JP2020517255A5 - - Google Patents
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- Publication number
- JP2020517255A5 JP2020517255A5 JP2019556884A JP2019556884A JP2020517255A5 JP 2020517255 A5 JP2020517255 A5 JP 2020517255A5 JP 2019556884 A JP2019556884 A JP 2019556884A JP 2019556884 A JP2019556884 A JP 2019556884A JP 2020517255 A5 JP2020517255 A5 JP 2020517255A5
- Authority
- JP
- Japan
- Prior art keywords
- nucleic acid
- viral vector
- acid sequence
- engineered
- sequence encoding
- 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
- 239000013603 viral vector Substances 0.000 claims 8
- 150000007523 nucleic acids Chemical group 0.000 claims 7
- 108091028043 Nucleic acid sequence Proteins 0.000 claims 6
- 108020004511 Recombinant DNA Proteins 0.000 claims 5
- 239000008194 pharmaceutical composition Substances 0.000 claims 5
- 108091033319 polynucleotide Proteins 0.000 claims 4
- 102000040430 polynucleotide Human genes 0.000 claims 4
- 239000002157 polynucleotide Substances 0.000 claims 4
- 239000013607 AAV vector Substances 0.000 claims 3
- 241000702421 Dependoparvovirus Species 0.000 claims 2
- 108020004999 messenger RNA Proteins 0.000 claims 2
- 108090000623 proteins and genes Proteins 0.000 claims 2
- 101710172072 Kexin Proteins 0.000 claims 1
- 108090000787 Subtilisin Proteins 0.000 claims 1
- 125000003275 alpha amino acid group Chemical group 0.000 claims 1
- 239000003937 drug carrier Substances 0.000 claims 1
- 150000002632 lipids Chemical class 0.000 claims 1
- 210000004185 liver Anatomy 0.000 claims 1
- 239000002105 nanoparticle Substances 0.000 claims 1
- 108020004707 nucleic acids Proteins 0.000 claims 1
- 102000039446 nucleic acids Human genes 0.000 claims 1
- 102000004169 proteins and genes Human genes 0.000 claims 1
- 239000013598 vector Substances 0.000 claims 1
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2022145178A JP7214915B2 (ja) | 2017-04-21 | 2022-09-13 | Ｐｃｓｋ９遺伝子の認識配列に特異的な操作されたメガヌクレアーゼ |
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201762488403P | 2017-04-21 | 2017-04-21 | |
| US62/488,403 | 2017-04-21 | ||
| US201762516966P | 2017-06-08 | 2017-06-08 | |
| US62/516,966 | 2017-06-08 | ||
| PCT/US2018/028607 WO2018195449A1 (en) | 2017-04-21 | 2018-04-20 | Engineered meganucleases specific for recognition sequences in the pcsk9 gene |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022145178A Division JP7214915B2 (ja) | 2017-04-21 | 2022-09-13 | Ｐｃｓｋ９遺伝子の認識配列に特異的な操作されたメガヌクレアーゼ |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2020517255A JP2020517255A (ja) | 2020-06-18 |
| JP2020517255A5 true JP2020517255A5 (https=) | 2021-05-27 |
Family
ID=62117064
Family Applications (4)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2019556884A Pending JP2020517255A (ja) | 2017-04-21 | 2018-04-20 | Ｐｃｓｋ９遺伝子の認識配列に特異的な操作されたメガヌクレアーゼ |
| JP2022145178A Active JP7214915B2 (ja) | 2017-04-21 | 2022-09-13 | Ｐｃｓｋ９遺伝子の認識配列に特異的な操作されたメガヌクレアーゼ |
| JP2023005733A Pending JP2023058509A (ja) | 2017-04-21 | 2023-01-18 | Ｐｃｓｋ９遺伝子の認識配列に特異的な操作されたメガヌクレアーゼ |
| JP2024231599A Pending JP2025066725A (ja) | 2017-04-21 | 2024-12-27 | Ｐｃｓｋ９遺伝子の認識配列に特異的な操作されたメガヌクレアーゼ |
