JP2020522259A5 - - Google Patents
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- Publication number
- JP2020522259A5 JP2020522259A5 JP2019566601A JP2019566601A JP2020522259A5 JP 2020522259 A5 JP2020522259 A5 JP 2020522259A5 JP 2019566601 A JP2019566601 A JP 2019566601A JP 2019566601 A JP2019566601 A JP 2019566601A JP 2020522259 A5 JP2020522259 A5 JP 2020522259A5
- Authority
- JP
- Japan
- Prior art keywords
- polynucleotide
- sequence
- synthetic
- vector
- seq
- 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.)
- Granted
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- 108091033319 polynucleotide Proteins 0.000 claims 34
- 102000040430 polynucleotide Human genes 0.000 claims 34
- 239000002157 polynucleotide Substances 0.000 claims 34
- 239000013598 vector Substances 0.000 claims 10
- 239000002773 nucleotide Substances 0.000 claims 9
- 125000003729 nucleotide group Chemical group 0.000 claims 9
- 102100022641 Coagulation factor IX Human genes 0.000 claims 5
- 108091026898 Leader sequence (mRNA) Proteins 0.000 claims 5
- 108010076282 Factor IX Proteins 0.000 claims 4
- 229960004222 factor ix Drugs 0.000 claims 4
- 230000008488 polyadenylation Effects 0.000 claims 4
- 238000000034 method Methods 0.000 claims 3
- 229920001184 polypeptide Polymers 0.000 claims 3
- 102000004196 processed proteins & peptides Human genes 0.000 claims 3
- 108090000765 processed proteins & peptides Proteins 0.000 claims 3
- 210000004185 liver Anatomy 0.000 claims 2
- 230000035772 mutation Effects 0.000 claims 2
- 102000039446 nucleic acids Human genes 0.000 claims 2
- 108020004707 nucleic acids Proteins 0.000 claims 2
- 150000007523 nucleic acids Chemical class 0.000 claims 2
- FWMNVWWHGCHHJJ-SKKKGAJSSA-N 4-amino-1-[(2r)-6-amino-2-[[(2r)-2-[[(2r)-2-[[(2r)-2-amino-3-phenylpropanoyl]amino]-3-phenylpropanoyl]amino]-4-methylpentanoyl]amino]hexanoyl]piperidine-4-carboxylic acid Chemical compound C([C@H](C(=O)N[C@H](CC(C)C)C(=O)N[C@H](CCCCN)C(=O)N1CCC(N)(CC1)C(O)=O)NC(=O)[C@H](N)CC=1C=CC=CC=1)C1=CC=CC=C1 FWMNVWWHGCHHJJ-SKKKGAJSSA-N 0.000 claims 1
- 108010039209 Blood Coagulation Factors Proteins 0.000 claims 1
- 102000015081 Blood Coagulation Factors Human genes 0.000 claims 1
- 108091026890 Coding region Proteins 0.000 claims 1
- 241000702421 Dependoparvovirus Species 0.000 claims 1
- 208000032182 acquired hemophilia B Diseases 0.000 claims 1
- 230000002457 bidirectional effect Effects 0.000 claims 1
- 239000003114 blood coagulation factor Substances 0.000 claims 1
- 208000009429 hemophilia B Diseases 0.000 claims 1
- 229960000027 human factor ix Drugs 0.000 claims 1
- 230000009466 transformation Effects 0.000 claims 1
- 239000013603 viral vector Substances 0.000 claims 1
Priority Applications (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2022123665A JP2022159358A (ja) | 2017-05-31 | 2022-08-03 | 最適化されたヒト凝固第ｉｘ因子遺伝子発現カセットおよびそれらの使用 |
| JP2024076788A JP7869828B2 (ja) | 2017-05-31 | 2024-05-09 | 最適化されたヒト凝固第ｉｘ因子遺伝子発現カセットおよびそれらの使用 |
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201762512833P | 2017-05-31 | 2017-05-31 | |
| US62/512,833 | 2017-05-31 | ||
| PCT/US2018/035267 WO2018222792A1 (en) | 2017-05-31 | 2018-05-31 | Optimized human clotting factor ix gene expression cassettes and their use |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022123665A Division JP2022159358A (ja) | 2017-05-31 | 2022-08-03 | 最適化されたヒト凝固第ｉｘ因子遺伝子発現カセットおよびそれらの使用 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2020522259A JP2020522259A (ja) | 2020-07-30 |
| JP2020522259A5 true JP2020522259A5 (enExample) | 2021-07-26 |
| JP7119009B2 JP7119009B2 (ja) | 2022-08-16 |
Family
ID=64456331
