CA3177869A1 - Cross-species compatible adeno-associated virus compositions and methods of use thereof - Google Patents

Cross-species compatible adeno-associated virus compositions and methods of use thereof Download PDF

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Publication number
CA3177869A1
CA3177869A1 CA3177869A CA3177869A CA3177869A1 CA 3177869 A1 CA3177869 A1 CA 3177869A1 CA 3177869 A CA3177869 A CA 3177869A CA 3177869 A CA3177869 A CA 3177869A CA 3177869 A1 CA3177869 A1 CA 3177869A1
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Canada
Prior art keywords
aav
seq
capsid protein
sequence
vector
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Pending
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CA3177869A
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English (en)
French (fr)
Inventor
Aravind Asokan
Trevor GONZALEZ
Lawrence Patrick HAVLIK
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Duke University
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Individual
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Publication of CA3177869A1 publication Critical patent/CA3177869A1/en
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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
    • 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
    • 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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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Virology (AREA)
  • Medicinal Chemistry (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Peptides Or Proteins (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Epidemiology (AREA)
  • Urology & Nephrology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
CA3177869A 2020-05-05 2021-05-05 Cross-species compatible adeno-associated virus compositions and methods of use thereof Pending CA3177869A1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202063020062P 2020-05-05 2020-05-05
US63/020,062 2020-05-05
PCT/US2021/030937 WO2021226267A2 (en) 2020-05-05 2021-05-05 Cross-species compatible adeno-associated virus compositions and methods of use thereof

Publications (1)

Publication Number Publication Date
CA3177869A1 true CA3177869A1 (en) 2021-11-11

Family

ID=78468375

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3177869A Pending CA3177869A1 (en) 2020-05-05 2021-05-05 Cross-species compatible adeno-associated virus compositions and methods of use thereof

Country Status (13)

Country Link
US (3) US12577586B2 (https=)
EP (1) EP4125977A4 (https=)
JP (1) JP7756439B2 (https=)
KR (1) KR20230029616A (https=)
CN (1) CN116096377B (https=)
AR (1) AR122013A1 (https=)
AU (1) AU2021268946A1 (https=)
BR (1) BR112022022644A2 (https=)
CA (1) CA3177869A1 (https=)
IL (1) IL297577A (https=)
MX (1) MX2022013963A (https=)
TW (1) TWI910162B (https=)
WO (1) WO2021226267A2 (https=)

Families Citing this family (11)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA3177869A1 (en) * 2020-05-05 2021-11-11 Duke University Cross-species compatible adeno-associated virus compositions and methods of use thereof
KR20240095539A (ko) 2021-10-08 2024-06-25 디노 테라퓨틱스, 인코포레이티드 캡시드 변이체 및 이의 사용 방법
US20260055144A1 (en) * 2022-08-24 2026-02-26 Regenxbio Inc. Recombinant adeno-associated viruses and uses thereof
WO2024168089A2 (en) * 2023-02-07 2024-08-15 Duke University Compositions comprising aavs and methods for treating gastrointestinal diseases
EP4661916A1 (en) * 2023-02-08 2025-12-17 Ginkgo Bioworks, Inc. Gene therapy for angelman syndrome
WO2024191778A1 (en) 2023-03-10 2024-09-19 Dyno Therapeutics, Inc. Capsid polypeptides and methods of use thereof
AU2024242227A1 (en) * 2023-03-31 2025-11-13 Duke University Compositions comprising brain-tropic aavs and methods of use thereof
AU2024332181A1 (en) 2023-08-31 2026-02-12 Dyno Therapeutics, Inc. Capsid polypeptides and methods of use thereof
TW202545971A (zh) 2024-02-08 2025-12-01 美商戴諾治療公司 殼體多肽及其使用方法
WO2025250454A1 (en) * 2024-05-28 2025-12-04 University Of Rochester Adeno-associated viruses evolved to specifically target human glial progenitor cells in vivo
WO2026064442A2 (en) 2024-09-18 2026-03-26 Dyno Therapeutics, Inc. Capsid polypeptides and methods of use thereof

Family Cites Families (25)

