JP2024539123A - アデノ随伴ウイルス(aav)送達を改善する方法 - Google Patents

アデノ随伴ウイルス(aav)送達を改善する方法 Download PDF

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JP2024539123A
JP2024539123A JP2024523575A JP2024523575A JP2024539123A JP 2024539123 A JP2024539123 A JP 2024539123A JP 2024523575 A JP2024523575 A JP 2024523575A JP 2024523575 A JP2024523575 A JP 2024523575A JP 2024539123 A JP2024539123 A JP 2024539123A
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aav
aavhu
aavrh
aav42
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JP2024539123A5 (https=
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マンズフィールド,キース
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ノバルティス アーゲー
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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
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    • A61K31/33Heterocyclic compounds
    • A61K31/395Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/41Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having five-membered rings with two or more ring hetero atoms, at least one of which being nitrogen, e.g. tetrazole
    • A61K31/41641,3-Diazoles
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    • A61K31/395Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
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    • A61K31/41641,3-Diazoles
    • A61K31/4174Arylalkylimidazoles, e.g. oxymetazolin, naphazoline, miconazole
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    • A61K31/41Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having five-membered rings with two or more ring hetero atoms, at least one of which being nitrogen, e.g. tetrazole
    • A61K31/433Thidiazoles
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    • A61K31/435Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom
    • A61K31/44Non condensed pyridines; Hydrogenated derivatives thereof
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    • A61K31/44Non condensed pyridines; Hydrogenated derivatives thereof
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JP2024523575A 2021-10-25 2022-10-24 アデノ随伴ウイルス(aav)送達を改善する方法 Pending JP2024539123A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202163271487P 2021-10-25 2021-10-25
US63/271,487 2021-10-25
PCT/IB2022/060189 WO2023073526A1 (en) 2021-10-25 2022-10-24 Methods for improving adeno-associated virus (aav) delivery

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JP2024539123A true JP2024539123A (ja) 2024-10-28
JP2024539123A5 JP2024539123A5 (https=) 2025-10-31
JPWO2023073526A5 JPWO2023073526A5 (https=) 2025-10-31

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US (1) US20250235554A1 (https=)
EP (1) EP4422613A1 (https=)
JP (1) JP2024539123A (https=)
CN (1) CN118043039A (https=)
WO (1) WO2023073526A1 (https=)

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JPWO2024071362A1 (https=) * 2022-09-30 2024-04-04
WO2025101959A1 (en) * 2023-11-08 2025-05-15 Renbio, Inc. Plasmid dna constructs for peptide hormone expression
WO2025250457A1 (en) * 2024-05-28 2025-12-04 University Of Rochester Enhanced brain transduction by gene therapeutics
CN118743768A (zh) * 2024-06-13 2024-10-08 浙江大学 一种血小板-aav包装复合体及其制备方法和应用

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WO1998033917A1 (en) 1994-11-14 1998-08-06 The Ludwig Institute For Cancer Research Vascular endothelial growth factor c (vegf-c) protein and gene, mutants thereof, and uses thereof
US6001650A (en) 1995-08-03 1999-12-14 Avigen, Inc. High-efficiency wild-type-free AAV helper functions
US6410300B1 (en) 1998-01-12 2002-06-25 The University Of North Carolina At Chapel Hill Methods and formulations for mediating adeno-associated virus (AAV) attachment and infection and methods for purifying AAV
EP1751275B1 (en) 2004-06-01 2017-08-16 Avigen, Inc. Compositions and methods to prevent aav vector aggregation
SI2548560T1 (sl) 2005-06-23 2015-12-31 Isis Pharmaceuticals, Inc. Sestavki in postopki za moduliranje izrezovanja smn2
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PT3305302T (pt) 2009-06-17 2018-12-14 Biogen Ma Inc Composições e métodos de modulação de excisões de smn2 em um sujeito
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LT3702466T (lt) 2013-08-27 2023-02-27 Research Institute At Nationwide Children's Hospital Produktai ir būdai, skirti šoninės amiotrofinės sklerozės gydymui
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EP3245221A4 (en) 2015-01-16 2018-06-13 Voyager Therapeutics, Inc. Central nervous system targeting polynucleotides
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MX2020004830A (es) 2017-11-08 2020-11-11 Avexis Inc Medios y metodo para preparar vectores virales y usos de los mismos.
TW202015742A (zh) 2018-05-15 2020-05-01 美商航海家醫療公司 投遞腺相關病毒(aav)之組成物和方法
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US12311035B2 (en) * 2018-10-04 2025-05-27 University Of Rochester Glymphatic delivery by manipulating plasma osmolarity

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US20250235554A1 (en) 2025-07-24
WO2023073526A1 (en) 2023-05-04
CN118043039A (zh) 2024-05-14

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