JP2021521833A - 筋疾患の治療のための改変ａａｖカプシドポリペプチド - Google Patents

筋疾患の治療のための改変ａａｖカプシドポリペプチド Download PDF

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JP2021521833A
JP2021521833A JP2020560127A JP2020560127A JP2021521833A JP 2021521833 A JP2021521833 A JP 2021521833A JP 2020560127 A JP2020560127 A JP 2020560127A JP 2020560127 A JP2020560127 A JP 2020560127A JP 2021521833 A JP2021521833 A JP 2021521833A
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aav capsid
capsid polypeptide
muscle
aav
amino acid
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JP2021521833A5 (https=
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グリム，ディルク
ヴァインマン，ヨーナス
アンダリ，ヨーゼフ エル
アンダリ，ヨーゼフ エル
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ウニベルジテート ハイデルベルク
ウニベルジテート ハイデルベルク
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Publication of JP2021521833A publication Critical patent/JP2021521833A/ja
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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
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    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
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    • C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
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    • A61K38/00—Medicinal preparations containing peptides
    • 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
    • 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
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/035—Fusion polypeptide containing a localisation/targetting motif containing a signal for targeting to the external surface of a cell, e.g. to the outer membrane of Gram negative bacteria, GPI- anchored eukaryote proteins
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/33—Fusion polypeptide fusions for targeting to specific cell types, e.g. tissue specific targeting, targeting of a bacterial subspecies
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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14122—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
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    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
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    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
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    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
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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
    • C12N2750/14145—Special targeting system for viral vectors

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  • Proteomics, Peptides & Aminoacids (AREA)
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  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
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JP2020560127A 2018-04-27 2019-04-26 筋疾患の治療のための改変ａａｖカプシドポリペプチド Pending JP2021521833A (ja)

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JP2023191983A JP7707261B2 (ja) 2018-04-27 2023-11-10 筋疾患の治療のための改変ａａｖカプシドポリペプチド

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Application Number Priority Date Filing Date Title
EP18169822.6 2018-04-27
EP18169822 2018-04-27
PCT/EP2019/060790 WO2019207132A1 (en) 2018-04-27 2019-04-26 Modified aav capsid polypeptides for treatment of muscular diseases

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JP2021521833A true JP2021521833A (ja) 2021-08-30
JP2021521833A5 JP2021521833A5 (https=) 2022-05-09

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JP2023191983A Active JP7707261B2 (ja) 2018-04-27 2023-11-10 筋疾患の治療のための改変ａａｖカプシドポリペプチド

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US (1) US20210363193A1 (https=)
EP (1) EP3784288A1 (https=)
JP (2) JP2021521833A (https=)
CN (1) CN112040988A (https=)
AU (2) AU2019258830B2 (https=)
CA (1) CA3097375A1 (https=)
WO (1) WO2019207132A1 (https=)

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JP2022551487A (ja) * 2019-10-10 2022-12-09 ソリッド・バイオサイエンシーズ・インコーポレーテッド 改変されたａａｖカプシドおよびその使用
JP2022551986A (ja) * 2019-10-16 2022-12-14 ザ・ブロード・インスティテュート・インコーポレイテッド 修飾筋肉標的化組成物

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EP4028064A1 (en) * 2019-09-12 2022-07-20 The Broad Institute, Inc. Engineered adeno-associated virus capsids
JP7651562B2 (ja) * 2019-09-19 2025-03-26 ジェネトン Ｆｋｒｐの心毒性を軽減する遺伝子治療発現系
TWI897882B (zh) * 2019-09-20 2025-09-21 英商Ｕｃｌ商業有限責任公司 右心室心律失常性心肌病之基因療法組合物及治療
JP2023523573A (ja) 2020-04-14 2023-06-06 ジェネトン 酸性セラミダーゼ欠乏症を処置するためのベクター
US12552838B2 (en) 2020-04-20 2026-02-17 Tenaya Therapeutics, Inc. Adeno-associated virus with engineered capsid
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