KR20230049663A - saRNA 및 mRNA 조절제들에 의한 SMA의 조합 치료 - Google Patents

saRNA 및 mRNA 조절제들에 의한 SMA의 조합 치료 Download PDF

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KR20230049663A
KR20230049663A KR1020237006996A KR20237006996A KR20230049663A KR 20230049663 A KR20230049663 A KR 20230049663A KR 1020237006996 A KR1020237006996 A KR 1020237006996A KR 20237006996 A KR20237006996 A KR 20237006996A KR 20230049663 A KR20230049663 A KR 20230049663A
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dna
artificial sequence
smn2
rna
sarna
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KR1020237006996A
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Korean (ko)
Inventor
롱쳉 리
무림 강
지안쳉 우
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락티젠 세러퓨틱스
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    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
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    • 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
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    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/33—Heterocyclic compounds
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    • 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
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    • 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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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/35—Nature of the modification
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    • C12N2310/3521—Methyl
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    • C12N2310/35—Nature of the modification
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    • C12N2310/3525—MOE, methoxyethoxy
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    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
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    • C12N2310/3533—Halogen
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    • C12N2320/33—Alteration of splicing

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  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
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  • Medicinal Chemistry (AREA)
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  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Physics & Mathematics (AREA)
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  • Plant Pathology (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Neurology (AREA)
  • 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)
KR1020237006996A 2020-07-31 2021-07-29 saRNA 및 mRNA 조절제들에 의한 SMA의 조합 치료 Pending KR20230049663A (ko)

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 (1)

Publication Number Publication Date
KR20230049663A true KR20230049663A (ko) 2023-04-13

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KR1020237006996A Pending KR20230049663A (ko) 2020-07-31 2021-07-29 saRNA 및 mRNA 조절제들에 의한 SMA의 조합 치료

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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

Family Cites Families (12)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
MX2021007932A (es) * 2018-12-29 2021-08-16 Ractigen Therapeutics Molecula oligomerica de acido nucleico y uso de esta.
US20200237931A1 (en) * 2019-01-25 2020-07-30 Oxford University Innovation Limited Molecules targeting survival motor neuron 2

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Publication number Publication date
EP4189091A4 (en) 2024-10-02
JP2023535832A (ja) 2023-08-21
EP4189091A1 (en) 2023-06-07
WO2022022617A1 (en) 2022-02-03
TW202214255A (zh) 2022-04-16
CA3190509A1 (en) 2022-02-03
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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