JP2023535832A - Ｓａｒｎａとｍｒｎａ調節剤によるｓｍａの併用治療 - Google Patents

Ｓａｒｎａとｍｒｎａ調節剤によるｓｍａの併用治療 Download PDF

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JP2023535832A
JP2023535832A JP2023506316A JP2023506316A JP2023535832A JP 2023535832 A JP2023535832 A JP 2023535832A JP 2023506316 A JP2023506316 A JP 2023506316A JP 2023506316 A JP2023506316 A JP 2023506316A JP 2023535832 A JP2023535832 A JP 2023535832A
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smn2
sarna
mrna
seq
strand
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JP2023535832A5 (enExample
Inventor
ロンチョン リ，
ムーリン カン，
チエンチョン ウー，
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ラクティゲン セラピューティクス
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • 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
    • 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
    • A61K31/33—Heterocyclic compounds
    • A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/495—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with two or more nitrogen atoms as the only ring heteroatoms, e.g. piperazine or tetrazines
    • A61K31/50—Pyridazines; Hydrogenated pyridazines
    • A61K31/501—Pyridazines; Hydrogenated pyridazines not condensed and containing further heterocyclic rings
    • 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
    • A61K31/33—Heterocyclic compounds
    • A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/495—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with two or more nitrogen atoms as the only ring heteroatoms, e.g. piperazine or tetrazines
    • A61K31/505—Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim
    • A61K31/519—Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim ortho- or peri-condensed with heterocyclic rings
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
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    • C12N2310/11—Antisense
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
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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
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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
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/33—Chemical structure of the base
    • C12N2310/334—Modified C
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3521—Methyl
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
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    • C12N2310/3525—MOE, methoxyethoxy
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/353—Nature of the modification linked to the nucleic acid via an atom other than carbon
    • C12N2310/3533—Halogen
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    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/31—Combination therapy
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    • C12N2320/30—Special therapeutic applications
    • C12N2320/33—Alteration of splicing

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  • Molecular Biology (AREA)
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  • General Engineering & Computer Science (AREA)
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  • Physics & Mathematics (AREA)
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  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Developing Agents For Electrophotography (AREA)
  • Electrotherapy Devices (AREA)
  • Control Of El Displays (AREA)
JP2023506316A 2020-07-31 2021-07-29 Ｓａｒｎａとｍｒｎａ調節剤によるｓｍａの併用治療 Pending JP2023535832A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
CN2020106200 2020-07-31
CNPCT/CN2020/106200 2020-07-31
PCT/CN2021/109146 WO2022022617A1 (en) 2020-07-31 2021-07-29 Combinatory treatment of sma with sarna and mrna modulators

Publications (2)

Publication Number Publication Date
JP2023535832A true JP2023535832A (ja) 2023-08-21
JP2023535832A5 JP2023535832A5 (enExample) 2024-08-05

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JP2023506316A Pending JP2023535832A (ja) 2020-07-31 2021-07-29 Ｓａｒｎａとｍｒｎａ調節剤によるｓｍａの併用治療

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US (1) US20230287416A1 (enExample)
EP (1) EP4189091A4 (enExample)
JP (1) JP2023535832A (enExample)
KR (1) KR20230049663A (enExample)
CN (1) CN116490216A (enExample)
AU (1) AU2021318560A1 (enExample)
CA (1) CA3190509A1 (enExample)
IL (1) IL300287A (enExample)
MX (1) MX2023001349A (enExample)
WO (1) WO2022022617A1 (enExample)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2022166849A1 (en) * 2021-02-08 2022-08-11 Ractigen Therapeutics Multi-valent oligonucleotide agent and methods of use thereof
EP4750897A1 (en) * 2023-07-27 2026-06-03 Ractigen Therapeutics Glycerol nucleic acid modified oligonucleotide agents and use thereof

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2020135677A1 (zh) * 2018-12-29 2020-07-02 中美瑞康核酸技术(南通)研究院有限公司 寡聚核酸分子及其应用
US20200237931A1 (en) * 2019-01-25 2020-07-30 Oxford University Innovation Limited Molecules targeting survival motor neuron 2

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KR20220084437A (ko) * 2009-06-17 2022-06-21 바이오젠 엠에이 인코포레이티드 대상에게서 ｓｍｎ２ 스플라이싱을 조정하기 위한 조성물 및 방법
US8729263B2 (en) * 2012-08-13 2014-05-20 Novartis Ag 1,4-disubstituted pyridazine analogs there of and methods for treating SMN-deficiency-related conditions
EP3286318A2 (en) * 2015-04-22 2018-02-28 Mina Therapeutics Limited Sarna compositions and methods of use
GB201507926D0 (en) * 2015-05-08 2015-06-24 Proqr Therapeutics N V Improved treatments using oligonucleotides
WO2017075030A1 (en) * 2015-10-26 2017-05-04 Rana Therapeutics, Inc. Methods and compositions for increasing smn expression
JP2019509272A (ja) * 2016-02-23 2019-04-04 インディアナ ユニバーシティー リサーチ アンド テクノロジー コーポレーションＩｎｄｉａｎａ Ｕｎｉｖｅｒｓｉｔｙ Ｒｅｓｅａｒｃｈ Ａｎｄ Ｔｅｃｈｎｏｌｏｇｙ Ｃｏｒｐｏｒａｔｉｏｎ 脊髄性筋萎縮症の治療のための併用療法
US20190161535A1 (en) * 2016-05-09 2019-05-30 Annexon, Inc. Compositions and methods for treating spinal muscular atrophy
US20190136192A1 (en) * 2017-11-09 2019-05-09 Cell Medicine, Inc. Mesenchymal stem cell therapy for spinal muscular atrophy
CN110959042B (zh) * 2018-04-10 2022-10-18 中美瑞康核酸技术(南通)研究院有限公司 一种新型小激活rna
JP2021534154A (ja) * 2018-08-15 2021-12-09 バイオジェン・エムエイ・インコーポレイテッドBiogen MA Inc. 脊髄性筋萎縮症のための併用療法

Patent Citations (2)

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Publication number Priority date Publication date Assignee Title
WO2020135677A1 (zh) * 2018-12-29 2020-07-02 中美瑞康核酸技术(南通)研究院有限公司 寡聚核酸分子及其应用
US20200237931A1 (en) * 2019-01-25 2020-07-30 Oxford University Innovation Limited Molecules targeting survival motor neuron 2

Non-Patent Citations (2)

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Title
J NEUROCHEM, vol. 153, JPN6025031025, 2019, pages 264 - 275, ISSN: 0005874565 *
NEURON, vol. 93, JPN6025031024, 2017, pages 66 - 79, ISSN: 0005874566 *

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Publication number Publication date
EP4189091A4 (en) 2024-10-02
EP4189091A1 (en) 2023-06-07
WO2022022617A1 (en) 2022-02-03
TW202214255A (zh) 2022-04-16
CA3190509A1 (en) 2022-02-03
KR20230049663A (ko) 2023-04-13
IL300287A (en) 2023-04-01
CN116490216A (zh) 2023-07-25
MX2023001349A (es) 2023-02-27
US20230287416A1 (en) 2023-09-14
AU2021318560A1 (en) 2023-03-30

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