JPWO2021097157A5 - - Google Patents
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- Publication number
- JPWO2021097157A5 JPWO2021097157A5 JP2022527987A JP2022527987A JPWO2021097157A5 JP WO2021097157 A5 JPWO2021097157 A5 JP WO2021097157A5 JP 2022527987 A JP2022527987 A JP 2022527987A JP 2022527987 A JP2022527987 A JP 2022527987A JP WO2021097157 A5 JPWO2021097157 A5 JP WO2021097157A5
- Authority
- JP
- Japan
- Prior art keywords
- vector construct
- c1ei
- composition
- functional
- mammal
- 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
- 239000013598 vector Substances 0.000 claims description 57
- 239000002245 particle Substances 0.000 claims description 46
- 229940009550 c1 esterase inhibitor Drugs 0.000 claims description 40
- 102000055157 Complement C1 Inhibitor Human genes 0.000 claims description 36
- 108050007539 Plasma protease C1 inhibitor Proteins 0.000 claims description 36
- 238000000034 method Methods 0.000 claims description 30
- 241000124008 Mammalia Species 0.000 claims description 26
- 241000702421 Dependoparvovirus Species 0.000 claims description 25
- 210000004185 liver Anatomy 0.000 claims description 18
- 210000004027 cell Anatomy 0.000 claims description 16
- 210000000234 capsid Anatomy 0.000 claims description 12
- 239000008194 pharmaceutical composition Substances 0.000 claims description 12
- 230000001105 regulatory effect Effects 0.000 claims description 12
- 230000002103 transcriptional effect Effects 0.000 claims description 12
- 150000007523 nucleic acids Chemical group 0.000 claims description 10
- 239000002773 nucleotide Substances 0.000 claims description 10
- 125000003729 nucleotide group Chemical group 0.000 claims description 10
- 108091028043 Nucleic acid sequence Proteins 0.000 claims description 8
- 239000003623 enhancer Substances 0.000 claims description 8
- 102000013918 Apolipoproteins E Human genes 0.000 claims description 6
- 108010025628 Apolipoproteins E Proteins 0.000 claims description 6
- 108090000565 Capsid Proteins Proteins 0.000 claims description 6
- 102100023321 Ceruloplasmin Human genes 0.000 claims description 6
- 206010019860 Hereditary angioedema Diseases 0.000 claims description 6
- 239000008280 blood Substances 0.000 claims description 6
- 210000004369 blood Anatomy 0.000 claims description 6
- 230000008488 polyadenylation Effects 0.000 claims description 6
- 102000004169 proteins and genes Human genes 0.000 claims description 6
- 108090000623 proteins and genes Proteins 0.000 claims description 6
- 239000002753 trypsin inhibitor Substances 0.000 claims description 6
- 108700028146 Genetic Enhancer Elements Proteins 0.000 claims description 4
- 125000003275 alpha amino acid group Chemical group 0.000 claims description 4
- 150000001413 amino acids Chemical class 0.000 claims description 4
- 230000007812 deficiency Effects 0.000 claims description 4
- 206010030113 Oedema Diseases 0.000 claims description 3
- 208000024891 symptom Diseases 0.000 claims description 3
- 241000702423 Adeno-associated virus - 2 Species 0.000 claims description 2
- 241000282693 Cercopithecidae Species 0.000 claims description 2
- 102000001554 Hemoglobins Human genes 0.000 claims description 2
- 108010054147 Hemoglobins Proteins 0.000 claims description 2
- 241000238631 Hexapoda Species 0.000 claims description 2
- 102000002265 Human Growth Hormone Human genes 0.000 claims description 2
- 108010000521 Human Growth Hormone Proteins 0.000 claims description 2
- 239000000854 Human Growth Hormone Substances 0.000 claims description 2
- 241001504519 Papio ursinus Species 0.000 claims description 2
- 108700029229 Transcriptional Regulatory Elements Proteins 0.000 claims description 2
- 239000007900 aqueous suspension Substances 0.000 claims description 2
- 230000036765 blood level Effects 0.000 claims description 2
- 239000002131 composite material Substances 0.000 claims description 2
- 239000003246 corticosteroid Substances 0.000 claims description 2
- 238000012258 culturing Methods 0.000 claims description 2
- 239000012634 fragment Substances 0.000 claims description 2
- 210000004962 mammalian cell Anatomy 0.000 claims description 2
- 108020004707 nucleic acids Proteins 0.000 claims description 2
- 102000039446 nucleic acids Human genes 0.000 claims description 2
- 239000000546 pharmaceutical excipient Substances 0.000 claims description 2
- 239000013608 rAAV vector Substances 0.000 claims description 2
- 230000010076 replication Effects 0.000 claims description 2
- 238000007920 subcutaneous administration Methods 0.000 claims description 2
- 238000002560 therapeutic procedure Methods 0.000 claims description 2
- 230000010415 tropism Effects 0.000 claims description 2
- 239000000203 mixture Substances 0.000 claims 20
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 JP2023503850A5 (https=) | 2023-11-20 |
| JPWO2021097157A5 true 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)
| 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)
| 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 |
-
2020
- 2020-11-13 CN CN202080087888.0A patent/CN114829391A/zh active Pending
- 2020-11-13 WO PCT/US2020/060337 patent/WO2021097157A1/en not_active Ceased
- 2020-11-13 MX MX2022005869A patent/MX2022005869A/es unknown
- 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 JP JP2022527987A patent/JP2023503850A/ja active Pending
- 2020-11-13 BR BR112022009279A patent/BR112022009279A2/pt not_active IP Right Cessation
- 2020-11-13 CA CA3161154A patent/CA3161154A1/en active Pending
- 2020-11-13 EP EP20824369.1A patent/EP4058475A1/en not_active Withdrawn
- 2020-11-13 KR KR1020227019436A patent/KR20220098210A/ko active Pending
- 2020-11-13 AU AU2020384294A patent/AU2020384294A1/en not_active Abandoned
-
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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