JP2024545052A - 修飾骨格化学を有するスプライススイッチャーアンチセンスオリゴヌクレオチド - Google Patents
修飾骨格化学を有するスプライススイッチャーアンチセンスオリゴヌクレオチド Download PDFInfo
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- JP2024545052A JP2024545052A JP2024532968A JP2024532968A JP2024545052A JP 2024545052 A JP2024545052 A JP 2024545052A JP 2024532968 A JP2024532968 A JP 2024532968A JP 2024532968 A JP2024532968 A JP 2024532968A JP 2024545052 A JP2024545052 A JP 2024545052A
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- C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
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- C12N2310/32—Chemical structure of the sugar
- C12N2310/323—Chemical structure of the sugar modified ring structure
- C12N2310/3231—Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
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- C12N2310/00—Structure or type of the nucleic acid
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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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- C12N2310/34—Spatial arrangement of the modifications
- C12N2310/344—Position-specific modifications, e.g. on every purine, at the 3'-end
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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
- C12N2310/3525—MOE, methoxyethoxy
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- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
- C12N2320/33—Alteration of splicing
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- Animal Behavior & Ethology (AREA)
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- Bioinformatics & Cheminformatics (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Gastroenterology & Hepatology (AREA)
- Veterinary Medicine (AREA)
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- Pharmacology & Pharmacy (AREA)
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- Neurosurgery (AREA)
- Neurology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Saccharide Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicinal Preparation (AREA)
- Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
Applications Claiming Priority (15)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202163285628P | 2021-12-03 | 2021-12-03 | |
| US202163285933P | 2021-12-03 | 2021-12-03 | |
| US202163285786P | 2021-12-03 | 2021-12-03 | |
| US202163285631P | 2021-12-03 | 2021-12-03 | |
| US63/285,631 | 2021-12-03 | ||
| US63/285,933 | 2021-12-03 | ||
| US63/285,786 | 2021-12-03 | ||
| US63/285,628 | 2021-12-03 | ||
| US202263350206P | 2022-06-08 | 2022-06-08 | |
| US63/350,206 | 2022-06-08 | ||
| US202263398987P | 2022-08-18 | 2022-08-18 | |
| US202263398992P | 2022-08-18 | 2022-08-18 | |
| US63/398,992 | 2022-08-18 | ||
| US63/398,987 | 2022-08-18 | ||
| PCT/US2022/051740 WO2023102242A2 (en) | 2021-12-03 | 2022-12-02 | Splice switcher antisense oligonucleotides with modified backbone chemistries |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2024545052A true JP2024545052A (ja) | 2024-12-05 |
| JP2024545052A5 JP2024545052A5 (https=) | 2026-01-19 |
| JPWO2023102242A5 JPWO2023102242A5 (https=) | 2026-01-19 |
Family
ID=86613046
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2024532968A Pending JP2024545052A (ja) | 2021-12-03 | 2022-12-02 | 修飾骨格化学を有するスプライススイッチャーアンチセンスオリゴヌクレオチド |
Country Status (9)
| Country | Link |
|---|---|
| EP (1) | EP4441226A2 (https=) |
| JP (1) | JP2024545052A (https=) |
| KR (1) | KR20240129626A9 (https=) |
| CN (1) | CN119522282A (https=) |
| AU (1) | AU2022402929A1 (https=) |
| CA (1) | CA3241870A1 (https=) |
| IL (1) | IL313141A (https=) |
| MX (1) | MX2024006612A (https=) |
| WO (1) | WO2023102242A2 (https=) |
Families Citing this family (4)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| AU2023356317A1 (en) * | 2022-10-05 | 2025-05-15 | Trace Neuroscience, Inc. | Unc13a antisense oligonucleotides and uses thereof |
| WO2024178223A1 (en) * | 2023-02-24 | 2024-08-29 | Northwestern University | Antisense oligonucleotides for preventing unc13a misplicing |
| GB202315883D0 (en) * | 2023-10-17 | 2023-11-29 | Univ Oxford Innovation Ltd | Method |
| WO2025186619A1 (en) * | 2024-03-05 | 2025-09-12 | Takeda Pharmaceutical Company Limited | Compositions and methods for splicing modulation of unc13a |
Family Cites Families (4)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| AU2003232071A1 (en) * | 2002-05-06 | 2003-11-17 | Genelabs Technologies, Inc. | Nucleoside derivatives for treating hepatitis c virus infection |
| EP3260540A1 (en) * | 2010-11-12 | 2017-12-27 | The General Hospital Corporation | Polycomb-associated non-coding rnas |
| WO2020150290A2 (en) * | 2019-01-14 | 2020-07-23 | President And Fellows Of Harvard College | Methods and compositions for restoring stmn2 levels |
| JP7795914B2 (ja) * | 2019-06-03 | 2026-01-08 | クラリス コーポレーション | 神経学的疾患を処置するためのオリゴヌクレオチドおよびその使用方法 |
-
2022
- 2022-12-02 AU AU2022402929A patent/AU2022402929A1/en active Pending
- 2022-12-02 KR KR1020247021691A patent/KR20240129626A9/ko active Pending
- 2022-12-02 EP EP22902271.0A patent/EP4441226A2/en active Pending
- 2022-12-02 CA CA3241870A patent/CA3241870A1/en active Pending
- 2022-12-02 IL IL313141A patent/IL313141A/en unknown
- 2022-12-02 CN CN202280090925.2A patent/CN119522282A/zh active Pending
- 2022-12-02 JP JP2024532968A patent/JP2024545052A/ja active Pending
- 2022-12-02 WO PCT/US2022/051740 patent/WO2023102242A2/en not_active Ceased
- 2022-12-02 MX MX2024006612A patent/MX2024006612A/es unknown
Also Published As
| Publication number | Publication date |
|---|---|
| EP4441226A2 (en) | 2024-10-09 |
| IL313141A (en) | 2024-07-01 |
| WO2023102242A2 (en) | 2023-06-08 |
| KR20240129626A9 (ko) | 2025-12-10 |
| AU2022402929A1 (en) | 2024-06-13 |
| CA3241870A1 (en) | 2023-06-08 |
| MX2024006612A (es) | 2024-06-12 |
| WO2023102242A3 (en) | 2023-10-12 |
| WO2023102242A9 (en) | 2024-10-31 |
| KR20240129626A (ko) | 2024-08-27 |
| CN119522282A (zh) | 2025-02-25 |
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