JPWO2020176552A5 - - Google Patents
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- Publication number
- JPWO2020176552A5 JPWO2020176552A5 JP2021549698A JP2021549698A JPWO2020176552A5 JP WO2020176552 A5 JPWO2020176552 A5 JP WO2020176552A5 JP 2021549698 A JP2021549698 A JP 2021549698A JP 2021549698 A JP2021549698 A JP 2021549698A JP WO2020176552 A5 JPWO2020176552 A5 JP WO2020176552A5
- Authority
- JP
- Japan
- Prior art keywords
- nucleic acid
- molecule
- grna
- rho
- grna molecule
- 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
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201962810320P | 2019-02-25 | 2019-02-25 | |
| US62/810,320 | 2019-02-25 | ||
| PCT/US2020/019766 WO2020176552A1 (en) | 2019-02-25 | 2020-02-25 | Crispr/rna-guided nuclease-related methods and compositions for treating rho-associated autosomal-dominant retinitis pigmentosa (adrp) |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2022521764A JP2022521764A (ja) | 2022-04-12 |
| JPWO2020176552A5 true JPWO2020176552A5 (enExample) | 2023-03-07 |
Family
ID=69960739
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021549698A Pending JP2022521764A (ja) | 2019-02-25 | 2020-02-25 | Ｒｈｏ関連常染色体優性網膜色素変性症（ａｄｒｐ）を治療するためのｃｒｉｓｐｒ／ｒｎａ誘導ヌクレアーゼ関連の方法及び組成物 |
Country Status (9)
| Country | Link |
|---|---|
| US (1) | US20220133768A1 (enExample) |
| EP (1) | EP3931326A1 (enExample) |
| JP (1) | JP2022521764A (enExample) |
| KR (1) | KR20210133993A (enExample) |
| CN (1) | CN113631710A (enExample) |
| AU (1) | AU2020227740A1 (enExample) |
| CA (1) | CA3130515A1 (enExample) |
| IL (1) | IL285680A (enExample) |
| WO (1) | WO2020176552A1 (enExample) |
Families Citing this family (10)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN113272436B (zh) * | 2018-11-08 | 2024-08-23 | 国立大学法人东海国立大学机构 | 使用利用单一的aav载体的基因组编辑的基因治疗 |
| EP4077674A1 (en) * | 2019-12-18 | 2022-10-26 | Alia Therapeutics S.R.L. | Compositions and methods for treating retinitis pigmentosa |
| US20230399638A1 (en) * | 2020-11-01 | 2023-12-14 | University of South Alabama Foundation for Research and Commercialization | Barcoded Cells Engineered With Heterozygous Genetic Diversity |
| EP4323522A1 (en) * | 2021-04-16 | 2024-02-21 | Editas Medicine, Inc. | Crispr/rna-guided nuclease-related methods and compositions for treating rho-associated autosomal-dominant retinitis pigmentosa (adrp) |
| WO2023285431A1 (en) * | 2021-07-12 | 2023-01-19 | Alia Therapeutics Srl | Compositions and methods for allele specific treatment of retinitis pigmentosa |
| WO2024036366A1 (en) * | 2022-08-16 | 2024-02-22 | The University Of Adelaide | Agent for treating or preventing a dominantly-inherited disease |
| EP4587564A2 (en) * | 2022-09-16 | 2025-07-23 | Alia Therapeutics Srl | Enqp type ii cas proteins and applications thereof |
| EP4590819A2 (en) * | 2022-09-20 | 2025-07-30 | The Board of Trustees of the Leland Stanford Junior University | Methods and compositions to control gene using genome editing |
| CN116555259B (zh) * | 2023-04-20 | 2024-02-27 | 云舟生物科技(广州)股份有限公司 | 核酸分子及其作为启动子的应用 |
| WO2025090637A2 (en) * | 2023-10-23 | 2025-05-01 | Prime Medicine, Inc. | Genome editing compositions and methods for treatment of retinitis pigmentosa |
