JP7244424B2 - エンドソーム切断可能なリンカー - Google Patents
エンドソーム切断可能なリンカー Download PDFInfo
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- JP7244424B2 JP7244424B2 JP2019538617A JP2019538617A JP7244424B2 JP 7244424 B2 JP7244424 B2 JP 7244424B2 JP 2019538617 A JP2019538617 A JP 2019538617A JP 2019538617 A JP2019538617 A JP 2019538617A JP 7244424 B2 JP7244424 B2 JP 7244424B2
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- 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
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- 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
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
- A61P25/04—Centrally acting analgesics, e.g. opioids
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P29/00—Non-central analgesic, antipyretic or antiinflammatory agents, e.g. antirheumatic agents; Non-steroidal antiinflammatory drugs [NSAID]
- A61P29/02—Non-central analgesic, antipyretic or antiinflammatory agents, e.g. antirheumatic agents; Non-steroidal antiinflammatory drugs [NSAID] without antiinflammatory effect
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
- A61P31/04—Antibacterial agents
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
- A61P31/10—Antimycotics
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
- A61P31/12—Antivirals
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P33/00—Antiparasitic agents
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P35/00—Antineoplastic agents
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P37/00—Drugs for immunological or allergic disorders
- A61P37/02—Immunomodulators
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- 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
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- 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
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- 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]
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- 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]
- C12N2310/141—MicroRNAs, miRNAs
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- 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
- C12N2310/31—Chemical structure of the backbone
- C12N2310/314—Phosphoramidates
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- 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
- C12N2310/31—Chemical structure of the backbone
- C12N2310/315—Phosphorothioates
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- 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
- C12N2310/31—Chemical structure of the backbone
- C12N2310/318—Chemical structure of the backbone where the PO2 is completely replaced, e.g. MMI or formacetal
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- 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
- C12N2310/31—Chemical structure of the backbone
- C12N2310/318—Chemical structure of the backbone where the PO2 is completely replaced, e.g. MMI or formacetal
- C12N2310/3183—Diol linkers, e.g. glycols or propanediols
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- 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
- C12N2310/3519—Fusion with another nucleic acid
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- 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
- C12N2310/50—Physical structure
- C12N2310/51—Physical structure in polymeric form, e.g. multimers, concatemers
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- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Organic Chemistry (AREA)
- Engineering & Computer Science (AREA)
- General Health & Medical Sciences (AREA)
- Veterinary Medicine (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Medicinal Chemistry (AREA)
