DK3265571T3 - Fler-vektorsystem og anvendelse heraf - Google Patents

Fler-vektorsystem og anvendelse heraf Download PDF

Info

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
Authority
DK
Denmark
Prior art keywords
vector system
multiple vector
vector
Prior art date
Application number
DK16710113.8T
Other languages
English (en)
Inventor
Pasqualina Colella
Alberto Auricchio
Ivana Trapani
Original Assignee
Fond 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 Fond Telethon filed Critical Fond Telethon
Application granted granted Critical
Publication of DK3265571T3 publication Critical patent/DK3265571T3/da

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)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Virology (AREA)
  • Physics & Mathematics (AREA)
  • Immunology (AREA)
  • Cell Biology (AREA)
  • Toxicology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Neurology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Hematology (AREA)
  • Urology & Nephrology (AREA)
  • Vascular Medicine (AREA)
  • Cardiology (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 (19)

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)
CY (1) CY1125385T1 (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)
SM (1) SMT202200269T1 (da)
WO (1) WO2016139321A1 (da)

Families Citing this family (29)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP2986635B1 (en) 2013-04-18 2018-10-03 Fondazione Telethon Effective delivery of large genes by dual aav vectors
US20180327779A1 (en) * 2015-03-03 2018-11-15 Fondazione Telethon (IT/IT) Multiple vector system and uses thereof
AU2016228751B2 (en) * 2015-03-11 2022-01-13 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Rp2 vectors for treating x-linked retinitis pigmentosa
EP3393522B1 (en) * 2015-12-22 2021-07-07 INSERM (Institut National de la Santé et de la Recherche Médicale) Improved hybrid dual recombinant aav vector systems for gene therapy
EP3510161A4 (en) 2016-08-23 2020-04-22 Akouos, Inc. COMPOSITIONS AND METHODS FOR TREATING PERSONAL HEARING LOSS IN A PERSON
JP7240675B2 (ja) * 2017-05-05 2023-03-16 ユニバーシティー オブ フロリダ リサーチ ファンデーション， インク． オトフェルリンを発現させるための組成物および方法
IL276764B2 (en) * 2018-02-22 2026-05-01 Akouos Inc Compositions and methods for treating non-age-related hearing impairment in humans
CN113056561A (zh) * 2018-04-05 2021-06-29 牛津大学科技创新有限公司 用于治疗黄斑营养不良的组合物和方法
MX2020010959A (es) 2018-04-17 2021-01-15 Univ Pennsylvania Moleculas de empalme trans.
US11660353B2 (en) 2018-04-27 2023-05-30 Decibel Therapeutics, Inc. Compositions and methods for treating sensorineural hearing loss using otoferlin dual vector systems
CN119410636A (zh) 2018-04-27 2025-02-11 分贝治疗公司 肌球蛋白15启动子及其用途
JP2022512718A (ja) * 2018-10-15 2022-02-07 フォンダッツィオーネ・テレソン インテインタンパク質及びその使用
AU2019363593A1 (en) * 2018-10-25 2021-04-29 Takeda Pharmaceutical Company Limited AAV triple-plasmid system
WO2020093018A1 (en) 2018-11-01 2020-05-07 University Of Florida Research Foundation, Incorporated A codon optimized otoferlin aav dual vector gene therapy
JP7562544B2 (ja) 2019-02-08 2024-10-07 デシベル セラピューティクス インコーポレイテッド ミオシン１５プロモーター及びその使用
EP3921032A4 (en) 2019-02-08 2022-11-30 Decibel Therapeutics, Inc. COMPOSITIONS AND METHODS FOR TREATMENT OF SENSORINEURAL HEARING LOSS USING OTOFERLIN DOUBLE VECTOR SYSTEMS
EP3956453A4 (en) * 2019-04-19 2023-02-22 University of Massachusetts GENE THERAPIES FOR USHER SYNDROME (USH1B)
US12612645B2 (en) 2019-04-26 2026-04-28 President And Fellows Of Harvard College AAV vectors encoding mini-PCDH15 and uses thereof
JP7665609B2 (ja) 2019-10-30 2025-04-21 デシベル セラピューティクス インコーポレイテッド オトフェリン二重ベクター系及びそれを含む感音難聴を治療するための組成物
WO2021116138A1 (en) * 2019-12-09 2021-06-17 Ucl Business Ltd Gene therapy composition and treatment for myh7-linked cardiomyopathy
US20230090778A1 (en) * 2020-02-07 2023-03-23 The Children's Medical Center Corporation Large gene vectors and delivery and uses thereof
CN116096905A (zh) 2020-02-21 2023-05-09 阿库斯股份有限公司 用于治疗人受试者的非年龄相关性听力损伤的组合物和方法
EP3885440A1 (en) * 2020-03-26 2021-09-29 Splicebio, S.L. Split inteins and their uses
CA3174500A1 (en) 2020-04-01 2021-10-07 University Of Florida Research Foundation, Incorporated Dual aav-myo7a vectors with improved safety for the treatment of ush1b
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
US20240425877A1 (en) * 2021-05-07 2024-12-26 Ucl Business Ltd Abca4 genome editing
EP4611784A2 (en) * 2022-11-04 2025-09-10 National Resilience, Inc. Methods and compositions for preparing recombinant adeno associated viruses and uses thereof
EP4724470A1 (en) 2023-06-12 2026-04-15 Children's Hospital Medical Center Aav-cftr vectors and methods of using same
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 (17)

