EA201892467A1 - Олигонуклеотидные аналоги, нацеленные на lmna человека - Google Patents

Олигонуклеотидные аналоги, нацеленные на lmna человека

Info

Publication number
EA201892467A1
EA201892467A1 EA201892467A EA201892467A EA201892467A1 EA 201892467 A1 EA201892467 A1 EA 201892467A1 EA 201892467 A EA201892467 A EA 201892467A EA 201892467 A EA201892467 A EA 201892467A EA 201892467 A1 EA201892467 A1 EA 201892467A1
Authority
EA
Eurasian Patent Office
Prior art keywords
aimed
oligonucleotide analogues
lmna
human lmna
human
Prior art date
Application number
EA201892467A
Other languages
English (en)
Russian (ru)
Inventor
Майкл Р. Эрдос
Кань Цао
Рисзард Коул
Ричард Кит Бествик
Фрэнсис С. Коллинз
Лесли Б. Гордон
Original Assignee
Сарепта Терапьютикс, Инк.
Дзе Юнайтед Стейтс Оф Эмерика, Эз Репрезентед Бай Дзе Секретэри, Депт. Оф Хелт Энд Хьюман Сервисиз
Юниверсити оф Мэриленд
Дзе Проджерия Рисерч Фаундейшн
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Сарепта Терапьютикс, Инк., Дзе Юнайтед Стейтс Оф Эмерика, Эз Репрезентед Бай Дзе Секретэри, Депт. Оф Хелт Энд Хьюман Сервисиз, Юниверсити оф Мэриленд, Дзе Проджерия Рисерч Фаундейшн filed Critical Сарепта Терапьютикс, Инк.
Publication of EA201892467A1 publication Critical patent/EA201892467A1/ru

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    • C—CHEMISTRY; METALLURGY
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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
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • A61P9/10—Drugs for disorders of the cardiovascular system for treating ischaemic or atherosclerotic diseases, e.g. antianginal drugs, coronary vasodilators, drugs for myocardial infarction, retinopathy, cerebrovascula insufficiency, renal arteriosclerosis
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K7/00—Peptides having 5 to 20 amino acids in a fully defined sequence; Derivatives thereof
    • C07K7/04—Linear peptides containing only normal peptide links
    • C07K7/06—Linear peptides containing only normal peptide links having 5 to 11 amino acids
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
    • 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
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/314—Phosphoramidates
    • C12N2310/3145—Phosphoramidates with the nitrogen in 3' or 5'-position
    • 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
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/318—Chemical structure of the backbone where the PO2 is completely replaced, e.g. MMI or formacetal
    • C12N2310/3183—Diol linkers, e.g. glycols or propanediols
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    • 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
    • C12N2310/323—Chemical structure of the sugar modified ring structure
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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
    • 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
    • C12N2310/351—Conjugate
    • 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
    • C12N2310/351—Conjugate
    • C12N2310/3513—Protein; Peptide
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • Medicinal Chemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Biochemistry (AREA)
  • Veterinary Medicine (AREA)
  • Biophysics (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Urology & Nephrology (AREA)
  • Vascular Medicine (AREA)
  • Cardiology (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Epidemiology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
  • Peptides Or Proteins (AREA)
EA201892467A 2016-04-29 2017-04-28 Олигонуклеотидные аналоги, нацеленные на lmna человека EA201892467A1 (ru)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201662330027P 2016-04-29 2016-04-29
PCT/US2017/030174 WO2017190041A1 (en) 2016-04-29 2017-04-28 Oligonucleotide analogues targeting human lmna

Publications (1)

Publication Number Publication Date
EA201892467A1 true EA201892467A1 (ru) 2019-05-31

Family

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Family Applications (1)

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EA201892467A EA201892467A1 (ru) 2016-04-29 2017-04-28 Олигонуклеотидные аналоги, нацеленные на lmna человека

Country Status (15)

Country Link
US (2) US10822608B2 (US07794700-20100914-C00152.png)
EP (1) EP3449000A1 (US07794700-20100914-C00152.png)
JP (2) JP7042255B2 (US07794700-20100914-C00152.png)
KR (1) KR102329187B1 (US07794700-20100914-C00152.png)
CN (1) CN109477109B (US07794700-20100914-C00152.png)
AU (2) AU2017258642B2 (US07794700-20100914-C00152.png)
BR (1) BR112018072279A2 (US07794700-20100914-C00152.png)
CA (1) CA3022303A1 (US07794700-20100914-C00152.png)
CO (1) CO2018012873A2 (US07794700-20100914-C00152.png)
EA (1) EA201892467A1 (US07794700-20100914-C00152.png)
MA (1) MA45616A (US07794700-20100914-C00152.png)
MX (1) MX2018013157A (US07794700-20100914-C00152.png)
NZ (1) NZ747685A (US07794700-20100914-C00152.png)
SG (1) SG11201809468XA (US07794700-20100914-C00152.png)
WO (1) WO2017190041A1 (US07794700-20100914-C00152.png)

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CA3022303A1 (en) 2017-11-02
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AU2023274161A1 (en) 2023-12-21
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