AR125467A1 - PIG DERIVED ADENO ASSOCIATED VIRUS CAPSIDS AND THEIR USES - Google Patents

PIG DERIVED ADENO ASSOCIATED VIRUS CAPSIDS AND THEIR USES

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Publication number
AR125467A1
AR125467A1 ARP220101102A ARP220101102A AR125467A1 AR 125467 A1 AR125467 A1 AR 125467A1 AR P220101102 A ARP220101102 A AR P220101102A AR P220101102 A ARP220101102 A AR P220101102A AR 125467 A1 AR125467 A1 AR 125467A1
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Argentina
Prior art keywords
seq
sequence
aav
capsid
raav
Prior art date
Application number
ARP220101102A
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Spanish (es)
Inventor
Qiang Wang
James M Wilson
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Univ Pennsylvania
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Publication date
Application filed by Univ Pennsylvania filed Critical Univ Pennsylvania
Publication of AR125467A1 publication Critical patent/AR125467A1/en

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    • 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
    • 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
    • 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
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector

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  • Life Sciences & Earth Sciences (AREA)
  • Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Virology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Biophysics (AREA)
  • Biomedical Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Biochemistry (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Medicinal Chemistry (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)

Abstract

Se proporcionan nuevas cápsides de virus adenoasociados (AAV) derivadas de porcinos y vectores AAV recombinantes que los comprenden. Además, se proporcionan métodos para el suministro de un transgén con los vectores AAV recombinantes descritos en el presente documento. Reivindicación 1: Un virus adenoasociado recombinante (rAAV) que tiene una cápside caracterizado porque comprende una proteína de la cápside que tiene una secuencia de vp1, vp2 y/o vp3 de AAVpoG015 (SEQ ID Nº 26), AAVpoG001 (SEQ ID Nº 2), AAVpoG002 (SEQ ID Nº 4), AAVpoG003 (SEQ ID Nº 6), AAVpoG004 (SEQ ID Nº 8), AAVpoG005 (SEQ ID Nº 10), AAVpoG006 (SEQ ID Nº 12), AAVpoG007 (SEQ ID Nº 14), AAVpoG008 (SEQ ID Nº 16), AAVpoG009 (SEQ ID Nº 18), AAVpoG012 (SEQ ID Nº 20), AAVpoG013 (SEQ ID Nº 22), AAVpoG014 (SEQ ID Nº 24), AAVpoG016 (SEQ ID Nº 28), AAVpoG017 (SEQ ID Nº 30), AAVpoG018 (SEQ ID Nº 32), AAVpoG019 (SEQ ID Nº 34), AAVpoG020 (SEQ ID Nº 36), AAVpoG021 (SEQ ID Nº 38), AAVpoG022 (SEQ ID Nº 40), AAVpoG023 (SEQ ID Nº 42), AAVpoG024 (SEQ ID Nº 44) o AAVpoG025 (SEQ ID Nº 46) o una secuencia que comparte al menos 98% o al menos 99% de identidad con una cualquiera de la SEQ ID Nº 2, 4, 6, 8 o una secuencia que comparte al menos 96%, al menos 97%, al menos 98% o al menos 99% de identidad con una cualquiera de la SEQ ID Nº 12, 14, 16, 18, 20, 22, 24, 26, 28, 30 o una secuencia que comparte al menos 90%, al menos 95%, al menos 96%, al menos 97%, al menos 98% o al menos 99% de identidad con una cualquiera de la SEQ ID Nº 32, 34, 36, 38, 40, 42, 44 o 46 y que tiene empaquetado en dicha cápside un genoma vectorial que comprende una secuencia de ácidos nucleicos no AAV. Reivindicación 2: Un rAAV que tiene una cápside caracterizado porque comprende una proteína de la cápside codificada por una secuencia de vp1, vp2 y/o vp3 de AAVpoG015 (SEQ ID Nº 25), AAVpoG001 (SEQ ID Nº 1), AAVpoG002 (SEQ ID Nº 3), AAVpoG003 (SEQ ID Nº 