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) | KR102620399B1 (enExample) |
| CN (2) | CN110769845B (enExample) |
| AU (2) | AU2018254576B2 (enExample) |
| CA (1) | CA3060112A1 (enExample) |
| ES (1) | ES2953153T3 (enExample) |
| IL (3) | IL301059B1 (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 |
| US20220195407A1 (en) | 2019-05-07 | 2022-06-23 | Precision Biosciences, Inc. | Optimization of engineered meganucleases for recognition sequences |
| EP4143308A4 (en) * | 2020-04-27 | 2024-06-26 | The Trustees of The University of Pennsylvania | COMPOSITIONS AND METHODS FOR REDUCING NUCLEASE EXPRESSION AND OFF-TARGET ACTIVITY USING A PROMOTER WITH LOW TRANSCRIPTION ACTIVITY |
| WO2021222249A1 (en) * | 2020-04-27 | 2021-11-04 | 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 |
| EP4162059A1 (en) | 2020-05-12 | 2023-04-12 | The Trustees of The University of Pennsylvania | Compositions for drg-specific reduction of transgene expression |
| CN117042787A (zh) | 2020-10-29 | 2023-11-10 | 宾夕法尼亚州大学信托人 | Aav衣壳和含有aav衣壳的组合物 |
| WO2022104062A1 (en) * | 2020-11-12 | 2022-05-19 | Precision Biosciences, Inc. | Engineered meganucleases having specificity for recognition sequences in the dystrophin gene |
| WO2022232267A1 (en) | 2021-04-27 | 2022-11-03 | The Trustees Of The University Of Pennsylvania | Porcine-derived adeno-associated virus capsids and uses thereof |
| TW202304528A (zh) * | 2021-04-27 | 2023-02-01 | 賓州大學委員會 | 用於治療遺傳疾病的體內核酸酶媒介的基因靶向之組成物及方法 |
| US20250295807A1 (en) | 2021-11-15 | 2025-09-25 | The Trustees Of The University Of Pennsylvania | Compositions for drg-specific reduction of transgene expression |
| EP4314267A1 (en) | 2022-06-07 | 2024-02-07 | 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 |
| 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 |
| 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 |
| WO2003052051A2 (en) | 2001-12-17 | 2003-06-26 | The Trustees Of The University Of Pennsylvania | Adeno-associated virus (aav) serotype 8 sequences |
| WO2004067753A2 (en) * | 2003-01-28 | 2004-08-12 | Cellectis | Use of meganucleases for inducing homologous recombination ex vivo and in toto in vertebrate somatic tissues and application 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 |
| CA2626262C (en) | 2005-10-18 | 2015-09-08 | Homme W. Hellinga | Rationally-designed meganucleases with altered sequence specificity and dna-binding affinity |
| 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 |
| AU2013203677C1 (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 |
| EP3578646A3 (en) | 2007-10-31 | 2020-03-18 | 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 |
| EP3495478A3 (en) * | 2008-07-14 | 2019-07-24 | 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 |
| WO2012167192A2 (en) | 2011-06-01 | 2012-12-06 | Precision Biosciences, Inc. | Methods and products for producing engineered mammalian cell lines with amplified transgenes |
| WO2014089486A1 (en) * | 2012-12-07 | 2014-06-12 | Shire Human Genetic Therapies, Inc. | Lipidic nanoparticles for mrna delivering |
| US10329574B2 (en) * | 2013-03-12 | 2019-06-25 | E I Du Pont De Nemours And Company | Methods for the identification of variant recognition sites for rare-cutting engineered double-strand-break-inducing agents and compositions and uses thereof |
| KR20240172759A (ko) | 2013-06-17 | 2024-12-10 | 더 브로드 인스티튜트, 인코퍼레이티드 | 간의 표적화 및 치료를 위한 ＣＲＩＳＰＲＣａｓ 시스템, 벡터 및 조성물의 전달 및 용도 |
| KR102592370B1 (ko) * | 2014-02-11 | 2023-10-25 | 알닐람 파마슈티칼스 인코포레이티드 | 케토헥소키나제(ＫＨＫ) ｉＲＮＡ 조성물 및 그의 사용 방법 |
| 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 |
| AU2015259191B2 (en) * | 2014-05-13 | 2019-03-21 | Sangamo Therapeutics, Inc. | Methods and compositions for prevention or treatment of a disease |
| EP4530354A3 (en) * | 2015-09-08 | 2025-09-10 | Precision Biosciences, Inc. | Treatment of retinitis pigmentosa using engineered meganucleases |
| DK3359184T3 (da) * | 2015-10-05 | 2020-06-15 | Prec Biosciences Inc | Genetisk modificerede celler, der omfatter et modificeret gen fra konstant alfaregion i human t-cellereceptor |
-
2018
- 2018-04-20 MX MX2019012452A patent/MX2019012452A/es unknown
- 2018-04-20 ES ES18723219T patent/ES2953153T3/es active Active
- 2018-04-20 CN CN201880038349.0A patent/CN110769845B/zh active Active
- 2018-04-20 US US16/606,856 patent/US11680254B2/en active Active
- 2018-04-20 IL IL301059A patent/IL301059B1/en unknown
- 2018-04-20 CA CA3060112A patent/CA3060112A1/en active Pending
- 2018-04-20 EP EP23172361.0A patent/EP4269596A3/en active Pending
- 2018-04-20 EP EP18723219.4A patent/EP3612205B9/en active Active
- 2018-04-20 KR KR1020197033913A patent/KR102620399B1/ko active Active
- 2018-04-20 IL IL324738A patent/IL324738A/en unknown
- 2018-04-20 KR KR1020237041504A patent/KR20230170125A/ko active Pending
- 2018-04-20 CN CN202311029425.0A patent/CN117264929A/zh active Pending
- 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 JP JP2019556884A patent/JP2020517255A/ja active Pending
-
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 active Pending
- 2023-05-04 US US18/312,328 patent/US20240101985A1/en not_active Abandoned
- 2023-05-04 US US18/312,334 patent/US20240101986A1/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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