JP2023537358A - 核酸構築物及び脊髄性筋委縮症治療のためのその使用 - Google Patents

核酸構築物及び脊髄性筋委縮症治療のためのその使用 Download PDF

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JP2023537358A
JP2023537358A JP2023507927A JP2023507927A JP2023537358A JP 2023537358 A JP2023537358 A JP 2023537358A JP 2023507927 A JP2023507927 A JP 2023507927A JP 2023507927 A JP2023507927 A JP 2023507927A JP 2023537358 A JP2023537358 A JP 2023537358A
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nucleic acid
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acid sequence
mir
hsa
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JP2023537358A5 (enExample
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ズフ ペイシン
イエ グオジエ
ウー ズヘンフア
ワング リジュン
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ハングズホウ エクセジェネシス バイオ エルティーディー．
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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
    • A61K48/0066—Manipulation 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
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    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
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    • A61K38/1709—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • 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
    • A61K48/0025—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 wherein the non-active part clearly interacts with the delivered nucleic acid
    • A61K48/0041—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 wherein the non-active part clearly interacts with the delivered nucleic acid the non-active part being polymeric
    • 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
    • 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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
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    • C—CHEMISTRY; METALLURGY
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    • C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
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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
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
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    • A01K2217/07—Animals genetically altered by homologous recombination
    • A01K2217/075—Animals genetically altered by homologous recombination inducing loss of function, i.e. knock out
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
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    • 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
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    • A01K2267/0306—Animal model for genetic diseases
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
    • C12N2310/141—MicroRNAs, miRNAs
    • C—CHEMISTRY; METALLURGY
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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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    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination

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  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
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JP2023507927A 2020-08-05 2021-08-04 核酸構築物及び脊髄性筋委縮症治療のためのその使用 Pending JP2023537358A (ja)

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JP2026003690A JP2026086420A (ja) 2020-08-05 2026-01-13 核酸構築物及び脊髄性筋委縮症治療のためのその使用

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CN2020107173 2020-08-05
CNPCT/CN2020/107173 2020-08-05
CNPCT/CN2020/138056 2020-12-21
CN2020138056 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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JP2026003690A Pending JP2026086420A (ja) 2020-08-05 2026-01-13 核酸構築物及び脊髄性筋委縮症治療のためのその使用

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EP (1) EP4192518A4 (enExample)
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KR (1) KR20230044522A (enExample)
CN (1) CN116685329B (enExample)
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WO2021216415A1 (en) * 2020-04-20 2021-10-28 Regents Of The University Of Minnesota Compositions and methods for increasing sodium current in cardiac cells
KR102717399B1 (ko) * 2022-05-10 2024-10-16 서울대학교산학협력단 인간 smn1 단백질 변이체 및 이의 용도
WO2024044340A1 (en) * 2022-08-24 2024-02-29 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

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN108795946A (zh) * 2018-06-28 2018-11-13 北京瑞希罕见病基因治疗技术研究所 携带设计smn1基因表达框的重组腺相关病毒及应用

Family Cites Families (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2007016548A2 (en) * 2005-08-01 2007-02-08 The Ohio State University Research Foundation Micro-rna-based methods and compositions for the diagnosis, prognosis and treatment of breast cancer
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
DK2561073T3 (en) * 2010-04-23 2016-12-12 Univ Massachusetts Aav vectors targeted to central nervous system and methods of use thereof
FR3004463A1 (fr) * 2013-04-11 2014-10-17 Genethon Systeme d'expression pour une therapie genique selective
BR112015027336A2 (pt) * 2013-05-01 2017-09-26 Genzyme Corp composições e processos para tratamento de atrofia muscular espinhal
JP7112414B2 (ja) * 2017-02-08 2022-08-03 フリードリッヒ ミーシェー インスティトゥート フォー バイオメディカル リサーチ 網膜神経節細胞中の遺伝子の特異的発現のためのプロモーターｓｙｎｐ８８
JOP20190200A1 (ar) * 2017-02-28 2019-08-27 Univ Pennsylvania تركيبات نافعة في معالجة ضمور العضل النخاعي
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

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN108795946A (zh) * 2018-06-28 2018-11-13 北京瑞希罕见病基因治疗技术研究所 携带设计smn1基因表达框的重组腺相关病毒及应用

Non-Patent Citations (4)

* Cited by examiner, † Cited by third party
Title
CONNOLLY, M ET AL., PLOS ONE, [ONLINE], JPN6025042158, 28 February 2020 (2020-02-28), pages 1 - 10, ISSN: 0005834263 *
LIANG, Y ET AL., BMC GENOMICS, [ONLINE], JPN6025042157, 2007, ISSN: 0005834264 *
NUCLEIC ACIDS RESEARCH, vol. 44, no. 8, JPN6025042154, 2016, pages 3865 - 3877, ISSN: 0005834261 *
WIRES SYSTEMS BIOLOGY AND MEDICINE, vol. vol.3(2), JPN6025042156, 2011, pages 183 - 190, ISSN: 0005834262 *

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AU2021323289A1 (en) 2023-03-23
US20230293730A1 (en) 2023-09-21
CA3190601A1 (en) 2022-02-10
CN116685329A (zh) 2023-09-01
JP2026086420A (ja) 2026-05-26
CN116685329B (zh) 2025-03-18
EP4192518A4 (en) 2024-12-25
KR20230044522A (ko) 2023-04-04
IL300277A (en) 2023-04-01
EP4192518A1 (en) 2023-06-14
WO2022028472A1 (en) 2022-02-10

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