JP7659550B2 - アデノ随伴ウイルスベクターに基づく遺伝性血管浮腫のための遺伝子治療 - Google Patents

アデノ随伴ウイルスベクターに基づく遺伝性血管浮腫のための遺伝子治療 Download PDF

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JP7659550B2
JP7659550B2 JP2022523915A JP2022523915A JP7659550B2 JP 7659550 B2 JP7659550 B2 JP 7659550B2 JP 2022523915 A JP2022523915 A JP 2022523915A JP 2022523915 A JP2022523915 A JP 2022523915A JP 7659550 B2 JP7659550 B2 JP 7659550B2
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JP2022553083A (ja
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マティアス クラグマン，
フランチスカ ホーリング，
ヨハネス レングラー，
パトリース ドゥイヤール，
フリードリヒ シェイフリンガー，
ハンスペーター ロッテンシュタイナー，
バギラス ガンガダラン，
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シャイアー ヒューマン ジェネティック セラピーズ インコーポレイテッド
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    • A—HUMAN NECESSITIES
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    • 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
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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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    • C12N2830/48—Vector systems having a special element relevant for transcription regulating transport or export of RNA, e.g. RRE, PRE, WPRE, CTE

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JP2022523915A 2019-10-23 2020-10-23 アデノ随伴ウイルスベクターに基づく遺伝性血管浮腫のための遺伝子治療 Active JP7659550B2 (ja)

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US201962924877P 2019-10-23 2019-10-23
US62/924,877 2019-10-23
PCT/US2020/056984 WO2021081280A1 (en) 2019-10-23 2020-10-23 Adeno-associated virus vectors based gene therapy for hereditary angioedema

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JP2025055764A Ceased JP2025098221A (ja) 2019-10-23 2025-03-28 アデノ随伴ウイルスベクターに基づく遺伝性血管浮腫のための遺伝子治療

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US (1) US20230043051A1 (https=)
EP (1) EP4048800A1 (https=)
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JP2023503850A (ja) * 2019-11-14 2023-02-01 バイオマリン ファーマシューティカル インコーポレイテッド 肝臓特異的遺伝子療法ベクターによる遺伝性血管浮腫の治療
WO2022130014A1 (en) * 2020-12-16 2022-06-23 Takeda Pharmaceutical Company Limited Adeno associated viral vector delivery of antibodies for the treatment of disease mediated by dysregulated plasma kallikrein
CA3207268A1 (en) * 2021-01-27 2022-08-04 Spark Therapeutics, Inc. Compositions and methods for treating hereditary angioedema
US20240408240A1 (en) * 2021-10-01 2024-12-12 Biomarin Pharmaceutical Inc. Treatment of hereditary angioedema with aav gene therapy vectors and therapeutic formulations
CN117947040A (zh) 2022-10-31 2024-04-30 苏州荷光科汇生物科技有限公司 用于目的基因的表达盒及其应用

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CN115176022A (zh) 2022-10-11
US20230043051A1 (en) 2023-02-09
JP2022553083A (ja) 2022-12-21
WO2021081280A1 (en) 2021-04-29
EP4048800A1 (en) 2022-08-31
JP2025098221A (ja) 2025-07-01

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