JP2024540181A - 骨格筋に再標的化されたウイルス粒子 - Google Patents

骨格筋に再標的化されたウイルス粒子 Download PDF

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JP2024540181A
JP2024540181A JP2024525653A JP2024525653A JP2024540181A JP 2024540181 A JP2024540181 A JP 2024540181A JP 2024525653 A JP2024525653 A JP 2024525653A JP 2024525653 A JP2024525653 A JP 2024525653A JP 2024540181 A JP2024540181 A JP 2024540181A
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protein
capsid
aav
modified
binding pair
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JP2024540181A5 (https=
Inventor
リア サビン，
マイケル ステック，
アンドリュー ジェイ． マーフィー，
クリストス カラツオス，
スヴェン モーラー－タンク，
ポウラミ サマイ，
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Regeneron Pharmaceuticals Inc
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Regeneron Pharmaceuticals Inc
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Publication of JP2024540181A publication Critical patent/JP2024540181A/ja
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    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
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    • A01K67/00—Rearing or breeding animals, not otherwise provided for; New or modified breeds of animals
    • A01K67/027—New or modified breeds of vertebrates
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    • A—HUMAN NECESSITIES
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    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
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    • A61K35/76—Viruses; Subviral particles; Bacteriophages
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
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    • A61K47/64—Drug-peptide, drug-protein or drug-polyamino acid conjugates, i.e. the modifying agent being a peptide, protein or polyamino acid which is covalently bonded or complexed to a therapeutically active agent
    • 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
    • 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
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A61P21/04—Drugs for disorders of the muscular or neuromuscular system for myasthenia gravis
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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
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    • C12N5/0602—Vertebrate cells
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    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
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    • A01K2207/00—Modified animals
    • A01K2207/15—Humanized animals
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    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
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JP2024525653A 2021-11-04 2022-11-04 骨格筋に再標的化されたウイルス粒子 Pending JP2024540181A (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US202163275731P 2021-11-04 2021-11-04
US63/275,731 2021-11-04
US202263369761P 2022-07-28 2022-07-28
US63/369,761 2022-07-28
PCT/US2022/079339 WO2023081850A1 (en) 2021-11-04 2022-11-04 Viral particles retargeted to skeletal muscle

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JP2024540181A true JP2024540181A (ja) 2024-10-31
JP2024540181A5 JP2024540181A5 (https=) 2025-11-11

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US (1) US20250304918A1 (https=)
EP (1) EP4426359A1 (https=)
JP (1) JP2024540181A (https=)
KR (1) KR20240095211A (https=)
CN (1) CN118201643A (https=)
AU (1) AU2022380841A1 (https=)
CA (1) CA3233698A1 (https=)
CL (2) CL2024001307A1 (https=)
CO (1) CO2024005350A2 (https=)
IL (1) IL312598A (https=)
MX (1) MX2024005314A (https=)
PE (1) PE20241212A1 (https=)
WO (1) WO2023081850A1 (https=)

Families Citing this family (6)

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Publication number Priority date Publication date Assignee Title
EP4612184A1 (en) * 2022-11-04 2025-09-10 Regeneron Pharmaceuticals, Inc. Calcium voltage-gated channel auxiliary subunit gamma 1 (cacng1) binding proteins and cacng1-mediated delivery to skeletal muscle
EP4665764A1 (en) * 2023-02-13 2025-12-24 Regeneron Pharmaceuticals, Inc. Treatment of muscle related disorders with anti-human cacng1 antibodies
CN121568720A (zh) * 2023-07-10 2026-02-24 瑞泽恩制药公司 抗人类cacng1抗体-药物缀合物及其用途
TW202528546A (zh) * 2023-09-07 2025-07-16 美商再生元醫藥公司 經共價表面修飾之腺相關病毒的生產及純化
WO2025087345A1 (en) * 2023-10-25 2025-05-01 Suzhou Cure Genetics Biosciences Co., Limited Targeted viral particles
WO2026037757A1 (en) 2024-08-13 2026-02-19 F. Hoffmann-La Roche Ag Modified aav particles

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WO1998011244A2 (en) 1996-09-11 1998-03-19 The Government Of The United States Of America, Represented By The Secretary, Department Of Health And Human Services Aav4 vector and uses thereof
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WO1999061601A2 (en) 1998-05-28 1999-12-02 The Government Of The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Aav5 vector and uses thereof
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ES2602610T3 (es) 2007-05-31 2017-02-21 Medigene Ag Proteína estructural mutada de un parvovirus
US8236557B2 (en) 2008-05-28 2012-08-07 University Of Missouri-Columbia Hybrid-AAV vectors to deliver large gene expression cassette
ES2724975T3 (es) 2009-12-10 2019-09-18 Regeneron Pharma Ratones que producen anticuerpos de cadena pesada
GB201002362D0 (en) 2010-02-11 2010-03-31 Isis Innovation Peptide tag systems that spontaneously form an irreversible link to protein partners via isopeptide bonds
CA3124228C (en) 2014-03-21 2024-05-14 Regeneron Pharmaceuticals, Inc. Non-human animals that make single domain binding proteins
EP3151866B1 (en) 2014-06-09 2023-03-08 Voyager Therapeutics, Inc. Chimeric capsids
JP6832280B2 (ja) 2015-01-16 2021-02-24 ユニバーシティ オブ ワシントンＵｎｉｖｅｒｓｉｔｙ ｏｆ Ｗａｓｈｉｎｇｔｏｎ 新規のマイクロジストロフィンおよび使用の関連する方法
CN117887804A (zh) 2017-02-02 2024-04-16 纽约基因组研究中心公司 用于识别或量化在生物样品中的靶标的方法和组合物
PE20200488A1 (es) 2017-06-27 2020-03-03 Regeneron Pharma Vectores virales recombinantes con tropismo modificado y usos de estos para la introduccion dirigida de material genetico a celulas humanas
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LT3645553T (lt) 2017-06-27 2023-04-25 Regeneron Pharmaceuticals, Inc. Modifikuoto tropizmo rekombinantinės viruso dalelės ir jų panaudojimas genetinės medžiagos tiksliniam įvedimui į žmogaus ląsteles
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CN118201643A (zh) 2024-06-14
IL312598A (en) 2024-07-01
EP4426359A1 (en) 2024-09-11
PE20241212A1 (es) 2024-06-06
AU2022380841A1 (en) 2024-05-02
CL2025000839A1 (es) 2025-05-30
WO2023081850A1 (en) 2023-05-11
MX2024005314A (es) 2024-05-17
CO2024005350A2 (es) 2024-05-10
KR20240095211A (ko) 2024-06-25
CA3233698A1 (en) 2023-05-11
CL2024001307A1 (es) 2024-10-11
US20250304918A1 (en) 2025-10-02

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