JP2024540085A5 - - Google Patents
Info
- Publication number
- JP2024540085A5 JP2024540085A5 JP2024525344A JP2024525344A JP2024540085A5 JP 2024540085 A5 JP2024540085 A5 JP 2024540085A5 JP 2024525344 A JP2024525344 A JP 2024525344A JP 2024525344 A JP2024525344 A JP 2024525344A JP 2024540085 A5 JP2024540085 A5 JP 2024540085A5
- Authority
- JP
- Japan
- Prior art keywords
- factor
- item
- coding sequence
- protein
- protein coding
- 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 (7)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202163272324P | 2021-10-27 | 2021-10-27 | |
| US63/272,324 | 2021-10-27 | ||
| US202263306037P | 2022-02-02 | 2022-02-02 | |
| US63/306,037 | 2022-02-02 | ||
| US202263369864P | 2022-07-29 | 2022-07-29 | |
| US63/369,864 | 2022-07-29 | ||
| PCT/US2022/078798 WO2023077012A1 (en) | 2021-10-27 | 2022-10-27 | Compositions and methods for expressing factor ix for hemophilia b therapy |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2024540085A JP2024540085A (ja) | 2024-10-31 |
| JP2024540085A5 true JP2024540085A5 (enExample) | 2025-10-30 |
Family
ID=84439883
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2024525344A Pending JP2024540085A (ja) | 2021-10-27 | 2022-10-27 | 血友病ｂ療法のための第ｉｘ因子を発現させるための組成物及び方法 |
Country Status (12)
| Country | Link |
|---|---|
| US (1) | US20230149563A1 (enExample) |
| EP (1) | EP4423119A1 (enExample) |
| JP (1) | JP2024540085A (enExample) |
| KR (1) | KR20240099358A (enExample) |
| AU (1) | AU2022379633A1 (enExample) |
| CA (1) | CA3232470A1 (enExample) |
| CL (1) | CL2024001251A1 (enExample) |
| CO (1) | CO2024005281A2 (enExample) |
| IL (1) | IL312000A (enExample) |
| MX (1) | MX2024005124A (enExample) |
| TW (1) | TW202334194A (enExample) |
| WO (1) | WO2023077012A1 (enExample) |
Families Citing this family (4)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN121693566A (zh) * | 2023-07-28 | 2026-03-17 | 瑞泽恩制药公司 | 使用bGH-SV40L串联PolyA在单向基因插入期间增强转基因表达 |
| WO2025160324A2 (en) | 2024-01-26 | 2025-07-31 | Regeneron Pharmaceuticals, Inc. | Methods and compositions for using plasma cell depleting agents and/or b cell depleting agents to suppress host anti-aav antibody response and enable aav transduction and re-dosing |
| US20250276092A1 (en) | 2024-03-01 | 2025-09-04 | Regeneron Pharmaceuticals, Inc. | Methods and compositions for re-dosing aav using anti-cd40 antagonistic antibody to suppress host anti-aav antibody response |
| WO2025265017A1 (en) * | 2024-06-20 | 2025-12-26 | Regeneron Pharmaceuticals, Inc. | Ass1 gene insertion for the treatment of citrullinemia type i |
Family Cites Families (32)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2003087341A2 (en) | 2002-01-23 | 2003-10-23 | The University Of Utah Research Foundation | Targeted chromosomal mutagenesis using zinc finger nucleases |
| WO2003080809A2 (en) | 2002-03-21 | 2003-10-02 | Sangamo Biosciences, Inc. | Methods and compositions for using zinc finger endonucleases to enhance homologous recombination |
| EP2806025B1 (en) | 2002-09-05 | 2019-04-03 | California Institute of Technology | Use of zinc finger nucleases to stimulate gene targeting |
| US8409861B2 (en) | 2003-08-08 | 2013-04-02 | Sangamo Biosciences, Inc. | Targeted deletion of cellular DNA sequences |
| US7888121B2 (en) | 2003-08-08 | 2011-02-15 | Sangamo Biosciences, Inc. | Methods and compositions for targeted cleavage and recombination |
| US7972854B2 (en) | 2004-02-05 | 2011-07-05 | Sangamo Biosciences, Inc. | Methods and compositions for targeted cleavage and recombination |
| AU2005287278B2 (en) | 2004-09-16 | 2011-08-04 | Sangamo Biosciences, Inc. | Compositions and methods for protein production |
| EP2027262B1 (en) | 2006-05-25 | 2010-03-31 | Sangamo Biosciences Inc. | Variant foki cleavage half-domains |
| ES2465996T3 (es) | 2006-05-25 | 2014-06-09 | Sangamo Biosciences, Inc. | Métodos y composiciones para la inactivación genética |
| US8022187B2 (en) | 2007-02-01 | 2011-09-20 | Baxter International Inc. | FVIII-independent FIX-mutant proteins for hemophilia A treatment |
