BR112019002904A2 - métodos e composições para transferência gênica direcionada - Google Patents

métodos e composições para transferência gênica direcionada

Info

Publication number
BR112019002904A2
BR112019002904A2 BR112019002904A BR112019002904A BR112019002904A2 BR 112019002904 A2 BR112019002904 A2 BR 112019002904A2 BR 112019002904 A BR112019002904 A BR 112019002904A BR 112019002904 A BR112019002904 A BR 112019002904A BR 112019002904 A2 BR112019002904 A2 BR 112019002904A2
Authority
BR
Brazil
Prior art keywords
compositions
methods
gene transfer
directed gene
viral
Prior art date
Application number
BR112019002904A
Other languages
English (en)
Inventor
Woodard Kenton
Jude Samulski Richard
Original Assignee
Univ North Carolina Chapel Hill
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 Univ North Carolina Chapel Hill filed Critical Univ North Carolina Chapel Hill
Publication of BR112019002904A2 publication Critical patent/BR112019002904A2/pt

Links

Classifications

    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00—Drugs for disorders of the senses
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00—Drugs for disorders of the senses
    • A61P27/02—Ophthalmic agents
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
    • 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
    • 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
    • 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
    • C12N2750/14122—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
    • 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
    • C12N2750/14123—Virus like particles [VLP]
    • 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
    • C12N2750/14142—Use of virus, viral particle or viral elements as a vector virus or viral particle as vehicle, e.g. encapsulating small organic molecule
    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biotechnology (AREA)
  • Medicinal Chemistry (AREA)
  • Molecular Biology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Virology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Epidemiology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Ophthalmology & Optometry (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Peptides Or Proteins (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)

Abstract

a presente invenção refere-se às proteínas de capsídeo de aav compreendendo uma modificação na sequência de aminoácidos e capsídeos virais e vetores virais compreendendo a proteína de capsídeo de aav modificado. a invenção também proporciona métodos de administração dos vetores virais e capsídeos virais da invenção a uma célula ou a um indivíduo in vivo.
BR112019002904A 2016-08-16 2017-08-16 métodos e composições para transferência gênica direcionada BR112019002904A2 (pt)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201662375666P 2016-08-16 2016-08-16
PCT/US2017/047123 WO2018035213A1 (en) 2016-08-16 2017-08-16 Methods and compositions for targeted gene transfer

Publications (1)

Publication Number Publication Date
BR112019002904A2 true BR112019002904A2 (pt) 2019-06-25

Family

ID=61197363

Family Applications (1)

Application Number Title Priority Date Filing Date
BR112019002904A BR112019002904A2 (pt) 2016-08-16 2017-08-16 métodos e composições para transferência gênica direcionada

Country Status (8)

Country Link
US (1) US20190262373A1 (pt)
EP (1) EP3500676A4 (pt)
JP (2) JP7226801B2 (pt)
CN (1) CN109890966A (pt)
AU (1) AU2017313064B2 (pt)
BR (1) BR112019002904A2 (pt)
CA (1) CA3033125A1 (pt)
WO (1) WO2018035213A1 (pt)

Families Citing this family (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
ES2865487T3 (es) 2015-09-28 2021-10-15 Univ North Carolina Chapel Hill Métodos y composiciones para vectores virales que evaden los anticuerpos
KR20210006357A (ko) 2018-04-03 2021-01-18 스트라이드바이오 인코포레이티드 항체-회피 바이러스 벡터
TW202005978A (zh) * 2018-05-14 2020-02-01 美商拜奧馬林製藥公司 新穎肝靶向腺相關病毒載體
KR20220011616A (ko) 2019-03-21 2022-01-28 스트라이드바이오 인코포레이티드 재조합 아데노 관련 바이러스 벡터
EP4045525A1 (en) 2019-10-17 2022-08-24 Stridebio, Inc. Adeno-associated viral vectors for treatment of niemann-pick disease type c
TW202142552A (zh) * 2020-01-29 2021-11-16 美商健臻公司 用於眼部基因療法的經修飾的腺相關病毒衣殼蛋白及其使用方法
MX2022014256A (es) 2020-05-13 2023-01-11 Voyager Therapeutics Inc Redirección del tropismo de las cápsides de aav.
WO2023140837A1 (en) * 2022-01-19 2023-07-27 Massachusetts Eye And Ear Infirmary Optimized aav-based vaccine
WO2023178311A1 (en) * 2022-03-18 2023-09-21 The University Of North Carolina At Chapel Hill Methods for treating disorders under hypothermia and compositions relating to the same

