JPWO2021034985A5 - - Google Patents
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- Publication number
- JPWO2021034985A5 JPWO2021034985A5 JP2022511203A JP2022511203A JPWO2021034985A5 JP WO2021034985 A5 JPWO2021034985 A5 JP WO2021034985A5 JP 2022511203 A JP2022511203 A JP 2022511203A JP 2022511203 A JP2022511203 A JP 2022511203A JP WO2021034985 A5 JPWO2021034985 A5 JP WO2021034985A5
- Authority
- JP
- Japan
- Prior art keywords
- fold
- premrna
- composition
- target protein
- exon
- 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 |
|---|---|---|---|
| US201962888887P | 2019-08-19 | 2019-08-19 | |
| US62/888,887 | 2019-08-19 | ||
| US202063049262P | 2020-07-08 | 2020-07-08 | |
| US63/049,262 | 2020-07-08 | ||
| PCT/US2020/047081 WO2021034985A1 (en) | 2019-08-19 | 2020-08-19 | Compositions and methods for modulating splicing and protein expression |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2022544702A JP2022544702A (ja) | 2022-10-20 |
| JPWO2021034985A5 true JPWO2021034985A5 (zh) | 2023-08-28 |
Family
ID=74659533
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022511203A Pending JP2022544702A (ja) | 2019-08-19 | 2020-08-19 | スプライシングおよびタンパク質発現を調節するための組成物および方法 |
Country Status (11)
| Country | Link |
|---|---|
| US (1) | US20220290142A1 (zh) |
| EP (1) | EP4017979A4 (zh) |
| JP (1) | JP2022544702A (zh) |
| KR (1) | KR20220104677A (zh) |
| CN (1) | CN114746550A (zh) |
| AU (1) | AU2020334067A1 (zh) |
| BR (1) | BR112022002905A2 (zh) |
| CA (1) | CA3147970A1 (zh) |
| IL (1) | IL290595A (zh) |
| MX (1) | MX2022002198A (zh) |
| WO (1) | WO2021034985A1 (zh) |
Families Citing this family (9)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP2022534086A (ja) * | 2019-05-27 | 2022-07-27 | ビジョン ファーマ ピーティーワイ リミテッド | 新規の網膜色素変性処置 |
| US20230174984A1 (en) * | 2020-05-11 | 2023-06-08 | The Florey Indtitute of Neuroscience and Mental Health | Compositions and methods for treating disorders associated with loss-of-function mutations in syngap1 |
| WO2022271699A2 (en) * | 2021-06-21 | 2022-12-29 | Stoke Therapeutics, Inc. | Antisense oligomers for treatment of non-sense mediated rna decay based conditions and diseases |
| WO2023004021A2 (en) * | 2021-07-23 | 2023-01-26 | The Children's Medical Center Corporation | Zinc finger ccch-type containing 14 (zc3h14) mutants and methods of use |
| WO2023163801A1 (en) * | 2022-02-24 | 2023-08-31 | Q-State Biosciences, Inc. | Therapeutics for syngap haploinsufficiency |
| WO2023196841A2 (en) * | 2022-04-05 | 2023-10-12 | The Johns Hopkins University | Methods and materials for treating syngap1-associated neurodevelopmental disorders |
| WO2023212625A1 (en) * | 2022-04-28 | 2023-11-02 | AcuraStem Incorporated | Syf2 antisense oligonucleotides |
| WO2023220727A1 (en) * | 2022-05-13 | 2023-11-16 | The Trustees Of The University Of Pennsylvania | Compositions for treating syngap-1 related neurodevelopmental disorders |
| WO2024005715A1 (en) * | 2022-06-28 | 2024-01-04 | Agency For Science, Technology And Research | Oligonucleotides |
Family Cites Families (13)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US8637478B2 (en) * | 2007-11-13 | 2014-01-28 | Isis Pharmaceuticals, Inc. | Compounds and methods for modulating protein expression |
| CA2735129C (en) * | 2008-11-07 | 2012-06-26 | Centre Hospitalier Universitaire Sainte-Justine | Syngap1 dysfunctions and uses thereof in diagnostic and therapeutic applications for mental retardation |
| CN107109411B (zh) * | 2014-10-03 | 2022-07-01 | 冷泉港实验室 | 核基因输出的定向增加 |
| IL308174A (en) * | 2015-10-09 | 2024-01-01 | Univ Southampton | Gene expression modulation and dysregulated protein expression scanning |
| WO2017106283A1 (en) * | 2015-12-14 | 2017-06-22 | Cold Spring Harbor Laboratory | Compositions and methods for treatment of liver diseases |
| EP3390666A4 (en) * | 2015-12-14 | 2019-08-07 | Cold Spring Harbor Laboratory | COMPOSITIONS AND METHODS FOR TREATING RENAL DISEASES |
| WO2017106375A1 (en) * | 2015-12-14 | 2017-06-22 | Cold Spring Harbor Laboratory | Antisense oligomers for treatment of tuberous sclerosis complex |
| CA3005256A1 (en) * | 2015-12-14 | 2017-06-22 | Cold Spring Harbor Laboratory | Antisense oligomers for treatment of autosomal dominant mental retardation-5 and dravet syndrome |
| CA3005128A1 (en) * | 2015-12-14 | 2017-06-22 | Cold Spring Harbor Laboratory | Compositions and methods for treatment of eye diseases |
| JP2018538287A (ja) * | 2015-12-14 | 2018-12-27 | コールド スプリング ハーバー ラボラトリー | 多発性嚢胞腎の処置のためのアンチセンスオリゴマー |
| CA3043755A1 (en) * | 2016-11-28 | 2018-05-31 | Ptc Therapeutics, Inc. | Methods for modulating rna splicing |
| HUE064635T2 (hu) * | 2017-08-25 | 2024-04-28 | Stoke Therapeutics Inc | Antisensz oligomerek állapotok és betegségek kezelésére |
| GB2610100B (en) * | 2017-10-23 | 2023-08-16 | Stoke Therapeutics Inc | Antisense oligomers for treatment of non-sense mediated RNA decay based conditions and diseases |
-
2020
- 2020-08-19 AU AU2020334067A patent/AU2020334067A1/en active Pending
- 2020-08-19 EP EP20855637.3A patent/EP4017979A4/en active Pending
- 2020-08-19 CN CN202080073358.0A patent/CN114746550A/zh active Pending
- 2020-08-19 MX MX2022002198A patent/MX2022002198A/es unknown
- 2020-08-19 JP JP2022511203A patent/JP2022544702A/ja active Pending
- 2020-08-19 WO PCT/US2020/047081 patent/WO2021034985A1/en unknown
- 2020-08-19 KR KR1020227008965A patent/KR20220104677A/ko unknown
- 2020-08-19 BR BR112022002905A patent/BR112022002905A2/pt unknown
- 2020-08-19 CA CA3147970A patent/CA3147970A1/en active Pending
-
2022
- 2022-02-13 IL IL290595A patent/IL290595A/en unknown
- 2022-02-16 US US17/673,226 patent/US20220290142A1/en active Pending
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