5), AAVpoG004 (SEQ ID Nº 7), AAVpoG005 (SEQ ID Nº 9), AAVpoG006 (SEQ ID Nº 11), AAVpoG007 (SEQ ID Nº 13), AAVpoG008 (SEQ ID Nº 15), AAVpoG009 (SEQ ID Nº 17), AAVpoG012 (SEQ ID Nº 19), AAVpoG013 (SEQ ID Nº 21), AAVpoG014 (SEQ ID Nº 23), AAVpoG016 (SEQ ID Nº 27), AAVpoG017 (SEQ ID Nº 29), AAVpoG018 (SEQ ID Nº 31), AAVpoG019 (SEQ ID Nº 33), AAVpoG020 (SEQ ID Nº 35), AAVpoG021 (SEQ ID Nº 37), AAVpoG022 (SEQ ID Nº 39), AAVpoG023 (SEQ ID Nº 41), AAVpoG024 (SEQ ID Nº 43) o AAVpoG025 (SEQ ID Nº 45), o una secuencia que comparte al menos 70% de identidad con la SEQ ID Nº 1, 3, 5, 7, 9, 11, 13, 15, 17, 19, 21, 23, 25, 27, 29, 31, 33, 35, 37, 39, 41, 43 o 45 y que tiene empaquetado en dicha cápside un genoma vectorial que comprende una secuencia de ácidos nucleicos no AAV. Reivindicación 7: Una célula hospedadora caracterizada porque contiene el rAAV según una cualquiera de las reivindicaciones 1 a 6. Reivindicación 8: Una composición farmacéutica caracterizada porque comprende un rAAV según una cualquiera de las reivindicaciones 1 a 6, y un transportador, amortiguador, adyuvante y/o diluyente fisiológicamente aceptable. Reivindicación 10: Un método para generar un rAAV caracterizado porque comprende una cápside de AAV, un método para generar que comprende cultivar una célula hospedadora que contiene: (a) un ácido nucleico que comprende la secuencia de vp1, vp2 y/o vp3 de AAV de AAVpoG015 (SEQ ID Nº 25), AAVpoG001 (SEQ ID Nº 1), AAVpoG002 (SEQ ID Nº 3), AAVpoG003 (SEQ ID Nº 5), AAVpoG004 (SEQ ID Nº 7), AAVpoG005 (SEQ ID Nº 9), AAVpoG006 (SEQ ID Nº 11), AAVpoG007 (SEQ ID Nº 13), AAVpoG008 (SEQ ID Nº 15), AAVpoG009 (SEQ ID Nº 17), AAVpoG012 (SEQ ID Nº 19), AAVpoG013 (SEQ ID Nº 21), AAVpoG014 (SEQ ID Nº 23), AAVpoG016 (SEQ ID Nº 27), AAVpoG017 (SEQ ID Nº 29), AAVpoG018 (SEQ ID Nº 31), AAVpoG019 (SEQ ID Nº 33), AAVpoG020 (SEQ ID Nº 35), AAVpoG021 (SEQ ID Nº 37), AAVpoG022 (SEQ ID Nº 39), AAVpoG023 (SEQ ID Nº 41), AAVpoG024 (SEQ ID Nº 43) o AAVpoG025 (SEQ ID Nº 45) o una secuencia que comparte al menos 70%, al menos 75%, al menos 80%, al menos 85%, al menos 90%, al menos 95%, al menos 96%, al menos 97%, al menos 98% o al menos 99% de identidad con una secuencia de nucleótidos vp1, vp2 y/o vp3 presentada en la SEQ ID Nº 1, 3, 5, 7, 9, 11, 13, 15, 17, 19, 21, 23, 25, 27, 29, 31, 33, 35, 37, 39, 41, 43 o 45, (b) un gen rep funcional; (c) un minigén que comprende repeticiones terminales invertidas (ITR) de AAV y un transgén; y (d) funciones auxiliares suficiente para permitir la encapsidación del minigén en la cápside del AAV. Reivindicación 12: Una composición que comprende una estirpe del rAAV generada según el método para generar de la reivindicación 10 o 11.Novel porcine-derived adeno-associated virus (AAV) capsids and recombinant AAV vectors comprising them are provided. In addition, methods for delivery of a transgene with the recombinant AAV vectors described herein are provided. Claim 1: A recombinant adeno-associated virus (rAAV) having a capsid characterized in that it comprises a capsid protein having a vp1, vp2 and/or vp3 sequence of AAVpoG015 (SEQ ID No. 26), AAVpoG001 (SEQ ID No. 2) , AAVpoG002 (SEQ ID No. 4), AAVpoG003 (SEQ ID No. 6), AAVpoG004 (SEQ ID No. 8), AAVpoG005 (SEQ ID No. 10), AAVpoG006 (SEQ ID No. 12), AAVpoG007 (SEQ ID No. 14), AAVpoG008 (SEQ ID No. 16), AAVpoG009 (SEQ ID No. 18), AAVpoG012 (SEQ ID No. 20), AAVpoG013 (SEQ ID No. 22), AAVpoG014 (SEQ ID No. 24), AAVpoG016 (SEQ ID No. 28), AAVpoG017 (SEQ ID No. ID No. 30), AAVpoG018 (SEQ ID No. 32), AAVpoG019 (SEQ ID No. 34), AAVpoG020 (SEQ ID No. 36), AAVpoG021 (SEQ ID No. 38), AAVpoG022 (SEQ ID No. 40), AAVpoG023 (SEQ ID No. 