JP2021505159A5 - - Google Patents
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- JP2021505159A5 JP2021505159A5 JP2020531057A JP2020531057A JP2021505159A5 JP 2021505159 A5 JP2021505159 A5 JP 2021505159A5 JP 2020531057 A JP2020531057 A JP 2020531057A JP 2020531057 A JP2020531057 A JP 2020531057A JP 2021505159 A5 JP2021505159 A5 JP 2021505159A5
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- Prior art keywords
- item
- cedna
- nuclease
- gene
- sequence
- Prior art date
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- 238000000034 method Methods 0.000 claims description 148
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- ISAKRJDGNUQOIC-UHFFFAOYSA-N Uracil Chemical compound O=C1C=CNC(=O)N1 ISAKRJDGNUQOIC-UHFFFAOYSA-N 0.000 description 2
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- 101710169336 5'-deoxyadenosine deaminase Proteins 0.000 description 1
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- 102100026735 Coagulation factor VIII Human genes 0.000 description 1
- 102100026846 Cytidine deaminase Human genes 0.000 description 1
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- 102000004190 Enzymes Human genes 0.000 description 1
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- 201000003542 Factor VIII deficiency Diseases 0.000 description 1
- 229940113491 Glycosylase inhibitor Drugs 0.000 description 1
- 208000009292 Hemophilia A Diseases 0.000 description 1
- 208000028782 Hereditary disease Diseases 0.000 description 1
- 101000911390 Homo sapiens Coagulation factor VIII Proteins 0.000 description 1
- 102000018697 Membrane Proteins Human genes 0.000 description 1
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- 108091058545 Secretory proteins Proteins 0.000 description 1
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Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2023195038A JP2024003220A (ja) | 2017-12-06 | 2023-11-16 | 修飾型閉端ｄｎａ（ｃｅｄｎａ）を使用する遺伝子編集 |
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201762595328P | 2017-12-06 | 2017-12-06 | |
| US62/595,328 | 2017-12-06 | ||
| US201762607069P | 2017-12-18 | 2017-12-18 | |
| US62/607,069 | 2017-12-18 | ||
| PCT/US2018/064242 WO2019113310A1 (en) | 2017-12-06 | 2018-12-06 | Gene editing using a modified closed-ended dna (cedna) |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2023195038A Division JP2024003220A (ja) | 2017-12-06 | 2023-11-16 | 修飾型閉端ｄｎａ（ｃｅｄｎａ）を使用する遺伝子編集 |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2021505159A JP2021505159A (ja) | 2021-02-18 |
| JP2021505159A5 true JP2021505159A5 (enExample) | 2022-01-11 |
Family
ID=66751200
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2020531057A Pending JP2021505159A (ja) | 2017-12-06 | 2018-12-06 | 修飾型閉端ｄｎａ（ｃｅｄｎａ）を使用する遺伝子編集 |
| JP2023195038A Pending JP2024003220A (ja) | 2017-12-06 | 2023-11-16 | 修飾型閉端ｄｎａ（ｃｅｄｎａ）を使用する遺伝子編集 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2023195038A Pending JP2024003220A (ja) | 2017-12-06 | 2023-11-16 | 修飾型閉端ｄｎａ（ｃｅｄｎａ）を使用する遺伝子編集 |
Country Status (14)
| Country | Link |
|---|---|
| US (1) | US20220290186A1 (enExample) |
| EP (1) | EP3720952A4 (enExample) |
| JP (2) | JP2021505159A (enExample) |
| KR (1) | KR20200093635A (enExample) |
| CN (1) | CN111527200A (enExample) |
| AU (1) | AU2018378672A1 (enExample) |
| BR (1) | BR112020009858A2 (enExample) |
| CA (1) | CA3084185A1 (enExample) |
| IL (1) | IL274845A (enExample) |
| MA (1) | MA51113A (enExample) |
| MX (1) | MX2020005808A (enExample) |
| PH (1) | PH12020550771A1 (enExample) |
| SG (2) | SG11202005281XA (enExample) |
| WO (1) | WO2019113310A1 (enExample) |
Families Citing this family (43)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US10704021B2 (en) | 2012-03-15 | 2020-07-07 | Flodesign Sonics, Inc. | Acoustic perfusion devices |
| WO2015105955A1 (en) | 2014-01-08 | 2015-07-16 | Flodesign Sonics, Inc. | Acoustophoresis device with dual acoustophoretic chamber |
| US11708572B2 (en) | 2015-04-29 | 2023-07-25 | Flodesign Sonics, Inc. | Acoustic cell separation techniques and processes |
