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 (enExample) | 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 (enExample) |
| EP (2) | EP4269596A3 (enExample) |
| JP (4) | JP2020517255A (enExample) |
| KR (2) | KR20230170125A (enExample) |
| CN (2) | CN117264929A (enExample) |
| AU (3) | AU2018254576B2 (enExample) |
| CA (1) | CA3060112A1 (enExample) |
| ES (1) | ES2953153T3 (enExample) |
| IL (3) | IL324738A (enExample) |
| MX (2) | MX2019012452A (enExample) |
| WO (1) | WO2018195449A1 (enExample) |
Families Citing this family (14)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US20220298500A1 (en) * | 2019-04-15 | 2022-09-22 | 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 |
| US20230167464A1 (en) * | 2020-04-27 | 2023-06-01 | 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 |
| WO2021231259A1 (en) | 2020-05-11 | 2021-11-18 | Precision Biosciences, Inc. | Self-limiting viral vectors encoding nucleases |
| IL298138A (en) | 2020-05-12 | 2023-01-01 | Univ Pennsylvania | Compositions for drg-specific reduction of transgene expression |
| MX2023005113A (es) | 2020-10-29 | 2023-08-04 | Univ Pennsylvania | Cápsides de aav y composiciones que las contienen. |
| KR20250099412A (ko) * | 2020-11-12 | 2025-07-01 | 프리시젼 바이오사이언시스 인코포레이티드 | 디스트로핀 유전자 내의 인식 서열에 대해 특이성을 갖는 조작된 메가뉴클레아제 |
| TW202309066A (zh) | 2021-04-27 | 2023-03-01 | 賓州大學委員會 | 衍生自豬的腺相關病毒衣殼及其用途 |
| CA3216285A1 (en) * | 2021-04-27 | 2022-11-03 | Lili Wang | Compositions and methods for in vivo nuclease-mediated gene targeting for the treatment of genetic disorders |
| WO2023087019A2 (en) | 2021-11-15 | 2023-05-19 | The Trustees Of The University Of Pennsylvania | Compositions for drg-specific reduction of transgene expression |
| CN119998446A (zh) | 2022-06-07 | 2025-05-13 | 斯克里贝治疗公司 | 用于靶向pcsk9的组合物和方法 |
| WO2024015966A2 (en) | 2022-07-15 | 2024-01-18 | 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 |
| US7767216B2 (en) | 1999-04-28 | 2010-08-03 | The Regents Of The University Of Michigan | Antimicrobial compositions and methods of use |
| US6635676B2 (en) | 1999-04-28 | 2003-10-21 | 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 |
| US6559189B2 (en) | 1999-04-28 | 2003-05-06 | Regents Of The University Of Michigan | Non-toxic antimicrobial compositions and methods of use |
| US6555674B2 (en) | 2000-08-09 | 2003-04-29 | Nsgene A/S | JeT promoter |
| DK2359869T3 (en) | 2001-12-17 | 2019-04-15 | Univ Pennsylvania | Sequences of adeno-associated virus (AAV) serotype 8, vectors containing these, and uses thereof |
| US20060206949A1 (en) | 2003-01-28 | 2006-09-14 | Sylvain Arnould | 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 |
| DK3246403T3 (da) | 2005-10-18 | 2020-10-26 | Prec Biosciences | Rationelt designede meganucleaser med ændret sekvens-specificitet og dna-bindende affinitet |
| ES2366973T3 (es) * | 2006-05-05 | 2011-10-27 | Isis Pharmaceuticals, Inc. | Compuestos y procedimiento para modular la expresión génica. |
| AU2013201688B2 (en) * | 2006-11-07 | 2016-03-03 | Merck Sharp & Dohme Llc | Antagonists of PCSK9 |
| AU2013203725A1 (en) * | 2007-08-23 | 2013-05-02 | 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 |
| ES2422291T3 (es) | 2007-10-31 | 2013-09-10 | Prec Biosciences Inc | Meganucleasas monocatenarias diseñadas racionalmente con secuencias de reconocimiento no palindrómicas |
| US7928189B2 (en) * | 2008-05-05 | 2011-04-19 | Ottawa Health Research Institute | PCSK9 polypeptide fragment |
| JP2011527906A (ja) | 2008-07-14 | 2011-11-10 | プレシジョン バイオサイエンシズ，インク． | Ｉ−ＣｒｅＩ由来メガヌクレアーゼの認識配列およびその使用 |
| 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 |
| EP3683319A1 (en) | 2011-06-01 | 2020-07-22 | Precision Biosciences, Inc. | Methods and products for producing engineered mammalian cell lines with amplified transgenes |
| US20150366997A1 (en) * | 2012-12-07 | 2015-12-24 | Shire Human Genetics Therapies, Inc. | COMPOSITIONS AND METHODS FOR mRNA DELIVERY |
| CN105189788B (zh) * | 2013-03-12 | 2018-07-13 | 纳幕尔杜邦公司 | 用于鉴定罕见切割工程化双链断裂诱导剂的变体识别位点的方法及其组合物和用途 |
| JP6738728B2 (ja) * | 2013-06-17 | 2020-08-19 | ザ・ブロード・インスティテュート・インコーポレイテッド | 肝臓ターゲティングおよび治療のためのＣＲＩＳＰＲ−Ｃａｓ系、ベクターおよび組成物の送達および使用 |
| EP3105331B1 (en) * | 2014-02-11 | 2021-06-23 | Alnylam Pharmaceuticals, Inc. | Ketohexokinase (khk) irna compositions and methods of use thereof |
| US11555059B2 (en) * | 2014-04-25 | 2023-01-17 | 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 |
| CA2997909A1 (en) * | 2015-09-08 | 2017-03-16 | Precision Biosciences, Inc. | Treatment of retinitis pigmentosa using engineered meganucleases |
| ES2803728T3 (es) * | 2015-10-05 | 2021-01-29 | Prec Biosciences Inc | Células genéticamente modificadas que comprenden un gen de región constante alfa del receptor de linfocitos T humano modificado |
-
2018
- 2018-04-20 EP EP23172361.0A patent/EP4269596A3/en active Pending
- 2018-04-20 KR KR1020237041504A patent/KR20230170125A/ko active Pending
- 2018-04-20 CN CN202311029425.0A patent/CN117264929A/zh active Pending
- 2018-04-20 IL IL324738A patent/IL324738A/en unknown
- 2018-04-20 KR KR1020197033913A patent/KR102620399B1/ko active Active
- 2018-04-20 US US16/606,856 patent/US11680254B2/en active Active
- 2018-04-20 MX MX2019012452A patent/MX2019012452A/es unknown
- 2018-04-20 AU AU2018254576A patent/AU2018254576B2/en active Active
- 2018-04-20 ES ES18723219T patent/ES2953153T3/es active Active
- 2018-04-20 EP EP18723219.4A patent/EP3612205B9/en active Active
- 2018-04-20 WO PCT/US2018/028607 patent/WO2018195449A1/en not_active Ceased
- 2018-04-20 JP JP2019556884A patent/JP2020517255A/ja active Pending
- 2018-04-20 CA CA3060112A patent/CA3060112A1/en active Pending
- 2018-04-20 IL IL301059A patent/IL301059B2/en unknown
- 2018-04-20 CN CN201880038349.0A patent/CN110769845B/zh active Active
-
2019
- 2019-10-17 IL IL270020A patent/IL270020B2/en unknown
- 2019-10-17 MX MX2023001877A patent/MX2023001877A/es 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
-
2026
- 2026-04-10 AU AU2026202721A patent/AU2026202721A1/en active Pending
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