JP7842850B2 - 1’-アルキル修飾リボース誘導体及び使用方法 - Google Patents
1’-アルキル修飾リボース誘導体及び使用方法Info
- Publication number
- JP7842850B2 JP7842850B2 JP2024507164A JP2024507164A JP7842850B2 JP 7842850 B2 JP7842850 B2 JP 7842850B2 JP 2024507164 A JP2024507164 A JP 2024507164A JP 2024507164 A JP2024507164 A JP 2024507164A JP 7842850 B2 JP7842850 B2 JP 7842850B2
- Authority
- JP
- Japan
- Prior art keywords
- alkyl
- optionally substituted
- conjugate
- halogens
- compound
- 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.)
- Active
Links
Classifications
-
- 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
- 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
-
- 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 (3)
| Publication Number | Publication Date |
|---|---|
| JP2024529067A JP2024529067A (ja) | 2024-08-01 |
| JP2024529067A5 JP2024529067A5 (https=) | 2025-08-08 |
| JP7842850B2 true JP7842850B2 (ja) | 2026-04-08 |
Family
ID=83049985
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2024507164A Active JP7842850B2 (ja) | 2021-08-05 | 2022-08-05 | 1’-アルキル修飾リボース誘導体及び使用方法 |
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=) |
Families Citing this family (13)
| 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 |
| 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 |
| 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 |
Citations (3)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP2014523884A (ja) | 2011-06-24 | 2014-09-18 | セントローズ, エルエルシー | 細胞外標的化薬物複合体 |
| WO2015006740A2 (en) | 2013-07-11 | 2015-01-15 | Alnylam Pharmaceuticals, Inc. | Oligonucleotide-ligand conjugates and process for their preparation |
| JP2018500027A (ja) | 2014-12-15 | 2018-01-11 | ダイセルナ ファーマシューティカルズ, インコーポレイテッドDicerna Pharmaceuticals, Inc. | リガンド修飾二本鎖核酸 |
Family Cites Families (35)
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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 |
| EP0786522A2 (en) | 1992-07-17 | 1997-07-30 | Ribozyme Pharmaceuticals, Inc. | Enzymatic RNA molecules for treatment of stenotic conditions |
| IL108367A0 (en) | 1993-01-27 | 1994-04-12 | Hektoen Inst For Medical Resea | Antisense polynzcleotide inhibition of human growth factor-sensitive cancer cells |
| US5591317A (en) | 1994-02-16 | 1997-01-07 | Pitts, Jr.; M. Michael | Electrostatic device for water treatment |
| US5631359A (en) | 1994-10-11 | 1997-05-20 | Ribozyme Pharmaceuticals, Inc. | Hairpin ribozymes |
| WO1996011266A2 (en) | 1994-10-05 | 1996-04-18 | Amgen Inc. | Method for inhibiting smooth muscle cell proliferation and oligonucleotides for use therein |
| US5783683A (en) | 1995-01-10 | 1998-07-21 | Genta Inc. | Antisense oligonucleotides which reduce expression of the FGFRI gene |
| US5747470A (en) | 1995-06-07 | 1998-05-05 | Gen-Probe Incorporated | Method for inhibiting cellular proliferation using antisense oligonucleotides to gp130 mRNA |
| US5763263A (en) | 1995-11-27 | 1998-06-09 | Dehlinger; Peter J. | Method and apparatus for producing position addressable combinatorial libraries |
| US5739119A (en) | 1996-11-15 | 1998-04-14 | Galli; Rachel L. | Antisense oligonucleotides specific for the muscarinic type 2 acetylcholine receptor MRNA |
| CA2518475C (en) | 2003-03-07 | 2014-12-23 | Alnylam Pharmaceuticals, Inc. | Irna agents comprising asymmetrical modifications |
| ES2702942T3 (es) | 2003-04-17 | 2019-03-06 | Alnylam Pharmaceuticals Inc | Agentes de ARNi modificados |
| AU2006279906B2 (en) | 2005-08-10 | 2012-05-10 | Alnylam Pharmaceuticals, Inc. | Chemically modified oligonucleotides for use in modulating micro RNA and uses thereof |
| 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 |
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| AU2013299717B2 (en) * | 2012-08-06 | 2018-06-28 | Alnylam Pharmaceuticals, Inc. | Carbohydrate conjugated RNA agents and process for their preparation |
| MX2018015109A (es) | 2016-06-06 | 2019-04-22 | Arrowhead Pharmaceuticals Inc | Nucleotidos modificados con 5'-ciclo-fosfonato. |
| 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
Patent Citations (3)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP2014523884A (ja) | 2011-06-24 | 2014-09-18 | セントローズ, エルエルシー | 細胞外標的化薬物複合体 |
| WO2015006740A2 (en) | 2013-07-11 | 2015-01-15 | Alnylam Pharmaceuticals, Inc. | Oligonucleotide-ligand conjugates and process for their preparation |
| JP2018500027A (ja) | 2014-12-15 | 2018-01-11 | ダイセルナ ファーマシューティカルズ, インコーポレイテッドDicerna Pharmaceuticals, Inc. | リガンド修飾二本鎖核酸 |
Also Published As
| Publication number | Publication date |
|---|---|
| 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 |
| CN117980315A (zh) | 2024-05-03 |
| AU2022324471A1 (en) | 2024-02-15 |
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