- General Chemical & Material Sciences (AREA)
- Genetics & Genomics (AREA)
- Biomedical Technology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Molecular Biology (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- Biochemistry (AREA)
- Oncology (AREA)
- Communicable Diseases (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Neurosurgery (AREA)
- Pain & Pain Management (AREA)
- Neurology (AREA)
- Immunology (AREA)
- Rheumatology (AREA)
- Virology (AREA)
- Tropical Medicine & Parasitology (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicinal Preparation (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Peptides Or Proteins (AREA)
Priority Applications (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2022185398A JP7450008B2 (ja) | 2017-01-18 | 2022-11-21 | エンドソーム切断可能なリンカー |
| JP2024031869A JP2024052946A (ja) | 2017-01-18 | 2024-03-04 | エンドソーム切断可能なリンカー |
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201762447786P | 2017-01-18 | 2017-01-18 | |
| US62/447,786 | 2017-01-18 | ||
| PCT/US2018/014213 WO2018136620A2 (en) | 2017-01-18 | 2018-01-18 | Endosomal cleavable linkers |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022185398A Division JP7450008B2 (ja) | 2017-01-18 | 2022-11-21 | エンドソーム切断可能なリンカー |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2020505032A JP2020505032A (ja) | 2020-02-20 |
| JP2020505032A5 JP2020505032A5 (enExample) | 2021-02-25 |
| JP7244424B2 true JP7244424B2 (ja) | 2023-03-22 |
Family
ID=62908671
Family Applications (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2019538617A Active JP7244424B2 (ja) | 2017-01-18 | 2018-01-18 | エンドソーム切断可能なリンカー |
| JP2022185398A Active JP7450008B2 (ja) | 2017-01-18 | 2022-11-21 | エンドソーム切断可能なリンカー |
| JP2024031869A Pending JP2024052946A (ja) | 2017-01-18 | 2024-03-04 | エンドソーム切断可能なリンカー |
Family Applications After (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022185398A Active JP7450008B2 (ja) | 2017-01-18 | 2022-11-21 | エンドソーム切断可能なリンカー |
| JP2024031869A Pending JP2024052946A (ja) | 2017-01-18 | 2024-03-04 | エンドソーム切断可能なリンカー |
Country Status (6)
| Country | Link |
|---|---|
| US (2) | US11286482B2 (enExample) |
| EP (1) | EP3570892A4 (enExample) |
| JP (3) | JP7244424B2 (enExample) |
| AU (2) | AU2018209934B2 (enExample) |
| CA (1) | CA3049133A1 (enExample) |
| WO (1) | WO2018136620A2 (enExample) |
Cited By (1)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP2024052946A (ja) * | 2017-01-18 | 2024-04-12 | アルニラム ファーマスーティカルズ インコーポレイテッド | エンドソーム切断可能なリンカー |
Families Citing this family (8)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| SG11202010910QA (en) | 2018-05-07 | 2020-12-30 | Alnylam Pharmaceuticals Inc | Extrahepatic delivery |
| CA3138627A1 (en) * | 2019-06-21 | 2020-12-24 | Alexander V. KEL'IN | Structurally defined sirna-dual variable domain immunoglobulin conjugates |
| CA3150715A1 (en) * | 2019-09-10 | 2021-03-18 | Makoto Koizumi | Conjugate of galnac-oligonucleotide for delivery to liver and manufacturing method thereof |
| MX2022010980A (es) | 2020-03-04 | 2022-12-02 | Verve Therapeutics Inc | Composiciones y metodos para el suministro de arn dirigido. |
| WO2022011214A1 (en) | 2020-07-10 | 2022-01-13 | Alnylam Pharmaceuticals, Inc. | Circular sirnas |
| JP2024527584A (ja) | 2021-07-09 | 2024-07-25 | アルナイラム ファーマシューティカルズ, インコーポレイテッド | CNS送達のためのBis-RNAi化合物 |
| US20250304957A1 (en) | 2022-05-13 | 2025-10-02 | Alnylam Pharmaceuticals, Inc. | Single-stranded loop oligonucleotides |
| WO2024238385A2 (en) | 2023-05-12 | 2024-11-21 | Alnylam Pharmaceuticals, Inc. | Single-stranded loop oligonucleotides |
Citations (4)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP2012508284A (ja) | 2008-11-06 | 2012-04-05 | ユニヴァーシティ オブ ワシントン | マルチブロック共重合体 |
| JP2015502931A (ja) | 2011-11-18 | 2015-01-29 | アルナイラム ファーマシューティカルズ, インコーポレイテッドAlnylam Pharmaceuticals, Inc. | 修飾RNAi剤 |
