JP2024516183A - Ｓｏｓ遺伝子発現を調節するための組成物及び方法 - Google Patents

Ｓｏｓ遺伝子発現を調節するための組成物及び方法 Download PDF

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JP2024516183A
JP2024516183A JP2023565227A JP2023565227A JP2024516183A JP 2024516183 A JP2024516183 A JP 2024516183A JP 2023565227 A JP2023565227 A JP 2023565227A JP 2023565227 A JP2023565227 A JP 2023565227A JP 2024516183 A JP2024516183 A JP 2024516183A
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composition
antisense oligonucleotide
mrna
nucleic acid
sos1
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JP2024516183A5 (https=
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リーシャン チェン
ボハン ジン
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モレキュラー アクシオム エルエルシー
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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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/7105—Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/7115—Nucleic acids or oligonucleotides having modified bases, i.e. other than adenine, guanine, cytosine, uracil or thymine
    • A—HUMAN NECESSITIES
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    • A61K31/712—Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
    • A—HUMAN NECESSITIES
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    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/7125—Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
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    • 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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    • C12N2310/11—Antisense
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/313—Phosphorodithioates
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/322—2'-R Modification
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/323—Chemical structure of the sugar modified ring structure
    • C12N2310/3231—Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/323—Chemical structure of the sugar modified ring structure
    • C12N2310/3233—Morpholino-type ring

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  • Biomedical Technology (AREA)
  • Veterinary Medicine (AREA)
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  • Animal Behavior & Ethology (AREA)
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  • Wood Science & Technology (AREA)
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  • General Engineering & Computer Science (AREA)
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  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
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  • Chemical Kinetics & Catalysis (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Peptides Or Proteins (AREA)
JP2023565227A 2021-04-22 2022-04-22 Ｓｏｓ遺伝子発現を調節するための組成物及び方法 Pending JP2024516183A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202163178420P 2021-04-22 2021-04-22
US63/178,420 2021-04-22
PCT/US2022/026052 WO2022226377A1 (en) 2021-04-22 2022-04-22 Compositions and methods for modulating sos gene expressions

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JP2024516183A true JP2024516183A (ja) 2024-04-12
JP2024516183A5 JP2024516183A5 (https=) 2025-04-30

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US (1) US20240182894A1 (https=)
EP (1) EP4326878A4 (https=)
JP (1) JP2024516183A (https=)
KR (1) KR20240032184A (https=)
CN (1) CN117751190A (https=)
WO (1) WO2022226377A1 (https=)

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP4396349A4 (en) * 2021-09-02 2025-10-22 Molecular Axiom Llc COMPOSITIONS AND METHODS FOR MODULATING NLRP3 OR NLRP1 EXPRESSION
CN119855910A (zh) 2022-07-06 2025-04-18 美国微哲默理有限责任公司 用于治疗胰腺癌的组合物和方法
WO2024138135A2 (en) * 2022-12-22 2024-06-27 Molecular Axiom, Llc Compositions and methods for modulating sos gene expressions
WO2025111565A1 (en) * 2023-11-22 2025-05-30 Empirico Inc. Treatment of sos2 related diseases and disorders

Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20050070490A1 (en) * 2002-06-26 2005-03-31 Chiron Corporation Sos1 inhibitors
WO2010091308A2 (en) * 2009-02-06 2010-08-12 Isis Pharmaceuticals, Inc. Oligomeric compounds and methods
US20110269820A1 (en) * 2010-04-28 2011-11-03 Iowa State University Research Foundation, Inc. Spinal muscular atrophy treatment via targeting smn2 catalytic core
JP2019525918A (ja) * 2016-07-15 2019-09-12 アイオーニス ファーマシューティカルズ， インコーポレーテッドＩｏｎｉｓ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ，Ｉｎｃ． Ｓｍｎ２の調節のための化合物及び方法

Family Cites Families (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2003295600A1 (en) * 2002-11-14 2004-06-15 Dharmacon, Inc. Functional and hyperfunctional sirna
WO2005116204A1 (ja) * 2004-05-11 2005-12-08 Rnai Co., Ltd. Ｒｎａ干渉を生じさせるポリヌクレオチド、および、これを用いた遺伝子発現抑制方法
US7901882B2 (en) * 2006-03-31 2011-03-08 Affymetrix, Inc. Analysis of methylation using nucleic acid arrays
AU2014227824B2 (en) * 2013-03-15 2018-09-27 Dana-Farber Cancer Institute, Inc. Stabilized SOS1 peptides
CA2954791C (en) * 2014-07-14 2025-11-18 The Regents Of The University Of California CRISPR/CAS TRANSCRIPTIONAL MODULATION
US10676792B2 (en) * 2015-04-01 2020-06-09 The Regents Of The University Of California Prognostic and diagnostic methods for colorectal cancer
CN119855910A (zh) * 2022-07-06 2025-04-18 美国微哲默理有限责任公司 用于治疗胰腺癌的组合物和方法
WO2024138135A2 (en) * 2022-12-22 2024-06-27 Molecular Axiom, Llc Compositions and methods for modulating sos gene expressions

Patent Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20050070490A1 (en) * 2002-06-26 2005-03-31 Chiron Corporation Sos1 inhibitors
WO2010091308A2 (en) * 2009-02-06 2010-08-12 Isis Pharmaceuticals, Inc. Oligomeric compounds and methods
US20110269820A1 (en) * 2010-04-28 2011-11-03 Iowa State University Research Foundation, Inc. Spinal muscular atrophy treatment via targeting smn2 catalytic core
JP2019525918A (ja) * 2016-07-15 2019-09-12 アイオーニス ファーマシューティカルズ， インコーポレーテッドＩｏｎｉｓ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ，Ｉｎｃ． Ｓｍｎ２の調節のための化合物及び方法

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Publication number Publication date
EP4326878A1 (en) 2024-02-28
EP4326878A4 (en) 2025-07-02
WO2022226377A1 (en) 2022-10-27
US20240182894A1 (en) 2024-06-06
CN117751190A (zh) 2024-03-22
KR20240032184A (ko) 2024-03-11

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