CN116490216A - Sarna和mrna调节剂联合治疗sma - Google Patents

Sarna和mrna调节剂联合治疗sma Download PDF

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Publication number
CN116490216A
CN116490216A CN202180058670.7A CN202180058670A CN116490216A CN 116490216 A CN116490216 A CN 116490216A CN 202180058670 A CN202180058670 A CN 202180058670A CN 116490216 A CN116490216 A CN 116490216A
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Prior art keywords
dna
artificial sequence
smn2
rna
sarna
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CN202180058670.7A
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English (en)
Chinese (zh)
Inventor
李龙承
姜武林
吴建成
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Sino American Ruikang Nucleic Acid Technology Nantong Research Institute Co ltd
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Sino American Ruikang Nucleic Acid Technology Nantong Research Institute Co ltd
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Publication of CN116490216A publication Critical patent/CN116490216A/zh
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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
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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
    • 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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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/11—Antisense
    • C—CHEMISTRY; METALLURGY
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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
    • 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
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/322—2'-R Modification
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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
    • C12N2310/3341—5-Methylcytosine
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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
    • C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
    • 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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    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/33—Alteration of splicing

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  • Health & Medical Sciences (AREA)
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  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • 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)
CN202180058670.7A 2020-07-31 2021-07-29 Sarna和mrna调节剂联合治疗sma Pending CN116490216A (zh)

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
CN116490216A true CN116490216A (zh) 2023-07-25

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CN202180058670.7A Pending CN116490216A (zh) 2020-07-31 2021-07-29 Sarna和mrna调节剂联合治疗sma

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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 (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN102665731A (zh) * 2009-06-17 2012-09-12 Isis制药公司 用于在对象中调节smn2剪接的组合物和方法
WO2016180784A1 (en) * 2015-05-08 2016-11-17 Proqr Therapeutics Ii B.V. Improved treatments using oligonucleotides
US20190136192A1 (en) * 2017-11-09 2019-05-09 Cell Medicine, Inc. Mesenchymal stem cell therapy for spinal muscular atrophy
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

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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
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
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
US20200237931A1 (en) * 2019-01-25 2020-07-30 Oxford University Innovation Limited Molecules targeting survival motor neuron 2

Patent Citations (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN102665731A (zh) * 2009-06-17 2012-09-12 Isis制药公司 用于在对象中调节smn2剪接的组合物和方法
CN106983768A (zh) * 2009-06-17 2017-07-28 冷泉港实验室 用于在对象中调节smn2剪接的组合物和方法
WO2016180784A1 (en) * 2015-05-08 2016-11-17 Proqr Therapeutics Ii B.V. Improved treatments using oligonucleotides
US20190136192A1 (en) * 2017-11-09 2019-05-09 Cell Medicine, Inc. Mesenchymal stem cell therapy for spinal muscular atrophy
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

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Title
SITHARA RAMDAS等: "New treatments in spinal muscular atrophy: an overview of currently available data", 《EXPERT OPIN PHARMACOTHER. 》, vol. 21, no. 3, pages 308 *
潘启超等: "《肿瘤药理学及化学治疗学》", 31 December 1989, 广东高等教育出版社, pages: 232 *
盛丹菁等: "《临床袖珍手册》", 31 May 1994, 上海医科大学出版社, pages: 141 *

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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
KR20230049663A (ko) 2023-04-13
IL300287A (en) 2023-04-01
MX2023001349A (es) 2023-02-27
US20230287416A1 (en) 2023-09-14
AU2021318560A1 (en) 2023-03-30

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