AU2019325332B2 - Compositions and methods for modulating transduction efficiency of adeno-associated viruses - Google Patents

Compositions and methods for modulating transduction efficiency of adeno-associated viruses

Info

Publication number
AU2019325332B2
AU2019325332B2 AU2019325332A AU2019325332A AU2019325332B2 AU 2019325332 B2 AU2019325332 B2 AU 2019325332B2 AU 2019325332 A AU2019325332 A AU 2019325332A AU 2019325332 A AU2019325332 A AU 2019325332A AU 2019325332 B2 AU2019325332 B2 AU 2019325332B2
Authority
AU
Australia
Prior art keywords
gpr108
cell
aav
cells
transduction
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.)
Active
Application number
AU2019325332A
Other languages
English (en)
Other versions
AU2019325332A1 (en
Inventor
Amanda DUDEK
Nerea Zabaleta LASARTE
Luk H. VANDENBERGHE
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.)
The Schepens Eye Research Institute Inc
Massachusetts Eye and Ear Infirmary
Original Assignee
Massachusetts Eye and Ear Infirmary
The Schepens Eye Research Institute Inc
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 Massachusetts Eye and Ear Infirmary, The Schepens Eye Research Institute Inc filed Critical Massachusetts Eye and Ear Infirmary
Publication of AU2019325332A1 publication Critical patent/AU2019325332A1/en
Application granted granted Critical
Publication of AU2019325332B2 publication Critical patent/AU2019325332B2/en
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

Classifications

    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/62—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being a protein, peptide or polyamino acid
    • A61K47/64—Drug-peptide, drug-protein or drug-polyamino acid conjugates, i.e. the modifying agent being a peptide, protein or polyamino acid which is covalently bonded or complexed to a therapeutically active agent
    • 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
    • C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • 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
    • C12N2750/14121—Viruses as such, e.g. new isolates, mutants or their genomic sequences
    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Biochemistry (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Virology (AREA)
  • Medicinal Chemistry (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Immunology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Toxicology (AREA)
  • Cell Biology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicinal Preparation (AREA)
  • Peptides Or Proteins (AREA)
AU2019325332A 2018-08-21 2019-08-21 Compositions and methods for modulating transduction efficiency of adeno-associated viruses Active AU2019325332B2 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201862720859P 2018-08-21 2018-08-21
US62/720,859 2018-08-21
PCT/US2019/047546 WO2020041498A1 (en) 2018-08-21 2019-08-21 Compositions and methods for modulating transduction efficiency of adeno-associated viruses

Publications (2)

Publication Number Publication Date
AU2019325332A1 AU2019325332A1 (en) 2021-03-18
AU2019325332B2 true AU2019325332B2 (en) 2025-11-13

Family

ID=69591093

Family Applications (1)

Application Number Title Priority Date Filing Date
AU2019325332A Active AU2019325332B2 (en) 2018-08-21 2019-08-21 Compositions and methods for modulating transduction efficiency of adeno-associated viruses

Country Status (5)

Country Link
US (1) US12195745B2 (enExample)
EP (1) EP3841109A4 (enExample)
JP (1) JP7451497B2 (enExample)
AU (1) AU2019325332B2 (enExample)
WO (1) WO2020041498A1 (enExample)

