IL291240A - Variants of aav vector for ocular gene transfer - Google Patents

Variants of aav vector for ocular gene transfer

Info

Publication number
IL291240A
IL291240A IL291240A IL29124022A IL291240A IL 291240 A IL291240 A IL 291240A IL 291240 A IL291240 A IL 291240A IL 29124022 A IL29124022 A IL 29124022A IL 291240 A IL291240 A IL 291240A
Authority
IL
Israel
Prior art keywords
gene delivery
aav vector
vector variants
ocular gene
ocular
Prior art date
Application number
IL291240A
Other languages
English (en)
Hebrew (he)
Original Assignee
Universit?T Bern
Medizinische Hochschule Hannover
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 Universit?T Bern, Medizinische Hochschule Hannover filed Critical Universit?T Bern
Publication of IL291240A publication Critical patent/IL291240A/en

Links

Classifications

    • 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0075Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the delivery route, e.g. oral, subcutaneous
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/0012Galenical forms characterised by the site of application
    • A61K9/0019Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/0012Galenical forms characterised by the site of application
    • A61K9/0048Eye, e.g. artificial tears
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00Drugs for disorders of the senses
    • A61P27/02Ophthalmic agents
    • 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2319/00Fusion polypeptide
    • C07K2319/33Fusion polypeptide fusions for targeting to specific cell types, e.g. tissue specific targeting, targeting of a bacterial subspecies
    • 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
    • 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
    • C12N2750/14145Special targeting system for viral vectors
    • 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
    • C12N2810/00Vectors comprising a targeting moiety
    • C12N2810/40Vectors comprising a peptide as targeting moiety, e.g. a synthetic peptide, from undefined source

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • General Engineering & Computer Science (AREA)
  • Virology (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • Biomedical Technology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Physics & Mathematics (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Ophthalmology & Optometry (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Dermatology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Peptides Or Proteins (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
IL291240A 2019-10-31 2022-03-09 Variants of aav vector for ocular gene transfer IL291240A (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
EP19206603 2019-10-31
PCT/EP2020/080704 WO2021084133A1 (en) 2019-10-31 2020-11-02 Aav vector variants for ocular gene delivery

Publications (1)

Publication Number Publication Date
IL291240A true IL291240A (en) 2022-05-01

Family

ID=68426130

Family Applications (1)

Application Number Title Priority Date Filing Date
IL291240A IL291240A (en) 2019-10-31 2022-03-09 Variants of aav vector for ocular gene transfer

Country Status (9)

Country Link
US (1) US12509705B2 (https=)
EP (1) EP4051694B1 (https=)
JP (1) JP7791546B2 (https=)
KR (1) KR20220093163A (https=)
CN (1) CN114729009A (https=)
AU (1) AU2020373326A1 (https=)
CA (1) CA3157464A1 (https=)
IL (1) IL291240A (https=)
WO (1) WO2021084133A1 (https=)

Families Citing this family (11)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20240252679A1 (en) * 2021-05-28 2024-08-01 Shanghai Regenelead Therapies Co., Ltd. Recombinant adeno-associated virus having variant capsid, and application thereof
KR20240095539A (ko) 2021-10-08 2024-06-25 디노 테라퓨틱스, 인코포레이티드 캡시드 변이체 및 이의 사용 방법
AU2023220219A1 (en) 2022-02-17 2024-09-05 Skyline Therapeutics Limited Recombinant adeno-associated virus with modified aav capsid polypeptides
WO2024191778A1 (en) 2023-03-10 2024-09-19 Dyno Therapeutics, Inc. Capsid polypeptides and methods of use thereof
AU2024276934A1 (en) * 2023-05-23 2025-12-18 Innovec Biotherapeutics Adeno-associated virus capsid protein, adeno-associated virus containing same, vector and use thereof
AU2024332181A1 (en) 2023-08-31 2026-02-12 Dyno Therapeutics, Inc. Capsid polypeptides and methods of use thereof
WO2025051292A1 (zh) * 2024-01-16 2025-03-13 广州译码基因科技有限公司 提高aav病毒对眼组织感染能力的衣壳蛋白突变体及其应用
CN118459557B (zh) * 2024-01-16 2024-11-22 沈阳兴齐眼药股份有限公司 提高aav病毒视网膜穿透能力的衣壳蛋白突变体及其应用
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
WO2026033140A1 (en) * 2024-08-09 2026-02-12 Revvity Gene Delivery Gmbh Variant aav capsid polypeptides targeting the eye
WO2026064442A2 (en) 2024-09-18 2026-03-26 Dyno Therapeutics, Inc. Capsid polypeptides and methods of use thereof

