CA3152529A1 - Asialoglycoprotein receptor mediated delivery of therapeutically active conjugates - Google Patents
Asialoglycoprotein receptor mediated delivery of therapeutically active conjugates Download PDFInfo
- Publication number
- CA3152529A1 CA3152529A1 CA3152529A CA3152529A CA3152529A1 CA 3152529 A1 CA3152529 A1 CA 3152529A1 CA 3152529 A CA3152529 A CA 3152529A CA 3152529 A CA3152529 A CA 3152529A CA 3152529 A1 CA3152529 A1 CA 3152529A1
- Authority
- CA
- Canada
- Prior art keywords
- alkyl
- nhac
- group
- phosphate
- pharmaceutical composition
- 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
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- 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
- 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
- A61K47/56—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 macromolecular compound, e.g. an oligomeric, polymeric or dendrimeric molecule
-
- 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
- C07H15/02—Acyclic radicals, not substituted by cyclic structures
- C07H15/04—Acyclic radicals, not substituted by cyclic structures attached to an oxygen atom of the saccharide radical
-
- 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
- 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
- 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
- C12N15/111—General methods applicable to biologically active non-coding nucleic 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
- 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
- C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
-
- 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
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/35—Nature of the modification
- C12N2310/351—Conjugate
- C12N2310/3515—Lipophilic moiety, e.g. cholesterol
-
- 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
-
- 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
- C12N2330/00—Production
- C12N2330/30—Production chemically synthesised
Landscapes
- Health & Medical Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Organic Chemistry (AREA)
- Molecular Biology (AREA)
- Biomedical Technology (AREA)
- Biotechnology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- General Health & Medical Sciences (AREA)
- Biochemistry (AREA)
- General Engineering & Computer Science (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- Physics & Mathematics (AREA)
- Plant Pathology (AREA)
- Biophysics (AREA)
- Microbiology (AREA)
- Pharmacology & Pharmacy (AREA)
- Medicinal Chemistry (AREA)
- Epidemiology (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Crystallography & Structural Chemistry (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic 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 |
|---|---|---|---|
| US201962895417P | 2019-09-03 | 2019-09-03 | |
| US62/895,417 | 2019-09-03 | ||
| PCT/US2020/049261 WO2021046260A1 (en) | 2019-09-03 | 2020-09-03 | Asialoglycoprotein receptor mediated delivery of therapeutically active conjugates |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| CA3152529A1 true CA3152529A1 (en) | 2021-03-11 |
Family
ID=74680487
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CA3152529A Pending CA3152529A1 (en) | 2019-09-03 | 2020-09-03 | Asialoglycoprotein receptor mediated delivery of therapeutically active conjugates |
Country Status (9)
| Country | Link |
|---|---|
| US (1) | US12290567B2 (https=) |
| EP (1) | EP4025252A4 (https=) |
| JP (1) | JP7672394B2 (https=) |
| KR (1) | KR20220058578A (https=) |
| CN (1) | CN114929282B (https=) |
| AU (1) | AU2020340987B2 (https=) |
| CA (1) | CA3152529A1 (https=) |
| IL (1) | IL290766B2 (https=) |
| WO (1) | WO2021046260A1 (https=) |
Families Citing this family (12)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2021257916A1 (en) * | 2020-06-18 | 2021-12-23 | Arcturus Therapeutics, Inc. | Una oligomers for the treatment of polyglutamine diseases |
| US20230227826A1 (en) * | 2020-06-18 | 2023-07-20 | Arcturus Therapeutics, Inc. | Una oligomers for the treatment of polyglutamine diseases |
| US20240398940A1 (en) | 2021-09-03 | 2024-12-05 | CureVac SE | Novel lipid nanoparticles for delivery of nucleic acids |
| EP4422698A1 (en) | 2021-10-29 | 2024-09-04 | CureVac SE | Improved circular rna for expressing therapeutic proteins |
| US20250099614A1 (en) | 2022-01-28 | 2025-03-27 | CureVac SE | Nucleic acid encoded transcription factor inhibitors |
| US20250345407A1 (en) | 2022-05-25 | 2025-11-13 | CureVac SE | Nucleic acid based vaccine encoding an escherichia coli fimh antigenic polypeptide |
| EP4608442A1 (en) | 2022-10-28 | 2025-09-03 | GlaxoSmithKline Biologicals S.A. | Nucleic acid based vaccine |
| WO2024104386A1 (zh) * | 2022-11-16 | 2024-05-23 | 南京明德新药研发有限公司 | 一类含七元杂环的三齿缀合基团 |
| WO2024184500A1 (en) | 2023-03-08 | 2024-09-12 | CureVac SE | Novel lipid nanoparticle formulations for delivery of nucleic acids |