Families Citing this family (16)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
IL278598B2 (en) 2018-05-11 2025-11-01 Massachusetts Eye & Ear Infirmary Altering tissue tropism of adeno-associated viruses
CA3153972A1 (en) 2019-09-09 2021-03-18 Massachusetts Eye And Ear Infirmary Methods and compositions for modulating the interaction between adeno-associated virus (aav) and the aav receptor (aavr) for altered bio-distribution of aav
WO2021205028A1 (en) 2020-04-09 2021-10-14 University Of Sheffield Gene therapy treatment
US20230295658A1 (en) * 2020-11-10 2023-09-21 The Children's Hospital Of Philadelphia Compositions and methods for in vivo gene transfer
TW202325840A (zh) * 2021-08-30 2023-07-01 美商艾維克羅姆公司 與人肝臟嵌合性組合的腺相關病毒受體（ａａｖｒ）剔除小鼠及彼之使用方法和產製
GB202215198D0 (en) 2022-10-14 2022-11-30 Univ Sheffield Gene therapy treatment
EP4762358A1 (en) 2023-11-30 2026-06-24 Cytomos Limited Cell selection
WO2025188755A1 (en) 2024-03-04 2025-09-12 Kate Therapeutics, Inc. Adeno-associated virus compositions for the treatment of duchenne muscular dystrophy
CN118207258A (zh) * 2024-03-12 2024-06-18 浙江恒驭生物科技有限公司 一种检测腺相关病毒转导滴度的敏感细胞系、构建方法及其应用
WO2025226842A1 (en) 2024-04-24 2025-10-30 Kate Therapeutics, Inc. Expression control by drg-expressed mirnas
WO2025226841A1 (en) 2024-04-24 2025-10-30 Kate Therapeutics, Inc. Gene therapy approach for treating disorders associated with tnnt2
WO2026011008A1 (en) 2024-07-02 2026-01-08 Kate Therapeutics, Inc. Expression control by skeletal muscle-expressed mirnas
WO2026011009A1 (en) 2024-07-02 2026-01-08 Kate Therapeutics, Inc. Compositions and methods for muscle disorders
WO2026030242A1 (en) 2024-07-29 2026-02-05 Kate Therapeutics, Inc. Adeno-associated virus compositions for the treatment of facioscapulohumeral muscular dystrophy
WO2026030244A1 (en) 2024-07-30 2026-02-05 Kate Therapeutics, Inc. Adeno-associated virus compositions for the treatment of limb girdle muscular dystrophy 2a
WO2026050402A1 (en) 2024-08-29 2026-03-05 Kate Therapeutics, Inc. Liver de-targeted muscle tropic capsids

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2005072364A2 (en) * 2004-01-27 2005-08-11 University Of Florida A modified baculovirus expression system for production of pseudotyped raav vector

Family Cites Families (15)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6855314B1 (en) * 2000-03-22 2005-02-15 The United States Of America As Represented By The Department Of Health And Human Services AAV5 vector for transducing brain cells and lung cells
EP1572893B1 (en) 2001-11-09 2009-01-07 THE GOVERNMENT OF THE UNITED STATES OF AMERICA as represented by THE SECRETARY of the DEPARTMENT OF HEALTH AND HUMAN SERVICES Production of adeno-associated virus in insect cells
SI1496944T1 (sl) * 2002-05-01 2009-02-28 Univ Florida Izboljšani sistemi ekspresije RAAV za genetsko modifikacijo specifičnih kapsidnih proteinov
DE23193606T1 (de) * 2005-04-07 2024-02-15 The Trustees Of The University Of Pennsylvania Verfahren zur erhöhung der funktion eines aav-vektors
WO2014124282A1 (en) * 2013-02-08 2014-08-14 The Trustees Of The University Of Pennsylvania Enhanced aav-mediated gene transfer for retinal therapies
WO2016065001A1 (en) * 2014-10-21 2016-04-28 University Of Massachusetts Recombinant aav variants and uses thereof
US10081659B2 (en) * 2015-04-06 2018-09-25 The United States Of America, As Represented By The Secretary, Dept. Of Health And Human Services Adeno-associated vectors for enhanced transduction and reduced immunogenicity
US10633662B2 (en) 2015-11-10 2020-04-28 The Board Of Trustees Of The Leland Stanford Junior University Methods and compositions for modulating AAV infection
EP3448874A4 (en) * 2016-04-29 2020-04-22 Voyager Therapeutics, Inc. COMPOSITIONS FOR TREATING A DISEASE
WO2017197355A2 (en) * 2016-05-13 2017-11-16 4D Molecular Therapeutics Inc. Adeno-associated virus variant capsids and methods of use thereof
JP6994018B2 (ja) 2016-07-26 2022-01-14 バイオマリン ファーマシューティカル インコーポレイテッド 新規アデノ随伴ウイルスキャプシドタンパク質
HUE053747T2 (hu) * 2016-08-15 2021-07-28 Genzyme Corp Eljárások AAV kimutatására
US20230048025A1 (en) * 2016-12-22 2023-02-16 Oregon Health & Science University Adeno associated viral vectors
US20210292373A1 (en) 2018-07-10 2021-09-23 University Of Florida Research Foundation, Incorporated Aav vp1u chimeras
TWI862583B (zh) * 2019-04-26 2024-11-21 美商愛德維仁生物科技公司 用於玻璃體內遞送之變異體aav蛋白殼

