CN113952472A - 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
CN113952472A
CN113952472A CN202010705069.XA CN202010705069A CN113952472A CN 113952472 A CN113952472 A CN 113952472A CN 202010705069 A CN202010705069 A CN 202010705069A CN 113952472 A CN113952472 A CN 113952472A
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CN
China
Prior art keywords
sequence
promoter
composition
aav
rpgr
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
CN202010705069.XA
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English (en)
Chinese (zh)
Inventor
赵巍
施中东
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Insperry Co ltd
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Insperry Co ltd
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.)
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Publication date
Application filed by Insperry Co ltd filed Critical Insperry Co ltd
Priority to CN202010705069.XA priority Critical patent/CN113952472A/zh
Priority to CN202180064480.6A priority patent/CN116615552A/zh
Priority to JP2023512665A priority patent/JP2023542279A/ja
Priority to AU2021312379A priority patent/AU2021312379A1/en
Priority to PCT/IB2021/000509 priority patent/WO2022018518A1/en
Priority to CA3186826A priority patent/CA3186826A1/en
Priority to EP21845856.0A priority patent/EP4185319A4/en
Publication of CN113952472A publication Critical patent/CN113952472A/zh
Priority to US18/157,589 priority patent/US20230321281A1/en
Pending legal-status Critical Current

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    • 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
    • A61K48/005Medicinal 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
    • A61K48/0058Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/162Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from virus
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/1703Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • A61K38/1709Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • 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
    • A61K48/005Medicinal 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
    • 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00Drugs for disorders of the cardiovascular system
    • A61P9/10Drugs for disorders of the cardiovascular system for treating ischaemic or atherosclerotic diseases, e.g. antianginal drugs, coronary vasodilators, drugs for myocardial infarction, retinopathy, cerebrovascula insufficiency, renal arteriosclerosis
    • 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
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C07K14/4701Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4702Regulators; Modulating activity
    • 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
    • A01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01KANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2217/00Genetically modified animals
    • A01K2217/07Animals genetically altered by homologous recombination
    • A01K2217/075Animals genetically altered by homologous recombination inducing loss of function, i.e. knock out
    • AHUMAN NECESSITIES
    • A01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01KANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2227/00Animals characterised by species
    • A01K2227/10Mammal
    • A01K2227/105Murine
    • AHUMAN NECESSITIES
    • A01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01KANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2267/00Animals characterised by purpose
    • A01K2267/03Animal model, e.g. for test or diseases
    • A01K2267/0306Animal model for genetic diseases
    • 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
    • C12N2830/00Vector systems having a special element relevant for transcription
    • C12N2830/008Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination
    • 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
    • C12N2830/00Vector systems having a special element relevant for transcription
    • C12N2830/42Vector systems having a special element relevant for transcription being an intron or intervening sequence for splicing and/or stability of RNA
    • 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
    • C12N2830/00Vector systems having a special element relevant for transcription
    • C12N2830/50Vector systems having a special element relevant for transcription regulating RNA stability, not being an intron, e.g. poly A signal

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Genetics & Genomics (AREA)
  • Medicinal Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Molecular Biology (AREA)
  • Zoology (AREA)
  • Biochemistry (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • Epidemiology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Biophysics (AREA)
  • General Engineering & Computer Science (AREA)
  • Virology (AREA)
  • Wood Science & Technology (AREA)
  • Ophthalmology & Optometry (AREA)
  • Immunology (AREA)
  • Cardiology (AREA)
  • Microbiology (AREA)
  • Urology & Nephrology (AREA)
  • Vascular Medicine (AREA)
  • Physics & Mathematics (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Plant Pathology (AREA)
  • Toxicology (AREA)
  • Marine Sciences & Fisheries (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
CN202010705069.XA 2020-07-21 2020-07-21 用于治疗眼部疾病的组合物和方法 Pending CN113952472A (zh)

Priority Applications (8)

Application Number Priority Date Filing Date Title
CN202010705069.XA CN113952472A (zh) 2020-07-21 2020-07-21 用于治疗眼部疾病的组合物和方法
CN202180064480.6A CN116615552A (zh) 2020-07-21 2021-07-20 用于治疗眼疾病的组合物和方法
JP2023512665A JP2023542279A (ja) 2020-07-21 2021-07-20 眼疾患の処置のための組成物および方法
AU2021312379A AU2021312379A1 (en) 2020-07-21 2021-07-20 Compositions and methods for the treatment of eye diseases
PCT/IB2021/000509 WO2022018518A1 (en) 2020-07-21 2021-07-20 Compositions and methods for the treatment of eye diseases
CA3186826A CA3186826A1 (en) 2020-07-21 2021-07-20 Compositions and methods for the treatment of eye diseases
EP21845856.0A EP4185319A4 (en) 2020-07-21 2021-07-20 COMPOSITIONS AND METHODS FOR THE TREATMENT OF OPHTHALMIC DISEASES
US18/157,589 US20230321281A1 (en) 2020-07-21 2023-01-20 Compositions and methods for the treatment of eye diseases

