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 PDF

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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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molecule
multitargeting
gene
effector
ligand
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JP2020505032A5 (enExample
JP2020505032A (ja
Inventor
ケイ ナイル,ジャヤプラカシュ
ジー ラジーヴ,カラントッタティル
レンティーニ,スコット
タイレ,クリストファー
マイヤー,マーティン
ズラテフ,イヴァン
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Alnylam Pharmaceuticals Inc
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Priority to JP2022185398A priority Critical patent/JP7450008B2/ja
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    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07HSUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • C07H21/02Compounds 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/04Centrally acting analgesics, e.g. opioids
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P29/00Non-central analgesic, antipyretic or antiinflammatory agents, e.g. antirheumatic agents; Non-steroidal antiinflammatory drugs [NSAID]
    • A61P29/02Non-central analgesic, antipyretic or antiinflammatory agents, e.g. antirheumatic agents; Non-steroidal antiinflammatory drugs [NSAID] without antiinflammatory effect
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/04Antibacterial agents
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/10Antimycotics
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61P31/00Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12Antivirals
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P33/00Antiparasitic agents
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00Antineoplastic agents
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61P37/00Drugs for immunological or allergic disorders
    • A61P37/02Immunomodulators
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/111General methods applicable to biologically active non-coding nucleic acids
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/14Type of nucleic acid interfering nucleic acids [NA]
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
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    • C12N2310/141MicroRNAs, miRNAs
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    • C12N2310/00Structure or type of the nucleic acid
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    • C12N2310/00Structure or type of the nucleic acid
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/31Chemical structure of the backbone
    • C12N2310/318Chemical structure of the backbone where the PO2 is completely replaced, e.g. MMI or formacetal
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/31Chemical structure of the backbone
    • C12N2310/318Chemical structure of the backbone where the PO2 is completely replaced, e.g. MMI or formacetal
    • C12N2310/3183Diol linkers, e.g. glycols or propanediols
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/35Nature of the modification
    • C12N2310/351Conjugate
    • C12N2310/3519Fusion with another nucleic acid
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/50Physical structure
    • C12N2310/51Physical structure in polymeric form, e.g. multimers, concatemers

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  • Health & Medical Sciences (AREA)
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  • 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)
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  • 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)
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  • 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)
JP2019538617A 2017-01-18 2018-01-18 エンドソーム切断可能なリンカー Active JP7244424B2 (ja)

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)

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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

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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

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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)

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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)

* Cited by examiner, † Cited by third party
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)

* Cited by examiner, † Cited by third party
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)

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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

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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

Patent Citations (4)

* Cited by examiner, † Cited by third party
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)

* Cited by examiner, † Cited by third party
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

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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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