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2005072364A2 (en) * 2004-01-27 2005-08-11 University Of Florida A modified baculovirus expression system for production of pseudotyped raav vector

Also Published As

Publication number Publication date
WO2020041498A1 (en) 2020-02-27
US20210317478A1 (en) 2021-10-14
EP3841109A4 (en) 2022-09-21
US12195745B2 (en) 2025-01-14
EP3841109A1 (en) 2021-06-30
JP2021533795A (ja) 2021-12-09
JP7451497B2 (ja) 2024-03-18
AU2019325332A1 (en) 2021-03-18

Similar Documents

Publication Publication Date Title
US12195745B2 (en) Compositions and methods for modulating transduction efficiency of adeno-associated viruses
Büning et al. Capsid modifications for targeting and improving the efficacy of AAV vectors
KR102743219B1 (ko) 아데노-관련 바이러스 변이체 캡시드 및 그 용도
JP7081767B2 (ja) アデノ随伴ウイルス（ａａｖ）キャプシドタンパク質の変異体
US20240384294A1 (en) Bocaparvovirus small noncoding rna and uses thereof
JP7558575B2 (ja) レトロウイルスインテグラーゼ－Ｃａｓ９融合タンパク質を使用した指向性非相同ＤＮＡ挿入によるゲノム編集
KR20220066225A (ko) 선택적 유전자 조절을 위한 조성물 및 방법
JP2020536505A (ja) 誘導性のＡＡＶ Ｒｅｐ遺伝子
CA3153972A1 (en) Methods and compositions for modulating the interaction between adeno-associated virus (aav) and the aav receptor (aavr) for altered bio-distribution of aav
US20250332288A1 (en) Cardioprotective heart disease therapies
JP2023546113A (ja) 同時遺伝子活性化のための核酸構築物
Gonçalves et al. Transduction of myogenic cells by retargeted dual high-capacity hybrid viral vectors: robust dystrophin synthesis in duchenne muscular dystrophy muscle cells
KR20250166360A (ko) 페이로드 전달을 위한 rgd-함유 펩티드
Gonçalves A concise peer into the background, initial thoughts and practices of human gene therapy
WO2020187268A1 (zh) 一种增强基因编辑的融合蛋白及其应用
AU2016338565A1 (en) Nucleic acid molecules containing spacers and methods of use thereof
Hao et al. Identification of host essential factors for recombinant AAV transduction of the polarized human airway epithelium
CN117377500A (zh) 具有改善的组织向性的腺相关病毒载体衣壳
Go et al. Enhancing transduction efficiency of adeno-associated virus 9 by cell line engineering: implication for gene therapy potency assay
WO2025024460A2 (en) Methods and compositions for modulating aav infection
US12410436B2 (en) Adeno-associated virus (AAV) producer cell lines
García Gallardo Developing cardiomyocyte specific vectors
WO2024215655A1 (en) Cardioprotective bag3 therapies
JP2024542982A (ja) タウオパチーの治療様式としてのｌｅｍｄ２、ｌｅｍｄ３、又はｃｈｍｐ７の過剰発現
WO2025207948A1 (en) Aav capsids for targeting human transferrin receptor

Legal Events

Date Code Title Description
FGA Letters patent sealed or granted (standard patent)