Family Cites Families (30)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5284746A (en) 1990-02-08 1994-02-08 Zymogenetics, Inc. Methods of producing hybrid G protein-coupled receptors
ES2308779T3 (es) 1995-07-26 2008-12-01 Astrazeneca Ab Receptores quimericos y metodos para identificar compuestos activos en los receptores de glutamato metabotropico y el uso de tales compuestos en el tratamiento de trastornos y enfermedades neurologicas.
US20040248202A1 (en) 1998-09-03 2004-12-09 Wisconsin Alumni Research Foundation Bacteriorhodopsin/G protein-coupled receptor chimeras
US9284363B2 (en) 2006-07-26 2016-03-15 Case Western Reserve University System and method for controlling G-protein coupled receptor pathways
MY152003A (en) 2008-05-29 2014-08-15 Univ Leland Stanford Junior Cell line, system and method for optical control of secondary messengers
WO2012051599A2 (en) 2010-10-15 2012-04-19 Eos Neuroscience, Inc. Modulation neural pathways
CN105755044A (zh) * 2011-04-22 2016-07-13 加利福尼亚大学董事会 具有变异衣壳的腺相关病毒病毒体及其使用方法
EP3029063B1 (en) 2011-06-24 2019-10-16 Arctos Medical AG Light-sensitive chimeric gpcr protein
CA2839938A1 (en) 2011-06-28 2013-01-03 University Of Rochester Photoactivatable receptors and their uses
US20140087463A1 (en) 2012-09-25 2014-03-27 The Washington University Optical control of cell signaling
EP3024497B1 (en) 2013-07-26 2021-01-13 University of Iowa Research Foundation Methods and compositions for treating brain diseases
BR112016017817A2 (pt) 2014-02-06 2017-10-10 Genzyme Corp composições e métodos para o tratamento e a prevenção de degeneração macular
US20170007720A1 (en) 2014-02-21 2017-01-12 University Of Florida Research Foundation, Inc. Methods and compositions for gene delivery to on bipolar cells
GB201403260D0 (en) 2014-02-25 2014-04-09 Univ Manchester Treatment of retinal degeneration using gene therapy
EP4678754A3 (en) 2014-03-11 2026-04-08 Wayne State University A modified mglur6 promoter and methods of use
AU2016219789B2 (en) 2015-02-20 2021-03-25 Fondazione Telethon Methods and compositions for treating genetic eye diseases
US11021519B2 (en) * 2015-03-02 2021-06-01 Adverum Biotechnologies, Inc. Compositions and methods for intravitreal delivery of polynucleotides to retinal cones
US10533187B2 (en) 2015-03-18 2020-01-14 University Of Florida Research Foundation, Incorporated Methods and compositions for restoration of cone function in BCM
CN105154470A (zh) 2015-07-22 2015-12-16 华东师范大学 一种高效筛查g蛋白-偶联受体表达的融合基因构建方法
US11364308B2 (en) * 2016-05-13 2022-06-21 4D Molecular Therapeutics Inc. Adeno-associated virus variant capsids and methods of use thereof
AU2017302013B2 (en) 2016-07-29 2022-05-26 The Regents Of The University Of California Adeno-associated virus virions with variant capsid and methods of use thereof
EP4653537A3 (en) 2016-09-02 2026-01-07 Keio University Rhodopsin gene therapy for regenerating visual function or for preventing deterioration in visual function
US10376265B2 (en) 2017-01-30 2019-08-13 Ethicon Llc Non-magnetic fragmentable tissue compression devices
CA3053154A1 (en) 2017-06-30 2019-01-03 The Regents Of The University Of California Adeno-associated virus virions with variant capsids and methods of use thereof
US12043848B2 (en) * 2017-10-16 2024-07-23 Vigeneron Gmbh AAV vectors
JP7029160B2 (ja) 2017-11-24 2022-03-03 公立大学法人大阪 新規な光受容タンパク質
CN111770999A (zh) * 2017-11-27 2020-10-13 4D分子治疗有限公司 腺相关病毒变体衣壳和用于抑制血管生成的应用
WO2020148913A1 (ja) 2019-01-18 2020-07-23 俊英 栗原 網膜疾患の予防および進行抑制、視覚認知行動機能の改善、および視覚機能強化
CN115052988A (zh) 2019-11-18 2022-09-13 伯尔尼大学 用于眼部基因递送的给光型双极细胞特异性启动子
US20240174743A1 (en) 2019-11-29 2024-05-30 Universität Bern Chimeric opsin gpcr proteins

Also Published As

Publication number Publication date
EP4051694C0 (en) 2025-07-02
CN114729009A (zh) 2022-07-08
JP2023504773A (ja) 2023-02-07
CA3157464A1 (en) 2021-05-06
WO2021084133A1 (en) 2021-05-06
JP7791546B2 (ja) 2025-12-24
EP4051694B1 (en) 2025-07-02
US12509705B2 (en) 2025-12-30
EP4051694A1 (en) 2022-09-07
AU2020373326A1 (en) 2022-06-02
US20240287542A1 (en) 2024-08-29
KR20220093163A (ko) 2022-07-05

Similar Documents

Publication Publication Date Title
IL291240A (en) Variants of aav vector for ocular gene transfer
SG11202110789VA (en) Gene therapy for eye pathologies
IL287438A (en) Variant aav capsids for intravitreal administration
IL277667A (en) Virus vectors for ocular tissues
IL284742A (en) AAV-mediated gene therapy restoring the autoperlin gene
IL277270A (en) Augmenting tissue-specific gene delivery through capsid modification
GB201907302D0 (en) Delivery system
EP4013770A4 (en) AAV CAPSID VARIANTS FOR GENE THERAPY
PL3693325T3 (pl) System dostawczy
GB201905301D0 (en) Gene therapy
IL287956A (en) Parthenogenesis garden
IL279225A (en) Cardiac AAV gene therapy for myocardial disease
EP3634986A4 (en) GENE THERAPY FOR EYE DISORDERS
IL268946A (en) Gene therapy for ocular disorders
EP3765030A4 (en) CHIMERIC PEPTIDES FOR ANTISENSE ADMINISTRATION
IL292881A (en) Bipolar cell-specific promoters for ocular gene delivery
IL290944A (en) A system for regulating gene expression
EP4073087A4 (en) Recombinant adeno-associated viral vector for gene delivery
HK40079698A (en) Aav vector variants for ocular gene delivery
GB201916884D0 (en) Nanoparticle-like delivery system
EP3717024B8 (en) Gene therapy for ocular improvement
EP4043038A4 (en) ARNSI EXPRESSION VECTOR
HK40078512A (en) On-bipolar cell-specific promoters for ocular gene delivery
HK40065561A (en) Variant aav capsids for intravitreal delivery
HK40068650A (en) Gene therapy for eye pathologies