Family Applications After (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022145178A Active JP7214915B2 (ja) | 2017-04-21 | 2022-09-13 | Ｐｃｓｋ９遺伝子の認識配列に特異的な操作されたメガヌクレアーゼ |
| JP2023005733A Pending JP2023058509A (ja) | 2017-04-21 | 2023-01-18 | Ｐｃｓｋ９遺伝子の認識配列に特異的な操作されたメガヌクレアーゼ |
| JP2024231599A Pending JP2025066725A (ja) | 2017-04-21 | 2024-12-27 | Ｐｃｓｋ９遺伝子の認識配列に特異的な操作されたメガヌクレアーゼ |
Country Status (11)
| Country | Link |
|---|---|
| US (4) | US11680254B2 (https=) |
| EP (2) | EP4269596A3 (https=) |
| JP (4) | JP2020517255A (https=) |
| KR (2) | KR20230170125A (https=) |
| CN (2) | CN117264929A (https=) |
| AU (2) | AU2018254576B2 (https=) |
| CA (1) | CA3060112A1 (https=) |
| ES (1) | ES2953153T3 (https=) |
| IL (3) | IL301059B2 (https=) |
| MX (2) | MX2019012452A (https=) |
| WO (1) | WO2018195449A1 (https=) |
Families Citing this family (14)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2020214724A1 (en) * | 2019-04-15 | 2020-10-22 | The Trustees Of The University Of Pennsylvania | Compositions for regulating and self-inactivating enzyme expression and methods for modulating off-target activity of enzymes |
| CN114026228A (zh) | 2019-05-07 | 2022-02-08 | 精密生物科学公司 | 用于识别序列的工程化大范围核酸酶的优化 |
| EP4142801A4 (en) * | 2020-04-27 | 2024-09-25 | The Trustees of The University of Pennsylvania | Compositions and methods for reducing nuclease expression and off-target activity using a promoter with low transcriptional activity |
| US20230175014A1 (en) * | 2020-04-27 | 2023-06-08 | The Trustees Of The University Of Pennsylvania | Compositions and methods for reducing nuclease expression and off-target activity using a promoter with low transcriptional activity |
| US20230183664A1 (en) | 2020-05-11 | 2023-06-15 | Precision Biosciences, Inc. | Self-limiting viral vectors encoding nucleases |
| MX2022014258A (es) | 2020-05-12 | 2023-02-22 | Univ Pennsylvania | Composiciones para la reducción específica de un fármaco de la expresión de transgén. |
| JP2023550581A (ja) | 2020-10-29 | 2023-12-04 | ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア | Ａａｖカプシド及びそれを含有する組成物 |
| JP7642809B2 (ja) * | 2020-11-12 | 2025-03-10 | プレシジョン バイオサイエンシズ，インク． | ジストロフィン遺伝子内の認識配列に特異性を有する遺伝子操作されたメガヌクレアーゼ |
| WO2022232267A1 (en) | 2021-04-27 | 2022-11-03 | The Trustees Of The University Of Pennsylvania | Porcine-derived adeno-associated virus capsids and uses thereof |
| MX2023012747A (es) * | 2021-04-27 | 2024-01-05 | Univ Pennsylvania | Composiciones y métodos para el direccionamiento genético in vivo mediado por nucleasas para el tratamiento de trastornos genéticos. |
| US20250295807A1 (en) | 2021-11-15 | 2025-09-25 | The Trustees Of The University Of Pennsylvania | Compositions for drg-specific reduction of transgene expression |
| GB2634837A (en) | 2022-06-07 | 2025-04-23 | Scribe Therapeutics Inc | Compositions and methods for the targeting of PCSK9 |
| US20260021205A1 (en) | 2022-07-15 | 2026-01-22 | The Trustees Of The University Of Pennsylvania | Recombinant aav having aav clade d and clade e capsids and compositions containing same |
