JP2022544702A - 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 - Google Patents
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 Download PDFInfo
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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
- C12N15/1138—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 against receptors or cell surface proteins
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- C12N15/09—Recombinant DNA-technology
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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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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/32—Chemical structure of the sugar
- C12N2310/323—Chemical structure of the sugar modified ring structure
- C12N2310/3233—Morpholino-type ring
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/34—Spatial arrangement of the modifications
- C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications
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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
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- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
- C12N2320/33—Alteration of splicing
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- Health & Medical Sciences (AREA)
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- Chemical & Material Sciences (AREA)
- Molecular Biology (AREA)
- Organic Chemistry (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Wood Science & Technology (AREA)
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- General Health & Medical Sciences (AREA)
- Biochemistry (AREA)
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- Pharmacology & Pharmacy (AREA)
- Epidemiology (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
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- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
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 (3)
| Publication Number | Publication Date |
|---|---|
| JP2022544702A true JP2022544702A (ja) | 2022-10-20 |
| JPWO2021034985A5 JPWO2021034985A5 (https=) | 2023-08-28 |
| JP2022544702A5 JP2022544702A5 (https=) | 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 (https=) |
| EP (1) | EP4017979A4 (https=) |
| JP (1) | JP2022544702A (https=) |
| KR (1) | KR20220104677A (https=) |
| CN (1) | CN114746550A (https=) |
| AU (1) | AU2020334067A1 (https=) |
| BR (1) | BR112022002905A2 (https=) |
| CA (1) | CA3147970A1 (https=) |
| IL (1) | IL290595A (https=) |
| MX (1) | MX2022002198A (https=) |
| WO (1) | WO2021034985A1 (https=) |
Cited By (1)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP2022547023A (ja) * | 2019-09-03 | 2022-11-10 | ザ・リージエンツ・オブ・ザ・ユニバーシテイ・オブ・コロラド、ア・ボデイー・コーポレイト | 感染の宿主rnaバイオマーカーの迅速な早期検出及びヒトにおけるcovid-19コロナウイルス感染の早期同定のためのシステム、方法、及び組成物。 |
Families Citing this family (12)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2020237294A1 (en) * | 2019-05-27 | 2020-12-03 | Murdoch University | Novel retinitis pigmentosa treatment |
| AU2021272832A1 (en) * | 2020-05-11 | 2022-12-15 | The Florey Institute Of Neuroscience And Mental Health | Compositions and methods for treating disorders associated with loss-of-function mutations in SYNGAP1 |
| EP4359538A4 (en) * | 2021-06-21 | 2025-09-17 | Stoke Therapeutics Inc | ANTISENSE OLIGOMERS FOR THE TREATMENT OF CONDITIONS AND DISEASES BASED ON THE DEGRADATION OF NONSENSE MRNA |
| 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 |
| US20250230467A1 (en) * | 2022-04-05 | 2025-07-17 | 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 |
| CN119948160A (zh) * | 2022-06-28 | 2025-05-06 | 新加坡科技研究局 | 寡核苷酸 |
| WO2025090633A1 (en) * | 2023-10-23 | 2025-05-01 | Regeneron Pharmaceuticals, Inc. | Compositions and methods comprising small nuclear rna (snrna) for treating genetic epilepsies |
| WO2025111249A2 (en) * | 2023-11-20 | 2025-05-30 | Dana-Farber Cancer Institute, Inc. | Methods, kits and systems for determining sarcomatoid differentiation of renal cell carcinoma and methods for treating based on the same |
| GB202401412D0 (en) * | 2024-02-02 | 2024-03-20 | Harness Therapeutics Ltd | Functional nucleic acid |