| WO2024230934A1 (en) | 2023-05-11 | 2024-11-14 | CureVac SE | Therapeutic nucleic acid for the treatment of ophthalmic diseases |
| WO2025049634A1 (en) * | 2023-08-29 | 2025-03-06 | Ganna Bio, Inc. | Glycan conjugate compositions and methods |
| CN117466959A (zh) * | 2023-10-30 | 2024-01-30 | 烟台药物研究所 | 一种肝靶向化合物、缀合物及应用 |
Family Cites Families (24)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2009073809A2 (en) | 2007-12-04 | 2009-06-11 | Alnylam Pharmaceuticals, Inc. | Carbohydrate conjugates as delivery agents for oligonucleotides |
| CA2721183C (en) * | 2008-04-11 | 2019-07-16 | Alnylam Pharmaceuticals, Inc. | Site-specific delivery of nucleic acids by combining targeting ligands with endosomolytic components |
| WO2013033230A1 (en) | 2011-08-29 | 2013-03-07 | Isis Pharmaceuticals, Inc. | Oligomer-conjugate complexes and their use |
| AU2013299717B2 (en) * | 2012-08-06 | 2018-06-28 | Alnylam Pharmaceuticals, Inc. | Carbohydrate conjugated RNA agents and process for their preparation |
| DK2992098T3 (da) | 2013-05-01 | 2019-06-17 | Ionis Pharmaceuticals Inc | Sammensætninger og fremgangsmåder til modulering af hbv- og ttr-ekspression |
| EP3564374A1 (en) | 2013-06-21 | 2019-11-06 | Ionis Pharmaceuticals, Inc. | Compositions and methods for modulation of target nucleic acids |
| WO2015042447A1 (en) * | 2013-09-20 | 2015-03-26 | Isis Pharmaceuticals, Inc. | Targeted therapeutic nucleosides and their use |
| CA2935426C (en) | 2014-01-30 | 2023-07-25 | F. Hoffmann-La Roche Ag | Polyoligomer compound with biocleavable conjugates for reducing or inhibiting expression of a nucleic acid target |
| US10513710B2 (en) | 2014-04-18 | 2019-12-24 | University Of Massachusetts | Exosomal loading using hydrophobically modified oligonucleotides |
| US10098959B2 (en) | 2014-05-01 | 2018-10-16 | Ionis Pharmaceuticals, Inc. | Method for synthesis of reactive conjugate clusters |
| EA036496B1 (ru) | 2014-05-01 | 2020-11-17 | Ионис Фармасьютикалз, Инк. | Конъюгированные олигонуклеотиды для модулирования экспрессии фактора комплемента b |
| BR112016022742B1 (pt) | 2014-05-01 | 2022-06-14 | Ionis Pharmaceuticals, Inc | Composto químico, composição compreendendo o composto e uso dos mesmos |
| US10588980B2 (en) | 2014-06-23 | 2020-03-17 | Novartis Ag | Fatty acids and their use in conjugation to biomolecules |
| DK3569711T3 (da) | 2014-12-15 | 2021-02-22 | Dicerna Pharmaceuticals Inc | Ligandmodificerede dobbeltstrengede nukleinsyrer |
| US9834510B2 (en) | 2015-12-30 | 2017-12-05 | Arcturus Therapeutics, Inc. | Aromatic ionizable cationic lipid |
| CA3011946A1 (en) | 2016-03-07 | 2017-09-14 | Arrowhead Pharmaceuticals, Inc. | Targeting ligands for therapeutic compounds |
| EP3228326A1 (en) | 2016-04-05 | 2017-10-11 | Silence Therapeutics GmbH | Nucleic acid linked to a trivalent glycoconjugate |
| WO2018013525A1 (en) * | 2016-07-11 | 2018-01-18 | Translate Bio Ma, Inc. | Nucleic acid conjugates and uses thereof |
| SG11201901841TA (en) * | 2016-09-02 | 2019-03-28 | Arrowhead Pharmaceuticals Inc | Targeting ligands |
| US11400161B2 (en) | 2016-10-06 | 2022-08-02 | Ionis Pharmaceuticals, Inc. | Method of conjugating oligomeric compounds |
| US10383952B2 (en) | 2016-12-21 | 2019-08-20 | Arcturus Therapeutics, Inc. | Ionizable cationic lipid for RNA delivery |
| JP7420727B2 (ja) | 2018-02-17 | 2024-01-23 | アローヘッド ファーマシューティカルズ インコーポレイテッド | トリアルキン結合剤及び使用方法 |
| US20230227826A1 (en) * | 2020-06-18 | 2023-07-20 | Arcturus Therapeutics, Inc. | Una oligomers for the treatment of polyglutamine diseases |
| WO2021257916A1 (en) * | 2020-06-18 | 2021-12-23 | Arcturus Therapeutics, Inc. | Una oligomers for the treatment of polyglutamine diseases |
-
2020
- 2020-09-03 JP JP2022514209A patent/JP7672394B2/ja active Active
- 2020-09-03 US US17/011,880 patent/US12290567B2/en active Active
- 2020-09-03 CA CA3152529A patent/CA3152529A1/en active Pending
- 2020-09-03 KR KR1020227010731A patent/KR20220058578A/ko active Pending
- 2020-09-03 IL IL290766A patent/IL290766B2/en unknown
- 2020-09-03 AU AU2020340987A patent/AU2020340987B2/en active Active
- 2020-09-03 WO PCT/US2020/049261 patent/WO2021046260A1/en not_active Ceased
- 2020-09-03 CN CN202080074997.9A patent/CN114929282B/zh active Active
- 2020-09-03 EP EP20860366.2A patent/EP4025252A4/en active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| EP4025252A4 (en) | 2023-10-18 |
| WO2021046260A1 (en) | 2021-03-11 |
| AU2020340987B2 (en) | 2026-02-12 |
| KR20220058578A (ko) | 2022-05-09 |
| IL290766B1 (en) | 2025-11-01 |
| IL290766B2 (en) | 2026-03-01 |
| JP2022546557A (ja) | 2022-11-04 |
| CN114929282A (zh) | 2022-08-19 |
| JP7672394B2 (ja) | 2025-05-07 |
| US20210060168A1 (en) | 2021-03-04 |
| IL290766A (en) | 2022-04-01 |
| US12290567B2 (en) | 2025-05-06 |
| EP4025252A1 (en) | 2022-07-13 |
| CN114929282B (zh) | 2025-09-19 |
| AU2020340987A1 (en) | 2022-04-14 |
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