Family Applications (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2019566601A Active JP7119009B2 (ja) | 2017-05-31 | 2018-05-31 | 最適化されたヒト凝固第ｉｘ因子遺伝子発現カセットおよびそれらの使用 |
| JP2022123665A Pending JP2022159358A (ja) | 2017-05-31 | 2022-08-03 | 最適化されたヒト凝固第ｉｘ因子遺伝子発現カセットおよびそれらの使用 |
| JP2024076788A Active JP7869828B2 (ja) | 2017-05-31 | 2024-05-09 | 最適化されたヒト凝固第ｉｘ因子遺伝子発現カセットおよびそれらの使用 |
Family Applications After (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022123665A Pending JP2022159358A (ja) | 2017-05-31 | 2022-08-03 | 最適化されたヒト凝固第ｉｘ因子遺伝子発現カセットおよびそれらの使用 |
| JP2024076788A Active JP7869828B2 (ja) | 2017-05-31 | 2024-05-09 | 最適化されたヒト凝固第ｉｘ因子遺伝子発現カセットおよびそれらの使用 |
Country Status (9)
| Country | Link |
|---|---|
| US (2) | US11530402B2 (enExample) |
| EP (1) | EP3630978A4 (enExample) |
| JP (3) | JP7119009B2 (enExample) |
| KR (2) | KR20240010098A (enExample) |
| CN (2) | CN114875051A (enExample) |
| AU (1) | AU2018275549B2 (enExample) |
| CA (1) | CA3064450A1 (enExample) |
| MX (2) | MX2019014379A (enExample) |
| WO (1) | WO2018222792A1 (enExample) |
Families Citing this family (18)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| RS61387B2 (sr) | 2013-02-15 | 2024-06-28 | Bioverativ Therapeutics Inc | Gen optimizovanog faktora viii |
| US11008561B2 (en) | 2014-06-30 | 2021-05-18 | Bioverativ Therapeutics Inc. | Optimized factor IX gene |
| GB201420139D0 (en) | 2014-11-12 | 2014-12-24 | Ucl Business Plc | Factor IX gene therapy |
| US10842885B2 (en) | 2018-08-20 | 2020-11-24 | Ucl Business Ltd | Factor IX encoding nucleotides |
| GB201813528D0 (en) | 2018-08-20 | 2018-10-03 | Ucl Business Plc | Factor IX encoding nucleotides |
| JP7803014B2 (ja) * | 2019-09-30 | 2026-01-21 | バイオベラティブ セラピューティクス インコーポレイテッド | レンチウイルスベクター製剤 |
| US20220411821A1 (en) * | 2019-10-28 | 2022-12-29 | University Of Florida Research Foundation, Incorporated | Gene therapy vectors |
| WO2021207077A1 (en) * | 2020-04-06 | 2021-10-14 | Homology Medicines, Inc. | Adeno-associated virus compositions for ids gene transfer and methods of use thereof |
| CN113817759B (zh) * | 2020-07-10 | 2023-06-02 | 南京吉迈生物技术有限公司 | 修饰的因子ix、组合物、方法及其在基因治疗中的应用 |
| CN115109788A (zh) * | 2021-03-23 | 2022-09-27 | 北京锦篮基因科技有限公司 | Aav介导的人脂蛋白脂酶肝脏异位表达载体及其用途 |
| BR112023026176A2 (pt) * | 2021-06-16 | 2024-03-05 | Mediphage Bioceuticals Inc | Vetores de expressão, vetores livres de sequência bacteriana e métodos de fabricação e uso dos mesmos |
| AU2022298827A1 (en) * | 2021-06-23 | 2024-02-08 | Frontera Therapeutics, Inc. | Composition and method for treating hemophilia b |
| CN114277057B (zh) * | 2021-07-09 | 2023-10-13 | 上海天泽云泰生物医药有限公司 | 用于治疗或预防b型血友病的重组腺相关病毒载体和方法 |
| KR20240099358A (ko) * | 2021-10-27 | 2024-06-28 | 리제너론 파마슈티칼스 인코포레이티드 | B형 혈우병 치료를 위한 인자 ix를 발현하기 위한 조성물 및 방법 |
| CN118460618A (zh) * | 2022-04-19 | 2024-08-09 | 康霖生物科技（杭州）有限公司 | 一种用于遗传性凝血因子缺乏病治疗的核酸构建体 |
| CN115029360B (zh) * | 2022-05-30 | 2024-08-02 | 上海勉亦生物科技有限公司 | 用于治疗粘多糖贮积症iiia型的转基因表达盒 |
| CN115948408A (zh) * | 2022-09-23 | 2023-04-11 | 上海信致医药科技有限公司 | 改进的人凝血因子viii基因表达盒及其应用 |
| WO2025143742A1 (ko) * | 2023-12-26 | 2025-07-03 | 삼성바이오에피스 주식회사 | 혈액응고인자 IX를 암호화하는 코돈-최적화된 폴리뉴클레오티드 및 이를 포함하는 rAAV 생산용 플라스미드 |
Family Cites Families (19)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| FR2650838A1 (fr) * | 1989-08-09 | 1991-02-15 | Transgene Sa | Vecteurs d'expression du facteur ix dans une cellule eucaryote superieure, procede de preparation de facteur ix par des animaux transgeniques et facteur ix obtenu |
| WO1994029471A1 (en) * | 1993-06-10 | 1994-12-22 | Genetic Therapy, Inc. | Adenoviral vectors for treatment of hemophilia |
| ES2436741T3 (es) | 2004-09-22 | 2014-01-07 | St. Jude Children's Research Hospital | Expresión mejorada de Factor IX en vectores de terapia génica |
