CN109843306A - 使用自身互补型重组腺相关病毒治疗病症的方法和组合物 - Google Patents

使用自身互补型重组腺相关病毒治疗病症的方法和组合物 Download PDF

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Publication number
CN109843306A
CN109843306A CN201780061659.XA CN201780061659A CN109843306A CN 109843306 A CN109843306 A CN 109843306A CN 201780061659 A CN201780061659 A CN 201780061659A CN 109843306 A CN109843306 A CN 109843306A
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vector
raav
modified
gene
capsid
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Chinese (zh)
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J·S·巴特利特
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University of Florida Research Foundation Inc
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Karin Muse Ltd By Share Ltd
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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
    • 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
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/19—Cytokines; Lymphokines; Interferons
    • A61K38/20—Interleukins [IL]
    • A61K38/2006—IL-1
    • 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
    • A61K35/12—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
    • A61K35/32—Bones; Osteocytes; Osteoblasts; Tendons; Tenocytes; Teeth; Odontoblasts; Cartilage; Chondrocytes; Synovial membrane
    • 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
    • A61K35/761—Adenovirus
    • 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
    • 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
    • A61K48/0008—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • 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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P19/00—Drugs for skeletal disorders
    • A61P19/02—Drugs for skeletal disorders for joint disorders, e.g. arthritis, arthrosis
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P29/00—Non-central analgesic, antipyretic or antiinflammatory agents, e.g. antirheumatic agents; Non-steroidal antiinflammatory drugs [NSAID]
    • 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
    • C07K14/52—Cytokines; Lymphokines; Interferons
    • C07K14/54—Interleukins [IL]
    • C07K14/545—IL-1
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • 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

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Immunology (AREA)
  • Zoology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Genetics & Genomics (AREA)
  • Molecular Biology (AREA)
  • Rheumatology (AREA)
  • Biotechnology (AREA)
  • Virology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Toxicology (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Dermatology (AREA)
  • Biomedical Technology (AREA)
  • Cell Biology (AREA)
  • Developmental Biology & Embryology (AREA)
  • Pain & Pain Management (AREA)
  • Microbiology (AREA)
  • Mycology (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
CN201780061659.XA 2016-08-19 2017-08-18 使用自身互补型重组腺相关病毒治疗病症的方法和组合物 Pending CN109843306A (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201662377297P 2016-08-19 2016-08-19
US62/377297 2016-08-19
PCT/US2017/047589 WO2018035451A1 (en) 2016-08-19 2017-08-18 Methods and compositions for treating conditions using recombinant self-complementary adeno-associated virus

Publications (1)

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CN109843306A true CN109843306A (zh) 2019-06-04

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US (2) US11207382B2 (enExample)
EP (1) EP3500278B1 (enExample)
JP (2) JP7165410B2 (enExample)
KR (2) KR102759317B1 (enExample)
CN (1) CN109843306A (enExample)
AU (1) AU2017313844B2 (enExample)
CA (1) CA3073137A1 (enExample)
ES (1) ES2982489T3 (enExample)
WO (1) WO2018035451A1 (enExample)

Cited By (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN110225923A (zh) * 2016-12-07 2019-09-10 佛罗里达大学研究基金会有限公司 IL-1Ra cDNA
CN116096904A (zh) * 2019-12-31 2023-05-09 天鹅生物疗法有限公司 改进的aav-abcd1构建体和用于治疗或预防肾上腺脑白质营养不良(ald)和/或肾上腺脊髓神经病(amn)的用途

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KR102759317B1 (ko) 2016-08-19 2025-01-31 유니버시티 오브 플로리다 리서치 파운데이션, 인코포레이티드 재조합 자기-상보적인 아데노-부속 바이러스를 이용한 병태의 치료 방법 및 조성물
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US12370240B2 (en) 2018-11-28 2025-07-29 Genascence Corporation Methods and compositions for treating osteoarthritis
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HRP20241647T1 (hr) * 2019-07-15 2025-02-14 Meiragtx Uk Ii Limited Modificirani proteini aav kapsida za liječenje artritične bolesti
TW202525832A (zh) 2023-08-31 2025-07-01 美商戴諾治療公司 衣殼多肽及其使用方法(二)

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Cited By (3)

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Publication number Priority date Publication date Assignee Title
CN110225923A (zh) * 2016-12-07 2019-09-10 佛罗里达大学研究基金会有限公司 IL-1Ra cDNA
US11958886B2 (en) 2016-12-07 2024-04-16 University Of Florida Research Foundation, Incorporated IL-1RA cDNAs
CN116096904A (zh) * 2019-12-31 2023-05-09 天鹅生物疗法有限公司 改进的aav-abcd1构建体和用于治疗或预防肾上腺脑白质营养不良(ald)和/或肾上腺脊髓神经病(amn)的用途

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KR20230130155A (ko) 2023-09-11
CA3073137A1 (en) 2018-02-22
JP2019524890A (ja) 2019-09-05
JP7653150B2 (ja) 2025-03-28
ES2982489T3 (es) 2024-10-16
JP2022130745A (ja) 2022-09-06
US20220175887A1 (en) 2022-06-09
EP3500278B1 (en) 2024-05-22
EP3500278A1 (en) 2019-06-26
US11207382B2 (en) 2021-12-28
AU2017313844A1 (en) 2019-04-11
WO2018035451A1 (en) 2018-02-22
EP3500278A4 (en) 2020-04-01
AU2017313844B2 (en) 2023-10-12
US20190381140A1 (en) 2019-12-19
JP7165410B2 (ja) 2022-11-04
KR20190055086A (ko) 2019-05-22
EP3500278C0 (en) 2024-05-22
KR102759317B1 (ko) 2025-01-31

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