CA3157464A1 - Aav vector variants for ocular gene delivery - Google Patents

Aav vector variants for ocular gene delivery

Info

Publication number
CA3157464A1
CA3157464A1 CA3157464A CA3157464A CA3157464A1 CA 3157464 A1 CA3157464 A1 CA 3157464A1 CA 3157464 A CA3157464 A CA 3157464A CA 3157464 A CA3157464 A CA 3157464A CA 3157464 A1 CA3157464 A1 CA 3157464A1
Authority
CA
Canada
Prior art keywords
seq
capsid
aav
acid sequence
nucleic acid
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
CA3157464A
Other languages
English (en)
French (fr)
Inventor
Sonja Kleinlogel
Anand DAVID
Hildegard Buning
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.)
Universitaet Bern
Medizinische Hochschule Hannover
Original Assignee
Universitaet 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 Universitaet Bern, Medizinische Hochschule Hannover filed Critical Universitaet Bern
Publication of CA3157464A1 publication Critical patent/CA3157464A1/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
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0075—Medicinal 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0048—Eye, e.g. artificial tears
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00—Drugs for disorders of the senses
    • A61P27/02—Ophthalmic agents
    • 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
    • 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
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/33—Fusion polypeptide fusions for targeting to specific cell types, e.g. tissue specific targeting, targeting of a bacterial subspecies
    • 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
    • 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
    • C12N2810/00—Vectors comprising a targeting moiety
    • C12N2810/40—Vectors 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)
CA3157464A 2019-10-31 2020-11-02 Aav vector variants for ocular gene delivery Pending CA3157464A1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
EP19206603.3 2019-10-31
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
CA3157464A1 true CA3157464A1 (en) 2021-05-06

Family

ID=68426130

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3157464A Pending CA3157464A1 (en) 2019-10-31 2020-11-02 Aav vector variants for ocular gene delivery

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
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
IL291240A (en) 2022-05-01
KR20220093163A (ko) 2022-07-05

Similar Documents

Publication Publication Date Title
EP4051694B1 (en) Aav vector variants for ocular gene delivery
RU2742724C1 (ru) Вирионы аденоассоциированного вируса с вариантным капсидом и способы их использования
AU2016219789B2 (en) Methods and compositions for treating genetic eye diseases
KR20170140185A (ko) 다중 벡터 시스템 및 이의 용도
CA3244355A1 (en) RECOMBINANT ADENOVIRUS-ASSOCIATED VIRUS (AAV) COMPRISING MODIFIED AAV CAPSID POLYPEPTIDES
KR20240010489A (ko) 시력 기능 향상을 위한 조성물 및 방법
KR20240023126A (ko) 망막 장애
CN116368228A (zh) 用于治疗眼部疾病的组合物和方法
KR20240005950A (ko) 벡터 시스템
CN112522276B (zh) 一种emc1核苷酸序列及其应用
US20240307559A1 (en) Kcnv2 gene therapy
US20260041791A1 (en) Compositions and methods for treatment of achromotopsia
CN117337331A (zh) 用于增强视觉功能的组合物和方法
WO2024160770A1 (en) An aav9 capsid variant for targeted gene transfer
IL321968A (en) Recombinant adeno-associated virus vector
HK40035863A (en) Adeno-associated virus virions with variant capsid and methods of use thereof
HK40035863B (en) Adeno-associated virus virions with variant capsid and methods of use thereof
CN119894920A (zh) 用于靶向神经系统的新型aav衣壳及其用途
CN112063625A (zh) 编码arl2bp的核酸及其应用
HK1247855B (en) Adeno-associated virus virions with variant capsid and methods of use thereof
HK1194982A (en) Adeno-associated virus virions with variant capsid and methods of use thereof
HK1194982B (en) Adeno-associated virus virions with variant capsid and methods of use thereof

Legal Events

Date Code Title Description
EEER Examination request

Effective date: 20220920

W00 Other event occurred

Free format text: ST27 STATUS EVENT CODE: A-2-2-W10-W00-W100 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: LETTER SENT

Effective date: 20250825

U13 Renewal or maintenance fee not paid

Free format text: ST27 STATUS EVENT CODE: N-2-6-U10-U13-U300 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: DEEMED ABANDONED - FAILURE TO RESPOND TO MAINTENANCE FEE NOTICE

Effective date: 20251027

W00 Other event occurred

Free format text: ST27 STATUS EVENT CODE: N-6-6-W10-W00-W100 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: LETTER SENT

Effective date: 20251229

W00 Other event occurred

Free format text: ST27 STATUS EVENT CODE: N-6-6-W10-W00-W100 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: LETTER SENT

Effective date: 20251231