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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China
Prior art keywords
sequence
promoter
composition
aav
rpgr
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Pending
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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
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Priority to CN202010705069.XA priority Critical patent/CN113952472A/zh
Priority to EP21845856.0A priority patent/EP4185319A4/en
Priority to CN202180064480.6A priority patent/CN116615552A/zh
Priority to PCT/IB2021/000509 priority patent/WO2022018518A1/en
Priority to CA3186826A priority patent/CA3186826A1/en
Priority to AU2021312379A priority patent/AU2021312379A1/en
Priority to JP2023512665A priority patent/JP2023542279A/ja
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
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    • 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)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Zoology (AREA)
  • Biochemistry (AREA)
  • Biomedical Technology (AREA)
  • Epidemiology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Biophysics (AREA)
  • Virology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Ophthalmology & Optometry (AREA)
  • Immunology (AREA)
  • Vascular Medicine (AREA)
  • Toxicology (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Physics & Mathematics (AREA)
  • Urology & Nephrology (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Cardiology (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
EP21845856.0A EP4185319A4 (en) 2020-07-21 2021-07-20 COMPOSITIONS AND METHODS FOR THE TREATMENT OF OPHTHALMIC DISEASES
CN202180064480.6A CN116615552A (zh) 2020-07-21 2021-07-20 用于治疗眼疾病的组合物和方法
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
AU2021312379A AU2021312379A1 (en) 2020-07-21 2021-07-20 Compositions and methods for the treatment of eye diseases
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
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)

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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 (enExample)
EP (1) EP4185319A4 (enExample)
JP (1) JP2023542279A (enExample)
CN (2) CN113952472A (enExample)
AU (1) AU2021312379A1 (enExample)
CA (1) CA3186826A1 (enExample)
WO (1) WO2022018518A1 (enExample)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR102825852B1 (ko) * 2020-09-02 2025-06-26 4디 몰레큘러 테라퓨틱스 아이엔씨. 코돈 최적화된 rpgrorf15 유전자 및 이의 용도

Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA2507036A1 (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

Family Cites Families (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP6199965B2 (ja) * 2012-07-11 2017-09-20 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア Rpgrx連鎖性網膜変性のaav媒介型遺伝子治療
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)
LT3137497T (lt) * 2014-05-02 2021-07-26 Genzyme Corporation Aav vektoriai, skirti tinklainės ir cns genų terapijai
KR20230039779A (ko) * 2016-07-29 2023-03-21 더 리젠츠 오브 더 유니버시티 오브 캘리포니아 변이체 캡시드를 갖는 아데노-관련된 바이러스 비리온 및 이의 사용 방법
CN110461368A (zh) * 2017-06-30 2019-11-15 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
KR102824021B1 (ko) * 2018-01-31 2025-06-24 더 리서치 인스티튜트 앳 네이션와이드 칠드런스 하스피탈 2c형 지대근이영양증에 대한 유전자 치료
JOP20210054A1 (ar) * 2018-09-21 2020-03-21 Nightstarx Ltd تركيبات وطرق لعلاج التهاب الشبكية الصباغي
EP4087617A4 (en) * 2020-01-10 2024-05-01 Solid Biosciences Inc. VIRAL VECTOR FOR COMBINATION THERAPY

Patent Citations (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA2507036A1 (en) * 2002-12-02 2004-06-17 Genvec, Inc. Materials and methods for treating ocular-related disorders
WO2004050027A2 (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

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
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 *

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

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Application publication date: 20220121