PE20212267A1 - Nuevas capsides de aav y composiciones que las contienen - Google Patents

Nuevas capsides de aav y composiciones que las contienen

Info

Publication number
PE20212267A1
PE20212267A1 PE2021001795A PE2021001795A PE20212267A1 PE 20212267 A1 PE20212267 A1 PE 20212267A1 PE 2021001795 A PE2021001795 A PE 2021001795A PE 2021001795 A PE2021001795 A PE 2021001795A PE 20212267 A1 PE20212267 A1 PE 20212267A1
Authority
PE
Peru
Prior art keywords
novel aav
compositions containing
capsides
aav capsides
novel
Prior art date
Application number
PE2021001795A
Other languages
English (en)
Inventor
Kalyani Nambiar
James M Wilson
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 PE20212267A1 publication Critical patent/PE20212267A1/es

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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)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (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)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (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

En la presente se proporcionan nuevas capsides de AAV y rAAV que las comprenden. En una forma de realizacion, los vectores que utilizan una nueva capside de AAV demuestran una transduccion mayor de un tejido diana seleccionado en comparacion con un AAV del arte previo
PE2021001795A 2019-04-29 2020-04-28 Nuevas capsides de aav y composiciones que las contienen PE20212267A1 (es)

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
PE20212267A1 true PE20212267A1 (es) 2021-11-30

Family

ID=73028724

Family Applications (3)

Application Number Title Priority Date Filing Date
PE2021001789A PE20212334A1 (es) 2019-04-29 2020-04-28 Nuevas capsides de aav y composiciones que las contienen
PE2021001788A PE20220014A1 (es) 2019-04-29 2020-04-28 Nuevas capsides de aav y composiciones que las contienen
PE2021001795A PE20212267A1 (es) 2019-04-29 2020-04-28 Nuevas capsides de aav y composiciones que las contienen

Family Applications Before (2)

Application Number Title Priority Date Filing Date
PE2021001789A PE20212334A1 (es) 2019-04-29 2020-04-28 Nuevas capsides de aav y composiciones que las contienen
PE2021001788A PE20220014A1 (es) 2019-04-29 2020-04-28 Nuevas capsides de aav y composiciones que las contienen

Country Status (15)

Country Link
US (3) US20220204990A1 (es)
EP (3) EP3962507A4 (es)
JP (3) JP2022530633A (es)
KR (3) KR20220004681A (es)
CN (3) CN113853207A (es)
AU (3) AU2020264976A1 (es)
BR (3) BR112021020545A2 (es)
CA (3) CA3134468A1 (es)
CL (3) CL2021002768A1 (es)
CO (3) CO2021015928A2 (es)
IL (3) IL287492A (es)
MX (3) MX2021013268A (es)
PE (3) PE20212334A1 (es)
SG (3) SG11202111102SA (es)
WO (3) WO2020223232A1 (es)

Families Citing this family (13)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
TW202214695A (zh) 2020-06-17 2022-04-16 賓州大學委員會 用於治療基因療法患者之組成物及方法
MX2023002293A (es) 2020-08-24 2023-05-19 Univ Pennsylvania Vectores virales que codifican fusiones de agonistas del receptor de glp-1 y usos de estos en el tratamiento de enfermedades metabólica.
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
JP2023551911A (ja) 2020-12-01 2023-12-13 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 アンジェルマン症候群の治療のための組成物及びその使用
EP4256065A2 (en) 2020-12-01 2023-10-11 The Trustees of The University of Pennsylvania Novel compositions with tissue-specific targeting motifs and compositions containing same
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
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