| US11377651B2 (en) | 2016-10-19 | 2022-07-05 | Flodesign Sonics, Inc. | Cell therapy processes utilizing acoustophoresis |
| NZ762100A (en) | 2017-08-09 | 2025-12-19 | Bioverativ Therapeutics Inc | Nucleic acid molecules and uses thereof |
| EP3690046A2 (en) * | 2017-09-28 | 2020-08-05 | Toolgen Incorporated | Composition for treatment of hemophilia, comprising crispr/cas system having coagulation factor viii gene inversion correction potential |
| KR20200120649A (ko) * | 2018-02-14 | 2020-10-21 | 제너레이션 바이오 컴퍼니 | 비-바이러스 dna 벡터 및 항체 및 융합 단백질 생산을 위한 이의 용도 |
| CN109022389A (zh) * | 2018-07-19 | 2018-12-18 | 陕西慧康生物科技有限责任公司 | 一种大肠杆菌表达人艾杜糖醛酸-2-硫酸酯酶的生产方法 |
| EP3833766A1 (en) | 2018-08-09 | 2021-06-16 | Bioverativ Therapeutics Inc. | Nucleic acid molecules and uses thereof for non-viral gene therapy |
| WO2020097417A1 (en) * | 2018-11-09 | 2020-05-14 | Generation Bio Co. | Modified closed-ended dna (cedna) comprising symmetrical modified inverted terminal repeats |
| CN113939595A (zh) | 2019-06-07 | 2022-01-14 | 瑞泽恩制药公司 | 包括人源化白蛋白基因座的非人动物 |
| EP4022074A4 (en) * | 2019-08-27 | 2023-11-15 | The Trustees of Columbia University in the City of New York | Engineered exosomes for targeted delivery |
| WO2021042045A1 (en) * | 2019-08-30 | 2021-03-04 | The Trustees Of Columbia University In The City Of New York | Methods for scalable gene insertions |
| EP4054651A1 (en) | 2019-11-08 | 2022-09-14 | Regeneron Pharmaceuticals, Inc. | Crispr and aav strategies for x-linked juvenile retinoschisis therapy |
| BR112022017715A2 (pt) | 2020-03-04 | 2022-11-16 | Flagship Pioneering Innovations Vi Llc | Métodos e composições para modular um genoma |
| DE102020111571A1 (de) | 2020-03-11 | 2021-09-16 | Immatics US, Inc. | Wpre-mutantenkonstrukte, zusammensetzungen und zugehörige verfahren |
| MX2022011805A (es) * | 2020-03-24 | 2023-01-11 | Generation Bio Co | Vectores de adn no virales y usos de estos para expresar agentes terapéuticos con factor ix. |
| CN116033915A (zh) * | 2020-05-13 | 2023-04-28 | 利索基因公司 | 用于治疗gm1神经节苷脂贮积症及其它病症的组合物和方法 |
| US20230190893A1 (en) * | 2020-07-14 | 2023-06-22 | The Regents Of The University Of California | Compositions and methods for treating an inherited retinal disease |
| JP2023535632A (ja) | 2020-07-27 | 2023-08-18 | アンジャリウム バイオサイエンシズ エージー | Ｄｎａ分子の組成物、その作製方法、及びその使用方法 |
| WO2022061014A1 (en) * | 2020-09-16 | 2022-03-24 | Generation Bio Co. | Non-viral dna vectors and uses thereof for expressing fviii therapeutics |
| JP2023542131A (ja) * | 2020-09-16 | 2023-10-05 | ジェネレーション バイオ カンパニー | フェニルアラニンヒドロキシラーゼ（ｐａｈ）を発現させるための閉端ｄｎａベクター及びその使用 |
| US20250270545A1 (en) * | 2020-09-29 | 2025-08-28 | Albert-Ludwigs-Universitat Freiburg | Rescue of recombinant adenoviruses by crispr/cas-mediated in vivo terminal resolution |
| CN112481262B (zh) * | 2020-12-04 | 2022-08-19 | 中国农业科学院农业基因组研究所 | 一种基于CRISPR/Cas9基因编辑技术分析增强子细胞生物学功能的方法 |
| CN112852880B (zh) * | 2021-01-28 | 2022-12-06 | 中吉智药(南京)生物技术有限公司 | 一种基于诱导型昆虫细胞生产aav基因药物的方法 |
| AU2022216614A1 (en) | 2021-02-05 | 2023-02-23 | Christiana Care Gene Editing Institute, Inc. | Methods of and compositions for reducing gene expression and/or activity |
| AU2022237643A1 (en) * | 2021-03-19 | 2023-09-28 | Generation Bio Co. | Non-viral dna vectors and uses thereof for expressing pfic therapeutics |
| EP4330416A4 (en) * | 2021-04-26 | 2025-12-03 | Univ Massachusetts | RAAV-mediated direct in vivo gene editing of hematopoietic stem cells |
| EP4337177A1 (en) * | 2021-05-11 | 2024-03-20 | Modernatx, Inc. | Non-viral delivery of dna for prolonged polypeptide expression in vivo |
| CN117729926A (zh) * | 2021-05-28 | 2024-03-19 | 比姆医疗股份有限公司 | 用于使碱基编辑器自失活的组合物和方法 |
| US20240307557A1 (en) * | 2021-07-09 | 2024-09-19 | The Board Of Trustees Of The University Of Illinois | Therapeutic gene silencing with crispr-cas13 |
| AU2022332276A1 (en) * | 2021-08-23 | 2024-04-04 | Bioverativ Therapeutics Inc. | Optimized factor viii genes |
| IL311225A (en) | 2021-09-08 | 2024-05-01 | Flagship Pioneering Innovations Vi Llc | Methods and compositions for genome modulation |
