KR20230044522A - 척수성 근위축을 치료하기 위한 핵산 구축물 및 이의 용도 - Google Patents

척수성 근위축을 치료하기 위한 핵산 구축물 및 이의 용도 Download PDF

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KR20230044522A
KR20230044522A KR1020237007688A KR20237007688A KR20230044522A KR 20230044522 A KR20230044522 A KR 20230044522A KR 1020237007688 A KR1020237007688 A KR 1020237007688A KR 20237007688 A KR20237007688 A KR 20237007688A KR 20230044522 A KR20230044522 A KR 20230044522A
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nucleic acid
seq
mir
hsa
acid sequence
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Korean (ko)
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페이신 주
궈지에 예
젠후아 우
리준 왕
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항저우 엑제제네시스 바이오 엘티디.
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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
    • A61K48/0066Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
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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
    • 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
    • A61K48/0008Medicinal 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
    • A61K48/0025Medicinal 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 wherein the non-active part clearly interacts with the delivered nucleic acid
    • A61K48/0041Medicinal 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 wherein the non-active part clearly interacts with the delivered nucleic acid the non-active part being polymeric
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • 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
    • 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
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00Drugs for disorders of the muscular or neuromuscular system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • 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
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    • 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
    • C07K14/4707Muscular dystrophy
    • 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
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    • A01K2267/0306Animal model for genetic diseases
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/14Type of nucleic acid interfering nucleic acids [NA]
    • C12N2310/141MicroRNAs, miRNAs
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    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
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    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
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    • 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

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  • Health & Medical Sciences (AREA)
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  • Chemical & Material Sciences (AREA)
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  • General Health & Medical Sciences (AREA)
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  • Bioinformatics & Cheminformatics (AREA)
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  • Wood Science & Technology (AREA)
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  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Neurology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
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  • Physical Education & Sports Medicine (AREA)
  • Physics & Mathematics (AREA)
  • Gastroenterology & Hepatology (AREA)
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  • Neurosurgery (AREA)
  • Marine Sciences & Fisheries (AREA)
  • Immunology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
KR1020237007688A 2020-08-05 2021-08-04 척수성 근위축을 치료하기 위한 핵산 구축물 및 이의 용도 Pending KR20230044522A (ko)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
CNPCT/CN2020/107173 2020-08-05
CN2020107173 2020-08-05
CN2020138056 2020-12-21
CNPCT/CN2020/138056 2020-12-21
PCT/CN2021/110521 WO2022028472A1 (en) 2020-08-05 2021-08-04 Nucleic acid constructs and uses thereof for treating spinal muscular atrophy

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KR20230044522A true KR20230044522A (ko) 2023-04-04

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US (1) US20230293730A1 (https=)
EP (1) EP4192518A4 (https=)
JP (1) JP2023537358A (https=)
KR (1) KR20230044522A (https=)
CN (1) CN116685329B (https=)
AU (1) AU2021323289A1 (https=)
CA (1) CA3190601A1 (https=)
IL (1) IL300277A (https=)
WO (1) WO2022028472A1 (https=)

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US12612627B2 (en) * 2020-04-20 2026-04-28 Regents Of The University Of Minnesota Compositions and methods for increasing sodium current in cardiac cells
WO2023219394A1 (ko) * 2022-05-10 2023-11-16 서울대학교산학협력단 인간 smn1 단백질 변이체 및 이의 용도
EP4577662A1 (en) * 2022-08-24 2025-07-02 Beacon Therapeutics (USA), Inc. Methods and compositions for the production of recombinant adeno-associated virus (raav) vectors
EP4711461A1 (en) * 2023-05-12 2026-03-18 Genecombio Ltd. Viral vector carrying smn gene expression cassette and use thereof
CN118685413B (zh) * 2024-08-28 2025-02-07 杭州嘉因生物科技有限公司 下调内源性smn的诱导型稳定细胞株的构建及其应用

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CN103820562B (zh) * 2005-08-01 2015-05-13 俄亥俄州立大学研究基金会 用于乳腺癌的诊断、预后和治疗的基于MicroRNA的方法和组合物
CN101787373B (zh) * 2009-01-23 2013-06-19 中国人民解放军第二军医大学东方肝胆外科医院 一种携带外源基因在包装细胞中高效生产的重组病毒载体、其构建方法及其用途
EP2561073B1 (en) * 2010-04-23 2016-08-24 University of Massachusetts Cns targeting aav vectors and methods of use thereof
FR3004463A1 (fr) * 2013-04-11 2014-10-17 Genethon Systeme d'expression pour une therapie genique selective
MX380973B (es) * 2013-05-01 2025-03-12 Genzyme Corp Composiciones y metodos para tratar la atrofia muscular espinal.
WO2018146588A1 (en) * 2017-02-08 2018-08-16 Friedrich Miescher Institute For Biomedical Research Synp88, a promoter for the specific expression of genes in retinal ganglion cells
JOP20190200A1 (ar) * 2017-02-28 2019-08-27 Univ Pennsylvania تركيبات نافعة في معالجة ضمور العضل النخاعي
CN114908099B (zh) * 2018-06-28 2024-07-02 北京锦篮基因科技有限公司 携带设计smn1基因表达框的重组腺相关病毒及应用
CN111088284B (zh) * 2019-05-31 2022-02-01 北京京谱迪基因科技有限公司 携带截短抗肌萎缩蛋白基因表达框的aav载体、制备方法及应用
CN110616262A (zh) * 2019-08-14 2019-12-27 深圳市人民医院 外泌体中microRNA在评估脑胶质瘤放疗效果中的用途
CN113755524B (zh) * 2020-06-02 2023-11-03 舒泰神(北京)生物制药股份有限公司 用于治疗脊髓性肌萎缩的腺相关病毒载体及其用途

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CN116685329B (zh) 2025-03-18
JP2023537358A (ja) 2023-08-31
IL300277A (en) 2023-04-01
AU2021323289A1 (en) 2023-03-23
US20230293730A1 (en) 2023-09-21
EP4192518A1 (en) 2023-06-14
WO2022028472A1 (en) 2022-02-10
EP4192518A4 (en) 2024-12-25
CA3190601A1 (en) 2022-02-10
CN116685329A (zh) 2023-09-01

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