JP2024533163A5 - - Google Patents
Info
- Publication number
- JP2024533163A5 JP2024533163A5 JP2024513942A JP2024513942A JP2024533163A5 JP 2024533163 A5 JP2024533163 A5 JP 2024533163A5 JP 2024513942 A JP2024513942 A JP 2024513942A JP 2024513942 A JP2024513942 A JP 2024513942A JP 2024533163 A5 JP2024533163 A5 JP 2024533163A5
- Authority
- JP
- Japan
- Prior art keywords
- rna
- esnrna
- nucleic acid
- molecule
- composition
- 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 (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202163240428P | 2021-09-03 | 2021-09-03 | |
| US63/240,428 | 2021-09-03 | ||
| US202263345660P | 2022-05-25 | 2022-05-25 | |
| US63/345,660 | 2022-05-25 | ||
| PCT/US2022/042486 WO2023034582A1 (en) | 2021-09-03 | 2022-09-02 | Rna editing via recruitment of spliceosome components |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2024533163A JP2024533163A (ja) | 2024-09-12 |
| JP2024533163A5 true JP2024533163A5 (https=) | 2025-10-06 |
Family
ID=85412903
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2024513942A Pending JP2024533163A (ja) | 2021-09-03 | 2022-09-02 | スプライソソーム構成要素のリクルートを介したｒｎａ編集 |
Country Status (9)
| Country | Link |
|---|---|
| US (2) | US12152239B2 (https=) |
| EP (1) | EP4396350A4 (https=) |
| JP (1) | JP2024533163A (https=) |
| KR (1) | KR20240052034A (https=) |
| AU (1) | AU2022337146A1 (https=) |
| CA (1) | CA3230419A1 (https=) |
| IL (1) | IL311101A (https=) |
| MX (1) | MX2024002647A (https=) |
| WO (1) | WO2023034582A1 (https=) |
Families Citing this family (5)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2022220968A1 (en) * | 2021-04-15 | 2022-10-20 | U1 Bio, Inc. | High efficiency trans-splicing for replacement of targeted rna sequences in human cells |
| AU2022337146A1 (en) | 2021-09-03 | 2024-03-14 | Tacit Therapeutics, Inc. | Rna editing via recruitment of spliceosome components |
| EP4522758A4 (en) | 2022-05-13 | 2026-05-06 | Ascidian Therapeutics Inc | Abca4 trans-splicing molecules |
| WO2025085691A1 (en) | 2023-10-18 | 2025-04-24 | Tacit Therapeutics, Inc. | Trans-splicing nucleic acid molecules and methods of use |
| WO2025147110A1 (ko) * | 2024-01-02 | 2025-07-10 | 알지노믹스 주식회사 | Mecp2 전사체 특이적 트랜스-스플라이싱 리보자임 및 이의 용도 |
Family Cites Families (32)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US20020193580A1 (en) | 1995-12-15 | 2002-12-19 | Mitchell Lloyd G. | Methods and compositions for use in spliceosome mediated RNA trans-splicing |
| IT1283876B1 (it) * | 1996-01-12 | 1998-05-07 | Univ Roma | Molecole chimeriche ribozima-snrna ad attivita' catalitica per rna a localizzazione nucleare |
| US20090148906A1 (en) | 1998-09-29 | 2009-06-11 | Shire Human Genetic Therapies, Inc. A Delaware Corporation | Optimized messenger rna |
| US20040214263A1 (en) * | 2002-03-20 | 2004-10-28 | Mitchell Lloyd G. | Spliceosome mediated RNA trans-splicing |
| FR2874384B1 (fr) * | 2004-08-17 | 2010-07-30 | Genethon | Vecteur viral adeno-associe pour realiser du saut d'exons dans un gene codant une proteine a domaines dispensables |
| EP2151248A1 (en) | 2008-07-30 | 2010-02-10 | Johann Bauer | Improved pre-mRNA trans-splicing molecule (RTM) molecules and their uses |
| US9303078B2 (en) | 2009-10-08 | 2016-04-05 | Association Institut De Myologie | Nucleic acid molecules and methods for exchanging exon(s) by transsplicing |
| US8802642B2 (en) | 2010-04-28 | 2014-08-12 | Iowa State University Research Foundation, Inc. | Spinal muscular atrophy treatment via targeting SMN2 catalytic core |
| IT1400425B1 (it) * | 2010-06-08 | 2013-05-31 | Amsterdam Molecular Therapeutics Bv | Modified snrnas for use in therapy. |
| IT1405793B1 (it) * | 2010-10-15 | 2014-01-24 | Univ Ferrara | Molecola di u1snrna umano modificato, gene codificante per la molecola di u1snrna umano modificato, vettore di espressione includente il gene, e loro uso in terapia genica |
