AU2022256479A1 - Rational polyploid aav virions that cross the blood brain barrier and elicit reduced humoral response - Google Patents

Rational polyploid aav virions that cross the blood brain barrier and elicit reduced humoral response Download PDF

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Publication number
AU2022256479A1
AU2022256479A1 AU2022256479A AU2022256479A AU2022256479A1 AU 2022256479 A1 AU2022256479 A1 AU 2022256479A1 AU 2022256479 A AU2022256479 A AU 2022256479A AU 2022256479 A AU2022256479 A AU 2022256479A AU 2022256479 A1 AU2022256479 A1 AU 2022256479A1
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aav
population
serotype
rational
polyploid
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AU2022256479A9 (en
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Audry FERNANDEZ
Amaury Pupo MERINO
Lester SUAREZ
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AskBio Inc
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Asklepios Biopharmaceutical Inc
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Assigned to AskBio Inc. reassignment AskBio Inc. Amend patent request/document other than specification (104) Assignors: ASKLEPIOS BIOPHARMACEUTICAL, INC.
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    • A—HUMAN NECESSITIES
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    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
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    • A61K48/0025—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid
    • A61K48/0041—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid the non-active part being polymeric
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    • A61K39/00—Medicinal preparations containing antigens or antibodies
    • A61K2039/57—Medicinal preparations containing antigens or antibodies characterised by the type of response, e.g. Th1, Th2
    • A61K2039/577—Medicinal preparations containing antigens or antibodies characterised by the type of response, e.g. Th1, Th2 tolerising response
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    • 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
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    • 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
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    • 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
    • C12N2750/14145—Special targeting system for viral vectors

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  • Health & Medical Sciences (AREA)
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  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
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  • Organic Chemistry (AREA)
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  • Wood Science & Technology (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
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  • Microbiology (AREA)
  • Molecular Biology (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
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  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Immunology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Mycology (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Materials For Medical Uses (AREA)
  • Medical Preparation Storing Or Oral Administration Devices (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Peptides Or Proteins (AREA)
AU2022256479A 2021-04-16 2022-04-14 Rational polyploid aav virions that cross the blood brain barrier and elicit reduced humoral response Pending AU2022256479A1 (en)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US202163175954P 2021-04-16 2021-04-16
US63/175,954 2021-04-16
US202163180414P 2021-04-27 2021-04-27
US63/180,414 2021-04-27
PCT/US2022/024809 WO2022221529A1 (en) 2021-04-16 2022-04-14 Rational polyploid aav virions that cross the blood brain barrier and elicit reduced humoral response

Publications (2)

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AU2022256479A1 true AU2022256479A1 (en) 2023-11-30
AU2022256479A9 AU2022256479A9 (en) 2023-12-14

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US (1) US20240189447A1 (https=)
EP (1) EP4323015A4 (https=)
JP (1) JP2024515626A (https=)
AU (1) AU2022256479A1 (https=)
CA (1) CA3216491A1 (https=)
WO (1) WO2022221529A1 (https=)

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* Cited by examiner, † Cited by third party
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WO2024249692A2 (en) * 2023-05-30 2024-12-05 Asklepios Biopharmaceutical, Inc. Methods of recombinant adeno-associated virus kidney administration
WO2025121957A1 (ko) * 2023-12-06 2025-06-12 주식회사 글루진테라퓨틱스 뇌 성상교세포 특이적 유전자 전달이 가능한 아데노부속바이러스 변이체와 이의 응용

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