* 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
PT1071806E (pt) 1998-04-24 2004-09-30 Univ Florida Vector viral adeno-associado recombinante que codifica a alfa-1-antitripsina para terapia genetica
WO2003059396A1 (en) 2002-01-11 2003-07-24 Sergei Zolotukhin Adiponectin gene therapy
WO2003088916A2 (en) * 2002-04-19 2003-10-30 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
AU2007223427A1 (en) * 2006-03-01 2007-09-13 University Of Utah Research Foundation Methods and compositions related to cyclic peptide synthesis
WO2007127428A2 (en) 2006-04-28 2007-11-08 University Of Florida Research Foundation, Inc. Double-stranded/self-complementary vectors with a truncated cba promoter and methods of gene delivery
US8658777B2 (en) * 2007-05-09 2014-02-25 The University Of Tokyo Activated protease indicator
US8236557B2 (en) * 2008-05-28 2012-08-07 University Of Missouri-Columbia Hybrid-AAV vectors to deliver large gene expression cassette
AU2009316660B2 (en) * 2008-11-19 2015-01-29 Amrys, Inc. Compositions and methods for the assembly of polynucleotides
AU2010267963B2 (en) 2009-07-02 2015-09-24 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
EP2986635B1 (en) * 2013-04-18 2018-10-03 Fondazione Telethon Effective delivery of large genes by dual aav vectors
US20180327779A1 (en) * 2015-03-03 2018-11-15 Fondazione Telethon (IT/IT) Multiple vector system and uses thereof

Also Published As

Publication number Publication date
US20180327779A1 (en) 2018-11-15
JP2018512125A (ja) 2018-05-17
EP4089172A3 (en) 2023-03-01
US20220002749A1 (en) 2022-01-06
HRP20220776T1 (hr) 2022-09-16
AU2016227668B2 (en) 2019-06-27
MX2023004479A (es) 2023-05-04
MX2017011255A (es) 2018-08-01
PT3265571T (pt) 2022-06-29
SI3265571T1 (sl) 2022-10-28
CA2979120A1 (en) 2016-09-09
ES2919880T3 (es) 2022-07-28
PL3265571T3 (pl) 2022-09-05
WO2016139321A1 (en) 2016-09-09
CN107466325A (zh) 2017-12-12
CY1125385T1 (el) 2025-05-09
EP4089172A2 (en) 2022-11-16
JP7182873B2 (ja) 2022-12-05
EP3265571B1 (en) 2022-04-13
SMT202200269T1 (it) 2022-09-14
BR112017018728A2 (pt) 2018-04-17
EP3265571A1 (en) 2018-01-10
RS63416B1 (sr) 2022-08-31
JP2022174192A (ja) 2022-11-22
KR102240180B1 (ko) 2021-04-14
AU2016227668A1 (en) 2017-08-31
KR20170140185A (ko) 2017-12-20

Similar Documents

Publication Publication Date Title
EP3659138C0 (en) SELECTION SYSTEM AND METHOD
IL258437B (en) Growing systems and methods
DK3560098T3 (da) Fotovoltaik-anlæg og tilhørende anvendelse
DK3265947T3 (da) Fremgangsmåde og system til kryptering
DK3610005T3 (da) Peptidligase og anvendelse deraf
DK3380101T3 (da) Eif4-a-inhiberende forbindelser og fremgangsmåder relateret dertil
DK3009511T3 (da) Hidtil ukendte crispr-enzymer og systemer
SI3265571T1 (sl) Več vektorski sistem in njegove uporabe
DK3329001T3 (da) Genomredigeringssystemer og anvendelsesfremgangsmåder
DK3218358T3 (da) Ifluorometyl-aminopyridiner og difluorometyl-aminopyrimidiner
DK3380620T3 (da) Optimerede lentivirale transfervektorer og anvendelse heraf
DK3177259T3 (da) Forbindelses- og beholdersystem
DK3481581T3 (da) Metalwiretilførselssystem og fremgangsmåde
DK3161139T3 (da) Tlr-4-specifikke aptamerer og anvendelser heraf
DK3346056T3 (da) Rengøringssystem og fremgangsmåde
DK3101760T3 (da) Opladningsfremgangsmåde og system
DK3209773T3 (da) Prolintolerante tripeptidylpeptidaser og anvendelser deraf
DK3128005T3 (da) Sirp-alfa-variantkonstruktioner og anvendelser deraf
DK3692009T3 (da) Biocementationsfremgangsmåde og system
DK3256598T3 (da) Svampestammer og anvendelsesfremgangsmåder
DK3314589T3 (da) Alarmsystem og fremgangsmåde
DK3265641T3 (da) Stigørenhed og fremgangsmåde
EP3310319C0 (en) CONNECTION AND CONTAINER SYSTEM
DK3321279T3 (da) Exenatidmodificeringsmiddel og anvendelse deraf
DK3231016T3 (da) Fotovoltaikmodul og fotovoltaiksystem