Family Cites Families (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US7867484B2 (en) * 2006-01-27 2011-01-11 University Of North Carolina At Chapel Hill Heparin and heparan sulfate binding chimeric vectors
EP2016174A2 (en) * 2006-04-28 2009-01-21 The Trustees of the University of Pennsylvania Modified aav vectors having reduced capsid immunogenicity and use thereof
US9725485B2 (en) * 2012-05-15 2017-08-08 University Of Florida Research Foundation, Inc. AAV vectors with high transduction efficiency and uses thereof for gene therapy
WO2014144229A1 (en) * 2013-03-15 2014-09-18 The University Of North Carolina At Chapel Hill Methods and compositions for dual glycan binding aav vectors
KR102298579B1 (ko) * 2013-05-21 2021-09-07 유니버시티 오브 플로리다 리서치 파운데이션, 인코포레이티드 캡시드-변형된 raav3 벡터 조성물, 및 인간 간암의 유전자 요법에서의 용도
GB201403684D0 (en) * 2014-03-03 2014-04-16 King S College London Vector
DK3137497T3 (da) * 2014-05-02 2021-07-12 Genzyme Corp Aav-vektorer til retinal- og cns-genterapi
CA2961523A1 (en) * 2014-09-16 2016-03-24 Genzyme Corporation Adeno-associated viral vectors for treating myocilin (myoc) glaucoma
AU2016206624B2 (en) * 2015-01-14 2020-08-06 The University Of North Carolina At Chapel Hill Methods and compositions for targeted gene transfer
ES2865487T3 (es) * 2015-09-28 2021-10-15 Univ North Carolina Chapel Hill Métodos y composiciones para vectores virales que evaden los anticuerpos

Also Published As

Publication number Publication date
AU2017313064A1 (en) 2019-02-21
JP2023055843A (ja) 2023-04-18
CN109890966A (zh) 2019-06-14
WO2018035213A1 (en) 2018-02-22
CA3033125A1 (en) 2018-02-22
US20190262373A1 (en) 2019-08-29
JP7226801B2 (ja) 2023-02-21
JP2019533428A (ja) 2019-11-21
EP3500676A4 (en) 2020-07-01
EP3500676A1 (en) 2019-06-26
AU2017313064B2 (en) 2024-05-02

Similar Documents

Publication Publication Date Title
BR112018003665A2 (pt) métodos e composições para vetores virais de evasão de anticorpo
BR112019002904A2 (pt) métodos e composições para transferência gênica direcionada
MX2019009720A (es) Metodos y composiciones para la transferencia de genes a traves de la vasculatura.
CO2020004738A2 (es) Variantes de cápsides de virus adenoasociados y métodos de uso de estas
BR112021018776A2 (pt) Vetores de vírus adenoassociado recombinante
BR112018011975A2 (pt) composições úteis no tratamento de atrofia muscular espinhal
CL2018003196A1 (es) Cápsides variantes de virus adenoasociados y métodos de uso de estas.
MX2017012097A (es) Variantes de virus adenoasociado y métodos de uso de estas.
MX2020010466A (es) Vectores de virus que evitan anticuerpos.
BR112021019821A2 (pt) Sorotipo de vírus adenoassociado híbrido recombinante com modificação peptídica entre aav9 e aavrh74 com tropismo hepático reduzido e transdução muscular aumentada
BR112017017867A2 (pt) métodos e composições para tratamento de doenças oculares genéticas
BR112021005769A2 (pt) proteínas de ligação a dll3 e métodos de uso
MX2020010465A (es) Vectores de virus para direccionamiento a tejidos oftalmicos.
BR112018011881A2 (pt) proteínas capsidiais modificadas para liberação aumentada de vetores de parvovírus
EA201792573A1 (ru) Триспецифические связанные белки и способы их применения
BR112018068189A2 (pt) proteínas de ligação induzíveis e métodos de uso
BR112017020986A2 (pt) proteínas de ligação recombinantes e seu uso
EA201892561A1 (ru) Гибридные белки gdf15 и их применение
MX2020010464A (es) Vectores de virus que evitan anticuerpos.
WO2015158749A3 (de) Viraler vektor für den zielgerichteten gentransfer in gehirn und rückenmark
BR112022012324A2 (pt) Composições e métodos para modular simultaneamente expressão de genes
EA201691981A1 (ru) Улучшенные транспортные молекулы модулярного антигена и их применение
MX2017008087A (es) Virus de la viruela porcina recombinante y vacunas.
BR112017024283A2 (pt) vacinas contra a dengue
BR112014028951A8 (pt) Peptídeos ledgf, seu uso, composição, sequência de ácido nucleico, e vetor

Legal Events

Date Code Title Description
B350 Update of information on the portal [chapter 15.35 patent gazette]
B06W Patent application suspended after preliminary examination (for patents with searches from other patent authorities) chapter 6.23 patent gazette]