JP2024534502A - オリゴヌクレオチドのインビボ送達に使用するための２’－アルキル又は３’－アルキル修飾リボース誘導体 - Google Patents
オリゴヌクレオチドのインビボ送達に使用するための２’－アルキル又は３’－アルキル修飾リボース誘導体 Download PDFInfo
- Publication number
- JP2024534502A JP2024534502A JP2024517430A JP2024517430A JP2024534502A JP 2024534502 A JP2024534502 A JP 2024534502A JP 2024517430 A JP2024517430 A JP 2024517430A JP 2024517430 A JP2024517430 A JP 2024517430A JP 2024534502 A JP2024534502 A JP 2024534502A
- Authority
- JP
- Japan
- Prior art keywords
- scaffold
- compound
- conjugate
- optionally substituted
- nucleic acid
- 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
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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
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
- C07H19/00—Compounds containing a hetero ring sharing one ring hetero atom with a saccharide radical; Nucleosides; Mononucleotides; Anhydro-derivatives thereof
- C07H19/02—Compounds containing a hetero ring sharing one ring hetero atom with a saccharide radical; Nucleosides; Mononucleotides; Anhydro-derivatives thereof sharing nitrogen
- C07H19/04—Heterocyclic radicals containing only nitrogen atoms as ring hetero atom
- C07H19/06—Pyrimidine radicals
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
- C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
- C07H21/02—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with ribosyl as saccharide radical
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
- C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
- C07H21/04—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with deoxyribosyl as saccharide radical
Landscapes
- Chemical & Material Sciences (AREA)
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Organic Chemistry (AREA)
- Molecular Biology (AREA)
- Biochemistry (AREA)
- Engineering & Computer Science (AREA)
- General Health & Medical Sciences (AREA)
- Biotechnology (AREA)
- Genetics & Genomics (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Animal Behavior & Ethology (AREA)
- Medicinal Chemistry (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Pharmacology & Pharmacy (AREA)
- Epidemiology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Saccharide Compounds (AREA)
- Medicinal Preparation (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202163246870P | 2021-09-22 | 2021-09-22 | |
| US63/246,870 | 2021-09-22 | ||
| PCT/US2022/044377 WO2023049258A1 (en) | 2021-09-22 | 2022-09-22 | 2'-alkyl or 3'- alkyl modified ribose derivatives for use in the in-vivo delivery of oligonucleotides |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2024534502A true JP2024534502A (ja) | 2024-09-20 |
| JP2024534502A5 JP2024534502A5 (https=) | 2025-09-30 |
Family
ID=84053050
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2024517430A Pending JP2024534502A (ja) | 2021-09-22 | 2022-09-22 | オリゴヌクレオチドのインビボ送達に使用するための２’－アルキル又は３’－アルキル修飾リボース誘導体 |
Country Status (9)
| Country | Link |
|---|---|
| US (1) | US20230241224A1 (https=) |
| EP (1) | EP4405368A1 (https=) |
| JP (1) | JP2024534502A (https=) |
| KR (1) | KR20240082361A (https=) |
| CN (1) | CN118076619A (https=) |
| AU (1) | AU2022352676A1 (https=) |
| CA (1) | CA3233113A1 (https=) |
| IL (1) | IL311486A (https=) |
| WO (1) | WO2023049258A1 (https=) |
Families Citing this family (6)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| 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 |
| WO2025144872A2 (en) * | 2023-12-29 | 2025-07-03 | Adarx Pharmaceuticals, Inc. | Nucleoside-derived lipids for drug delivery |
| 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 |
Family Cites Families (36)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| 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 |
| 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. |
| 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 |
| US5801154A (en) | 1993-10-18 | 1998-09-01 | Isis Pharmaceuticals, Inc. | Antisense oligonucleotide modulation of multidrug resistance-associated protein |
| 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 |
| 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 |
| BR112013026345A2 (pt) * | 2011-04-13 | 2019-04-24 | Merck Sharp & Dohe Corp. | composto, composição farmacêutica, uso de um composto, e, método para tratar um paciente infectado com hcv |
| US8577565B2 (en) | 2011-12-16 | 2013-11-05 | GM Global Technology Operations LLC | Limiting branch pressure to a solenoid valve in a fluid circuit |
| WO2015006740A2 (en) | 2013-07-11 | 2015-01-15 | Alnylam Pharmaceuticals, Inc. | Oligonucleotide-ligand conjugates and process for their preparation |
| US10030042B2 (en) * | 2014-03-16 | 2018-07-24 | MiRagen Therapeutics, Inc. | Synthesis of bicyclic nucleosides |
| DK3569711T3 (da) | 2014-12-15 | 2021-02-22 | Dicerna Pharmaceuticals Inc | Ligandmodificerede dobbeltstrengede nukleinsyrer |
| CN106366146A (zh) * | 2015-07-13 | 2017-02-01 | 中科云和（北京）生物医药科技有限公司 | 2’-双烷基取代的核苷类似物及用途 |
| 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 |
| AU2020358016A1 (en) * | 2019-10-02 | 2022-04-21 | Dicerna Pharmaceuticals, Inc. | Chemical modifications of small interfering RNA with minimal fluorine content |
-
2022
- 2022-09-22 EP EP22800011.3A patent/EP4405368A1/en active Pending
- 2022-09-22 IL IL311486A patent/IL311486A/en unknown
- 2022-09-22 CN CN202280068035.1A patent/CN118076619A/zh active Pending
- 2022-09-22 US US17/950,520 patent/US20230241224A1/en active Pending
- 2022-09-22 WO PCT/US2022/044377 patent/WO2023049258A1/en not_active Ceased
- 2022-09-22 CA CA3233113A patent/CA3233113A1/en active Pending
- 2022-09-22 JP JP2024517430A patent/JP2024534502A/ja active Pending
- 2022-09-22 KR KR1020247013107A patent/KR20240082361A/ko active Pending
- 2022-09-22 AU AU2022352676A patent/AU2022352676A1/en active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| IL311486A (en) | 2024-05-01 |
| EP4405368A1 (en) | 2024-07-31 |
| CN118076619A (zh) | 2024-05-24 |
| AU2022352676A1 (en) | 2024-03-14 |
| KR20240082361A (ko) | 2024-06-10 |
| WO2023049258A1 (en) | 2023-03-30 |
| US20230241224A1 (en) | 2023-08-03 |
| CA3233113A1 (en) | 2023-03-30 |
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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: 20250919 |
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| A621 | Written request for application examination |
Free format text: JAPANESE INTERMEDIATE CODE: A621 Effective date: 20250919 |