JP2023518541A5 - - Google Patents
Info
- Publication number
- JP2023518541A5 JP2023518541A5 JP2022556229A JP2022556229A JP2023518541A5 JP 2023518541 A5 JP2023518541 A5 JP 2023518541A5 JP 2022556229 A JP2022556229 A JP 2022556229A JP 2022556229 A JP2022556229 A JP 2022556229A JP 2023518541 A5 JP2023518541 A5 JP 2023518541A5
- Authority
- JP
- Japan
- Prior art keywords
- sequence
- cells
- grna
- seq
- casx
- 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
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202062991403P | 2020-03-18 | 2020-03-18 | |
| US62/991,403 | 2020-03-18 | ||
| PCT/US2021/022840 WO2021188729A1 (en) | 2020-03-18 | 2021-03-17 | Compositions and methods for the targeting of c9orf72 |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2026059998A Division JP2026131603A (ja) | 2020-03-18 | 2026-04-01 | Ｃ９ｏｒｆ７２の標的化のための組成物及び方法 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2023518541A JP2023518541A (ja) | 2023-05-02 |
| JP2023518541A5 true JP2023518541A5 (enExample) | 2024-03-28 |
| JP7847853B2 JP7847853B2 (ja) | 2026-04-20 |
Family
ID=75478206
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022556229A Active JP7847853B2 (ja) | 2020-03-18 | 2021-03-17 | Ｃ９ｏｒｆ７２の標的化のための組成物及び方法 |
Country Status (12)
| Country | Link |
|---|---|
| US (1) | US20240309344A1 (enExample) |
| EP (1) | EP4121535A1 (enExample) |
| JP (1) | JP7847853B2 (enExample) |
| KR (1) | KR20230002401A (enExample) |
| CN (1) | CN116096885A (enExample) |
| AU (1) | AU2021237633A1 (enExample) |
| BR (1) | BR112022018673A2 (enExample) |
| CA (1) | CA3172178A1 (enExample) |
| CO (1) | CO2022014598A2 (enExample) |
| IL (1) | IL296477A (enExample) |
| MX (1) | MX2022011460A (enExample) |
| WO (1) | WO2021188729A1 (enExample) |
Families Citing this family (18)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| SG11202113253SA (en) | 2019-06-07 | 2021-12-30 | Scribe Therapeutics Inc | Engineered casx systems |
| AU2021392719B2 (en) | 2020-12-03 | 2025-08-14 | Scribe Therapeutics Inc. | Engineered class 2 type v crispr systems |
| GB2603454A (en) | 2020-12-09 | 2022-08-10 | Ucl Business Ltd | Novel therapeutics for the treatment of neurodegenerative disorders |
| AU2022216614B2 (en) | 2021-02-05 | 2026-03-05 | Christiana Care Gene Editing Institute, Inc. | Methods of and compositions for reducing gene expression and/or activity |
| WO2022261150A2 (en) | 2021-06-09 | 2022-12-15 | Scribe Therapeutics Inc. | Particle delivery systems |
| EP4423263A4 (en) * | 2021-10-29 | 2026-03-04 | Mammoth Biosciences Inc | EFFECTOR PROTEINS, COMPOSITIONS, SYSTEMS, DEVICES, KITS AND METHODS FOR THERECOMMENDATION |
| US20250011766A1 (en) * | 2021-11-09 | 2025-01-09 | Prime Medicine, Inc. | Genome Editing Compositions and Methods for Treatment of Amyotrophic Lateral Sclerosis |
| US20250313819A1 (en) * | 2022-05-12 | 2025-10-09 | The Regents Of The University Of California | Therapeutic crispr/cas9 gene editing approaches to the c9orf72 repeat expansion mutation in ipscs |
| WO2023235725A2 (en) * | 2022-05-31 | 2023-12-07 | Regeneron Pharmaceuticals, Inc. | Crispr-based therapeutics for c9orf72 repeat expansion disease |
| EP4536819A1 (en) | 2022-06-07 | 2025-04-16 | Scribe Therapeutics Inc. | Compositions and methods for the targeting of pcsk9 |
| 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 |
| EP4536286A1 (en) * | 2022-06-08 | 2025-04-16 | Scribe Therapeutics Inc. | Aav vectors for gene editing |
| WO2023240157A2 (en) * | 2022-06-08 | 2023-12-14 | Scribe Therapeutics Inc. | Compositions and methods for the targeting of dmd |
| EP4665848A2 (en) * | 2023-02-15 | 2025-12-24 | The Trustees Of Columbia University In The City Of New York | Compositions and methods for treating motor neuron diseases |