| GB201219762D0 (en) | 2012-11-02 | 2012-12-19 | Bauer Johann | A RNA trans-splicing molecule (RTM) for use in the treatment of cancer |
| MX388221B (es) | 2013-04-20 | 2025-03-19 | Res Institute At Nationwide Children´S Hospital | ADMINISTRACIÓN DE VIRUS ADENO-ASOCIADO RECOMBINANTE DE CONSTRUCCIONES DE POLINUCLÉOTIDOS U7snRNA DIRIGIDA AL EXÓN 2. |
| CA3005474A1 (en) | 2015-11-19 | 2017-05-26 | The Trustees Of The University Of Pennsylvania | Compositions and methods for correction of heritable ocular disease |
| CA3005968A1 (en) | 2015-11-23 | 2017-06-01 | The Regents Of The University Of California | Tracking and manipulating cellular rna via nuclear delivery of crispr/cas9 |
| PL3386484T3 (pl) | 2015-12-10 | 2022-07-25 | Modernatx, Inc. | Kompozycje i sposoby dostarczania środków terapeutycznych |
| EP3436590A4 (en) * | 2016-04-01 | 2019-12-04 | National University of Singapore | TRANSSPLEISSEN OF RNA (TSRNA) |
| CN110612353A (zh) | 2017-03-03 | 2019-12-24 | 加利福尼亚大学董事会 | 经由抑制性tRNAs和脱氨酶对突变进行RNA靶向 |
| US10476825B2 (en) | 2017-08-22 | 2019-11-12 | Salk Institue for Biological Studies | RNA targeting methods and compositions |
| KR102866133B1 (ko) | 2018-04-17 | 2025-09-30 | 더 트러스티스 오브 더 유니버시티 오브 펜실바니아 | 트랜스-스플라이싱 분자 |
| KR102918544B1 (ko) | 2018-07-12 | 2026-01-27 | 로켓 파마슈티컬스, 리미티드 | 다논병을 치료하기 위한 유전자 요법 벡터 |
| US20220010333A1 (en) | 2018-09-06 | 2022-01-13 | The Regents Of The University Of California | Rna and dna base editing via engineered adar recruitment |
| US20210388351A1 (en) | 2018-09-28 | 2021-12-16 | The Jackson Laboratory | Artificial rna-guided splicing factors |
| WO2020160486A1 (en) | 2019-02-01 | 2020-08-06 | Spark Therapeutics, Inc. | Aav vector treatment methods for late infantile neuronal ceroid lipofuscinosis type 2 |
| WO2020205604A1 (en) | 2019-03-29 | 2020-10-08 | Salk Institute For Biological Studies | High-efficiency reconstitution of rna molecules |
| BR112021020539A2 (pt) | 2019-04-17 | 2022-01-04 | Univ Pennsylvania | Terminador de hélice tripla para trans-splicing de rna eficiente |
| CN115023499A (zh) | 2019-08-16 | 2022-09-06 | 麻省理工学院 | 使用CRISPR/Cas13的靶向反式剪接 |
| WO2021076656A1 (en) | 2019-10-15 | 2021-04-22 | University Of Massachusetts | Rna editor-enhanced rna trans-splicing |
| IL292904A (en) * | 2019-11-11 | 2022-07-01 | Salk Inst For Biological Studi | Compositions and methods for high-efficiency recombination of rna molecules |
| CN115777020A (zh) | 2020-04-22 | 2023-03-10 | 塑造治疗公司 | 使用snrna组分的组合物和方法 |
| CA3177380A1 (en) | 2020-05-26 | 2021-12-02 | Shape Therapeutics Inc. | Compositions and methods for modifying target rnas |
| EP4217010A4 (en) | 2020-09-28 | 2025-01-08 | Tacit Therapeutics, Inc. | TRANS-SPLICING SYSTEM FOR TISSUE-SPECIFIC REPLACEMENT OF RNA SEQUENCES |
| AU2022337146A1 (en) | 2021-09-03 | 2024-03-14 | Tacit Therapeutics, Inc. | Rna editing via recruitment of spliceosome components |
-
2022
- 2022-09-02 AU AU2022337146A patent/AU2022337146A1/en active Pending
- 2022-09-02 MX MX2024002647A patent/MX2024002647A/es unknown
- 2022-09-02 WO PCT/US2022/042486 patent/WO2023034582A1/en not_active Ceased
- 2022-09-02 EP EP22865613.8A patent/EP4396350A4/en active Pending
- 2022-09-02 KR KR1020247010496A patent/KR20240052034A/ko active Pending
- 2022-09-02 CA CA3230419A patent/CA3230419A1/en active Pending
- 2022-09-02 IL IL311101A patent/IL311101A/en unknown
- 2022-09-02 JP JP2024513942A patent/JP2024533163A/ja active Pending
-
2023
- 2023-07-31 US US18/362,698 patent/US12152239B2/en active Active
-
2024
- 2024-10-10 US US18/911,917 patent/US20250051764A1/en active Pending
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