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
CN202010705069.XA CN113952472A (zh) 2020-07-21 2020-07-21 用于治疗眼部疾病的组合物和方法

Publications (1)

Publication Number Publication Date
CN113952472A true CN113952472A (zh) 2022-01-21

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CN202010705069.XA Pending CN113952472A (zh) 2020-07-21 2020-07-21 用于治疗眼部疾病的组合物和方法
CN202180064480.6A Pending CN116615552A (zh) 2020-07-21 2021-07-20 用于治疗眼疾病的组合物和方法

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US (1) US20230321281A1 (https=)
EP (1) EP4185319A4 (https=)
JP (1) JP2023542279A (https=)
CN (2) CN113952472A (https=)
AU (1) AU2021312379A1 (https=)
CA (1) CA3186826A1 (https=)
WO (1) WO2022018518A1 (https=)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US11345930B2 (en) * 2020-09-02 2022-05-31 4D Molecular Therapeutics Inc. Codon optimized RPGRORF15 genes and uses thereof

Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2004050027A2 (en) * 2002-12-02 2004-06-17 Genvec, Inc. Materials and methods for treating ocular-related disorders
CN105940109A (zh) * 2013-12-06 2016-09-14 国立健康与医学研究所 用于在受试者的视网膜色素上皮中表达目的多核苷酸的方法和药物组合物
CN105980569A (zh) * 2013-08-05 2016-09-28 莱顿大学医学中心附属莱顿教学医院 重组aav-crumbs同源物组合物及治疗lca-8和进行性rp的方法
CN108699118A (zh) * 2015-09-10 2018-10-23 牛津大学创新有限公司 色素性视网膜炎的治疗

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EP2872183B1 (en) * 2012-07-11 2018-09-26 The Trustees Of The University Of Pennsylvania Aav-mediated gene therapy for rpgr x-linked retinal degeneration
JP5971661B2 (ja) * 2012-09-19 2016-08-17 国立大学法人 東京大学 神経活動依存的プロモータ活性を有するdna、及びこれを含むベクター
CA2945965C (en) 2014-04-15 2022-11-01 Applied Genetic Technologies Corporation Codon optimized nucleic acid encoding a retinitis pigmentosa gtpase regulator (rpgr)
IL248102B (en) * 2014-05-02 2022-07-01 Genzyme Corp aav vectors for gene therapy of the central nervous system and retina
US20160346359A1 (en) * 2015-05-01 2016-12-01 Spark Therapeutics, Inc. Adeno-associated Virus-Mediated CRISPR-Cas9 Treatment of Ocular Disease
EP3827812B1 (en) * 2016-07-29 2025-10-29 The Regents of the University of California Adeno-associated virus virions with variant capsid and methods of use thereof
WO2019006182A1 (en) * 2017-06-30 2019-01-03 The Regents Of The University Of California VIRIONS OF ADENO-ASSOCIATED VIRUSES OF CAPSID VARIANTS AND METHODS OF USE THEREOF
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TW202028225A (zh) * 2018-09-21 2020-08-01 英商夜星克有限公司 用於治療色素性視網膜炎之組合物及方法
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WO2004050027A2 (en) * 2002-12-02 2004-06-17 Genvec, Inc. Materials and methods for treating ocular-related disorders
CA2507036A1 (en) * 2002-12-02 2004-06-17 Genvec, Inc. Materials and methods for treating ocular-related disorders
US20050220768A1 (en) * 2002-12-02 2005-10-06 Genvec, Inc. Materials and methods for treating ocular-related disorders
CN105980569A (zh) * 2013-08-05 2016-09-28 莱顿大学医学中心附属莱顿教学医院 重组aav-crumbs同源物组合物及治疗lca-8和进行性rp的方法
CN105940109A (zh) * 2013-12-06 2016-09-14 国立健康与医学研究所 用于在受试者的视网膜色素上皮中表达目的多核苷酸的方法和药物组合物
CN108699118A (zh) * 2015-09-10 2018-10-23 牛津大学创新有限公司 色素性视网膜炎的治疗

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VALE´RIE L. DUFOUR ET AL: "Toxicity and Efficacy Evaluation of an Adeno-Associated Virus Vector Expressing Codon-Optimized RPGR Delivered by Subretinal Injection in a Canine Model of X-linked Retinitis Pigmentosa", 《CLINICAL DEVELOPMENT》, vol. 31, no. 3, 29 February 2020 (2020-02-29), pages 253 - 266 *

Also Published As

Publication number Publication date
CA3186826A1 (en) 2022-01-27
EP4185319A4 (en) 2024-12-04
JP2023542279A (ja) 2023-10-06
AU2021312379A1 (en) 2023-03-23
US20230321281A1 (en) 2023-10-12
CN116615552A (zh) 2023-08-18
EP4185319A1 (en) 2023-05-31
WO2022018518A1 (en) 2022-01-27

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Application publication date: 20220121