BR112021020545A2 - Capsídeos de aav e composições contendo os mesmos - Google Patents

Capsídeos de aav e composições contendo os mesmos

Info

Publication number
BR112021020545A2
BR112021020545A2 BR112021020545A BR112021020545A BR112021020545A2 BR 112021020545 A2 BR112021020545 A2 BR 112021020545A2 BR 112021020545 A BR112021020545 A BR 112021020545A BR 112021020545 A BR112021020545 A BR 112021020545A BR 112021020545 A2 BR112021020545 A2 BR 112021020545A2
Authority
BR
Brazil
Prior art keywords
same
aav
compositions containing
aav capsids
capsids
Prior art date
Application number
BR112021020545A
Other languages
English (en)
Inventor
M Wilson James
Kalyani Nambiar
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 BR112021020545A2 publication Critical patent/BR112021020545A2/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

Landscapes

  • 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)
  • Wood Science & Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Virology (AREA)
  • Medicinal Chemistry (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Animal Behavior & Ethology (AREA)
  • Epidemiology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (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 aav e composições contendo os mesmos. a presente invenção refere-se a capsídeos de aav e raav compreendendo os mesmos. em uma modalidade, vetores usando um capsídeo de aav mostram transdução aumentada de um tecido alvo selecionado em comparação com um aav da técnica anterior.
BR112021020545A 2019-04-29 2020-04-28 Capsídeos de aav e composições contendo os mesmos BR112021020545A2 (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/US2020/030281 WO2020223236A1 (en) 2019-04-29 2020-04-28 Novel aav capsids and compositions containing same

Publications (1)

Publication Number Publication Date
BR112021020545A2 true BR112021020545A2 (pt) 2022-01-04

Family

ID=73028724

Family Applications (3)

Application Number Title Priority Date Filing Date
BR112021021502A BR112021021502A2 (pt) 2019-04-29 2020-04-28 Capsídeos de aav e composições contendo os mesmos
BR112021020545A BR112021020545A2 (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 (1)

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

Family Applications After (1)

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

Country Status (15)

Country Link
US (3) US20220204990A1 (pt)
EP (3) EP3976078A4 (pt)
JP (3) JP2022530656A (pt)
KR (3) KR20220004681A (pt)
CN (3) CN113853208A (pt)
AU (3) AU2020264976A1 (pt)
BR (3) BR112021021502A2 (pt)
CA (3) CA3134379A1 (pt)
CL (3) CL2021002768A1 (pt)
CO (3) CO2021015924A2 (pt)
IL (3) IL287492A (pt)
MX (3) MX2021013266A (pt)
PE (3) PE20212267A1 (pt)
SG (3) SG11202111373YA (pt)
WO (3) WO2020223232A1 (pt)

Families Citing this family (15)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
MX2022016528A (es) 2020-06-17 2023-06-02 Univ Pennsylvania Composiciones y métodos para el tratamiento de pacientes de terapia génica.
JP2023543125A (ja) 2020-08-24 2023-10-13 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 Ｇｌｐ－１受容体アゴニスト融合物をコードするウイルスベクター及び代謝性疾患の治療におけるその使用
WO2022094078A1 (en) 2020-10-28 2022-05-05 The Trustees Of The University Of Pennsylvania Compositions useful in treatment of rett syndrome
JP2023548432A (ja) 2020-11-11 2023-11-16 ヨーロピアン モレキュラー バイオロジー ラボラトリー 遺伝子療法のための改変ウイルス粒子
TW202237850A (zh) 2020-12-01 2022-10-01 賓州大學委員會 具有組織特異性靶向基序的新穎構成物及含有其之組成物
IL303239A (en) * 2020-12-01 2023-07-01 Univ Pennsylvania Compositions and their uses for the treatment of Engelmann syndrome
AU2022262771A1 (en) 2021-04-23 2023-11-02 The Trustees Of The University Of Pennsylvania Novel compositions with brain-specific targeting motifs and compositions containing same
WO2023147304A1 (en) 2022-01-25 2023-08-03 The Trustees Of The University Of Pennsylvania Aav capsids for improved heart transduction and detargeting of liver
WO2023168293A2 (en) * 2022-03-01 2023-09-07 The Trustees Of The University Of Pennsylvania 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