Family Cites Families (18)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
PL220644B1 (pl) * 2001-11-13 2015-11-30 Univ Pennsylvania Wirus stowarzyszony z adenowirusem (AAV), kompozycja, wyizolowane białko kapsydowe, wyizolowane lub syntetyczne cząsteczki kwasu nukleinowego, sposób wytwarzania zrekombinowanego wirusa, komórka gospodarza
US7906111B2 (en) * 2003-09-30 2011-03-15 The Trustees Of The University Of Pennsylvania Adeno-associated virus (AAV) clades, sequences, vectors containing same, and uses therefor
EP4234687A2 (en) * 2005-04-07 2023-08-30 The Trustees of the University of Pennsylvania Method of increasing the function of an aav vector
CN101495624A (zh) * 2006-04-28 2009-07-29 宾夕法尼亚州立大学托管会 衣壳免疫原性降低的经修饰aav载体及其用途
EP2826860B1 (en) * 2010-04-23 2018-08-22 University of Massachusetts CNS targeting AAV vectors and methods of use thereof
MX2016011585A (es) * 2014-03-10 2016-11-29 Uniqure Ip Bv Vectores aav mejorados adicionales producidos en celulas de insecto.
WO2015191508A1 (en) * 2014-06-09 2015-12-17 Voyager Therapeutics, Inc. Chimeric capsids
CA2970730A1 (en) * 2015-01-16 2016-07-21 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)
EP3368065A4 (en) * 2015-10-29 2019-03-20 Voyager Therapeutics, Inc. DISTRIBUTION OF POLYNUCLEOTIDES FOR TARGETING THE CENTRAL NERVOUS SYSTEM
WO2017189959A1 (en) * 2016-04-29 2017-11-02 Voyager Therapeutics, Inc. Compositions for the treatment of disease
CN110214187B (zh) * 2016-05-18 2024-01-30 沃雅戈治疗公司 调节性多核苷酸
AU2017345470B2 (en) * 2016-10-19 2023-08-03 Adverum Biotechnologies, Inc. Modified AAV capsids and uses thereof
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
WO2019028306A2 (en) * 2017-08-03 2019-02-07 Voyager Therapeutics, Inc. COMPOSITIONS AND METHODS FOR ADMINISTRATION OF ADENO-ASSOCIATED VIRUSES

Also Published As

Publication number Publication date
KR20220004680A (ko) 2022-01-11
CO2021015924A2 (es) 2021-11-30
EP3962507A4 (en) 2023-10-11
KR20220004681A (ko) 2022-01-11
SG11202111102SA (en) 2021-11-29
US20220220453A1 (en) 2022-07-14
PE20220014A1 (es) 2022-01-11
CN113853209A (zh) 2021-12-28
KR20220004695A (ko) 2022-01-11
CN113853208A (zh) 2021-12-28
CL2021002768A1 (es) 2022-05-27
CA3134379A1 (en) 2020-11-05
BR112021021502A2 (pt) 2022-07-19
WO2020223231A1 (en) 2020-11-05
US20220204990A1 (en) 2022-06-30
JP2022530656A (ja) 2022-06-30
EP3976078A1 (en) 2022-04-06
CA3134468A1 (en) 2020-11-05
EP3976077A4 (en) 2023-08-30
PE20212334A1 (es) 2021-12-14
CL2021002811A1 (es) 2022-05-27
JP2022530633A (ja) 2022-06-30
SG11202111375RA (en) 2021-11-29
CO2021015928A2 (es) 2021-11-30
CN113853207A (zh) 2021-12-28
EP3976077A1 (en) 2022-04-06
MX2021013268A (es) 2021-11-17
AU2020264976A1 (en) 2021-11-11
IL287492A (en) 2021-12-01
CA3134507A1 (en) 2020-11-05
BR112021020054A2 (pt) 2021-12-07
SG11202111373YA (en) 2021-11-29
US20220249705A1 (en) 2022-08-11
AU2020264975A1 (en) 2021-11-11
AU2020266523A1 (en) 2021-11-04
JP2022530544A (ja) 2022-06-29
MX2021013267A (es) 2021-11-17
WO2020223232A1 (en) 2020-11-05
IL287498A (en) 2021-12-01
MX2021013266A (es) 2021-11-17
BR112021020545A2 (pt) 2022-01-04
CO2021015916A2 (es) 2021-11-30
CL2021002824A1 (es) 2022-05-27
IL287491A (en) 2021-12-01
WO2020223236A1 (en) 2020-11-05
EP3962507A1 (en) 2022-03-09
EP3976078A4 (en) 2023-08-30

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