CN116368228A - 用于治疗眼部疾病的组合物和方法 - Google Patents

用于治疗眼部疾病的组合物和方法 Download PDF

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Publication number
CN116368228A
CN116368228A CN202180064406.4A CN202180064406A CN116368228A CN 116368228 A CN116368228 A CN 116368228A CN 202180064406 A CN202180064406 A CN 202180064406A CN 116368228 A CN116368228 A CN 116368228A
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China
Prior art keywords
sequence
seq
rpe65
promoter
cpg dinucleotides
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CN202180064406.4A
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English (en)
Chinese (zh)
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赵巍
施中东
塞巴斯蒂安·阿吉雷
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Fangtuo Biotechnology Co ltd
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Fangtuo Biotechnology Co ltd
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Priority claimed from CN202010704946.1A external-priority patent/CN113952471A/zh
Application filed by Fangtuo Biotechnology Co ltd filed Critical Fangtuo Biotechnology Co ltd
Publication of CN116368228A publication Critical patent/CN116368228A/zh
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    • 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
    • A61K48/005—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 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/52—Isomerases (5)
    • 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
    • A61K48/005—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 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
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    • 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
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    • C12N9/14—Hydrolases (3)
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    • C12N9/18—Carboxylic ester hydrolases (3.1.1)
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    • C12Y301/00—Hydrolases acting on ester bonds (3.1)
    • C12Y301/01—Carboxylic ester hydrolases (3.1.1)
    • C12Y301/01064—Retinoid isomerohydrolase (3.1.1.64)
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2217/00—Genetically modified animals
    • A01K2217/07—Animals genetically altered by homologous recombination
    • A01K2217/072—Animals genetically altered by homologous recombination maintaining or altering function, i.e. knock in
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2227/00—Animals characterised by species
    • A01K2227/10—Mammal
    • A01K2227/105—Murine
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2267/00—Animals characterised by purpose
    • A01K2267/03—Animal model, e.g. for test or diseases
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    • 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
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    • 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
    • C12N2750/14151—Methods of production or purification of viral material
    • C12N2750/14152—Methods of production or purification of viral material relating to complementing cells and packaging systems for producing virus or viral particles
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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

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  • Genetics & Genomics (AREA)
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  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Wood Science & Technology (AREA)
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  • General Engineering & Computer Science (AREA)
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  • Animal Behavior & Ethology (AREA)
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  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Ophthalmology & Optometry (AREA)
  • Virology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Immunology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Urology & Nephrology (AREA)
  • Vascular Medicine (AREA)
  • Cardiology (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
CN202180064406.4A 2020-07-21 2021-07-20 用于治疗眼部疾病的组合物和方法 Pending CN116368228A (zh)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
CN202010704946.1A CN113952471A (zh) 2020-07-21 2020-07-21 用于治疗眼部疾病的组合物和方法
CN2020107049461 2020-07-21
CN202110378979 2021-04-08
CN2021103789796 2021-04-08
PCT/CN2021/107284 WO2022017363A1 (en) 2020-07-21 2021-07-20 Compositions and methods for the treatment of ocular diseases

Publications (1)

Publication Number Publication Date
CN116368228A true CN116368228A (zh) 2023-06-30

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CN202180064406.4A Pending CN116368228A (zh) 2020-07-21 2021-07-20 用于治疗眼部疾病的组合物和方法

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US (1) US20230321280A1 (enExample)
EP (1) EP4185335A1 (enExample)
JP (1) JP2023535232A (enExample)
CN (1) CN116368228A (enExample)
AU (1) AU2021314400A1 (enExample)
CA (1) CA3186818A1 (enExample)
WO (1) WO2022017363A1 (enExample)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2023187822A1 (en) * 2022-03-28 2023-10-05 Indian Institute Of Technology Kanpur A system for an ocular gene therapy and a process for preparing thereof
CN120225686A (zh) 2022-10-04 2025-06-27 赛仁生物技术公司 用于重组病毒载体生产的修饰的CpG二核苷酸

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN107429252A (zh) * 2015-02-09 2017-12-01 Ucl商业有限公司 优化的rpe65启动子和编码序列
CN111118017A (zh) * 2018-11-01 2020-05-08 上海市第一人民医院 治疗Leber先天性黑蒙症的载体及其应用

Family Cites Families (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
HK1253299A1 (zh) * 2015-06-04 2019-06-14 INSERM (Institut National de la Santé et de la Recherche Médicale) 一种通过基因治疗治疗眼疾病的改进方法
CA3071769A1 (en) * 2017-07-31 2019-02-07 Reflection Biotechnologies Limited Cellular models of and therapies for ocular diseases

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN107429252A (zh) * 2015-02-09 2017-12-01 Ucl商业有限公司 优化的rpe65启动子和编码序列
CN111118017A (zh) * 2018-11-01 2020-05-08 上海市第一人民医院 治疗Leber先天性黑蒙症的载体及其应用

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
RUSLAN GRISHANIN等: "Preclinical Evaluation of ADVM-022, a Novel Gene Therapy Approach to Treating Wet Age-Related Macular Degeneration", MOLECULAR THERAPY, vol. 27, no. 1, 2 January 2019 (2019-01-02), pages 118 - 129 *
杜梦潭等: "腺相关病毒的生产方式及其在基因治疗中的应用", 生物技术进展, vol. 9, no. 4, 15 July 2019 (2019-07-15), pages 326 - 331 *

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Publication number Publication date
JP2023535232A (ja) 2023-08-16
WO2022017363A1 (en) 2022-01-27
AU2021314400A1 (en) 2023-03-23
US20230321280A1 (en) 2023-10-12
CA3186818A1 (en) 2022-01-27
EP4185335A1 (en) 2023-05-31

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