ES3059584T3 - Rhodopsin gene therapy for regenerating visual function or for preventing deterioration in visual function - Google Patents

Rhodopsin gene therapy for regenerating visual function or for preventing deterioration in visual function

Info

Publication number
ES3059584T3
ES3059584T3 ES17846710T ES17846710T ES3059584T3 ES 3059584 T3 ES3059584 T3 ES 3059584T3 ES 17846710 T ES17846710 T ES 17846710T ES 17846710 T ES17846710 T ES 17846710T ES 3059584 T3 ES3059584 T3 ES 3059584T3
Authority
ES
Spain
Prior art keywords
acid sequence
amino acid
visual function
loop
protein
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
ES17846710T
Other languages
English (en)
Spanish (es)
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.)
Keio University
Nagoya Institute of Technology
Original Assignee
Keio University
Nagoya Institute of Technology
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 Keio University, Nagoya Institute of Technology filed Critical Keio University
Application granted granted Critical
Publication of ES3059584T3 publication Critical patent/ES3059584T3/es
Active legal-status Critical Current
Anticipated expiration legal-status Critical

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Classifications

    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/66—Microorganisms or materials therefrom
    • A61K35/76—Viruses; Subviral particles; Bacteriophages
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • 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
    • 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
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/195—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from bacteria
    • 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
    • C07K14/72—Receptors; Cell surface antigens; Cell surface determinants for hormones
    • C07K14/723—G protein coupled receptor, e.g. TSHR-thyrotropin-receptor, LH/hCG receptor, FSH receptor
    • 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
    • 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
    • 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
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K19/00—Hybrid peptides, i.e. peptides covalently bound to nucleic acids, or non-covalently bound protein-protein complexes
    • 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
    • C12N2740/00—Reverse transcribing RNA viruses
    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
    • C12N2740/15011—Lentivirus, not HIV, e.g. FIV, SIV
    • C12N2740/15041—Use of virus, viral particle or viral elements as a vector
    • C12N2740/15043—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
    • C12N2740/00—Reverse transcribing RNA viruses
    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
    • C12N2740/16011—Human Immunodeficiency Virus, HIV
    • C12N2740/16041—Use of virus, viral particle or viral elements as a vector
    • C12N2740/16043—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
    • C12N2750/14143—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
    • C12N2750/14171—Demonstrated in vivo effect

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)
  • Molecular Biology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Wood Science & Technology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Epidemiology (AREA)
  • Microbiology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Plant Pathology (AREA)
  • Virology (AREA)
  • Physics & Mathematics (AREA)
  • Immunology (AREA)
  • Endocrinology (AREA)
  • Toxicology (AREA)
  • Cell Biology (AREA)
  • Ophthalmology & Optometry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Mycology (AREA)
  • Peptides Or Proteins (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
ES17846710T 2016-09-02 2017-09-01 Rhodopsin gene therapy for regenerating visual function or for preventing deterioration in visual function Active ES3059584T3 (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
JP2016172149 2016-09-02
PCT/JP2017/031579 WO2018043707A1 (ja) 2016-09-02 2017-09-01 視覚機能再生剤又は視覚機能低下予防剤

Publications (1)

Publication Number Publication Date
ES3059584T3 true ES3059584T3 (en) 2026-03-23

Family

ID=61301215

Family Applications (1)

Application Number Title Priority Date Filing Date
ES17846710T Active ES3059584T3 (en) 2016-09-02 2017-09-01 Rhodopsin gene therapy for regenerating visual function or for preventing deterioration in visual function

Country Status (6)

Country Link
US (2) US11932679B2 (enExample)
EP (2) EP3508212B1 (enExample)
JP (5) JP6757024B2 (enExample)
CN (5) CN109890404A (enExample)
ES (1) ES3059584T3 (enExample)
WO (1) WO2018043707A1 (enExample)