42), AAVpoG024 (SEQ ID No. 44) or AAVpoG025 (SEQ ID No. 46) or a sequence that shares at least 98% or at least 99% identity with any one of SEQ ID No. 2, 4, 6, 8 or a sequence that shares at least 96%, at least 97%, at least 98%, or at least 99% identity with any one of SEQ ID NO: 12, 14, 16, 18, 20, 22, 24, 26, 28 , 30 or a sequence that shares at least 90%, at least 95%, at least 96%, at least 97%, at least 98% or at least 99% identity with any one of SEQ ID Nos. 32, 34, 36, 38, 40, 42, 44 or 46 and having packaged in said capsid a vector genome comprising a non-AAV nucleic acid sequence. Claim 2: A rAAV having a capsid characterized in that it comprises a capsid protein encoded by a vp1, vp2 and/or vp3 sequence of AAVpoG015 (SEQ ID No. 25), AAVpoG001 (SEQ ID No. 1), AAVpoG002 (SEQ ID No. No. 3), AAVpoG003 (SEQ ID No. 5), AAVpoG004 (SEQ ID No. 7), AAVpoG005 (SEQ ID No. 9), AAVpoG006 (SEQ ID No. 11), AAVpoG007 (SEQ ID No. 13), AAVpoG008 (SEQ ID No. 15 ), AAVpoG009 (SEQ ID No. 17), AAVpoG012 (SEQ ID No. 19), AAVpoG013 (SEQ ID No. 21), AAVpoG014 (SEQ ID No. 23), AAVpoG016 (SEQ ID No. 27), AAVpoG017 (SEQ ID No. 29), AAVpoG018 (SEQ ID No. 31), AAVpoG019 (SEQ ID No. 33), AAVpoG020 (SEQ ID No. 35), AAVpoG021 (SEQ ID No. 37), AAVpoG022 (SEQ ID No. 39), AAVpoG023 (SEQ ID No. 41), AAVpoG024 ( SEQ ID Nº 43) or AAVpoG025 (SEQ ID Nº 45), or a sequence that shares at least 70% identity with SEQ ID Nº 1, 3, 5, 7, 9, 11, 13, 15, 17, 19, 21, 23, 25, 27, 29, 31, 33, 35, 37, 39, 41, 43 or 45 and having packaged in said capsid a vector genome comprising a non-AAV nucleic acid sequence. Claim 7: A host cell characterized in that it contains the rAAV according to any one of claims 1 to 6. Claim 8: A pharmaceutical composition characterized in that it comprises a rAAV according to any one of claims 1 to 6, and a transporter, buffer, adjuvant and /or physiologically acceptable diluent. Claim 10: A method for generating an rAAV characterized by comprising an AAV capsid, a method for generating comprising culturing a host cell containing: (a) a nucleic acid comprising the AAV vp1, vp2 and/or vp3 sequence of AAVpoG015 (SEQ ID No. 25), AAVpoG001 (SEQ ID No. 1), AAVpoG002 (SEQ ID No. 3), AAVpoG003 (SEQ ID No. 5), AAVpoG004 (SEQ ID No. 7), AAVpoG005 (SEQ ID No. 9), AAVpoG006 (SEQ ID No. 11), AAVpoG007 (SEQ ID No. 13), AAVpoG008 (SEQ ID No. 15), AAVpoG009 (SEQ ID No. 17), AAVpoG012 (SEQ ID No. 19), AAVpoG013 (SEQ ID No. 21), AAVpoG014 (SEQ ID No. 23), AAVpoG016 (SEQ ID No. 27), AAVpoG017 (SEQ ID No. 29), AAVpoG018 (SEQ ID No. 31), AAVpoG019 (SEQ ID No. 33), AAVpoG020 (SEQ ID No. 35), AAVpoG021 (SEQ ID No. 37), AAVpoG022 (SEQ ID No. 39), AAVpoG023 (SEQ ID No. 41), AAVpoG024 (SEQ ID No. 43) or AAVpoG025 (SEQ ID No. 45) or a sequence that shares at least 70%, at least 75%, at at least 80%, at least 85%, at least 90%, at least 95%, at least 96%, at least 97%, at least 98%, or at least 99% identity to a vp1, vp2, and/or nucleotide sequence or vp3 presented in SEQ ID No. 1, 3, 5, 7, 9, 11, 13, 15, 17, 19, 21, 23, 25, 27, 29, 31, 33, 35, 37, 39, 41, 43 or 45, (b) a functional rep gene; (c) a minigene comprising AAV inverted terminal repeats (ITRs) and a transgene; and (d) sufficient helper functions to allow encapsidation of the minigene into the AAV capsid. Claim 12: A composition comprising a rAAV strain generated according to the method of generating claim 10 or 11.

ARP220101102A 2021-04-27 2022-04-27 PIG DERIVED ADENO ASSOCIATED VIRUS CAPSIDS AND THEIR USES AR125467A1 (en)

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WO2022232267A1 (en) 2022-11-03

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