CN117980315A - 1’-烷基修饰的核糖衍生物及使用方法 - Google Patents
1’-烷基修饰的核糖衍生物及使用方法 Download PDFInfo
- Publication number
- CN117980315A CN117980315A CN202280061450.4A CN202280061450A CN117980315A CN 117980315 A CN117980315 A CN 117980315A CN 202280061450 A CN202280061450 A CN 202280061450A CN 117980315 A CN117980315 A CN 117980315A
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Classifications
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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
- C07H15/00—Compounds containing hydrocarbon or substituted hydrocarbon radicals directly attached to hetero atoms of saccharide radicals
- C07H15/26—Acyclic or carbocyclic radicals, substituted by hetero rings
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
- A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
- A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
- A61K47/54—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic compound
- A61K47/549—Sugars, nucleosides, nucleotides or nucleic acids
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07D—HETEROCYCLIC COMPOUNDS
- C07D307/00—Heterocyclic compounds containing five-membered rings having one oxygen atom as the only ring hetero atom
- C07D307/02—Heterocyclic compounds containing five-membered rings having one oxygen atom as the only ring hetero atom not condensed with other rings
- C07D307/04—Heterocyclic compounds containing five-membered rings having one oxygen atom as the only ring hetero atom not condensed with other rings having no double bonds between ring members or between ring members and non-ring members
- C07D307/18—Heterocyclic compounds containing five-membered rings having one oxygen atom as the only ring hetero atom not condensed with other rings having no double bonds between ring members or between ring members and non-ring members with hetero atoms or with carbon atoms having three bonds to hetero atoms with at the most one bond to halogen, e.g. ester or nitrile radicals, directly attached to ring carbon atoms
- C07D307/20—Oxygen atoms
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07F—ACYCLIC, CARBOCYCLIC OR HETEROCYCLIC COMPOUNDS CONTAINING ELEMENTS OTHER THAN CARBON, HYDROGEN, HALOGEN, OXYGEN, NITROGEN, SULFUR, SELENIUM OR TELLURIUM
- C07F9/00—Compounds containing elements of Groups 5 or 15 of the Periodic Table
- C07F9/02—Phosphorus compounds
- C07F9/547—Heterocyclic compounds, e.g. containing phosphorus as a ring hetero atom
- C07F9/655—Heterocyclic compounds, e.g. containing phosphorus as a ring hetero atom having oxygen atoms, with or without sulfur, selenium, or tellurium atoms, as the only ring hetero atoms
- C07F9/65515—Heterocyclic compounds, e.g. containing phosphorus as a ring hetero atom having oxygen atoms, with or without sulfur, selenium, or tellurium atoms, as the only ring hetero atoms the oxygen atom being part of a five-membered ring
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07F—ACYCLIC, CARBOCYCLIC OR HETEROCYCLIC COMPOUNDS CONTAINING ELEMENTS OTHER THAN CARBON, HYDROGEN, HALOGEN, OXYGEN, NITROGEN, SULFUR, SELENIUM OR TELLURIUM
- C07F9/00—Compounds containing elements of Groups 5 or 15 of the Periodic Table
- C07F9/02—Phosphorus compounds
- C07F9/547—Heterocyclic compounds, e.g. containing phosphorus as a ring hetero atom
- C07F9/6558—Heterocyclic compounds, e.g. containing phosphorus as a ring hetero atom containing at least two different or differently substituted hetero rings neither condensed among themselves nor condensed with a common carbocyclic ring or ring system
- C07F9/65586—Heterocyclic compounds, e.g. containing phosphorus as a ring hetero atom containing at least two different or differently substituted hetero rings neither condensed among themselves nor condensed with a common carbocyclic ring or ring system at least one of the hetero rings does not contain nitrogen as ring hetero atom
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
- C07H13/00—Compounds containing saccharide radicals esterified by carbonic acid or derivatives thereof, or by organic acids, e.g. phosphonic 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
- 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
-
- 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
- C12N2310/35—Nature of the modification
- C12N2310/351—Conjugate
-
- 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
Landscapes
- Chemical & Material Sciences (AREA)
