CA3227661A1 - 1'-alkyl modified ribose derivatives and methods of use - Google Patents
1'-alkyl modified ribose derivatives and methods of use Download PDFInfo
- Publication number
- CA3227661A1 CA3227661A1 CA3227661A CA3227661A CA3227661A1 CA 3227661 A1 CA3227661 A1 CA 3227661A1 CA 3227661 A CA3227661 A CA 3227661A CA 3227661 A CA3227661 A CA 3227661A CA 3227661 A1 CA3227661 A1 CA 3227661A1
- Authority
- CA
- Canada
- Prior art keywords
- halogen
- scaffold
- compound
- alkyl
- conjugate
- 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
-
- 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 |
|---|---|
| CA3227661A1 true CA3227661A1 (en) | 2023-02-09 |
Family
ID=83049985
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CA3227661A Pending CA3227661A1 (en) | 2021-08-05 | 2022-08-05 | 1'-alkyl modified ribose derivatives and methods of use |
Country Status (10)
| Country | Link |
|---|---|
| US (2) | US12465647B2 (enExample) |
| EP (1) | EP4380946A1 (enExample) |
| JP (2) | JP7842850B2 (enExample) |
| KR (1) | KR20240070519A (enExample) |
| CN (1) | CN117980315A (enExample) |
| AU (1) | AU2022324471A1 (enExample) |
| CA (1) | CA3227661A1 (enExample) |
| IL (1) | IL310547A (enExample) |
| MX (1) | MX2024001581A (enExample) |
| WO (1) | WO2023014938A1 (enExample) |
Families Citing this family (17)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US20230227480A1 (en) * | 2021-10-05 | 2023-07-20 | Sanegene Bio Usa Inc. | Polyhydroxylated cyclopentane derivatives and methods of use |
| WO2024081954A2 (en) | 2022-10-14 | 2024-04-18 | Sanegene Bio Usa Inc. | Small interfering rna targeting c3 and uses thereof |
| EP4705312A1 (en) * | 2023-05-03 | 2026-03-11 | Sanegene Bio USA Inc. | Lipid-based conjugates for systemic, central nervous system, peripheral nervous system, and ocular delivery |
| AU2024301344A1 (en) * | 2023-07-24 | 2026-01-29 | 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 |
| WO2025143262A1 (ja) | 2023-12-28 | 2025-07-03 | 日産化学株式会社 | カルバモイルエチル修飾を含有する二本鎖rna |
| TW202608456A (zh) * | 2024-05-09 | 2026-03-01 | 大陸商石藥集團中奇製藥技術(石家莊)有限公司 | GalNAc衍生物及其寡聚核苷酸綴合物 |
| 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 |
| WO2025264616A1 (en) * | 2024-06-18 | 2025-12-26 | 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 |
| WO2026092658A2 (en) | 2024-10-31 | 2026-05-07 | Sanegene Bio Usa Inc. | Small interfering rna targeting amyloid beta precursor protein (app) and uses thereof |
| WO2026102144A2 (en) * | 2024-11-06 | 2026-05-15 | Sanegene Bio Usa Inc. | Oligonucleotide conjugates of phosphocholine lipid derivatives |
| US20260226475A1 (en) | 2025-01-27 | 2026-08-06 | Sanegene Bio Usa Inc. | Small interfering rna targeting acvr1c and uses thereof |
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| Publication number | Priority date | Publication date | Assignee | Title |
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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 |
| CA2039718C (en) | 1989-08-31 | 2003-02-25 | John J. Rossi | 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 |
| AU649074B2 (en) | 1990-10-12 | 1994-05-12 | Max-Planck-Gesellschaft Zur Forderung Der Wissenschaften E.V. | Modified ribozymes |
| 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 |
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| US6172208B1 (en) * | 1992-07-06 | 2001-01-09 | Genzyme Corporation | Oligonucleotides modified with conjugate groups |
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| 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 |
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| AU3889595A (en) | 1994-10-05 | 1996-05-02 | Amgen, Inc. | Method for inhibiting smooth muscle cell proliferation and oligonucleotides for use therein |
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| EP2669377A3 (en) | 2003-04-17 | 2015-10-14 | Alnylam Pharmaceuticals Inc. | Modified iRNA agents |
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| EP3865576A1 (en) * | 2014-12-15 | 2021-08-18 | Dicerna Pharmaceuticals, Inc. | Ligand-modified double-stranded nucleic acids |
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| MX2019002075A (es) | 2016-08-23 | 2019-07-01 | Dicerna Pharmaceuticals Inc | Composiciones que comprenden oligonucleotidos modificados reversiblemente, y sus usos. |
| WO2018045317A1 (en) | 2016-09-02 | 2018-03-08 | Dicerna Pharmaceuticals, Inc. | 4'-phosphate analogs and oligonucleotides comprising the same |
| JP2021521140A (ja) * | 2018-04-12 | 2021-08-26 | ウェイブ ライフ サイエンシズ リミテッドWave Life Sciences Ltd. | オリゴヌクレオチド組成物及びその使用方法 |
-
2022
- 2022-08-05 CA CA3227661A patent/CA3227661A1/en active Pending
- 2022-08-05 AU AU2022324471A patent/AU2022324471A1/en active Pending
- 2022-08-05 WO PCT/US2022/039517 patent/WO2023014938A1/en not_active Ceased
- 2022-08-05 KR KR1020247007393A patent/KR20240070519A/ko active Pending
- 2022-08-05 MX MX2024001581A patent/MX2024001581A/es unknown
- 2022-08-05 JP JP2024507164A patent/JP7842850B2/ja active Active
- 2022-08-05 US US17/881,935 patent/US12465647B2/en active Active
- 2022-08-05 IL IL310547A patent/IL310547A/en unknown
- 2022-08-05 CN CN202280061450.4A patent/CN117980315A/zh active Pending
- 2022-08-05 EP EP22758376.2A patent/EP4380946A1/en active Pending
-
2025
- 2025-07-28 US US19/282,328 patent/US20260021188A1/en active Pending
-
2026
- 2026-03-27 JP JP2026054255A patent/JP2026095525A/ja active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| US20260021188A1 (en) | 2026-01-22 |
| WO2023014938A1 (en) | 2023-02-09 |
| JP2024529067A (ja) | 2024-08-01 |
| EP4380946A1 (en) | 2024-06-12 |
| JP2026095525A (ja) | 2026-06-11 |
| JP7842850B2 (ja) | 2026-04-08 |
| AU2022324471A1 (en) | 2024-02-15 |
| US12465647B2 (en) | 2025-11-11 |
| US20230138928A1 (en) | 2023-05-04 |
| IL310547A (en) | 2024-03-01 |
| KR20240070519A (ko) | 2024-05-21 |
| CN117980315A (zh) | 2024-05-03 |
| MX2024001581A (es) | 2024-04-18 |
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