CA3100066A1 - Viral vectors exhibiting improved gene delivery properties - Google Patents

Viral vectors exhibiting improved gene delivery properties Download PDF

Info

Publication number
CA3100066A1
CA3100066A1 CA3100066A CA3100066A CA3100066A1 CA 3100066 A1 CA3100066 A1 CA 3100066A1 CA 3100066 A CA3100066 A CA 3100066A CA 3100066 A CA3100066 A CA 3100066A CA 3100066 A1 CA3100066 A1 CA 3100066A1
Authority
CA
Canada
Prior art keywords
seq
mutation
capsid polypeptide
viral
cell
Prior art date
Legal status (The legal status 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 status listed.)
Pending
Application number
CA3100066A
Other languages
English (en)
French (fr)
Inventor
George M. Church
Eric KELSIC
Pierce OGDEN
Sam SINAI
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Harvard University
Original Assignee
Harvard University
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 Harvard University filed Critical Harvard University
Publication of CA3100066A1 publication Critical patent/CA3100066A1/en
Pending legal-status Critical Current

Links

Classifications

    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • 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
    • 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
    • A61K38/00—Medicinal preparations containing peptides
    • 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
    • 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
    • C12N2750/14145—Special targeting system for viral vectors

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Virology (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Medicinal Chemistry (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Plant Pathology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Immunology (AREA)
  • Epidemiology (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Peptides Or Proteins (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
CA3100066A 2018-05-15 2019-05-08 Viral vectors exhibiting improved gene delivery properties Pending CA3100066A1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201862671949P 2018-05-15 2018-05-15
US62/671,949 2018-05-15
PCT/US2019/031307 WO2019221992A1 (en) 2018-05-15 2019-05-08 Viral vectors exhibiting improved gene delivery properties

Publications (1)

Publication Number Publication Date
CA3100066A1 true CA3100066A1 (en) 2019-11-21

Family

ID=68540740

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3100066A Pending CA3100066A1 (en) 2018-05-15 2019-05-08 Viral vectors exhibiting improved gene delivery properties

Country Status (6)

Country Link
US (1) US12338267B2 (enExample)
EP (1) EP3813845A4 (enExample)
JP (2) JP2021522840A (enExample)
AU (1) AU2019270900A1 (enExample)
CA (1) CA3100066A1 (enExample)
WO (1) WO2019221992A1 (enExample)

Families Citing this family (11)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3461836A1 (en) * 2017-09-28 2019-04-03 Universität zu Köln Mutated adeno-associated viral capsid proteins for chemical coupling of ligands, nanoparticles or drugs via thioether binding and production method thereof
CN114981442A (zh) * 2019-12-04 2022-08-30 桑格摩生物治疗股份有限公司 用于生产重组aav的新颖组合物及方法
CN115989234A (zh) * 2020-01-29 2023-04-18 建新公司 用于眼部基因疗法的经修饰的腺相关病毒衣壳蛋白及其使用方法
CN111349148A (zh) * 2020-03-13 2020-06-30 辉大(上海)生物科技有限公司 一种腺相关病毒载体及其用途
EP4143206A1 (en) * 2020-05-01 2023-03-08 Trustees of Boston College Controlled modification of adeno-associated virus (aav) for enhanced gene therapy
JP2024525205A (ja) * 2021-06-23 2024-07-10 ダイノ セラピューティクス インコーポレイテッド カプシドバリアント及びそれを使用する方法
WO2023283962A1 (en) * 2021-07-16 2023-01-19 Huigene Therapeutics Co., Ltd. Modified aav capsid for gene therapy and methods thereof
AU2022358779A1 (en) 2021-10-08 2024-04-18 Dyno Therapeutics, Inc. Capsid variants and methods of using the same
US20250269055A1 (en) * 2022-04-15 2025-08-28 Dyno Therapeutics, Inc. Capsid variants and methods of using the same
WO2023201366A2 (en) * 2022-04-15 2023-10-19 Dyno Therapeutics, Inc. Capsid variants and methods of using the same
US12480098B1 (en) * 2024-10-18 2025-11-25 Aavatar Therapeutics Co., Ltd. Adeno-associated virus variant