| IL312184A (en) | 2021-10-18 | 2024-06-01 | Flagship Pioneering Innovations Vii Llc | Dna compositions and related methods |
| CN118541475A (zh) * | 2021-12-27 | 2024-08-23 | 亘喜生物科技（上海）有限公司 | 用于细胞修饰的系统和方法 |
| WO2024040222A1 (en) | 2022-08-19 | 2024-02-22 | Generation Bio Co. | Cleavable closed-ended dna (cedna) and methods of use thereof |
| EP4587454A2 (en) * | 2022-09-16 | 2025-07-23 | Joseph Fenton Lawler | Trans-splicing methods and compositions for generation of single sex offspring |
| WO2024229153A2 (en) * | 2023-05-01 | 2024-11-07 | Seattle Children's Hospital D/B/A Seattle Children's Research Institute | Systems and methods to provide therapeutic factor viii using ultrasound-mediated nucleic acid delivery |
| WO2025064507A1 (en) * | 2023-09-19 | 2025-03-27 | Poseida Therapeutics, Inc. | Compositions and methods for integration of viral vectors |
| WO2025085119A2 (en) * | 2023-10-17 | 2025-04-24 | Generation Bio Co. | Synthetic partially single-stranded nucleic acid compositions and uses and methods therefor |
| WO2025083617A1 (en) | 2023-10-18 | 2025-04-24 | Nanocell Therapeutics Holdings B.V. | Methods and compositions for nucleic construct delivery |
| WO2025106523A1 (en) * | 2023-11-14 | 2025-05-22 | Nchroma Bio, Inc. | Compositions and methods for epigenetic regulation of f8 expression |
| WO2025106834A1 (en) * | 2023-11-17 | 2025-05-22 | Temple University-Of The Commonwealth System Of Higher Education | A single-plasmid system for enhanced crispr-cas9 mediated homologous recombination |
Family Cites Families (9)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP2500434A1 (en) * | 2011-03-12 | 2012-09-19 | Association Institut de Myologie | Capsid-free AAV vectors, compositions, and methods for vector production and gene delivery |
| US10604771B2 (en) * | 2013-05-10 | 2020-03-31 | Sangamo Therapeutics, Inc. | Delivery methods and compositions for nuclease-mediated genome engineering |
| MX374529B (es) * | 2013-12-12 | 2025-03-06 | Broad Inst Inc | Suministro, uso y aplicaciones terapeuticas de sistemas y composiciones crispr-cas para edicion del genoma. |
| WO2015138620A1 (en) * | 2014-03-11 | 2015-09-17 | University Of Washington | Restricting nuclear protein to specific phases of the cell cycle |
| US20180112213A1 (en) * | 2015-03-25 | 2018-04-26 | Editas Medicine, Inc. | Crispr/cas-related methods, compositions and components |
| WO2016176191A1 (en) * | 2015-04-27 | 2016-11-03 | The Trustees Of The University Of Pennsylvania | Dual aav vector system for crispr/cas9 mediated correction of human disease |
| WO2016205728A1 (en) * | 2015-06-17 | 2016-12-22 | Massachusetts Institute Of Technology | Crispr mediated recording of cellular events |
| KR102336362B1 (ko) * | 2016-03-03 | 2021-12-08 | 보이저 테라퓨틱스, 인크. | 비-바이러스 유전자 전달을 위한 폐쇄형-말단 선형 듀플렉스 dna |
| CN111132699A (zh) * | 2017-09-08 | 2020-05-08 | 世代生物公司 | 修饰的封闭端dna(cedna) |
-
2018
- 2018-12-06 KR KR1020207018991A patent/KR20200093635A/ko not_active Ceased
- 2018-12-06 SG SG11202005281XA patent/SG11202005281XA/en unknown
- 2018-12-06 US US16/769,671 patent/US20220290186A1/en not_active Abandoned
- 2018-12-06 JP JP2020531057A patent/JP2021505159A/ja active Pending
- 2018-12-06 MX MX2020005808A patent/MX2020005808A/es unknown
- 2018-12-06 CN CN201880083624.0A patent/CN111527200A/zh active Pending
- 2018-12-06 AU AU2018378672A patent/AU2018378672A1/en not_active Abandoned
- 2018-12-06 WO PCT/US2018/064242 patent/WO2019113310A1/en not_active Ceased
- 2018-12-06 CA CA3084185A patent/CA3084185A1/en active Pending
- 2018-12-06 BR BR112020009858-2A patent/BR112020009858A2/pt unknown
- 2018-12-06 SG SG10202012132WA patent/SG10202012132WA/en unknown
- 2018-12-06 MA MA051113A patent/MA51113A/fr unknown
- 2018-12-06 EP EP18886653.7A patent/EP3720952A4/en active Pending
-
2020
- 2020-05-21 IL IL274845A patent/IL274845A/en unknown
- 2020-06-01 PH PH12020550771A patent/PH12020550771A1/en unknown
-
2023
- 2023-11-16 JP JP2023195038A patent/JP2024003220A/ja active Pending
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