| AU2008209985A1 (en) | 2007-02-01 | 2008-08-07 | Baxter Healthcare S.A. | Improved fix-mutant proteins for hemophilia B treatment |
| DE602008003684D1 (de) | 2007-04-26 | 2011-01-05 | Sangamo Biosciences Inc | Gezielte integration in die ppp1r12c-position |
| CA2720903C (en) | 2008-04-14 | 2019-01-15 | Sangamo Biosciences, Inc. | Linear donor constructs for targeted integration |
| EP2149603A1 (en) | 2008-07-28 | 2010-02-03 | DRK-Blutspendedienst Baden-Württemberg-Hessen gGmbH | Factor IX variants with clotting activity in absence of their cofactor and their use for treating bleeding disorders |
| SG191561A1 (en) | 2008-08-22 | 2013-07-31 | Sangamo Biosciences Inc | Methods and compositions for targeted single-stranded cleavage and targeted integration |
| US8586526B2 (en) | 2010-05-17 | 2013-11-19 | Sangamo Biosciences, Inc. | DNA-binding proteins and uses thereof |
| EP2660318A1 (en) | 2010-02-09 | 2013-11-06 | Sangamo BioSciences, Inc. | Targeted genomic modification with partially single-stranded donor molecules |
| US9567573B2 (en) | 2010-04-26 | 2017-02-14 | Sangamo Biosciences, Inc. | Genome editing of a Rosa locus using nucleases |
| CA2848417C (en) | 2011-09-21 | 2023-05-02 | Sangamo Biosciences, Inc. | Methods and compositions for regulation of transgene expression |
| CA3099582A1 (en) | 2011-10-27 | 2013-05-02 | Sangamo Biosciences, Inc. | Methods and compositions for modification of the hprt locus |
| AU2013266968B2 (en) | 2012-05-25 | 2017-06-29 | Emmanuelle CHARPENTIER | Methods and compositions for RNA-directed target DNA modification and for RNA-directed modulation of transcription |
| EP2881463A1 (en) | 2013-12-09 | 2015-06-10 | DRK-Blutspendedienst Baden-Württemberg-Hessen gGmbH | Factor IX variants with clotting activity in absence of their cofactor and/or with increased F.IX clotting activity and their use for treating bleeding disorders |
| CN105940013B (zh) * | 2013-12-09 | 2020-03-27 | 桑格摩生物科学股份有限公司 | 用于治疗血友病的方法和组合物 |
| MX390848B (es) * | 2015-06-23 | 2025-03-21 | Childrens Hospital Philadelphia | Factor ix modificado, y composiciones, metodos y usos para la transferencia de genes a celulas, organos y tejidos. |
| CN110945127B (zh) * | 2017-05-22 | 2024-07-12 | 武田药品工业株式会社 | 用于血友病b的基因疗法的编码具有增加的表达的重组fix的病毒载体 |
| JP7119009B2 (ja) * | 2017-05-31 | 2022-08-16 | ザ ユニバーシティ オブ ノース カロライナ アット チャペル ヒル | 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 |
| JP2021533799A (ja) * | 2018-08-20 | 2021-12-09 | ユーシーエル ビジネス リミテッド | 第ｉｘ因子をコードするヌクレオチド |
| US20200270617A1 (en) | 2018-10-18 | 2020-08-27 | Intellia Therapeutics, Inc. | Compositions and methods for transgene expression from an albumin locus |
| CN113272428A (zh) | 2018-10-18 | 2021-08-17 | 英特利亚治疗股份有限公司 | 核酸构建体和使用方法 |
| AU2019360270B2 (en) | 2018-10-18 | 2025-08-07 | Intellia Therapeutics, Inc. | Compositions and methods for expressing factor IX. |
| US11596671B2 (en) * | 2019-02-01 | 2023-03-07 | Takeda Pharmaceutical Company Limited | Gene therapy of hemophilia B using viral vectors encoding recombinant fix variants with increased expression |
| AU2020242945B2 (en) * | 2019-03-19 | 2025-06-26 | CSL Innovation Pty Ltd | Factor IX variants and uses thereof in therapy |
-
2022
- 2022-10-27 US US18/050,161 patent/US20230149563A1/en active Pending
- 2022-10-27 JP JP2024525344A patent/JP2024540085A/ja active Pending
- 2022-10-27 MX MX2024005124A patent/MX2024005124A/es unknown
- 2022-10-27 AU AU2022379633A patent/AU2022379633A1/en active Pending
- 2022-10-27 WO PCT/US2022/078798 patent/WO2023077012A1/en not_active Ceased
- 2022-10-27 KR KR1020247017650A patent/KR20240099358A/ko active Pending
- 2022-10-27 EP EP22818964.3A patent/EP4423119A1/en active Pending
- 2022-10-27 CA CA3232470A patent/CA3232470A1/en active Pending
- 2022-10-27 TW TW111140939A patent/TW202334194A/zh unknown
- 2022-10-27 IL IL312000A patent/IL312000A/en unknown
-
2024
- 2024-04-22 CL CL2024001251A patent/CL2024001251A1/es unknown
- 2024-04-25 CO CONC2024/0005281A patent/CO2024005281A2/es unknown
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