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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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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Publication of CA3177869A1 publication Critical patent/CA3177869A1/en
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    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14122New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143Use 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)
  • Bioinformatics & Cheminformatics (AREA)
  • Molecular Biology (AREA)
  • Wood Science & Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Zoology (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Virology (AREA)
  • Medicinal Chemistry (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (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)
  • Peptides Or Proteins (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Enzymes And Modification Thereof (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 (enExample)
EP (1) EP4125977A4 (enExample)
JP (1) JP7756439B2 (enExample)
KR (1) KR20230029616A (enExample)
CN (1) CN116096377B (enExample)
AR (1) AR122013A1 (enExample)
AU (1) AU2021268946A1 (enExample)
BR (1) BR112022022644A2 (enExample)
CA (1) CA3177869A1 (enExample)
IL (1) IL297577A (enExample)
MX (1) MX2022013963A (enExample)
TW (1) TWI910162B (enExample)
WO (1) WO2021226267A2 (enExample)

Families Citing this family (11)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
MX2022013963A (es) * 2020-05-05 2023-02-22 Univ Duke Composiciones de virus adenoasociados compatibles entre especies y metodos de uso de las mismas.
EP4413147A4 (en) 2021-10-08 2025-07-30 Dyno Therapeutics Inc CAPSID VARIANTS AND METHODS OF USE THEREOF
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
TW202434734A (zh) * 2023-02-08 2024-09-01 美商銀杏生物工作室有限公司 天使症候群的基因療法
WO2024191778A1 (en) 2023-03-10 2024-09-19 Dyno Therapeutics, Inc. Capsid polypeptides and methods of use thereof
WO2024206831A1 (en) * 2023-03-31 2024-10-03 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)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5478745A (en) 1992-12-04 1995-12-26 University Of Pittsburgh Recombinant viral vector system
ATE454445T1 (de) 1998-11-10 2010-01-15 Univ North Carolina Virusvektoren und verfahren für ihre herstellung und verabreichung.
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
HUE054805T2 (hu) 2003-09-30 2021-09-28 Univ Pennsylvania Adeno-asszociált vírus (AAV) kládok, szekvenciák, az azokat tartalmazó vektorok és alkalmazásaik
US9198984B2 (en) 2006-04-28 2015-12-01 The Trustees Of The University Of Pennsylvania Scalable production method for AAV
WO2010138263A2 (en) 2009-05-28 2010-12-02 University Of Massachusetts Novel aav 's and uses thereof
US9409953B2 (en) * 2011-02-10 2016-08-09 The University Of North Carolina At Chapel Hill Viral vectors with modified transduction profiles and methods of making and using the same
EP3470523A1 (en) 2012-05-09 2019-04-17 Oregon Health & Science University Adeno associated virus plasmids and vectors
CN105247044B (zh) * 2013-05-31 2021-05-07 加利福尼亚大学董事会 腺相关病毒变体及其使用方法
US10746742B2 (en) * 2014-04-25 2020-08-18 Oregon Health & Science University Methods of viral neutralizing antibody epitope mapping
US10577627B2 (en) 2014-06-09 2020-03-03 Voyager Therapeutics, Inc. Chimeric capsids
IL314171A (en) * 2015-09-28 2024-09-01 Univ Florida Methods and compositions for antibody-evading virus vectors
CA3052829A1 (en) 2017-02-15 2018-08-23 The University Of North Carolina At Chapel Hill Methods and compositions for gene transfer across the vasculature
CN110650733A (zh) 2017-02-28 2020-01-03 阿德夫拉姆生物技术股份有限公司 经过修饰的aav衣壳和其用途
US11332727B2 (en) 2017-03-14 2022-05-17 The Regents Of The University Of California Method for reducing an immune response by administering an immune evading adeno-associated AAV8 or AAVDJ viral vector
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
IL316259A (en) 2017-03-15 2024-12-01 Univ North Carolina Chapel Hill Polyploid gland associated with virus vectors and methods for preparing and using the same.
JOP20190269A1 (ar) 2017-06-15 2019-11-20 Voyager Therapeutics Inc بولي نوكليوتيدات aadc لعلاج مرض باركنسون
WO2019195444A1 (en) * 2018-04-03 2019-10-10 Stridebio, Inc. Antibody-evading virus vectors
WO2019195423A1 (en) 2018-04-03 2019-10-10 Stridebio, Inc. Virus vectors for targeting ophthalmic tissues
EP3793615A2 (en) 2018-05-16 2021-03-24 Voyager Therapeutics, Inc. Directed evolution of aav to improve tropism for cns
BR112021006060A2 (pt) * 2018-10-01 2021-07-20 The Trustees Of The University Of Pennsylvania composições úteis para tratamento de gangliosidose gm1
AR118465A1 (es) 2019-03-21 2021-10-06 Stridebio Inc Vectores de virus adenoasociados recombinantes
MX2022013963A (es) 2020-05-05 2023-02-22 Univ Duke Composiciones de virus adenoasociados compatibles entre especies y metodos de uso de las mismas.

Also Published As

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

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