CN119213005A - 用于提高siRNA生物利用度的GALNAC组合物 - Google Patents
用于提高siRNA生物利用度的GALNAC组合物 Download PDFInfo
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- CN119213005A CN119213005A CN202380040964.6A CN202380040964A CN119213005A CN 119213005 A CN119213005 A CN 119213005A CN 202380040964 A CN202380040964 A CN 202380040964A CN 119213005 A CN119213005 A CN 119213005A
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- YFTHZRPMJXBUME-UHFFFAOYSA-N tripropylamine Chemical compound CCCN(CCC)CCC YFTHZRPMJXBUME-UHFFFAOYSA-N 0.000 description 1
- LENZDBCJOHFCAS-UHFFFAOYSA-N tris Chemical compound OCC(N)(CO)CO LENZDBCJOHFCAS-UHFFFAOYSA-N 0.000 description 1
- 125000005455 trithianyl group Chemical group 0.000 description 1
- 238000002211 ultraviolet spectrum Methods 0.000 description 1
- 230000002861 ventricular Effects 0.000 description 1
- 125000000391 vinyl group Chemical group [H]C([*])=C([H])[H] 0.000 description 1
- 229920002554 vinyl polymer Polymers 0.000 description 1
- 239000001993 wax Substances 0.000 description 1
- 238000010626 work up procedure Methods 0.000 description 1
- BPICBUSOMSTKRF-UHFFFAOYSA-N xylazine Chemical compound CC1=CC=CC(C)=C1NC1=NCCCS1 BPICBUSOMSTKRF-UHFFFAOYSA-N 0.000 description 1
- 229960001600 xylazine Drugs 0.000 description 1
- 239000011701 zinc Substances 0.000 description 1
- 229910052725 zinc Inorganic materials 0.000 description 1
Classifications
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- C—CHEMISTRY; METALLURGY
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- 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
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- 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
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- A—HUMAN NECESSITIES
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- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
- A61P1/16—Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
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- A61P3/00—Drugs for disorders of the metabolism
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- 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
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- 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
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- C12N2310/00—Structure or type of the nucleic acid
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- C12N2310/321—2'-O-R Modification
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2310/3521—Methyl
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Applications Claiming Priority (7)
| Application Number | Priority Date | Filing Date | Title |
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| US202263320431P | 2022-03-16 | 2022-03-16 | |
| US63/320,431 | 2022-03-16 | ||
| US202263354359P | 2022-06-22 | 2022-06-22 | |
| US63/354,359 | 2022-06-22 | ||
| US202263430542P | 2022-12-06 | 2022-12-06 | |
| US63/430,542 | 2022-12-06 | ||
| PCT/US2023/064384 WO2023178144A2 (en) | 2022-03-16 | 2023-03-15 | Galnac compositions for improving sirna bioavailability |
Publications (1)
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|---|---|
| CN119213005A true CN119213005A (zh) | 2024-12-27 |
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| CN202380040964.6A Pending CN119213005A (zh) | 2022-03-16 | 2023-03-15 | 用于提高siRNA生物利用度的GALNAC组合物 |
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| EP (1) | EP4493570A2 (https=) |
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| WO2025199466A1 (en) | 2024-03-22 | 2025-09-25 | Purdue Research Foundation | Liver-specific asialoglycoprotein receptor targeting ligands, conjugates comprising same, and related compositions and methods of use |
| WO2025255269A1 (en) * | 2024-06-05 | 2025-12-11 | Empirico Inc. | Treatment of mtres1 related diseases and disorders |
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