Family Cites Families (18)

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EP2338900B1 (en) * 2001-11-13 2014-01-01 The Trustees of The University of Pennsylvania Adeno-associated virus (AAV) Cy.5 sequences, vectors containing the same and uses thereof
DK2292779T3 (en) * 2003-09-30 2017-02-27 Univ Pennsylvania ADENOASS-ASSOCIATED VIRUS (AAV) CLADES, SEQUENCES, VECTORS CONTAINING SAME AND APPLICATIONS THEREOF
CN106906241B (zh) * 2005-04-07 2020-09-11 宾夕法尼亚大学托管会 增强腺相关病毒载体功能的方法
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
JP5963743B2 (ja) * 2010-04-23 2016-08-03 ユニバーシティ オブ マサチューセッツ Ｃｎｓターゲティングａａｖベクターおよびその使用方法
MX2016011585A (es) * 2014-03-10 2016-11-29 Uniqure Ip Bv Vectores aav mejorados adicionales producidos en celulas de insecto.
US10577627B2 (en) * 2014-06-09 2020-03-03 Voyager Therapeutics, Inc. Chimeric capsids
US20180021364A1 (en) * 2015-01-16 2018-01-25 Voyager Therapeutics, Inc. Central nervous system targeting polynucleotides
DK3872085T3 (da) * 2015-07-30 2023-04-03 Massachusetts Eye & Ear Infirmary Stam-virussekvenser og anvendelser heraf
US20180230489A1 (en) * 2015-10-28 2018-08-16 Voyager Therapeutics, Inc. Regulatable expression using adeno-associated virus (aav)
CA3002406A1 (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
US11512327B2 (en) * 2017-08-03 2022-11-29 Voyager Therapeutics, Inc. Compositions and methods for delivery of AAV

Also Published As

Publication number Publication date
WO2020223231A1 (en) 2020-11-05
PE20212267A1 (es) 2021-11-30
US20220204990A1 (en) 2022-06-30
CL2021002824A1 (es) 2022-05-27
EP3962507A1 (en) 2022-03-09
EP3976078A4 (en) 2023-08-30
JP2022530544A (ja) 2022-06-29
CA3134468A1 (en) 2020-11-05
KR20220004681A (ko) 2022-01-11
SG11202111373YA (en) 2021-11-29
SG11202111375RA (en) 2021-11-29
CA3134507A1 (en) 2020-11-05
CN113853208A (zh) 2021-12-28
JP2022530656A (ja) 2022-06-30
JP2022530633A (ja) 2022-06-30
KR20220004695A (ko) 2022-01-11
BR112021021502A2 (pt) 2022-07-19
EP3976078A1 (en) 2022-04-06
MX2021013266A (es) 2021-11-17
CL2021002768A1 (es) 2022-05-27
CO2021015924A2 (es) 2021-11-30
KR20220004680A (ko) 2022-01-11
IL287498A (en) 2021-12-01
AU2020266523A1 (en) 2021-11-04
IL287491A (en) 2021-12-01
AU2020264975A1 (en) 2021-11-11
EP3976077A1 (en) 2022-04-06
CN113853209A (zh) 2021-12-28
EP3976077A4 (en) 2023-08-30
CL2021002811A1 (es) 2022-05-27
PE20212334A1 (es) 2021-12-14
BR112021020054A2 (pt) 2021-12-07
CN113853207A (zh) 2021-12-28
IL287492A (en) 2021-12-01
PE20220014A1 (es) 2022-01-11
US20220249705A1 (en) 2022-08-11
WO2020223232A1 (en) 2020-11-05
CO2021015916A2 (es) 2021-11-30
CO2021015928A2 (es) 2021-11-30
AU2020264976A1 (en) 2021-11-11
US20220220453A1 (en) 2022-07-14
MX2021013268A (es) 2021-11-17
CA3134379A1 (en) 2020-11-05
EP3962507A4 (en) 2023-10-11
WO2020223236A1 (en) 2020-11-05
SG11202111102SA (en) 2021-11-29
MX2021013267A (es) 2021-11-17

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