JP2018531624A5 - - Google Patents
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- Publication number
- JP2018531624A5 JP2018531624A5 JP2018531315A JP2018531315A JP2018531624A5 JP 2018531624 A5 JP2018531624 A5 JP 2018531624A5 JP 2018531315 A JP2018531315 A JP 2018531315A JP 2018531315 A JP2018531315 A JP 2018531315A JP 2018531624 A5 JP2018531624 A5 JP 2018531624A5
- Authority
- JP
- Japan
- Prior art keywords
- seq
- aav vector
- subunit
- promoter
- recombinant
- 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.)
- Granted
Links
- 239000013607 AAV vector Substances 0.000 claims 8
- 125000003275 alpha amino acid group Chemical group 0.000 claims 4
- 102200141512 rs104893768 Human genes 0.000 claims 4
- 108091033319 polynucleotide Proteins 0.000 claims 3
- 102000040430 polynucleotide Human genes 0.000 claims 3
- 239000002157 polynucleotide Substances 0.000 claims 3
- 108020004511 Recombinant DNA Proteins 0.000 claims 2
- 150000007523 nucleic acids Chemical group 0.000 claims 2
- 241000702421 Dependoparvovirus Species 0.000 claims 1
- 101001031598 Dictyostelium discoideum Probable serine/threonine-protein kinase fhkC Proteins 0.000 claims 1
- 101000887490 Homo sapiens Guanine nucleotide-binding protein G(z) subunit alpha Proteins 0.000 claims 1
- 101000829506 Homo sapiens Rhodopsin kinase GRK1 Proteins 0.000 claims 1
- 108091028043 Nucleic acid sequence Proteins 0.000 claims 1
- 102100023742 Rhodopsin kinase GRK1 Human genes 0.000 claims 1
- 210000004027 cell Anatomy 0.000 claims 1
- 102000052301 human GNAZ Human genes 0.000 claims 1
- 108020004707 nucleic acids Proteins 0.000 claims 1
- 102000039446 nucleic acids Human genes 0.000 claims 1
- 102000005962 receptors Human genes 0.000 claims 1
- 108020003175 receptors Proteins 0.000 claims 1
- 230000002207 retinal effect Effects 0.000 claims 1
- 210000000880 retinal rod photoreceptor cell Anatomy 0.000 claims 1
- 239000013598 vector Substances 0.000 claims 1
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201562215460P | 2015-09-08 | 2015-09-08 | |
| US62/215,460 | 2015-09-08 | ||
| PCT/US2016/050809 WO2017044649A1 (en) | 2015-09-08 | 2016-09-08 | Treatment of retinitis pigmentosa using engineered meganucleases |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2020168987A Division JP7068413B2 (ja) | 2015-09-08 | 2020-10-06 | 設計されたメガヌクレアーゼを使用した網膜色素変性症の治療 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2018531624A JP2018531624A (ja) | 2018-11-01 |
| JP2018531624A5 true JP2018531624A5 (enExample) | 2020-10-01 |
| JP6775584B2 JP6775584B2 (ja) | 2020-10-28 |
Family
ID=57003577
Family Applications (5)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2018531315A Active JP6775584B2 (ja) | 2015-09-08 | 2016-09-08 | 設計されたメガヌクレアーゼを使用した網膜色素変性症の治療 |
| JP2020168987A Active JP7068413B2 (ja) | 2015-09-08 | 2020-10-06 | 設計されたメガヌクレアーゼを使用した網膜色素変性症の治療 |
| JP2022074109A Pending JP2022110004A (ja) | 2015-09-08 | 2022-04-28 | 設計されたメガヌクレアーゼを使用した網膜色素変性症の治療 |
| JP2023182410A Pending JP2024023183A (ja) | 2015-09-08 | 2023-10-24 | 設計されたメガヌクレアーゼを使用した網膜色素変性症の治療 |
| JP2025079382A Pending JP2025131592A (ja) | 2015-09-08 | 2025-05-12 | 設計されたメガヌクレアーゼを使用した網膜色素変性症の治療 |
Family Applications After (4)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2020168987A Active JP7068413B2 (ja) | 2015-09-08 | 2020-10-06 | 設計されたメガヌクレアーゼを使用した網膜色素変性症の治療 |
| JP2022074109A Pending JP2022110004A (ja) | 2015-09-08 | 2022-04-28 | 設計されたメガヌクレアーゼを使用した網膜色素変性症の治療 |
| JP2023182410A Pending JP2024023183A (ja) | 2015-09-08 | 2023-10-24 | 設計されたメガヌクレアーゼを使用した網膜色素変性症の治療 |
| JP2025079382A Pending JP2025131592A (ja) | 2015-09-08 | 2025-05-12 | 設計されたメガヌクレアーゼを使用した網膜色素変性症の治療 |
Country Status (6)
| Country | Link |
|---|---|
| US (6) | US10603363B2 (enExample) |
| EP (2) | EP4530354A3 (enExample) |
| JP (5) | JP6775584B2 (enExample) |
| AU (3) | AU2016321244B2 (enExample) |
| CA (1) | CA2997909A1 (enExample) |
| WO (1) | WO2017044649A1 (enExample) |
Families Citing this family (13)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP3957735A1 (en) | 2014-03-05 | 2022-02-23 | Editas Medicine, Inc. | Crispr/cas-related methods and compositions for treating usher syndrome and retinitis pigmentosa |
| US12460231B2 (en) | 2014-04-02 | 2025-11-04 | Editas Medicine, Inc. | Crispr/CAS-related methods and compositions for treating primary open angle glaucoma |
| EP4019975A1 (en) | 2015-04-24 | 2022-06-29 | Editas Medicine, Inc. | Evaluation of cas9 molecule/guide rna molecule complexes |
| EP4530354A3 (en) | 2015-09-08 | 2025-09-10 | Precision Biosciences, Inc. | Treatment of retinitis pigmentosa using engineered meganucleases |
