JP2024545602A - Ｈｓｄ１７ｂ１３関連二本鎖オリゴヌクレオチド組成物及びそれに関連する方法 - Google Patents
Ｈｓｄ１７ｂ１３関連二本鎖オリゴヌクレオチド組成物及びそれに関連する方法 Download PDFInfo
- Publication number
- JP2024545602A JP2024545602A JP2024529302A JP2024529302A JP2024545602A JP 2024545602 A JP2024545602 A JP 2024545602A JP 2024529302 A JP2024529302 A JP 2024529302A JP 2024529302 A JP2024529302 A JP 2024529302A JP 2024545602 A JP2024545602 A JP 2024545602A
- Authority
- JP
- Japan
- Prior art keywords
- nucleotide
- hsd17b13
- terminal
- guide strand
- double
- 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
Classifications
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- 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
- C12N15/1137—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 enzymes
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/31—Chemical structure of the backbone
- C12N2310/315—Phosphorothioates
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/34—Spatial arrangement of the modifications
- C12N2310/344—Position-specific modifications, e.g. on every purine, at the 3'-end
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2320/00—Applications; Uses
- C12N2320/10—Applications; Uses in screening processes
- C12N2320/11—Applications; Uses in screening processes for the determination of target sites, i.e. of active nucleic acids
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
- C12N2320/32—Special delivery means, e.g. tissue-specific
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y101/00—Oxidoreductases acting on the CH-OH group of donors (1.1)
- C12Y101/01—Oxidoreductases acting on the CH-OH group of donors (1.1) with NAD+ or NADP+ as acceptor (1.1.1)
- C12Y101/01062—17Beta-estradiol 17-dehydrogenase (1.1.1.62)
Landscapes
- Life Sciences & Earth Sciences (AREA)
- Genetics & Genomics (AREA)
- Health & Medical Sciences (AREA)
- Engineering & Computer Science (AREA)
- Biomedical Technology (AREA)
- General Engineering & Computer Science (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Organic Chemistry (AREA)
- Chemical & Material Sciences (AREA)
- Biotechnology (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- Molecular Biology (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Virology (AREA)
- Biophysics (AREA)
- Biochemistry (AREA)
- General Health & Medical Sciences (AREA)
- Physics & Mathematics (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
Applications Claiming Priority (7)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202163264360P | 2021-11-19 | 2021-11-19 | |
| US63/264,360 | 2021-11-19 | ||
| US202263268775P | 2022-03-02 | 2022-03-02 | |
| US63/268,775 | 2022-03-02 | ||
| US202263377482P | 2022-09-28 | 2022-09-28 | |
| US63/377,482 | 2022-09-28 | ||
| PCT/US2022/050371 WO2023091644A2 (en) | 2021-11-19 | 2022-11-18 | Hsd17b13-related double stranded oligonucleotide compositions and methods relating thereto |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2024545602A true JP2024545602A (ja) | 2024-12-10 |
| JP2024545602A5 JP2024545602A5 (https=) | 2025-11-27 |
| JPWO2023091644A5 JPWO2023091644A5 (https=) | 2025-11-27 |
Family
ID=86397758
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2024529302A Pending JP2024545602A (ja) | 2021-11-19 | 2022-11-18 | Ｈｓｄ１７ｂ１３関連二本鎖オリゴヌクレオチド組成物及びそれに関連する方法 |
Country Status (4)
| Country | Link |
|---|---|
| US (1) | US20240384273A1 (https=) |
| EP (1) | EP4433596A4 (https=) |
| JP (1) | JP2024545602A (https=) |
| WO (1) | WO2023091644A2 (https=) |
Families Citing this family (4)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP3899024A4 (en) | 2018-12-21 | 2023-05-31 | Ionis Pharmaceuticals, Inc. | Modulators of hsd17b13 expression |
| US20240352464A1 (en) * | 2023-03-07 | 2024-10-24 | Aligos Therapeutics, Inc. | Modified Short Interfering Nucleic Acid (siNA) Molecules and Uses Thereof |
| CN118773194A (zh) * | 2023-06-16 | 2024-10-15 | 施能康医药科技(苏州)有限公司 | 靶向17-β-羟基类固醇脱氢酶13的核酸及其用途 |
| WO2025184661A1 (en) * | 2024-03-01 | 2025-09-04 | Wave Life Sciences Ltd. | Double stranded oligonucleotide compositions and methods relating thereto |
Family Cites Families (7)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| ES2917473T3 (es) * | 2014-01-16 | 2022-07-08 | Wave Life Sciences Ltd | Diseño quiral |
| IL314915B1 (en) * | 2017-01-23 | 2026-02-01 | Regeneron Pharma | Variants of 17-beta-hydroxysteroid dehydrogenase and their uses |
| US11603532B2 (en) * | 2017-06-02 | 2023-03-14 | Wave Life Sciences Ltd. | Oligonucleotide compositions and methods of use thereof |
| CA3086485A1 (en) * | 2017-12-21 | 2019-06-27 | Alnylam Pharmaceuticals, Inc. | Chirally-enriched double-stranded rna agents |
| JP7507093B2 (ja) * | 2018-03-21 | 2024-06-27 | リジェネロン・ファーマシューティカルズ・インコーポレイテッド | １７β－水酸化ステロイド脱水素酵素１３型（ＨＳＤ１７Ｂ１３）ｉＲＮＡ組成物およびその使用方法 |
| MX2021003077A (es) * | 2018-09-19 | 2021-05-27 | Arrowhead Pharmaceuticals Inc | Agentes de arni para inhibir la expresion de 17beta-hsd tipo 13 (hsd17b13), composiciones de dichos agentes, y metodos de uso. |
| EP3899024A4 (en) * | 2018-12-21 | 2023-05-31 | Ionis Pharmaceuticals, Inc. | Modulators of hsd17b13 expression |
-
2022
- 2022-11-18 JP JP2024529302A patent/JP2024545602A/ja active Pending
- 2022-11-18 WO PCT/US2022/050371 patent/WO2023091644A2/en not_active Ceased
- 2022-11-18 EP EP22896516.6A patent/EP4433596A4/en active Pending
-
2024
- 2024-05-17 US US18/667,824 patent/US20240384273A1/en active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| EP4433596A2 (en) | 2024-09-25 |
| WO2023091644A2 (en) | 2023-05-25 |
| EP4433596A4 (en) | 2026-02-11 |
| WO2023091644A3 (en) | 2024-01-11 |
| US20240384273A1 (en) | 2024-11-21 |
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Legal Events
| Date | Code | Title | Description |
|---|---|---|---|
| A521 | Request for written amendment filed |
Free format text: JAPANESE INTERMEDIATE CODE: A523 Effective date: 20251118 |
|
| A621 | Written request for application examination |
Free format text: JAPANESE INTERMEDIATE CODE: A621 Effective date: 20251118 |