| WO2025122552A1 (en) * | 2023-12-04 | 2025-06-12 | The Trustees Of The University Of Pennsylvania | In silico prediction tool for meganuclease off-target sites |
Family Cites Families (30)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US4873192A (en) | 1987-02-17 | 1989-10-10 | The United States Of America As Represented By The Department Of Health And Human Services | Process for site specific mutagenesis without phenotypic selection |
| US5652224A (en) * | 1995-02-24 | 1997-07-29 | The Trustees Of The University Of Pennsylvania | Methods and compositions for gene therapy for the treatment of defects in lipoprotein metabolism |
| US6015832A (en) | 1997-12-31 | 2000-01-18 | The Regents Of The University Of Michigan | Methods of inactivating bacteria including bacterial spores |
| US6559189B2 (en) | 1999-04-28 | 2003-05-06 | Regents Of The University Of Michigan | Non-toxic antimicrobial compositions and methods of use |
| US6506803B1 (en) | 1999-04-28 | 2003-01-14 | Regents Of The University Of Michigan | Methods of preventing and treating microbial infections |
| US6635676B2 (en) | 1999-04-28 | 2003-10-21 | Regents Of The University Of Michigan | Non-toxic antimicrobial compositions and methods of use |
| US7767216B2 (en) | 1999-04-28 | 2010-08-03 | The Regents Of The University Of Michigan | Antimicrobial compositions and methods of use |
| US6555674B2 (en) | 2000-08-09 | 2003-04-29 | Nsgene A/S | JeT promoter |
| PT1453547T (pt) | 2001-12-17 | 2016-12-28 | Univ Pennsylvania | Sequências do vírus adeno-associado (aav) do serotipo 8, vetores contendo as mesmas, e utilizações destas |
| EP3202899B1 (en) | 2003-01-28 | 2020-10-21 | Cellectis | Custom-made meganuclease and use thereof |
| US7404969B2 (en) | 2005-02-14 | 2008-07-29 | Sirna Therapeutics, Inc. | Lipid nanoparticle based compositions and methods for the delivery of biologically active molecules |
| ES2582091T3 (es) * | 2005-10-18 | 2016-09-09 | Precision Biosciences | Meganucleasas diseñadas racionalmente con especificidad de secuencia y afinidad de unión a ADN alteradas |
| ES2366974T3 (es) * | 2006-05-05 | 2011-10-27 | Isis Pharmaceuticals, Inc. | Compuestos y procedimientos para modular la expresión de sglt2. |
| AU2013201688B2 (en) * | 2006-11-07 | 2016-03-03 | Merck Sharp & Dohme Llc | Antagonists of PCSK9 |
| AU2013203748C1 (en) * | 2007-08-23 | 2017-05-25 | Amgen Inc. | Antigen Binding Proteins to Proprotein Convertase Subtilisin Kexin Type 9 (PCSK9) |
| CA2603615A1 (en) | 2007-09-21 | 2009-03-21 | Patrick Labonte | Methods of reducing viral infection and kits therefore |
| EP2215223B1 (en) * | 2007-10-31 | 2013-05-01 | Precision Biosciences, Inc. | Rationally-designed single-chain meganucleases with non-palindromic recognition sequences |
| US7928189B2 (en) * | 2008-05-05 | 2011-04-19 | Ottawa Health Research Institute | PCSK9 polypeptide fragment |
| EP2313498B1 (en) | 2008-07-14 | 2017-03-15 | Precision Biosciences, Inc. | Recognition sequences for i-crei-derived meganucleases and uses thereof |
| EP2180058A1 (en) * | 2008-10-23 | 2010-04-28 | Cellectis | Meganuclease recombination system |
| AU2013273674B2 (en) * | 2009-09-03 | 2015-11-05 | Pfizer Vaccines Llc | PCSK9 vaccine |
