IL300287A - Combined treatment of SMA with SARNA and mRNA modulators - Google Patents
Combined treatment of SMA with SARNA and mRNA modulatorsInfo
- Publication number
- IL300287A IL300287A IL300287A IL30028723A IL300287A IL 300287 A IL300287 A IL 300287A IL 300287 A IL300287 A IL 300287A IL 30028723 A IL30028723 A IL 30028723A IL 300287 A IL300287 A IL 300287A
- Authority
- IL
- Israel
- Prior art keywords
- seq
- smn2
- sarna
- mrna
- strand
- Prior art date
Links
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- C12N2310/315—Phosphorothioates
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/32—Chemical structure of the sugar
- C12N2310/321—2'-O-R Modification
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- C12N2310/00—Structure or type of the nucleic acid
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- C12N2310/32—Chemical structure of the sugar
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/33—Chemical structure of the base
- C12N2310/334—Modified C
- C12N2310/3341—5-Methylcytosine
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/35—Nature of the modification
- C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
- C12N2310/3521—Methyl
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/35—Nature of the modification
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- C12N2310/3525—MOE, methoxyethoxy
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/35—Nature of the modification
- C12N2310/353—Nature of the modification linked to the nucleic acid via an atom other than carbon
- C12N2310/3533—Halogen
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- C12N2320/00—Applications; Uses
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- C12N2320/30—Special therapeutic applications
- C12N2320/33—Alteration of splicing
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Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| CN2020106200 | 2020-07-31 | ||
| PCT/CN2021/109146 WO2022022617A1 (en) | 2020-07-31 | 2021-07-29 | Combinatory treatment of sma with sarna and mrna modulators |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| IL300287A true IL300287A (en) | 2023-04-01 |
Family
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Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| IL300287A IL300287A (en) | 2020-07-31 | 2021-07-29 | Combined treatment of SMA with SARNA and mRNA modulators |
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| EP4750897A1 (en) * | 2023-07-27 | 2026-06-03 | Ractigen Therapeutics | Glycerol nucleic acid modified oligonucleotide agents and use thereof |
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| KR20220084437A (ko) * | 2009-06-17 | 2022-06-21 | 바이오젠 엠에이 인코포레이티드 | 대상에게서 ｓｍｎ２ 스플라이싱을 조정하기 위한 조성물 및 방법 |
| US8729263B2 (en) * | 2012-08-13 | 2014-05-20 | Novartis Ag | 1,4-disubstituted pyridazine analogs there of and methods for treating SMN-deficiency-related conditions |
| EP3286318A2 (en) * | 2015-04-22 | 2018-02-28 | Mina Therapeutics Limited | Sarna compositions and methods of use |
| GB201507926D0 (en) * | 2015-05-08 | 2015-06-24 | Proqr Therapeutics N V | Improved treatments using oligonucleotides |
| WO2017075030A1 (en) * | 2015-10-26 | 2017-05-04 | Rana Therapeutics, Inc. | Methods and compositions for increasing smn expression |
| JP2019509272A (ja) * | 2016-02-23 | 2019-04-04 | インディアナ ユニバーシティー リサーチ アンド テクノロジー コーポレーションＩｎｄｉａｎａ Ｕｎｉｖｅｒｓｉｔｙ Ｒｅｓｅａｒｃｈ Ａｎｄ Ｔｅｃｈｎｏｌｏｇｙ Ｃｏｒｐｏｒａｔｉｏｎ | 脊髄性筋萎縮症の治療のための併用療法 |
| US20190161535A1 (en) * | 2016-05-09 | 2019-05-30 | Annexon, Inc. | Compositions and methods for treating spinal muscular atrophy |
| US20190136192A1 (en) * | 2017-11-09 | 2019-05-09 | Cell Medicine, Inc. | Mesenchymal stem cell therapy for spinal muscular atrophy |
| CN110959042B (zh) * | 2018-04-10 | 2022-10-18 | 中美瑞康核酸技术(南通)研究院有限公司 | 一种新型小激活rna |
| JP2021534154A (ja) * | 2018-08-15 | 2021-12-09 | バイオジェン・エムエイ・インコーポレイテッドBiogen MA Inc. | 脊髄性筋萎縮症のための併用療法 |
| MX2021007932A (es) * | 2018-12-29 | 2021-08-16 | Ractigen Therapeutics | Molecula oligomerica de acido nucleico y uso de esta. |
| US20200237931A1 (en) * | 2019-01-25 | 2020-07-30 | Oxford University Innovation Limited | Molecules targeting survival motor neuron 2 |
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- 2021-07-29 IL IL300287A patent/IL300287A/en unknown
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| EP4189091A4 (en) | 2024-10-02 |
| JP2023535832A (ja) | 2023-08-21 |
| EP4189091A1 (en) | 2023-06-07 |
| WO2022022617A1 (en) | 2022-02-03 |
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| CA3190509A1 (en) | 2022-02-03 |
| KR20230049663A (ko) | 2023-04-13 |
| CN116490216A (zh) | 2023-07-25 |
| MX2023001349A (es) | 2023-02-27 |
| US20230287416A1 (en) | 2023-09-14 |
| AU2021318560A1 (en) | 2023-03-30 |
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