| WO2024192291A1 (en) | 2023-03-15 | 2024-09-19 | Renagade Therapeutics Management Inc. | Delivery of gene editing systems and methods of use thereof |
| WO2025024285A1 (en) * | 2023-07-21 | 2025-01-30 | Mammoth Biosciences, Inc. | Compositions for the modification of the human c9orf72 gene |
| EP4771154A2 (en) | 2023-09-01 | 2026-07-08 | Renagade Therapeutics Management Inc. | Gene editing systems, compositions, and methods for treatment of vexas syndrome |
| WO2025174765A1 (en) | 2024-02-12 | 2025-08-21 | Renagade Therapeutics Management Inc. | Lipid nanoparticles comprising coding rna molecules for use in gene editing and as vaccines and therapeutic agents |
Family Cites Families (14)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5143854A (en) | 1989-06-07 | 1992-09-01 | Affymax Technologies N.V. | Large scale photolithographic solid phase synthesis of polypeptides and receptor binding screening thereof |
| US5173414A (en) | 1990-10-30 | 1992-12-22 | Applied Immune Sciences, Inc. | Production of recombinant adeno-associated virus vectors |
| US5412087A (en) | 1992-04-24 | 1995-05-02 | Affymax Technologies N.V. | Spatially-addressable immobilization of oligonucleotides and other biological polymers on surfaces |
| US5695937A (en) | 1995-09-12 | 1997-12-09 | The Johns Hopkins University School Of Medicine | Method for serial analysis of gene expression |
| WO2010075303A1 (en) | 2008-12-23 | 2010-07-01 | The United States Of America, As Represented By The Secretary, Department Of Health And Human Services | Splicing factors with a puf protein rna-binding domain and a splicing effector domain and uses of same |
| US9580714B2 (en) | 2010-11-24 | 2017-02-28 | The University Of Western Australia | Peptides for the specific binding of RNA targets |
| EP3374494A4 (en) | 2015-11-11 | 2019-05-01 | Coda Biotherapeutics, Inc. | CRISPR COMPOSITIONS AND METHODS OF USE THEREOF FOR GENE THERAPY |
| CA3009308A1 (en) * | 2015-12-23 | 2017-06-29 | Chad Albert COWAN | Materials and methods for treatment of amyotrophic lateral sclerosis and/or frontal temporal lobular degeneration |
| JP2019532644A (ja) * | 2016-09-30 | 2019-11-14 | ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア | Ｒｎａ誘導型核酸修飾酵素及びその使用方法 |
| US11773409B2 (en) | 2017-04-21 | 2023-10-03 | The Board Of Trustees Of The Leland Stanford Junior University | CRISPR/Cas 9-mediated integration of polynucleotides by sequential homologous recombination of AAV donor vectors |
| AU2018264996A1 (en) * | 2017-05-09 | 2019-12-05 | University Of Massachusetts | Methods of treating Amyotrophic Lateral Sclerosis (ALS) |
| WO2019030306A1 (en) * | 2017-08-08 | 2019-02-14 | Depixus | ISOLATION AND IN VITRO ENRICHMENT OF NUCLEIC ACIDS USING SITE-SPECIFIC NUCLEASES |
| WO2019084148A1 (en) * | 2017-10-25 | 2019-05-02 | Monsanto Technology Llc | TARGETED RNA GUIDED ENDONUCLEASE ENDONUCLEASE ACTIVITY IN EUKARYOTES |
| SG11202113253SA (en) | 2019-06-07 | 2021-12-30 | Scribe Therapeutics Inc | Engineered casx systems |
-
2021
- 2021-03-17 MX MX2022011460A patent/MX2022011460A/es unknown
- 2021-03-17 BR BR112022018673A patent/BR112022018673A2/pt unknown
- 2021-03-17 JP JP2022556229A patent/JP7847853B2/ja active Active
- 2021-03-17 IL IL296477A patent/IL296477A/en unknown
- 2021-03-17 CN CN202180034169.7A patent/CN116096885A/zh active Pending
- 2021-03-17 CA CA3172178A patent/CA3172178A1/en active Pending
- 2021-03-17 AU AU2021237633A patent/AU2021237633A1/en active Pending
- 2021-03-17 EP EP21718316.9A patent/EP4121535A1/en active Pending
- 2021-03-17 KR KR1020227035510A patent/KR20230002401A/ko active Pending
- 2021-03-17 WO PCT/US2021/022840 patent/WO2021188729A1/en not_active Ceased
-
2022
- 2022-09-16 US US17/932,798 patent/US20240309344A1/en active Pending
- 2022-10-14 CO CONC2022/0014598A patent/CO2022014598A2/es unknown
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