PE20212334A1 - Nuevas capsides de aav y composiciones que las contienen - Google Patents

Nuevas capsides de aav y composiciones que las contienen

Info

Publication number
PE20212334A1
PE20212334A1 PE2021001789A PE2021001789A PE20212334A1 PE 20212334 A1 PE20212334 A1 PE 20212334A1 PE 2021001789 A PE2021001789 A PE 2021001789A PE 2021001789 A PE2021001789 A PE 2021001789A PE 20212334 A1 PE20212334 A1 PE 20212334A1
Authority
PE
Peru
Prior art keywords
novel aav
compositions containing
capsides
novel
aav capsides
Prior art date
Application number
PE2021001789A
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 PE20212334A1 publication Critical patent/PE20212334A1/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

Abstract

En el presente documento se proporcionan nuevas capsides de AAV y rAAV que las comprenden. En una forma de realizacion, los vectores que emplean una nueva capside de AAV muestran una mayor transduccion de un tejido diana seleccionado en comparacion con un AAV de la tecnica anterior
PE2021001789A 2019-04-29 2020-04-28 Nuevas capsides de aav y composiciones que las contienen PE20212334A1 (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/US2020/030273 WO2020223232A1 (en) 2019-04-29 2020-04-28 Novel aav capsids and compositions containing same

Publications (1)

Publication Number Publication Date
PE20212334A1 true PE20212334A1 (es) 2021-12-14

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 After (2)

Application Number Title Priority Date Filing Date
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

Country Status (15)

Country Link
US (3) US20220204990A1 (es)
EP (3) EP3976078A4 (es)
JP (3) JP2022530633A (es)
KR (3) KR20220004695A (es)
CN (3) CN113853209A (es)
AU (3) AU2020264976A1 (es)
BR (3) BR112021020545A2 (es)
CA (3) CA3134468A1 (es)
CL (3) CL2021002768A1 (es)
CO (3) CO2021015916A2 (es)
IL (3) IL287491A (es)
MX (3) MX2021013267A (es)
PE (3) PE20212334A1 (es)
SG (3) SG11202111373YA (es)
WO (3) WO2020223231A1 (es)

Families Citing this family (13)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20230220069A1 (en) 2020-06-17 2023-07-13 The Trustees Of The University Of Pennsylvania Compositions and methods for treatment of gene therapy patients
CA3190399A1 (en) 2020-08-24 2022-03-03 James M. Wilson Viral vectors encoding glp-1 receptor agonist fusions and uses thereof in treating metabolic diseases
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
MX2023006445A (es) * 2020-12-01 2023-08-10 Univ Pennsylvania Composiciones y usos de estas para el tratamiento del síndrome de angelman.
AR124216A1 (es) 2020-12-01 2023-03-01 Univ Pennsylvania Composiciones nuevas con motivos selectivos específicos del tejido y composiciones que las contienen
WO2022226263A1 (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

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NZ532635A (en) * 2001-11-13 2007-05-31 Univ Pennsylvania A method of identifying unknown adeno-associated virus (AAV) sequences and a kit for the method
ES2648241T3 (es) * 2003-09-30 2017-12-29 The Trustees Of The University Of Pennsylvania Clados de virus adenoasociados (AAV), secuencias, vectores que contienen el mismo, y usos de los mismos
ES2525067T3 (es) * 2005-04-07 2014-12-17 The Trustees Of The University Of Pennsylvania Método de incremento de la función de un vector de AAV
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
DK2826860T3 (en) * 2010-04-23 2018-12-03 Univ Massachusetts CNS targeting AAV vectors and methods for their use
CN106459984B (zh) * 2014-03-10 2021-09-07 优尼科Ip有限公司 昆虫细胞中产生的进一步改善的aav载体
WO2015191508A1 (en) * 2014-06-09 2015-12-17 Voyager Therapeutics, Inc. Chimeric capsids
EP3245221A4 (en) * 2015-01-16 2018-06-13 Voyager Therapeutics, Inc. Central nervous system targeting polynucleotides
EP3872085B1 (en) * 2015-07-30 2023-03-01 Massachusetts Eye & Ear Infirmary Ancestral aav sequences and uses thereof
US20180230489A1 (en) * 2015-10-28 2018-08-16 Voyager Therapeutics, Inc. Regulatable expression using adeno-associated virus (aav)
US20190055578A1 (en) * 2015-10-29 2019-02-21 Voyager Therapeutics, Inc. Delivery of central nervous system targeting polynucleotides
WO2017189959A1 (en) * 2016-04-29 2017-11-02 Voyager Therapeutics, Inc. Compositions for the treatment of disease
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WO2018075798A1 (en) * 2016-10-19 2018-04-26 Adverum Biotechnologies, Inc. Modified aav capsids and uses thereof
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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
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Also Published As

Publication number Publication date
BR112021021502A2 (pt) 2022-07-19
US20220249705A1 (en) 2022-08-11
MX2021013268A (es) 2021-11-17
CL2021002811A1 (es) 2022-05-27
KR20220004680A (ko) 2022-01-11
WO2020223231A1 (en) 2020-11-05
EP3976077A1 (en) 2022-04-06
CL2021002768A1 (es) 2022-05-27
CN113853209A (zh) 2021-12-28
EP3962507A1 (en) 2022-03-09
CO2021015916A2 (es) 2021-11-30
CA3134379A1 (en) 2020-11-05
PE20220014A1 (es) 2022-01-11
WO2020223232A1 (en) 2020-11-05
SG11202111373YA (en) 2021-11-29
EP3976078A4 (en) 2023-08-30
CA3134468A1 (en) 2020-11-05
CL2021002824A1 (es) 2022-05-27
IL287492A (en) 2021-12-01
US20220204990A1 (en) 2022-06-30
EP3976078A1 (en) 2022-04-06
SG11202111375RA (en) 2021-11-29
SG11202111102SA (en) 2021-11-29
CN113853207A (zh) 2021-12-28
KR20220004695A (ko) 2022-01-11
US20220220453A1 (en) 2022-07-14
AU2020264975A1 (en) 2021-11-11
EP3976077A4 (en) 2023-08-30
WO2020223236A1 (en) 2020-11-05
MX2021013267A (es) 2021-11-17
PE20212267A1 (es) 2021-11-30
JP2022530656A (ja) 2022-06-30
JP2022530633A (ja) 2022-06-30
CO2021015928A2 (es) 2021-11-30
KR20220004681A (ko) 2022-01-11
BR112021020545A2 (pt) 2022-01-04
BR112021020054A2 (pt) 2021-12-07
IL287491A (en) 2021-12-01
AU2020266523A1 (en) 2021-11-04
MX2021013266A (es) 2021-11-17
CN113853208A (zh) 2021-12-28
CA3134507A1 (en) 2020-11-05
EP3962507A4 (en) 2023-10-11
AU2020264976A1 (en) 2021-11-11
JP2022530544A (ja) 2022-06-29
CO2021015924A2 (es) 2021-11-30
IL287498A (en) 2021-12-01

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