Family Cites Families (17)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5443505A (en) | 1993-11-15 | 1995-08-22 | Oculex Pharmaceuticals, Inc. | Biocompatible ocular implants |
| US5725493A (en) | 1994-12-12 | 1998-03-10 | Avery; Robert Logan | Intravitreal medicine delivery |
| US6299895B1 (en) | 1997-03-24 | 2001-10-09 | Neurotech S.A. | Device and method for treating ophthalmic diseases |
| US6416777B1 (en) | 1999-10-21 | 2002-07-09 | Alcon Universal Ltd. | Ophthalmic drug delivery device |
| DE60040876D1 (de) | 1999-10-21 | 2009-01-02 | Alcon Inc | Medikamentenzuführeinrichtung |
| 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 |
| EP3957735A1 (en) | 2014-03-05 | 2022-02-23 | Editas Medicine, Inc. | Crispr/cas-related methods and compositions for treating usher syndrome and retinitis pigmentosa |
| EP3152319A4 (en) * | 2014-06-05 | 2017-12-27 | Sangamo BioSciences, Inc. | Methods and compositions for nuclease design |
| EP3155101B1 (en) * | 2014-06-16 | 2020-01-29 | The Johns Hopkins University | Compositions and methods for the expression of crispr guide rnas using the h1 promoter |
| US20160324987A1 (en) * | 2015-04-15 | 2016-11-10 | Cedars-Sinai Medical Center | Use of crispr/cas9 as in vivo gene therapy to generate targeted genomic disruptions in genes bearing dominant mutations for retinitis pigmentosa |
| EP3289080B1 (en) * | 2015-04-30 | 2021-08-25 | The Trustees of Columbia University in the City of New York | Gene therapy for autosomal dominant diseases |
| WO2017165862A1 (en) | 2016-03-25 | 2017-09-28 | Editas Medicine, Inc. | Systems and methods for treating alpha 1-antitrypsin (a1at) deficiency |
| AU2017293773A1 (en) * | 2016-07-05 | 2019-02-21 | The Johns Hopkins University | CRISPR/Cas9-based compositions and methods for treating retinal degenerations |
| MX2019006475A (es) | 2016-12-05 | 2019-09-26 | Editas Medicine Inc | Sistemas y métodos para dirección con arn guía puntual (arngp) de adn endógeno y fuente. |
| WO2018209158A2 (en) * | 2017-05-10 | 2018-11-15 | Editas Medicine, Inc. | Crispr/rna-guided nuclease systems and methods |
| EP3714055A1 (en) * | 2017-11-21 | 2020-09-30 | CRISPR Therapeutics AG | Materials and methods for treatment of autosomal dominant retinitis pigmentosa |
| JP2021519067A (ja) * | 2018-03-23 | 2021-08-10 | ザ・トラスティーズ・オブ・コロンビア・ユニバーシティ・イン・ザ・シティ・オブ・ニューヨーク | 常染色体優性疾患のための遺伝子編集 |
-
2020
- 2020-02-25 US US17/433,975 patent/US20220133768A1/en active Pending
- 2020-02-25 JP JP2021549698A patent/JP2022521764A/ja active Pending
- 2020-02-25 WO PCT/US2020/019766 patent/WO2020176552A1/en not_active Ceased
- 2020-02-25 KR KR1020217030652A patent/KR20210133993A/ko active Pending
- 2020-02-25 CA CA3130515A patent/CA3130515A1/en active Pending
- 2020-02-25 CN CN202080025160.5A patent/CN113631710A/zh active Pending
- 2020-02-25 AU AU2020227740A patent/AU2020227740A1/en not_active Abandoned
- 2020-02-25 EP EP20714062.5A patent/EP3931326A1/en active Pending
-
2021
- 2021-08-17 IL IL285680A patent/IL285680A/en unknown
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