| US7943374B2 (en) * | 2005-08-21 | 2011-05-17 | Markus Hildinger | Super-size adeno-associated viral vector harboring a recombinant genome larger than 5.7 kb |
| WO2007078599A2 (en) * | 2005-12-16 | 2007-07-12 | The Board Of Trustees Of The Leland Stanford Junior University | Functional arrays for high throughput characterization of gene expression regulatory elements |
| CN101511373B (zh) * | 2006-06-19 | 2015-12-09 | 阿斯克肋匹奥生物制药公司 | 用于基因治疗的修饰的因子ⅷ和因子ⅸ基因和载体 |
| CN102628041B (zh) * | 2008-02-14 | 2013-09-11 | 财团法人牧岩生命工学研究所 | 适于表达用于基因治疗的编码序列的表达载体 |
| US9617548B2 (en) * | 2008-04-22 | 2017-04-11 | Vib Vzw | Liver-specific nucleic acid regulatory elements and methods and use thereof |
| US9700486B2 (en) * | 2013-04-24 | 2017-07-11 | Corning Incorporated | Delamination resistant pharmaceutical glass containers containing active pharmaceutical ingredients |
| RU2585532C2 (ru) * | 2014-01-31 | 2016-05-27 | Федеральное государственное учреждение "Федеральный исследовательский центр "Фундаментальные основы биотехнологии" Российской академии наук"(ФИЦ Биотехнологии РАН) | Плазмида для экспрессии рекомбинантного фактора свёртываемости крови ix человека, клетка сно - продуцент рекомбинантного фактора свёртываемости крови ix человека и способ получения указанного фактора |
| BR112016020688B1 (pt) | 2014-03-09 | 2024-01-30 | The Trustees Of The University Of Pennsylvania | Vetores virais recombinantes, vírus adenoassociado recombinante, composição farmacêutica, bem como uso dos mesmos |
| KR102244434B1 (ko) * | 2014-08-11 | 2021-04-23 | 삼성전자주식회사 | 재조합 벡터 및 이를 이용한 목적 폴리펩타이드의 생산 방법 |
| GB201420139D0 (en) * | 2014-11-12 | 2014-12-24 | Ucl Business Plc | Factor IX gene therapy |
| KR102554850B1 (ko) * | 2015-02-06 | 2023-07-13 | 더 유니버시티 오브 노쓰 캐롤라이나 엣 채플 힐 | 최적화된 인간 응고 인자 viii 유전자 발현 카세트 및 그의 용도 |
| EP4378487A3 (en) * | 2015-06-23 | 2024-09-04 | The Children's Hospital of Philadelphia | Modified factor ix, and compositions, methods and uses for gene transfer to cells, organs and tissues |
| WO2017049132A1 (en) * | 2015-09-18 | 2017-03-23 | DNARx | Systems and methods for nucleic acid expression in vivo |
| US11191847B2 (en) * | 2016-04-15 | 2021-12-07 | The Trustees Of The University Of Pennsylvania | Gene therapy for treating hemophilia B |
| CN116732066A (zh) * | 2016-10-14 | 2023-09-12 | 四川至善唯新生物科技有限公司 | 一种高活性凝血因子ix突变体、重组蛋白与融合蛋白的制备与应用 |
| EP3600428A4 (en) * | 2017-03-23 | 2020-08-26 | Dnarx | SYSTEMS AND METHODS FOR THE EXPRESSION OF NUCLEIC ACIDS IN VIVO |
-
2018
- 2018-05-31 JP JP2019566601A patent/JP7119009B2/ja active Active
- 2018-05-31 US US16/616,784 patent/US11530402B2/en active Active
- 2018-05-31 KR KR1020247001130A patent/KR20240010098A/ko not_active Ceased
- 2018-05-31 AU AU2018275549A patent/AU2018275549B2/en active Active
- 2018-05-31 WO PCT/US2018/035267 patent/WO2018222792A1/en not_active Ceased
- 2018-05-31 CA CA3064450A patent/CA3064450A1/en active Pending
- 2018-05-31 CN CN202210750672.9A patent/CN114875051A/zh active Pending
- 2018-05-31 CN CN201810547030.2A patent/CN108977452B/zh active Active
- 2018-05-31 EP EP18809332.2A patent/EP3630978A4/en active Pending
- 2018-05-31 MX MX2019014379A patent/MX2019014379A/es unknown
- 2018-05-31 KR KR1020197036397A patent/KR102625470B1/ko active Active
-
2019
- 2019-11-29 MX MX2024007348A patent/MX2024007348A/es unknown
-
2022
- 2022-08-03 JP JP2022123665A patent/JP2022159358A/ja active Pending
- 2022-11-17 US US18/056,424 patent/US12209263B2/en active Active
-
2024
- 2024-05-09 JP JP2024076788A patent/JP7869828B2/ja active Active
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