DK3265571T3 - Fler-vektorsystem og anvendelse heraf - Google Patents

Fler-vektorsystem og anvendelse heraf Download PDF

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Publication number
DK3265571T3
DK3265571T3 DK16710113.8T DK16710113T DK3265571T3 DK 3265571 T3 DK3265571 T3 DK 3265571T3 DK 16710113 T DK16710113 T DK 16710113T DK 3265571 T3 DK3265571 T3 DK 3265571T3
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DK
Denmark
Prior art keywords
vector system
multiple vector
vector
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DK16710113.8T
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English (en)
Inventor
Pasqualina Colella
Alberto Auricchio
Ivana Trapani
Original Assignee
Fond Telethon
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Publication of DK3265571T3 publication Critical patent/DK3265571T3/da

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    • 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
    • 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00Drugs for disorders of the muscular or neuromuscular system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00Drugs for disorders of the muscular or neuromuscular system
    • A61P21/04Drugs for disorders of the muscular or neuromuscular system for myasthenia gravis
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00Drugs for disorders of the blood or the extracellular fluid
    • A61P7/04Antihaemorrhagics; Procoagulants; Haemostatic agents; Antifibrinolytic agents
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00Drugs for disorders of the cardiovascular system
    • A61P9/10Drugs 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
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/705Receptors; Cell surface antigens; Cell surface determinants
    • 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
    • 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
    • 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
    • C12N2800/00Nucleic acids vectors
    • C12N2800/40Systems of functionally co-operating vectors
    • 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
    • C12N2840/00Vectors comprising a special translation-regulating system
    • C12N2840/20Vectors comprising a special translation-regulating system translation of more than one cistron
    • 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
    • C12N2840/00Vectors comprising a special translation-regulating system
    • C12N2840/44Vectors comprising a special translation-regulating system being a specific part of the splice mechanism, e.g. donor, acceptor
    • 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
    • C12N2840/00Vectors comprising a special translation-regulating system
    • C12N2840/44Vectors comprising a special translation-regulating system being a specific part of the splice mechanism, e.g. donor, acceptor
    • C12N2840/445Vectors 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

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  • 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)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Virology (AREA)
  • Physics & Mathematics (AREA)
  • Immunology (AREA)
  • Cell Biology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Toxicology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Neurology (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Vascular Medicine (AREA)
  • Diabetes (AREA)
  • Hematology (AREA)
DK16710113.8T 2015-03-03 2016-03-03 Fler-vektorsystem og anvendelse heraf DK3265571T3 (da)

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
DK3265571T3 true DK3265571T3 (da) 2022-06-27

Family

ID=56849204

Family Applications (1)

Application Number Title Priority Date Filing Date
DK16710113.8T DK3265571T3 (da) 2015-03-03 2016-03-03 Fler-vektorsystem og anvendelse heraf

Country Status (17)

Country Link
US (2) US20180327779A1 (da)
EP (2) EP3265571B1 (da)
JP (2) JP7182873B2 (da)
KR (1) KR102240180B1 (da)
CN (1) CN107466325A (da)
AU (1) AU2016227668B2 (da)
BR (1) BR112017018728A2 (da)
CA (1) CA2979120A1 (da)
DK (1) DK3265571T3 (da)
ES (1) ES2919880T3 (da)
HR (1) HRP20220776T1 (da)
MX (2) MX2017011255A (da)
PL (1) PL3265571T3 (da)
PT (1) PT3265571T (da)
RS (1) RS63416B1 (da)
SI (1) SI3265571T1 (da)
WO (1) WO2016139321A1 (da)

Families Citing this family (16)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
JP7007273B2 (ja) * 2015-12-22 2022-01-24 アンセルム(アンスティチュート・ナシオナル・ドゥ・ラ・サンテ・エ・ドゥ・ラ・ルシェルシュ・メディカル) 遺伝子治療用の改良された複合型二重組換えaavベクターシステム
JP2019530737A (ja) 2016-08-23 2019-10-24 アコーオス インコーポレイテッド ヒト対象において非加齢性聴力障害を治療するための組成物および方法
KR20230167138A (ko) * 2017-05-05 2023-12-07 유니버시티 오브 플로리다 리서치 파운데이션, 인코포레이티드 오토펄린을 발현시키기 위한 조성물 및 방법
JOP20200253A1 (ar) * 2018-04-05 2020-10-04 Univ Oxford Innovation Ltd تركيبات وطرق لعلاج الحثل البقعي
US11993776B2 (en) 2018-04-17 2024-05-28 Ascidian Therapeutics, Inc. Trans-splicing 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
CN113348249A (zh) * 2018-10-15 2021-09-03 特莱索恩基金会 内蛋白及其用途
JP2022511348A (ja) * 2018-10-25 2022-01-31 バクスアルタ インコーポレイテッド Aavトリプルプラスミドシステム
EP3956453A4 (en) * 2019-04-19 2023-02-22 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
EP4073257A1 (en) * 2019-12-09 2022-10-19 UCL Business Ltd Gene therapy composition and treatment for myh7-linked cardiomyopathy
JP2023512824A (ja) * 2020-02-07 2023-03-29 ザ チルドレンズ メディカル センター コーポレーション 大型遺伝子ベクターならびにその送達および使用の方法
BR112022016596A2 (pt) 2020-02-21 2022-11-16 Akouos Inc Composições e métodos para o tratamento de debilitação auditiva não associada à idade em um indivíduo humano
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

Family Cites Families (14)

* 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
DE69918090T2 (de) 1998-04-24 2005-06-16 University Of Florida, Gainesville Rekombinanter adeno-assoziierter viraler vektor, der alpha-1 antitrypsin kodiert, zur gentherapie
US6967018B2 (en) 2002-01-11 2005-11-22 Applied Genetic Technologies Corporation Adiponectin gene therapy
EP1496980A4 (en) * 2002-04-19 2005-06-22 Georgia Tech Res Inst COMPOSITIONS AND METHODS FOR ACCELERATING 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
AU2007223427A1 (en) * 2006-03-01 2007-09-13 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
BRPI0922187B1 (pt) * 2008-11-19 2021-05-04 Amyris, Inc Métodos para gerar um polinucleotídeo e para gerar uma célula hospedeira
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EP2986635B1 (en) * 2013-04-18 2018-10-03 Fondazione Telethon Effective delivery of large genes by dual aav vectors

Also Published As

Publication number Publication date
EP4089172A2 (en) 2022-11-16
US20180327779A1 (en) 2018-11-15
JP7182873B2 (ja) 2022-12-05
CA2979120A1 (en) 2016-09-09
AU2016227668B2 (en) 2019-06-27
AU2016227668A1 (en) 2017-08-31
MX2023004479A (es) 2023-05-04
WO2016139321A1 (en) 2016-09-09
JP2022174192A (ja) 2022-11-22
EP3265571A1 (en) 2018-01-10
CN107466325A (zh) 2017-12-12
PL3265571T3 (pl) 2022-09-05
BR112017018728A2 (pt) 2018-04-17
RS63416B1 (sr) 2022-08-31
KR20170140185A (ko) 2017-12-20
MX2017011255A (es) 2018-08-01
US20220002749A1 (en) 2022-01-06
EP3265571B1 (en) 2022-04-13
KR102240180B1 (ko) 2021-04-14
ES2919880T3 (es) 2022-07-28
PT3265571T (pt) 2022-06-29
JP2018512125A (ja) 2018-05-17
HRP20220776T1 (hr) 2022-09-16
SI3265571T1 (sl) 2022-10-28
EP4089172A3 (en) 2023-03-01

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