Family Cites Families (15)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6156303A (en) 1997-06-11 2000-12-05 University Of Washington Adeno-associated virus (AAV) isolates and AAV vectors derived therefrom
JP2003526377A (ja) 2000-03-14 2003-09-09 ニユーロロジクス・インコーポレーテツド キメラキャプシドベクターの製造
US6962815B2 (en) 2001-01-05 2005-11-08 Children's Hopital Inc. AAV2 vectors and methods
EP1453547B1 (en) 2001-12-17 2016-09-21 The Trustees Of The University Of Pennsylvania Adeno-associated virus (aav) serotype 8 sequences, vectors containing same, and uses therefor
EP1486567A1 (en) 2003-06-11 2004-12-15 Deutsches Krebsforschungszentrum Stiftung des öffentlichen Rechts Improved adeno-associated virus (AAV) vector for gene therapy
US9441244B2 (en) 2003-06-30 2016-09-13 The Regents Of The University Of California Mutant adeno-associated virus virions and methods of use thereof
ES2874298T3 (es) 2003-09-30 2021-11-04 Univ Pennsylvania Clados de virus adenoasociados (AAV), secuencias, vectores que contienen el mismo, y usos de los mismos
EP2191001B1 (en) * 2007-04-09 2016-06-08 University of Florida Research Foundation, Inc. Raav vector compositions having tyrosine-modified capsid proteins and methods for use
US9725485B2 (en) 2012-05-15 2017-08-08 University Of Florida Research Foundation, Inc. AAV vectors with high transduction efficiency and uses thereof for gene therapy
WO2009108274A2 (en) * 2008-02-26 2009-09-03 The University Of North Carolina At Chapel Hill Methods and compositions for adeno-associated virus (aav) with hi loop mutations
EP3470523A1 (en) * 2012-05-09 2019-04-17 Oregon Health & Science University Adeno associated virus plasmids and vectors
GB201403684D0 (en) * 2014-03-03 2014-04-16 King S College London Vector
AU2015252797C1 (en) * 2014-05-02 2021-04-22 Genzyme Corporation AAV vectors for retinal and CNS gene therapy
US11124546B2 (en) * 2015-12-14 2021-09-21 The University Of North Carolina At Chapel Hill Modified capsid proteins for enhanced delivery of parvovirus vectors
US20190071681A1 (en) * 2016-02-26 2019-03-07 University Of Florida Research Foundation, Incorporated Aav heparin mutants that display significantly improved eye and brain transduction

Also Published As

Publication number Publication date
JP2024051082A (ja) 2024-04-10
US20210230229A1 (en) 2021-07-29
WO2019221992A1 (en) 2019-11-21
EP3813845A4 (en) 2022-10-26
US12338267B2 (en) 2025-06-24
JP7741575B2 (ja) 2025-09-18
AU2019270900A1 (en) 2020-10-15
EP3813845A1 (en) 2021-05-05
JP2021522840A (ja) 2021-09-02

Similar Documents

Publication Publication Date Title
US12338267B2 (en) Viral vectors exhibiting improved gene delivery properties
Bantel-Schaal et al. Human adeno-associated virus type 5 is only distantly related to other known primate helper-dependent parvoviruses
US6593123B1 (en) Large-scale recombinant adeno-associated virus (rAAV) production and purification
US7419817B2 (en) Scalable purification of AAV2, AAV4 or AAV5 using ion-exchange chromatography
JP6868572B2 (ja) アフィニティー精製工程を含む組換えアデノ随伴ウイルス粒子の精製
Huang et al. Characterization of the adeno-associated virus 1 and 6 sialic acid binding site
Daya et al. Gene therapy using adeno-associated virus vectors
Samulski et al. AAV-mediated gene therapy for research and therapeutic purposes
Kaludov et al. Scalable purification of adeno-associated virus type 2, 4, or 5 using ion-exchange chromatography
AU780231B2 (en) Virus vectors and methods of making and administering the same
US20210079422A1 (en) Aav vector column purification methods
EP3310369B1 (en) Self-limiting viral vectors encoding nucleases
KR20190006951A (ko) 컬럼-기반 완전히 확장 가능한 rAAV 제조 공정
WO2008021140A2 (en) Recombinant aav production in mammalian cells
CN111808828B (zh) 腺相关病毒的分离纯化方法
Kochergin-Nikitsky et al. Tissue and cell-type-specific transduction using rAAV vectors in lung diseases
CA3223292A1 (en) Adeno-associated virus packaging systems
US20230013145A1 (en) Aav capsid-promoter interactions and cell selective gene expression
Romanovsky et al. Heparan sulfate proteoglycan affinity of adeno-associated virus vectors: Implications for retinal gene delivery
CN112218880A (zh) 逃避中和的肝趋向性重组aav6载体
Mary et al. Adeno-associated Virus Vectors in Gene Therapy
HK40044939A (en) Liver tropic recombinant aav6 vectors that evade neutralization
WO2025006957A2 (en) Non-neutralizing aav constructs and methods of generating and using same
Blouin Structure-function studies of the SF3 helicase domain of the adeno-associated virus type 2 non-structural proteins: Interactions with DNA
Zhou et al. 90. Evaluation of Novel Gene Transfer Vectors Derived from Infectious Molecular Clones of Primate AAVs

Legal Events

Date Code Title Description
EEER Examination request

Effective date: 20240506