- Health & Medical Sciences (AREA)
- Organic Chemistry (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Molecular Biology (AREA)
- General Health & Medical Sciences (AREA)
- Biochemistry (AREA)
- Genetics & Genomics (AREA)
- Biotechnology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Biomedical Technology (AREA)
- General Engineering & Computer Science (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- Medicinal Chemistry (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Pharmacology & Pharmacy (AREA)
- Epidemiology (AREA)
- Animal Behavior & Ethology (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicinal Preparation (AREA)
- Saccharide Compounds (AREA)
- Peptides Or Proteins (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202163229628P | 2021-08-05 | 2021-08-05 | |
| US63/229,628 | 2021-08-05 | ||
| PCT/US2022/039517 WO2023014938A1 (en) | 2021-08-05 | 2022-08-05 | 1'-alkyl modified ribose derivatives and methods of use |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| CN117980315A true CN117980315A (zh) | 2024-05-03 |
Family
ID=83049985
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CN202280061450.4A Pending CN117980315A (zh) | 2021-08-05 | 2022-08-05 | 1’-烷基修饰的核糖衍生物及使用方法 |
Country Status (10)
| Country | Link |
|---|---|
| US (2) | US12465647B2 (https=) |
| EP (1) | EP4380946A1 (https=) |
| JP (1) | JP7842850B2 (https=) |
| KR (1) | KR20240070519A (https=) |
| CN (1) | CN117980315A (https=) |
| AU (1) | AU2022324471A1 (https=) |
| CA (1) | CA3227661A1 (https=) |
| IL (1) | IL310547A (https=) |
| MX (1) | MX2024001581A (https=) |
| WO (1) | WO2023014938A1 (https=) |
Cited By (1)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2025256544A1 (zh) * | 2024-06-11 | 2025-12-18 | 福建盛迪医药有限公司 | 一种1-氨烷基修饰的呋喃核糖衍生物的制备方法 |
Families Citing this family (12)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP4413015A1 (en) * | 2021-10-05 | 2024-08-14 | Sanegene Bio USA Inc. | Polyhydroxylated cyclopentane derivatives and methods of use |
| KR20250088573A (ko) * | 2022-10-14 | 2025-06-17 | 사네진 바이오 유에스에이 인크. | C3을 표적화하는 소형 간섭 rna 및 그의 용도 |
| MX2025013094A (es) * | 2023-05-03 | 2026-02-03 | Sanegene Bio Usa Inc | Conjugados basados en lipidos para la administracion sistemica en el sistema nervioso central, el sistema nervioso periferico y ocular |
| US20250064939A1 (en) * | 2023-07-24 | 2025-02-27 | Sanegene Bio Usa Inc. | Lipid-based enhancement agent for rna delivery and therapy |
| CN116854754B (zh) | 2023-09-01 | 2023-12-12 | 北京悦康科创医药科技股份有限公司 | 一种含有核糖环或其衍生结构的GalNAc化合物及其寡核苷酸缀合物 |
| CN121263426A (zh) * | 2024-05-09 | 2026-01-02 | 石药集团中奇制药技术(石家庄)有限公司 | GalNAc衍生物及其寡聚核苷酸缀合物 |
| US20250382616A1 (en) * | 2024-06-18 | 2025-12-18 | Sanegene Bio Usa Inc. | Small interfering rna targeting cfb and uses thereof |
| WO2026017140A2 (en) | 2024-07-18 | 2026-01-22 | Sanegene Bio Usa Inc. | Small interfering rna targeting fxi and uses thereof |
| WO2026030554A1 (en) * | 2024-07-31 | 2026-02-05 | Sanegene Bio Usa Inc. | Saccharide-based conjugates for rna delivery and therapy |
| CN118638166B (zh) | 2024-08-15 | 2024-12-17 | 北京悦康科创医药科技股份有限公司 | 含有三氮唑结构的GalNAc化合物及其寡核苷酸缀合物 |
| WO2026041067A2 (en) | 2024-08-20 | 2026-02-26 | Sanegene Bio Usa Inc. | Small interfering rna targeting myostatin (mstn) and uses thereof |
| WO2026046411A1 (en) | 2024-09-02 | 2026-03-05 | Sanegene Bio Usa Inc. | Small interfering rna targeting plasminogen (plg) and uses thereof |
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| US154A (en) | 1837-03-30 | Improvement in machines for cutting the threads of wood-screws | ||
| US4522811A (en) | 1982-07-08 | 1985-06-11 | Syntex (U.S.A.) Inc. | Serial injection of muramyldipeptides and liposomes enhances the anti-infective activity of muramyldipeptides |
| US5453566A (en) | 1986-03-28 | 1995-09-26 | Calgene, Inc. | Antisense regulation of gene expression in plant/cells |
| US4987071A (en) | 1986-12-03 | 1991-01-22 | University Patents, Inc. | RNA ribozyme polymerases, dephosphorylases, restriction endoribonucleases and methods |
| CA1340323C (en) | 1988-09-20 | 1999-01-19 | Arnold E. Hampel | Rna catalyst for cleaving specific rna sequences |
| GB8822492D0 (en) | 1988-09-24 | 1988-10-26 | Considine J | Apparatus for removing tumours from hollow organs of body |
| AU637800B2 (en) | 1989-08-31 | 1993-06-10 | City Of Hope | Chimeric dna-rna catalytic sequences |
| US6365730B1 (en) | 1990-06-19 | 2002-04-02 | Gene Shears Pty. Limited | DNA-Armed ribozymes and minizymes |