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US5478745A (en) 1992-12-04 1995-12-26 University Of Pittsburgh Recombinant viral vector system
AU780231B2 (en) 1998-11-10 2005-03-10 University Of North Carolina At Chapel Hill, The Virus vectors and methods of making and administering the same
US7314912B1 (en) 1999-06-21 2008-01-01 Medigene Aktiengesellschaft AAv scleroprotein, production and use thereof
ATE318923T1 (de) 2000-06-01 2006-03-15 Univ North Carolina Doppelsträngige parvovirus-vektoren
EP2345731B1 (en) * 2003-09-30 2015-10-21 The Trustees of the University of Pennsylvania Adeno-associated virus (AAV) clades, sequences, vectors containing same, and uses thereof
EP2018421B1 (en) * 2006-04-28 2012-12-19 The Trustees of the University of Pennsylvania Scalable production method for aav
US8734809B2 (en) 2009-05-28 2014-05-27 University Of Massachusetts AAV's and uses thereof
WO2012109570A1 (en) 2011-02-10 2012-08-16 The University Of North Carolina At Chapel Hill Viral vectors with modified transduction profiles and methods of making and using the same
WO2013170078A1 (en) 2012-05-09 2013-11-14 Oregon Health & Science University Adeno associated virus plasmids and vectors
EP3003391B1 (en) 2013-05-31 2021-09-22 The Regents of The University of California Adeno-associated virus variants and methods of use thereof
WO2015164757A1 (en) 2014-04-25 2015-10-29 Oregon Health & Science University Methods of viral neutralizing antibody epitope mapping
EP3151866B1 (en) * 2014-06-09 2023-03-08 Voyager Therapeutics, Inc. Chimeric capsids
ES2865487T3 (es) 2015-09-28 2021-10-15 Univ North Carolina Chapel Hill Métodos y composiciones para vectores virales que evaden los anticuerpos
US11905312B2 (en) 2017-02-15 2024-02-20 The University Of North Carolina At Chapel Hill Methods and compositions for gene transfer across the vasculature
JP2020510424A (ja) * 2017-02-28 2020-04-09 アドヴェラム バイオテクノロジーズ， インコーポレイテッド 改変ａａｖカプシドおよびその使用
WO2018170015A1 (en) 2017-03-14 2018-09-20 The Regents Of The University Of California Engineering crispr cas9 immune stealth
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
NZ756587A (en) * 2017-03-15 2025-12-19 Univ North Carolina Chapel Hill Polyploid adeno-associated virus vectors and methods of making and using the same
JOP20190269A1 (ar) 2017-06-15 2019-11-20 Voyager Therapeutics Inc بولي نوكليوتيدات aadc لعلاج مرض باركنسون
MX2020010465A (es) 2018-04-03 2021-01-08 Vectores de virus para direccionamiento a tejidos oftalmicos.
EP3774852A1 (en) 2018-04-03 2021-02-17 Stridebio, Inc. Antibody-evading virus vectors
US20210214749A1 (en) 2018-05-16 2021-07-15 Voyager Therapeutics, Inc. Directed evolution
CA3114175A1 (en) * 2018-10-01 2020-04-09 The Trustees Of The University Of Pennsylvania Beta-galactosidase based aav for treating gm1 gangliosidosis
AR118465A1 (es) 2019-03-21 2021-10-06 Stridebio Inc Vectores de virus adenoasociados recombinantes
CA3177869A1 (en) 2020-05-05 2021-11-11 Duke University Cross-species compatible adeno-associated virus compositions and methods of use thereof

Also Published As

Publication number Publication date
AR122013A1 (es) 2022-08-03
JP7756439B2 (ja) 2025-10-20
US20240067988A1 (en) 2024-02-29
US12037598B2 (en) 2024-07-16
AU2021268946A1 (en) 2022-12-01
TW202208631A (zh) 2022-03-01
EP4125977A4 (en) 2024-09-25
MX2022013963A (es) 2023-02-22
JP2023524291A (ja) 2023-06-09
TWI910162B (zh) 2026-01-01
US12577586B2 (en) 2026-03-17
IL297577A (en) 2022-12-01
EP4125977A2 (en) 2023-02-08
US20230151389A1 (en) 2023-05-18
BR112022022644A2 (pt) 2023-01-17
US20240076695A1 (en) 2024-03-07
CN116096377A (zh) 2023-05-09
KR20230029616A (ko) 2023-03-03
CN116096377B (zh) 2026-01-02
WO2021226267A2 (en) 2021-11-11
US12146151B2 (en) 2024-11-19
WO2021226267A3 (en) 2021-12-16

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