| JP2015536323A (ja) | 2012-10-16 | 2015-12-21 | エンドサイト・インコーポレイテッドEndocyte, Inc. | 非天然アミノ酸を含む薬物送達結合体および使用方法 |
| WO2017015109A1 (en) | 2015-07-17 | 2017-01-26 | Alnylam Pharmaceuticals, Inc. | Multi-targeted single entity conjugates |
Family Cites Families (10)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5519134A (en) | 1994-01-11 | 1996-05-21 | Isis Pharmaceuticals, Inc. | Pyrrolidine-containing monomers and oligomers |
| US20070258986A1 (en) * | 2003-11-19 | 2007-11-08 | Govt of the US as represented by the secretary, | Targeted Delivery System for Bioactive Agents |
| AU2005289588B2 (en) | 2004-09-24 | 2011-12-22 | Alnylam Pharmaceuticals, Inc. | RNAi modulation of ApoB and uses thereof |
| US20060148740A1 (en) * | 2005-01-05 | 2006-07-06 | Prosensa B.V. | Mannose-6-phosphate receptor mediated gene transfer into muscle cells |
| WO2008109105A2 (en) * | 2007-03-06 | 2008-09-12 | Flagship Ventures | Methods and compositions for improved therapeutic effects with sirna |
| AU2009234266B2 (en) | 2008-04-11 | 2015-08-06 | Tekmira Pharmaceuticals Corporation | Site-specific delivery of nucleic acids by combining targeting ligands with endosomolytic components |
| WO2010093788A2 (en) * | 2009-02-11 | 2010-08-19 | Dicerna Pharmaceuticals, Inc. | Multiplex dicer substrate rna interference molecules having joining sequences |
| US10030243B2 (en) * | 2013-07-05 | 2018-07-24 | Bioneer Corporation | Nanoparticle type oligonucleotide structure having high efficiency and method for preparing same |
| 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 |
| AU2018209934B2 (en) * | 2017-01-18 | 2023-11-09 | Alnylam Pharmaceuticals, Inc. | Endosomal cleavable linkers |
-
2018
- 2018-01-18 AU AU2018209934A patent/AU2018209934B2/en active Active
- 2018-01-18 JP JP2019538617A patent/JP7244424B2/ja active Active
- 2018-01-18 US US16/474,776 patent/US11286482B2/en active Active
- 2018-01-18 CA CA3049133A patent/CA3049133A1/en active Pending
- 2018-01-18 WO PCT/US2018/014213 patent/WO2018136620A2/en not_active Ceased
- 2018-01-18 EP EP18741935.3A patent/EP3570892A4/en active Pending
-
2022
- 2022-02-10 US US17/668,917 patent/US20220177885A1/en active Pending
- 2022-11-21 JP JP2022185398A patent/JP7450008B2/ja active Active
-
2024
- 2024-02-07 AU AU2024200743A patent/AU2024200743A1/en active Pending
- 2024-03-04 JP JP2024031869A patent/JP2024052946A/ja active Pending
Patent Citations (4)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP2012508284A (ja) | 2008-11-06 | 2012-04-05 | ユニヴァーシティ オブ ワシントン | マルチブロック共重合体 |
| JP2015502931A (ja) | 2011-11-18 | 2015-01-29 | アルナイラム ファーマシューティカルズ, インコーポレイテッドAlnylam Pharmaceuticals, Inc. | 修飾RNAi剤 |
| JP2015536323A (ja) | 2012-10-16 | 2015-12-21 | エンドサイト・インコーポレイテッドEndocyte, Inc. | 非天然アミノ酸を含む薬物送達結合体および使用方法 |
| WO2017015109A1 (en) | 2015-07-17 | 2017-01-26 | Alnylam Pharmaceuticals, Inc. | Multi-targeted single entity conjugates |
Cited By (1)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP2024052946A (ja) * | 2017-01-18 | 2024-04-12 | アルニラム ファーマスーティカルズ インコーポレイテッド | エンドソーム切断可能なリンカー |
Also Published As
| Publication number | Publication date |
|---|---|
| CA3049133A1 (en) | 2018-07-26 |
| US11286482B2 (en) | 2022-03-29 |
| AU2018209934A1 (en) | 2019-08-15 |
| WO2018136620A3 (en) | 2018-11-08 |
| EP3570892A4 (en) | 2020-11-25 |
| AU2024200743A1 (en) | 2024-04-11 |
| JP2023010836A (ja) | 2023-01-20 |
| WO2018136620A2 (en) | 2018-07-26 |
| JP7450008B2 (ja) | 2024-03-14 |
| AU2018209934B2 (en) | 2023-11-09 |
| JP2020505032A (ja) | 2020-02-20 |
| EP3570892A2 (en) | 2019-11-27 |
| US20190330630A1 (en) | 2019-10-31 |
| JP2024052946A (ja) | 2024-04-12 |
| US20220177885A1 (en) | 2022-06-09 |
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