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BR112021020054A2 - Capsídeos de vírus adeno-associado (aav) e composições contendo os mesmos - Google Patents

Capsídeos de vírus adeno-associado (aav) e composições contendo os mesmos

Info

Publication number
BR112021020054A2
BR112021020054A2 BR112021020054A BR112021020054A BR112021020054A2 BR 112021020054 A2 BR112021020054 A2 BR 112021020054A2 BR 112021020054 A BR112021020054 A BR 112021020054A BR 112021020054 A BR112021020054 A BR 112021020054A BR 112021020054 A2 BR112021020054 A2 BR 112021020054A2
Authority
BR
Brazil
Prior art keywords
aav
capsids
same
adeno
compositions containing
Prior art date
Application number
BR112021020054A
Other languages
English (en)
Inventor
M Wilson James
Kalyani Nambiar
Qiang Wang
Original Assignee
Univ Pennsylvania
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 Pennsylvania filed Critical Univ Pennsylvania
Publication of BR112021020054A2 publication Critical patent/BR112021020054A2/pt

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    • 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
    • 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
    • A61K48/0075—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 delivery route, e.g. oral, subcutaneous
    • 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
    • C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00—Medicinal preparations containing antigens or antibodies
    • 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
    • C12N2750/14141—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
    • 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
    • C12N2750/14151—Methods of production or purification of viral material
    • 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
    • C12N2750/14151—Methods of production or purification of viral material
    • C12N2750/14152—Methods of production or purification of viral material relating to complementing cells and packaging systems for producing virus or viral particles

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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)
  • Biotechnology (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Biochemistry (AREA)
  • Virology (AREA)
  • Medicinal Chemistry (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Epidemiology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Immunology (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Peptides Or Proteins (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
  • Medicinal Preparation (AREA)
  • Materials For Medical Uses (AREA)

Abstract

capsídeos de vírus adeno-associado (aav) e composições contendo os mesmos. são fornecidos aqui capsídeos de aav e raav compreendendo os mesmos. em uma modalidade, vetores empregando um capsídeo de aav mostram transdução aumentada de um tecido alvo selecionado em comparação com um aav da técnica anterior.
BR112021020054A 2019-04-29 2020-04-28 Capsídeos de vírus adeno-associado (aav) e composições contendo os mesmos BR112021020054A2 (pt)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US201962840184P 2019-04-29 2019-04-29
US201962913314P 2019-10-10 2019-10-10
US201962924095P 2019-10-21 2019-10-21
PCT/US2020/030266 WO2020223231A1 (en) 2019-04-29 2020-04-28 Novel aav capsids and compositions containing same

Publications (1)

Publication Number Publication Date
BR112021020054A2 true BR112021020054A2 (pt) 2021-12-07

Family

ID=73028724

Family Applications (3)

Application Number Title Priority Date Filing Date
BR112021020545A BR112021020545A2 (pt) 2019-04-29 2020-04-28 Capsídeos de aav e composições contendo os mesmos
BR112021021502A BR112021021502A2 (pt) 2019-04-29 2020-04-28 Capsídeos de aav e composições contendo os mesmos
BR112021020054A BR112021020054A2 (pt) 2019-04-29 2020-04-28 Capsídeos de vírus adeno-associado (aav) e composições contendo os mesmos

Family Applications Before (2)

Application Number Title Priority Date Filing Date
BR112021020545A BR112021020545A2 (pt) 2019-04-29 2020-04-28 Capsídeos de aav e composições contendo os mesmos
BR112021021502A BR112021021502A2 (pt) 2019-04-29 2020-04-28 Capsídeos de aav e composições contendo os mesmos

Country Status (15)

