칠드런스 메디칼 센터 코포레이션 큰 유전자 벡터 및 그의 전달 및 용도
AU2021225035A1 (en) 2020-02-21 2022-10-13 Akouos, Inc. Compositions and methods for treating non-age-associated hearing impairment in a human subject
CN112501209B (zh) * 2020-12-07 2024-02-13 和元生物技术（上海）股份有限公司 外源基因可控表达的腺相关病毒包装方法
WO2022234295A1 (en) * 2021-05-07 2022-11-10 Ucl Business Ltd Abca4 genome editing
WO2024098035A2 (en) * 2022-11-04 2024-05-10 National Resilience, Inc. Methods and compositions for preparing recombinant adeno associated viruses and uses thereof
CN116925239B (zh) * 2023-07-17 2024-10-18 苏州星奥拓维生物技术有限公司 双载体系统表达Otof基因的组合物和方法

Family Cites Families (16)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5827702A (en) 1994-10-31 1998-10-27 Genentech, Inc. Ocular gene therapy
GB9720465D0 (en) * 1997-09-25 1997-11-26 Oxford Biomedica Ltd Dual-virus vectors
US6106826A (en) 1997-12-17 2000-08-22 Wisconsin Alumni Research Foundation Replication competent, avirulent Herpes simplex virus as a vector for neural and ocular gene therapy
US6461606B1 (en) 1998-04-24 2002-10-08 University Of Florida Research Foundation Materials and methods for gene therapy
US6967018B2 (en) 2002-01-11 2005-11-22 Applied Genetic Technologies Corporation Adiponectin gene therapy
US7250406B2 (en) * 2002-04-19 2007-07-31 Georgia Tech Research Corporation Compositions and methods for the acceleration of protein secretion dynamics
WO2006036465A2 (en) 2004-09-03 2006-04-06 University Of Florida Compositions and methods for treating cystic fibrosis
JP4948845B2 (ja) 2006-02-08 2012-06-06 財団法人 東京都医学総合研究所 神経変性疾患治療用物質のスクリーニング方法
US9365622B2 (en) * 2006-03-01 2016-06-14 University Of Utah Research Foundation Methods and compositions related to cyclic peptide synthesis
US8298818B2 (en) 2006-04-28 2012-10-30 University Of Florida Research Foundation, Inc. Self-complementary adeno-associated virus having a truncated CMV-chicken β-actin promoter
US8236557B2 (en) * 2008-05-28 2012-08-07 University Of Missouri-Columbia Hybrid-AAV vectors to deliver large gene expression cassette
KR101794298B1 (ko) * 2008-11-19 2017-11-20 아미리스 인코퍼레이티드 폴리뉴클레오타이드의 집합체 조성물 및 이에 대한 방법
WO2011000929A1 (en) 2009-07-02 2011-01-06 Merz Pharma Gmbh & Co. Kgaa Neurotoxins exhibiting shortened biological activity
WO2013075008A1 (en) * 2011-11-16 2013-05-23 University Of Florida Research Foundation Inc. Aav dual vector systems for gene therapy
US10494645B2 (en) * 2013-04-18 2019-12-03 Fondazione Telethon Effective delivery of large genes by dual AAV vectors
RS63416B1 (sr) * 2015-03-03 2022-08-31 Fond Telethon Sistem višestrukih vektora i njegove primene

Also Published As

Publication number Publication date
BR112017018728A2 (pt) 2018-04-17
CN107466325A (zh) 2017-12-12
SI3265571T1 (sl) 2022-10-28
WO2016139321A1 (en) 2016-09-09
ES2919880T3 (es) 2022-07-28
KR102240180B1 (ko) 2021-04-14
KR20170140185A (ko) 2017-12-20
JP2018512125A (ja) 2018-05-17
EP4089172A3 (en) 2023-03-01
HRP20220776T1 (hr) 2022-09-16
AU2016227668A1 (en) 2017-08-31
EP4089172A2 (en) 2022-11-16
MX2023004479A (es) 2023-05-04
DK3265571T3 (da) 2022-06-27
JP2022174192A (ja) 2022-11-22
CA2979120A1 (en) 2016-09-09
EP3265571A1 (en) 2018-01-10
US20220002749A1 (en) 2022-01-06
US20180327779A1 (en) 2018-11-15
EP3265571B1 (en) 2022-04-13
MX2017011255A (es) 2018-08-01
AU2016227668B2 (en) 2019-06-27
JP7182873B2 (ja) 2022-12-05
RS63416B1 (sr) 2022-08-31
PT3265571T (pt) 2022-06-29

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