| EP2714936B1 (en) | 2011-06-01 | 2018-12-12 | Precision Biosciences, Inc. | Methods and products for producing engineered mammalian cell lines with amplified transgenes |
| EP4331620A3 (en) * | 2012-12-07 | 2024-12-04 | Translate Bio, Inc. | Lipidic nanoparticles for mrna delivery |
| RU2694686C2 (ru) * | 2013-03-12 | 2019-07-16 | Е.И.Дюпон Де Немур Энд Компани | Способы идентификации вариантных сайтов распознавания для редкощепящих сконструированных средств для индукции двунитевого разрыва, и композиции с ними, и их применения |
| RU2716420C2 (ru) * | 2013-06-17 | 2020-03-11 | Те Брод Инститьют Инк. | Доставка и применение систем crispr-cas, векторов и композиций для целенаправленного воздействия и терапии в печени |
| CN113057959B (zh) * | 2014-02-11 | 2024-07-16 | 阿尔尼拉姆医药品有限公司 | 己酮糖激酶（KHK）iRNA组合物及其使用方法 |
| EP3134431B1 (en) * | 2014-04-25 | 2021-04-07 | The Trustees Of The University Of Pennsylvania | Ldlr variants and their use in compositions for reducing cholesterol levels |
| WO2015175642A2 (en) * | 2014-05-13 | 2015-11-19 | Sangamo Biosciences, Inc. | Methods and compositions for prevention or treatment of a disease |
| US10603363B2 (en) | 2015-09-08 | 2020-03-31 | Precision Biosciences, Inc. | Treatment of retinitis pigmentosa using engineered meganucleases |
| JP6816133B2 (ja) * | 2015-10-05 | 2021-01-20 | プレシジョン バイオサイエンシズ，インク． | 改変ヒトｔ細胞受容体アルファ定常領域遺伝子を含む遺伝子改変細胞 |
-
2018
- 2018-04-20 IL IL301059A patent/IL301059B2/en unknown
- 2018-04-20 AU AU2018254576A patent/AU2018254576B2/en active Active
- 2018-04-20 WO PCT/US2018/028607 patent/WO2018195449A1/en not_active Ceased
- 2018-04-20 US US16/606,856 patent/US11680254B2/en active Active
- 2018-04-20 KR KR1020237041504A patent/KR20230170125A/ko active Pending
- 2018-04-20 JP JP2019556884A patent/JP2020517255A/ja active Pending
- 2018-04-20 CA CA3060112A patent/CA3060112A1/en active Pending
- 2018-04-20 KR KR1020197033913A patent/KR102620399B1/ko active Active
- 2018-04-20 CN CN202311029425.0A patent/CN117264929A/zh active Pending
- 2018-04-20 MX MX2019012452A patent/MX2019012452A/es unknown
- 2018-04-20 ES ES18723219T patent/ES2953153T3/es active Active
- 2018-04-20 EP EP23172361.0A patent/EP4269596A3/en active Pending
- 2018-04-20 IL IL324738A patent/IL324738A/en unknown
- 2018-04-20 CN CN201880038349.0A patent/CN110769845B/zh active Active
- 2018-04-20 EP EP18723219.4A patent/EP3612205B9/en active Active
-
2019
- 2019-10-17 MX MX2023001877A patent/MX2023001877A/es unknown
- 2019-10-17 IL IL270020A patent/IL270020B2/en unknown
-
2022
- 2022-09-13 JP JP2022145178A patent/JP7214915B2/ja active Active
-
2023
- 2023-01-18 JP JP2023005733A patent/JP2023058509A/ja active Pending
- 2023-03-16 AU AU2023201658A patent/AU2023201658A1/en not_active Abandoned
- 2023-05-04 US US18/312,334 patent/US20240101986A1/en not_active Abandoned
- 2023-05-04 US US18/312,328 patent/US20240101985A1/en not_active Abandoned
-
2024
- 2024-09-26 US US18/898,073 patent/US20250154481A1/en active Pending
- 2024-12-27 JP JP2024231599A patent/JP2025066725A/ja active Pending
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