JP2023503850A5 - - Google Patents

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Publication number
JP2023503850A5
JP2023503850A5 JP2022527987A JP2022527987A JP2023503850A5 JP 2023503850 A5 JP2023503850 A5 JP 2023503850A5 JP 2022527987 A JP2022527987 A JP 2022527987A JP 2022527987 A JP2022527987 A JP 2022527987A JP 2023503850 A5 JP2023503850 A5 JP 2023503850A5
Authority
JP
Japan
Application number
JP2022527987A
Other languages
Japanese (ja)
Other versions
JP2023503850A (ja
JPWO2021097157A5 (https=
Filing date
Publication date
Application filed filed Critical
Priority claimed from PCT/US2020/060337 external-priority patent/WO2021097157A1/en
Publication of JP2023503850A publication Critical patent/JP2023503850A/ja
Publication of JP2023503850A5 publication Critical patent/JP2023503850A5/ja
Publication of JPWO2021097157A5 publication Critical patent/JPWO2021097157A5/ja
Pending legal-status Critical Current

Links

JP2022527987A 2019-11-14 2020-11-13 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 Pending JP2023503850A (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201962935359P 2019-11-14 2019-11-14
US62/935,359 2019-11-14
US202063016365P 2020-04-28 2020-04-28
US63/016,365 2020-04-28
PCT/US2020/060337 WO2021097157A1 (en) 2019-11-14 2020-11-13 Treatment of hereditary angioedema with liver-specific gene therapy vectors

Publications (3)

Publication Number Publication Date
JP2023503850A JP2023503850A (ja) 2023-02-01
JP2023503850A5 true JP2023503850A5 (https=) 2023-11-20
JPWO2021097157A5 JPWO2021097157A5 (https=) 2023-11-20

Family

ID=73834586

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2022527987A Pending JP2023503850A (ja) 2019-11-14 2020-11-13 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

Country Status (14)

Country Link
US (1) US20230340078A1 (https=)
EP (1) EP4058475A1 (https=)
JP (1) JP2023503850A (https=)
KR (1) KR20220098210A (https=)
CN (1) CN114829391A (https=)
AU (1) AU2020384294A1 (https=)
BR (1) BR112022009279A2 (https=)
CA (1) CA3161154A1 (https=)
CL (1) CL2022001260A1 (https=)
CO (1) CO2022005641A2 (https=)
IL (1) IL292717A (https=)
MX (1) MX2022005869A (https=)
TW (1) TW202128733A (https=)
WO (1) WO2021097157A1 (https=)

Families Citing this family (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20230407328A1 (en) 2020-11-02 2023-12-21 Biomarin Pharmaceutical Inc. Process for enriching adeno-associated virus
WO2022165027A2 (en) * 2021-01-27 2022-08-04 Spark Therapeutics, Inc. Compositions and methods for treating hereditary angioedema
JP2024523891A (ja) * 2021-06-17 2024-07-02 メイラグティーエックス ユーケー アイアイ リミティド Aav産生方法
AR127217A1 (es) 2021-10-01 2023-12-27 Biomarin Pharm Inc Tratamiento de angioedema hereditario con vectores de genoterapia de aav y formulaciones terapéuticas
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

Family Cites Families (18)

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Publication number Priority date Publication date Assignee Title
IE58011B1 (en) 1983-05-27 1993-06-16 Texas A & M Univ Sys Method for producing a recombinant baculovirus expression vector
US4745051A (en) 1983-05-27 1988-05-17 The Texas A&M University System Method for producing a recombinant baculovirus expression vector
ZA848495B (en) 1984-01-31 1985-09-25 Idaho Res Found Production of polypeptides in insect cells
US6204059B1 (en) 1994-06-30 2001-03-20 University Of Pittsburgh AAV capsid vehicles for molecular transfer
US7323324B2 (en) 2001-10-16 2008-01-29 National Institute Of Advanced Industrial Science And Technology N-Acetylglucosamine transferase, nucleic acid encoding the same, antibody against the same and use thereof for diagnosing cancer or tumor
US6723551B2 (en) 2001-11-09 2004-04-20 The United States Of America As Represented By The Department Of Health And Human Services Production of adeno-associated virus in insect cells
NZ532635A (en) 2001-11-13 2007-05-31 Univ Pennsylvania A method of identifying unknown adeno-associated virus (AAV) sequences and a kit for the method
WO2003074714A1 (en) 2002-03-05 2003-09-12 Stichting Voor De Technische Wetenschappen Baculovirus expression system
AU2003221733A1 (en) * 2002-04-17 2003-11-03 University Of Florida Research Foundation, Inc. Improved raav vectors
US7943379B2 (en) 2008-04-30 2011-05-17 Nationwide Children's Hospital, Inc. Production of rAAV in vero cells using particular adenovirus helpers
AU2013221212B2 (en) 2012-02-17 2018-08-09 The Children's Hospital Of Philadelphia AAV vector compositions and methods for gene transfer to cells, organs and tissues
KR20240090694A (ko) * 2013-07-22 2024-06-21 더 칠드런스 호스피탈 오브 필라델피아 변종 aav 및 조성물, 세포, 기관 및 조직으로의 유전자 전이를 위한 방법 및 용도
RS61039B1 (sr) * 2013-09-12 2020-12-31 Biomarin Pharm Inc Aav vektori koji sadrže gen koji kodira faktor viii
JP6573991B2 (ja) * 2015-05-28 2019-09-11 コーネル ユニヴァーシティー 血管性浮腫の治療としてのc1eiのアデノ随伴ウイルス介在性送達
PE20190401A1 (es) 2016-07-26 2019-03-13 Biomarin Pharm Inc Novedosas proteinas de la capside del virus adenoasociado
KR20200122320A (ko) * 2018-01-16 2020-10-27 씨엘에스 테라퓨틱스 리미티드 데옥시리보뉴클레아제 (dnase) 활성을 갖는 효소의 간 발현에 의한 질환의 치료
US20210379203A1 (en) * 2018-10-26 2021-12-09 Vrije Universiteit Brussel New Tools for Improving Gene Therapy and Use Thereof
EP4048800A1 (en) * 2019-10-23 2022-08-31 Shire Human Genetic Therapies, Inc. Adeno-associated virus vectors based gene therapy for hereditary angioedema

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