JP2024506266A5 - - Google Patents

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Publication number
JP2024506266A5
JP2024506266A5 JP2023545212A JP2023545212A JP2024506266A5 JP 2024506266 A5 JP2024506266 A5 JP 2024506266A5 JP 2023545212 A JP2023545212 A JP 2023545212A JP 2023545212 A JP2023545212 A JP 2023545212A JP 2024506266 A5 JP2024506266 A5 JP 2024506266A5
Authority
JP
Japan
Prior art keywords
sequence
seq
polynucleotide
signal peptide
inhibitor
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Withdrawn
Application number
JP2023545212A
Other languages
English (en)
Japanese (ja)
Other versions
JP2024506266A (ja
Filing date
Publication date
Application filed filed Critical
Priority claimed from PCT/US2022/014091 external-priority patent/WO2022165027A2/en
Publication of JP2024506266A publication Critical patent/JP2024506266A/ja
Publication of JP2024506266A5 publication Critical patent/JP2024506266A5/ja
Withdrawn legal-status Critical Current

Links

JP2023545212A 2021-01-27 2022-01-27 遺伝性血管性浮腫を治療するための組成物および方法 Withdrawn JP2024506266A (ja)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US202163142121P 2021-01-27 2021-01-27
US63/142,121 2021-01-27
US202163201466P 2021-04-30 2021-04-30
US63/201,466 2021-04-30
US202163261603P 2021-09-24 2021-09-24
US63/261,603 2021-09-24
PCT/US2022/014091 WO2022165027A2 (en) 2021-01-27 2022-01-27 Compositions and methods for treating hereditary angioedema

Publications (2)

Publication Number Publication Date
JP2024506266A JP2024506266A (ja) 2024-02-13
JP2024506266A5 true JP2024506266A5 (https=) 2025-01-30

Family

ID=82653909

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2023545212A Withdrawn JP2024506266A (ja) 2021-01-27 2022-01-27 遺伝性血管性浮腫を治療するための組成物および方法

Country Status (12)

Country Link
US (1) US20240110201A1 (https=)
EP (1) EP4284417A4 (https=)
JP (1) JP2024506266A (https=)
KR (1) KR20230136147A (https=)
AU (1) AU2022214192A1 (https=)
CA (1) CA3207268A1 (https=)
CL (1) CL2023002138A1 (https=)
CO (1) CO2023011154A2 (https=)
IL (1) IL304404A (https=)
MX (1) MX2023008801A (https=)
PE (1) PE20240692A1 (https=)
WO (1) WO2022165027A2 (https=)

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
AU2024257493A1 (en) * 2023-04-18 2025-12-04 Siren Biotechnology, Inc. Adeno-associated viral vector compositions and methods of use
WO2025110984A1 (en) * 2023-11-21 2025-05-30 Hydrogene Therapeutics, Inc. Compositions and methods for treating wilson's disease

Family Cites Families (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR101610268B1 (ko) * 2007-08-28 2016-04-07 애들레이드 리서치 앤드 이노베이션 피티와이 리미티드 외과용 하이드로겔
WO2013151665A2 (en) * 2012-04-02 2013-10-10 modeRNA Therapeutics Modified polynucleotides for the production of proteins associated with human disease
CN108064176A (zh) * 2015-04-22 2018-05-22 库瑞瓦格股份公司 用于治疗肿瘤疾病的含有rna的组合物
US10214731B2 (en) * 2015-05-28 2019-02-26 Cornell University Adeno-associated virus mediated delivery of C1E1 as a therapy for angioedema
WO2021081280A1 (en) * 2019-10-23 2021-04-29 Shire Human Genetic Therapies, Inc. Adeno-associated virus vectors based gene therapy for hereditary angioedema
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

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