| US5789573A (en) | 1990-08-14 | 1998-08-04 | Isis Pharmaceuticals, Inc. | Antisense inhibition of ICAM-1, E-selectin, and CMV IE1/IE2 |
| ES2061416T3 (es) | 1990-10-12 | 1997-03-01 | Max Planck Gesellschaft | Ribozimas modificadas. |
| DE4216134A1 (de) | 1991-06-20 | 1992-12-24 | Europ Lab Molekularbiolog | Synthetische katalytische oligonukleotidstrukturen |
| US5652094A (en) | 1992-01-31 | 1997-07-29 | University Of Montreal | Nucleozymes |
| EP0642589A4 (en) | 1992-05-11 | 1997-05-21 | Ribozyme Pharm Inc | METHOD AND REAGENT TO INHIBIT VIRAL REPLICATION. |
| US6172208B1 (en) * | 1992-07-06 | 2001-01-09 | Genzyme Corporation | Oligonucleotides modified with conjugate groups |
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| US5591317A (en) | 1994-02-16 | 1997-01-07 | Pitts, Jr.; M. Michael | Electrostatic device for water treatment |
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| WO1996011266A2 (en) | 1994-10-05 | 1996-04-18 | Amgen Inc. | Method for inhibiting smooth muscle cell proliferation and oligonucleotides for use therein |
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| US5739119A (en) | 1996-11-15 | 1998-04-14 | Galli; Rachel L. | Antisense oligonucleotides specific for the muscarinic type 2 acetylcholine receptor MRNA |
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| ES2702942T3 (es) | 2003-04-17 | 2019-03-06 | Alnylam Pharmaceuticals Inc | Agentes de ARNi modificados |
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| US20070213292A1 (en) | 2005-08-10 | 2007-09-13 | The Rockefeller University | Chemically modified oligonucleotides for use in modulating micro RNA and uses thereof |
| WO2009073809A2 (en) | 2007-12-04 | 2009-06-11 | Alnylam Pharmaceuticals, Inc. | Carbohydrate conjugates as delivery agents for oligonucleotides |
| CA3131967A1 (en) | 2010-12-29 | 2012-07-05 | F. Hoffman-La Roche Ag | Small molecule conjugates for intracellular delivery of nucleic acids |
| CN107080847A (zh) * | 2011-06-24 | 2017-08-22 | 森彻斯有限公司 | 细胞外靶向药物缀合物 |
| US8577565B2 (en) | 2011-12-16 | 2013-11-05 | GM Global Technology Operations LLC | Limiting branch pressure to a solenoid valve in a fluid circuit |
| AU2013299717B2 (en) * | 2012-08-06 | 2018-06-28 | Alnylam Pharmaceuticals, Inc. | Carbohydrate conjugated RNA agents and process for their preparation |
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| DK3569711T3 (da) * | 2014-12-15 | 2021-02-22 | Dicerna Pharmaceuticals Inc | Ligandmodificerede dobbeltstrengede nukleinsyrer |
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| JP7249080B2 (ja) | 2016-08-23 | 2023-03-30 | ディセルナ ファーマシューティカルズ インコーポレイテッド | 可逆的に修飾されたオリゴヌクレオチドを含む組成物及びその使用 |
| HUE059718T2 (hu) | 2016-09-02 | 2022-12-28 | Dicerna Pharmaceuticals Inc | 4'-foszfát analógok és azokat tartalmazó oligonukleotidok |
| SG11202009877XA (en) * | 2018-04-12 | 2020-11-27 | Wave Life Sciences Ltd | Oligonucleotide compositions and methods of use thereof |
-
2022
- 2022-08-05 US US17/881,935 patent/US12465647B2/en active Active
- 2022-08-05 AU AU2022324471A patent/AU2022324471A1/en active Pending
- 2022-08-05 CN CN202280061450.4A patent/CN117980315A/zh active Pending
- 2022-08-05 WO PCT/US2022/039517 patent/WO2023014938A1/en not_active Ceased
- 2022-08-05 MX MX2024001581A patent/MX2024001581A/es unknown
- 2022-08-05 KR KR1020247007393A patent/KR20240070519A/ko active Pending
- 2022-08-05 JP JP2024507164A patent/JP7842850B2/ja active Active
- 2022-08-05 CA CA3227661A patent/CA3227661A1/en active Pending
- 2022-08-05 EP EP22758376.2A patent/EP4380946A1/en active Pending
- 2022-08-05 IL IL310547A patent/IL310547A/en unknown
-
2025
- 2025-07-28 US US19/282,328 patent/US20260021188A1/en active Pending
Cited By (1)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2025256544A1 (zh) * | 2024-06-11 | 2025-12-18 | 福建盛迪医药有限公司 | 一种1-氨烷基修饰的呋喃核糖衍生物的制备方法 |
Also Published As
| Publication number | Publication date |
|---|---|
| JP7842850B2 (ja) | 2026-04-08 |
| US20260021188A1 (en) | 2026-01-22 |
| IL310547A (en) | 2024-03-01 |
| US20230138928A1 (en) | 2023-05-04 |
| US12465647B2 (en) | 2025-11-11 |
| WO2023014938A1 (en) | 2023-02-09 |
| EP4380946A1 (en) | 2024-06-12 |
| MX2024001581A (es) | 2024-04-18 |
| CA3227661A1 (en) | 2023-02-09 |
| KR20240070519A (ko) | 2024-05-21 |
| JP2024529067A (ja) | 2024-08-01 |
| AU2022324471A1 (en) | 2024-02-15 |
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