Families Citing this family (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3508212B1 (en) * 2016-09-02 2025-10-22 Keio University Rhodopsin gene therapy for regenerating visual function or for preventing deterioration in visual function
CA3127535A1 (en) * 2019-01-18 2020-07-23 Restore Vision Inc. Preventing, and suppressing progression of, retinal disease, improving visual cognitive behavioral function, and strengthening visual function
WO2021049634A1 (ja) * 2019-09-13 2021-03-18 株式会社レストアビジョン キメラロドプシンをコードする核酸コンストラクト
US11771741B2 (en) 2019-09-13 2023-10-03 Restore Vision Inc. Nucleic acid construct that encodes chimeric rhodopsin
CN114729009A (zh) 2019-10-31 2022-07-08 伯尔尼大学 用于眼部基因递送的aav载体变体
WO2022059736A1 (ja) * 2020-09-17 2022-03-24 株式会社レストアビジョン 小胞体ストレスまたはオールトランスレチナールに関連する疾患、障害または症状を治療または予防するため、または網膜厚を保護し、または網膜厚の萎縮もしくは萎縮進行を抑制するための組成物。
CN118717946A (zh) * 2023-03-29 2024-10-01 郑州大学第一附属医院 一种嵌合蛋白在治疗视神经变性疾病中的应用
CN118791624B (zh) * 2023-04-11 2026-04-03 苏州星明优健生物技术有限公司 用于治疗视网膜神经退行性疾病的新型gpcr嵌合视蛋白

Family Cites Families (12)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP3521382B2 (ja) 1997-02-27 2004-04-19 日本たばこ産業株式会社 細胞間接着及びシグナル伝達を媒介する細胞表面分子
US8470790B2 (en) * 2006-05-04 2013-06-25 Wayne State University Restoration of visual responses by in vivo delivery of rhodopsin nucleic acids
KR101197129B1 (ko) 2009-09-14 2012-11-07 서강대학교산학협력단 글레오박터 로돕신 및 ａｔｐ 합성효소가 방향성을 갖도록 장착된 ａｔｐ 합성용 조성물
EP3399024A1 (en) 2010-03-17 2018-11-07 The Board of Trustees of The Leland Stanford Junior University Light-sensitive ion-passing molecules
KR101061482B1 (ko) 2010-04-20 2011-09-02 서강대학교산학협력단 카로테노이드가 재구성된 글레오박터 로돕신
EP3486253A1 (en) 2010-11-05 2019-05-22 The Board of Trustees of The Leland Stanford Junior University Light-activated chimeric opsins and methods of using the same
AU2014243925B2 (en) * 2013-03-14 2018-12-06 Wayne State University Method of enhancing delivery of therapeutic compounds to the eye
US10307492B2 (en) * 2014-03-11 2019-06-04 Wayne State University Modified MGLUR6 promoter and methods of use
EP3508212B1 (en) 2016-09-02 2025-10-22 Keio University Rhodopsin gene therapy for regenerating visual function or for preventing deterioration in visual function
CA3127535A1 (en) * 2019-01-18 2020-07-23 Restore Vision Inc. Preventing, and suppressing progression of, retinal disease, improving visual cognitive behavioral function, and strengthening visual function
WO2021049634A1 (ja) 2019-09-13 2021-03-18 株式会社レストアビジョン キメラロドプシンをコードする核酸コンストラクト
US11771741B2 (en) 2019-09-13 2023-10-03 Restore Vision Inc. Nucleic acid construct that encodes chimeric rhodopsin

Also Published As

Publication number Publication date
CN109890404A (zh) 2019-06-14
US20240247049A1 (en) 2024-07-25
EP3508212A1 (en) 2019-07-10
US20190194294A1 (en) 2019-06-27
EP4653537A3 (en) 2026-01-07
JP2020117513A (ja) 2020-08-06
CN117701605A (zh) 2024-03-15
JP2022126804A (ja) 2022-08-30
EP3508212A4 (en) 2020-05-20
CA3035651A1 (en) 2018-03-08
EP4653537A2 (en) 2025-11-26
JP7427208B2 (ja) 2024-02-05
CN117695375A (zh) 2024-03-15
CN117701604A (zh) 2024-03-15
US11932679B2 (en) 2024-03-19
JP2026063282A (ja) 2026-04-10
JP2024026791A (ja) 2024-02-28
JPWO2018043707A1 (ja) 2019-07-25
EP3508212B1 (en) 2025-10-22
CN117701637A (zh) 2024-03-15
WO2018043707A1 (ja) 2018-03-08
JP6757024B2 (ja) 2020-09-16

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