| MX2019012452A (es) * | 2017-04-21 | 2020-08-03 | Prec Biosciences Inc | Meganucleasas diseñadas especificas para secuencias de reconocimiento en el gen pcsk9. |
| EP3714055A1 (en) | 2017-11-21 | 2020-09-30 | CRISPR Therapeutics AG | Materials and methods for treatment of autosomal dominant retinitis pigmentosa |
| US20220195407A1 (en) | 2019-05-07 | 2022-06-23 | Precision Biosciences, Inc. | Optimization of engineered meganucleases for recognition sequences |
| WO2021113763A1 (en) * | 2019-12-06 | 2021-06-10 | Scribe Therapeutics Inc. | Compositions and methods for the targeting of rhodopsin |
| WO2021212686A1 (zh) * | 2020-04-21 | 2021-10-28 | 北京中因科技有限公司 | RHO-adRP基于基因编辑的方法和组合物 |
| US20230183664A1 (en) | 2020-05-11 | 2023-06-15 | Precision Biosciences, Inc. | Self-limiting viral vectors encoding nucleases |
| US20230193230A1 (en) * | 2020-05-12 | 2023-06-22 | Precision Biosciences, Inc. | Treatment of retinitis pigmentosa using improved engineered meganucleases |
| MX2024004840A (es) * | 2021-10-19 | 2024-07-09 | Prec Biosciences Inc | Método de edición de genes para tratar la deficiencia de alfa-1 antitripsina (aat). |
| WO2025101946A1 (en) | 2023-11-08 | 2025-05-15 | Precision Biosciences, Inc. | Polypeptide linkers for use in engineered meganucleases |
Family Cites Families (16)
| 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 |
| 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 |
| 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 |
| US6506803B1 (en) | 1999-04-28 | 2003-01-14 | Regents Of The University Of Michigan | Methods of preventing and treating microbial infections |
| CA2626262C (en) | 2005-10-18 | 2015-09-08 | Homme W. Hellinga | Rationally-designed meganucleases with altered sequence specificity and dna-binding affinity |
| EP3578646A3 (en) | 2007-10-31 | 2020-03-18 | Precision Biosciences, Inc. | Rationally-designed single-chain meganucleases with non-palindromic recognition sequences |
| EP2531609A1 (en) * | 2010-02-02 | 2012-12-12 | INSERM (Institut National de la Santé et de la Recherche Médicale) | Methods for the diagnosis and therapy of retinitis pigmentosa |
| EP2569435A1 (en) * | 2010-05-12 | 2013-03-20 | Cellectis | Meganuclease variants cleaving a dna target sequence from the rhodopsin gene and uses thereof |
| US20120204282A1 (en) | 2011-02-04 | 2012-08-09 | Sangamo Biosciences, Inc. | Methods and compositions for treating occular disorders |
| WO2012167192A2 (en) | 2011-06-01 | 2012-12-06 | Precision Biosciences, Inc. | Methods and products for producing engineered mammalian cell lines with amplified transgenes |
| EP2776459A1 (en) | 2011-11-08 | 2014-09-17 | Novartis Forschungsstiftung, Zweigniederlassung Friedrich Miescher Institute For Biomedical Research | Rod cell-specific promoter |
| EP3653229A1 (en) * | 2013-12-12 | 2020-05-20 | The Broad Institute, Inc. | Delivery, use and therapeutic applications of the crispr-cas systems and compositions for genome editing |
| AU2015229299A1 (en) * | 2014-03-12 | 2016-09-22 | Precision Biosciences, Inc. | Dystrophin gene exon deletion using engineered nucleases |
| EP4530354A3 (en) | 2015-09-08 | 2025-09-10 | Precision Biosciences, Inc. | Treatment of retinitis pigmentosa using engineered meganucleases |
-
2016
- 2016-09-08 EP EP24212265.3A patent/EP4530354A3/en active Pending
- 2016-09-08 WO PCT/US2016/050809 patent/WO2017044649A1/en not_active Ceased
- 2016-09-08 JP JP2018531315A patent/JP6775584B2/ja active Active
- 2016-09-08 US US15/758,366 patent/US10603363B2/en active Active
- 2016-09-08 CA CA2997909A patent/CA2997909A1/en active Pending
- 2016-09-08 AU AU2016321244A patent/AU2016321244B2/en active Active
- 2016-09-08 EP EP16771036.7A patent/EP3347463B1/en active Active
-
2020
- 2020-02-14 US US16/791,687 patent/US10758595B2/en active Active
- 2020-07-17 US US16/932,653 patent/US20210000928A1/en not_active Abandoned
- 2020-10-06 JP JP2020168987A patent/JP7068413B2/ja active Active
- 2020-12-23 AU AU2020294264A patent/AU2020294264A1/en not_active Abandoned
-
2022
- 2022-01-26 US US17/585,527 patent/US20220143155A1/en not_active Abandoned
- 2022-04-28 JP JP2022074109A patent/JP2022110004A/ja active Pending
-
2023
- 2023-07-13 US US18/351,633 patent/US20240238387A1/en not_active Abandoned
- 2023-08-03 AU AU2023210619A patent/AU2023210619A1/en not_active Abandoned
- 2023-10-24 JP JP2023182410A patent/JP2024023183A/ja active Pending
-
2024
- 2024-12-19 US US18/987,584 patent/US20250121039A1/en active Pending
-
2025
- 2025-05-12 JP JP2025079382A patent/JP2025131592A/ja active Pending
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