Citations (4)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2018098446A1 (en) * | 2016-11-28 | 2018-05-31 | Ptc Therapeutics, Inc | Methods for modulating rna splicing |
| JP2019500345A (ja) * | 2015-12-14 | 2019-01-10 | コールド スプリング ハーバー ラボラトリー | 肝臓病の処置のための組成物および方法 |
| JP2019501892A (ja) * | 2015-12-14 | 2019-01-24 | コールド スプリング ハーバー ラボラトリー | 常染色体優性精神遅滞−5とドラベ症候群の処置のためのアンチセンスオリゴマー |
| WO2019084050A1 (en) * | 2017-10-23 | 2019-05-02 | Stoke Therapeutics, Inc. | ANTISENSE OLIGOMERS FOR THE TREATMENT OF CONDITIONS AND DISEASES BASED ON THE DECLINE OF NON-SENSE MEDIATED MRNA |
Family Cites Families (9)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2009064920A2 (en) * | 2007-11-13 | 2009-05-22 | Isis Pharmaceuticals, Inc. | Compounds and methods for modulating protein expression |
| JP2012507989A (ja) * | 2008-11-07 | 2012-04-05 | センター ホスピタライヤー ユニヴェルシテール サント−ジュスティーヌ | Syngap1機能不全ならびに精神遅滞の診断および治療用途でのその使用 |
| CN107109411B (zh) * | 2014-10-03 | 2022-07-01 | 冷泉港实验室 | 核基因输出的定向增加 |
| EP3359685B1 (en) * | 2015-10-09 | 2026-01-28 | University Of Southampton | Modulation of gene expression for deregulated protein expression |
| 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 |
| WO2017106375A1 (en) * | 2015-12-14 | 2017-06-22 | Cold Spring Harbor Laboratory | Antisense oligomers for treatment of tuberous sclerosis complex |
| EP3390666A4 (en) * | 2015-12-14 | 2019-08-07 | Cold Spring Harbor Laboratory | COMPOSITIONS AND METHODS OF TREATING NURSE DISEASES |
| EP3390634A4 (en) * | 2015-12-14 | 2019-08-14 | Cold Spring Harbor Laboratory | COMPOSITIONS AND METHODS FOR THE TREATMENT OF EYE DISEASES |
| SG11202001590RA (en) * | 2017-08-25 | 2020-03-30 | Stoke Therapeutics Inc | Antisense oligomers for treatment of conditions and diseases |
-
2020
- 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 AU AU2020334067A patent/AU2020334067A1/en active Pending
- 2020-08-19 KR KR1020227008965A patent/KR20220104677A/ko active Pending
- 2020-08-19 JP JP2022511203A patent/JP2022544702A/ja active Pending
- 2020-08-19 MX MX2022002198A patent/MX2022002198A/es unknown
- 2020-08-19 BR BR112022002905A patent/BR112022002905A2/pt unknown
- 2020-08-19 CA CA3147970A patent/CA3147970A1/en active Pending
- 2020-08-19 WO PCT/US2020/047081 patent/WO2021034985A1/en not_active Ceased
-
2022
- 2022-02-13 IL IL290595A patent/IL290595A/en unknown
- 2022-02-16 US US17/673,226 patent/US20220290142A1/en active Pending
Patent Citations (4)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP2019500345A (ja) * | 2015-12-14 | 2019-01-10 | コールド スプリング ハーバー ラボラトリー | 肝臓病の処置のための組成物および方法 |
| JP2019501892A (ja) * | 2015-12-14 | 2019-01-24 | コールド スプリング ハーバー ラボラトリー | 常染色体優性精神遅滞−5とドラベ症候群の処置のためのアンチセンスオリゴマー |
| WO2018098446A1 (en) * | 2016-11-28 | 2018-05-31 | Ptc Therapeutics, Inc | Methods for modulating rna splicing |
| WO2019084050A1 (en) * | 2017-10-23 | 2019-05-02 | Stoke Therapeutics, Inc. | ANTISENSE OLIGOMERS FOR THE TREATMENT OF CONDITIONS AND DISEASES BASED ON THE DECLINE OF NON-SENSE MEDIATED MRNA |
Non-Patent Citations (3)
| Title |
|---|
| BMC MEDICAL GENETICS, vol. 18, no. 62, JPN6025021509, 2017, pages 1 - 7, ISSN: 0005740564 * |
| MOLECULAR GENETICS & GENOMIC MEDICINE, vol. 1, no. 4, JPN6024044829, 2013, pages 246 - 259, ISSN: 0005454491 * |
| NATURE COMMUNICATIONS, vol. 11, no. 3501, JPN6024044828, July 2020 (2020-07-01), pages 1 - 13, ISSN: 0005454492 * |
Cited By (1)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP2022547023A (ja) * | 2019-09-03 | 2022-11-10 | ザ・リージエンツ・オブ・ザ・ユニバーシテイ・オブ・コロラド、ア・ボデイー・コーポレイト | 感染の宿主rnaバイオマーカーの迅速な早期検出及びヒトにおけるcovid-19コロナウイルス感染の早期同定のためのシステム、方法、及び組成物。 |
Also Published As
| Publication number | Publication date |
|---|---|
| BR112022002905A2 (pt) | 2022-07-12 |
| WO2021034985A1 (en) | 2021-02-25 |
| KR20220104677A (ko) | 2022-07-26 |
| IL290595A (en) | 2022-04-01 |
| EP4017979A4 (en) | 2024-03-27 |
| CN114746550A (zh) | 2022-07-12 |
| EP4017979A1 (en) | 2022-06-29 |
| CA3147970A1 (en) | 2021-02-25 |
| AU2020334067A1 (en) | 2022-03-17 |
| US20220290142A1 (en) | 2022-09-15 |
| MX2022002198A (es) | 2022-05-24 |
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