Country Link
US (3) US20220204990A1 (pt)
EP (3) EP3962507A4 (pt)
JP (3) JP2022530544A (pt)
KR (3) KR20220004680A (pt)
CN (3) CN113853209A (pt)
AU (3) AU2020264975A1 (pt)
BR (3) BR112021020545A2 (pt)
CA (3) CA3134468A1 (pt)
CL (3) CL2021002768A1 (pt)
CO (3) CO2021015916A2 (pt)
IL (3) IL287498A (pt)
MX (3) MX2021013267A (pt)
PE (3) PE20212334A1 (pt)
SG (3) SG11202111373YA (pt)
WO (3) WO2020223236A1 (pt)

Families Citing this family (15)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2023531451A (ja) 2020-06-17 2023-07-24 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア 遺伝子療法患者の治療のための組成物及び方法
BR112023003310A2 (pt) 2020-08-24 2023-05-02 Univ Pennsylvania Vetores virais que codificam fusões de agonistas do receptor de glp-1 e usos dos mesmos no tratamento de doenças metabólicas
WO2022094078A1 (en) 2020-10-28 2022-05-05 The Trustees Of The University Of Pennsylvania Compositions useful in treatment of rett syndrome
CA3201533A1 (en) 2020-11-11 2022-05-19 European Molecular Biology Laboratory Modified viral particles for gene therapy
US20230414785A1 (en) 2020-12-01 2023-12-28 The Trustees Of The University Of Pennsylvania Compositions and uses thereof for treatment of angelman syndrome
AR124216A1 (es) 2020-12-01 2023-03-01 Univ Pennsylvania Composiciones nuevas con motivos selectivos específicos del tejido y composiciones que las contienen
CA3216004A1 (en) 2021-04-23 2022-10-27 The Trustees Of The University Of Pennsylvania Novel compositions with brain-specific targeting motifs and compositions containing same
KR20240137067A (ko) 2022-01-25 2024-09-19 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 개선된 심장 형질도입 및 간의 탈표적화를 위한 aav 캡시드
IL315248A (en) * 2022-03-01 2024-10-01 Univ Pennsylvania A viral vector genome encoding an insulin fusion protein
WO2023168403A2 (en) * 2022-03-03 2023-09-07 The Trustees Of The University Of Pennsylvania Viral vectors encoding parathyroid hormone fusions and uses thereof in treating hypoparathyroidism
WO2023196893A1 (en) 2022-04-06 2023-10-12 The Trustees Of The University Of Pennsylvania Compositions and methods for treating her2 positive metastatic breast cancer and other cancers
WO2023196892A1 (en) 2022-04-06 2023-10-12 The Trustees Of The University Of Pennsylvania Passive immunization with anti- aav neutralizing antibodies to prevent off-target transduction of intrathecally delivered aav vectors
WO2024015966A2 (en) * 2022-07-15 2024-01-18 The Trustees Of The University Of Pennsylvania Recombinant aav having aav clade d and clade e capsids and compositions containing same
WO2024130070A2 (en) 2022-12-17 2024-06-20 The Trustees Of The University Of Pennsylvania Recombinant aav capsids with cardiac- and skeletal muscle- specific targeting motifs and uses thereof
WO2024130067A2 (en) 2022-12-17 2024-06-20 The Trustees Of The University Of Pennsylvania Recombinant aav mutant vectors with cardiac and skeletal muscle-specific targeting motifs and compositions containing same

