JP2023503850A5 - - Google Patents
Info
- 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
- Prior art keywords
- vector construct
- c1ei
- composition
- functional
- construct according
- 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.)
- Pending
Links
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 (2)
| Publication Number | Publication Date |
|---|---|
| JP2023503850A JP2023503850A (ja) | 2023-02-01 |
| JP2023503850A5 true JP2023503850A5 (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)
| 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 |
| CA3207268A1 (en) * | 2021-01-27 | 2022-08-04 | Spark Therapeutics, Inc. | Compositions and methods for treating hereditary angioedema |
| KR20240054958A (ko) * | 2021-06-17 | 2024-04-26 | 메이라지티엑스 유케이 Ii 리미티드 | Aav 제조 방법 |
| 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 |
Family Cites Families (18)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US4745051A (en) | 1983-05-27 | 1988-05-17 | The Texas A&M University System | Method for producing a recombinant baculovirus expression vector |
| EP0127839B1 (en) | 1983-05-27 | 1992-07-15 | 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 |
| JPWO2003033710A1 (ja) | 2001-10-16 | 2005-02-03 | 独立行政法人産業技術総合研究所 | 新規ｎ−アセチルグルコサミン転移酵素、それをコードする核酸及びそれに対する抗体並びにこれらの癌若しくは腫瘍診断用途 |
| 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 |
| MX359371B (es) | 2001-11-13 | 2018-09-25 | Univ Pennsylvania | Un metodo para detectar y/o identificar secuencias del virus adeno-asociado y aislamiento de secuencias novedosas identificadas de ese modo. |
| AU2003212708A1 (en) | 2002-03-05 | 2003-09-16 | 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 |
| MX367100B (es) | 2012-02-17 | 2019-08-05 | The Children´S Hospital Of Philadelphia | Composiciones del vector aav y metodos para su transferencia genetica a celulas, organos y tejidos. |
| IL297919A (en) * | 2013-07-22 | 2023-01-01 | Childrens Hospital Philadelphia | Variant aav and compositions, methods and uses for gene transfer to cells, organs and tissues |
| EP3044231B1 (en) * | 2013-09-12 | 2020-08-05 | BioMarin Pharmaceutical Inc. | Aav vectors comprising a gene encoding factor viii |
| US10214731B2 (en) * | 2015-05-28 | 2019-02-26 | Cornell University | Adeno-associated virus mediated delivery of C1E1 as a therapy for angioedema |
| IL305149A (en) | 2016-07-26 | 2023-10-01 | Biomarin Pharm Inc | Novel adeno-associated virus capsid proteins |
| AU2019209770B2 (en) * | 2018-01-16 | 2025-07-31 | Cls Therapeutics Limited | Treatment of diseases by liver expression of an enzyme which has a deoxyribonuclease (DNase) activity |
| CN113164560A (zh) * | 2018-10-26 | 2021-07-23 | Vrije布鲁塞尔大学 | 改善基因疗法的新工具及其用途 |
| WO2021081280A1 (en) * | 2019-10-23 | 2021-04-29 | Shire Human Genetic Therapies, Inc. | Adeno-associated virus vectors based gene therapy for hereditary angioedema |
-
2020
- 2020-11-13 JP JP2022527987A patent/JP2023503850A/ja active Pending
- 2020-11-13 AU AU2020384294A patent/AU2020384294A1/en not_active Abandoned
- 2020-11-13 US US17/777,690 patent/US20230340078A1/en not_active Abandoned
- 2020-11-13 TW TW109139830A patent/TW202128733A/zh unknown
- 2020-11-13 CA CA3161154A patent/CA3161154A1/en active Pending
- 2020-11-13 CN CN202080087888.0A patent/CN114829391A/zh active Pending
- 2020-11-13 KR KR1020227019436A patent/KR20220098210A/ko active Pending
- 2020-11-13 WO PCT/US2020/060337 patent/WO2021097157A1/en not_active Ceased
- 2020-11-13 EP EP20824369.1A patent/EP4058475A1/en not_active Withdrawn
- 2020-11-13 BR BR112022009279A patent/BR112022009279A2/pt not_active IP Right Cessation
- 2020-11-13 MX MX2022005869A patent/MX2022005869A/es unknown
-
2022
- 2022-04-29 CO CONC2022/0005641A patent/CO2022005641A2/es unknown
- 2022-05-03 IL IL292717A patent/IL292717A/en unknown
- 2022-05-13 CL CL2022001260A patent/CL2022001260A1/es unknown
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