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NZ600121A (en) * 2001-11-13 2013-12-20 Univ Pennsylvania A method of detecting and/or identifying adeno-associated virus (aav) sequences and isolating novel sequences identified thereby
JP5054975B2 (ja) * 2003-09-30 2012-10-24 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア アデノ随伴ウイルス（ａａｖ）の同源系統群、配列、それらを含有するベクターおよびそれらの用途
EP4282956A3 (en) * 2005-04-07 2024-03-13 The Trustees of the University of Pennsylvania Method of increasing the function of an aav vector
WO2008027084A2 (en) * 2006-04-28 2008-03-06 The Trustees Of The University Of Pennsylvania Modified aav vectors having reduced capsid immunogenicity and use thereof
EP3540055A1 (en) * 2010-04-23 2019-09-18 University of Massachusetts Cns targeting aav vectors and methods of use thereof
IL247729B2 (en) * 2014-03-10 2023-09-01 Uniqure Ip Bv Improved aav vectors produced in insect cells
WO2015191508A1 (en) * 2014-06-09 2015-12-17 Voyager Therapeutics, Inc. Chimeric capsids
EP3632923A1 (en) * 2015-01-16 2020-04-08 Voyager Therapeutics, Inc. Central nervous system targeting polynucleotides
DK3872085T5 (da) * 2015-07-30 2024-08-12 Massachusetts Eye & Ear Infirmary Stam-virussekvenser og anvendelser heraf
WO2017075335A1 (en) * 2015-10-28 2017-05-04 Voyager Therapeutics, Inc. Regulatable expression using adeno-associated virus (aav)
WO2017075338A2 (en) * 2015-10-29 2017-05-04 Voyager Therapeutics, Inc. Delivery of central nervous system targeting polynucleotides
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US10550405B2 (en) * 2017-03-15 2020-02-04 The University Of North Carolina At Chapel Hill Rational polyploid adeno-associated virus vectors and methods of making and using the same
WO2018200419A1 (en) * 2017-04-23 2018-11-01 The Trustees Of The University Of Pennsylvania Viral vectors comprising engineered aav capsids and compositions containing the same
JOP20190269A1 (ar) * 2017-06-15 2019-11-20 Voyager Therapeutics Inc بولي نوكليوتيدات aadc لعلاج مرض باركنسون
CN111448308A (zh) * 2017-08-03 2020-07-24 沃雅戈治疗公司 递送aav的组合物和方法

Also Published As

Publication number Publication date
PE20212334A1 (es) 2021-12-14
EP3976078A4 (en) 2023-08-30
IL287491A (en) 2021-12-01
CL2021002768A1 (es) 2022-05-27
IL287492A (en) 2021-12-01
CL2021002824A1 (es) 2022-05-27
AU2020266523A1 (en) 2021-11-04
KR20220004681A (ko) 2022-01-11
US20220204990A1 (en) 2022-06-30
CA3134468A1 (en) 2020-11-05
BR112021020545A2 (pt) 2022-01-04
EP3976078A1 (en) 2022-04-06
CO2021015928A2 (es) 2021-11-30
CA3134379A1 (en) 2020-11-05
EP3962507A4 (en) 2023-10-11
CN113853208A (zh) 2021-12-28
PE20220014A1 (es) 2022-01-11
EP3976077A4 (en) 2023-08-30
JP2022530544A (ja) 2022-06-29
US20220220453A1 (en) 2022-07-14
CA3134507A1 (en) 2020-11-05
CL2021002811A1 (es) 2022-05-27
US20220249705A1 (en) 2022-08-11
BR112021021502A2 (pt) 2022-07-19
CN113853209A (zh) 2021-12-28
WO2020223236A1 (en) 2020-11-05
IL287498A (en) 2021-12-01
KR20220004680A (ko) 2022-01-11
MX2021013268A (es) 2021-11-17
EP3976077A1 (en) 2022-04-06
MX2021013266A (es) 2021-11-17
PE20212267A1 (es) 2021-11-30
SG11202111373YA (en) 2021-11-29
JP2022530633A (ja) 2022-06-30
SG11202111375RA (en) 2021-11-29
AU2020264975A1 (en) 2021-11-11
CO2021015916A2 (es) 2021-11-30
AU2020264976A1 (en) 2021-11-11
WO2020223232A1 (en) 2020-11-05
CN113853207A (zh) 2021-12-28
JP2022530656A (ja) 2022-06-30
KR20220004695A (ko) 2022-01-11
CO2021015924A2 (es) 2021-11-30
EP3962507A1 (en) 2022-03-09
SG11202111102SA (en) 2021-11-29
MX2021013267A (es) 2021-11-17
WO2020223231A1 (en) 2020-11-05

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