EA202090584A1 - ANTI-SENSE OLIGOMERS FOR TREATMENT OF CONDITIONS AND DISEASES - Google Patents

ANTI-SENSE OLIGOMERS FOR TREATMENT OF CONDITIONS AND DISEASES

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Publication number
EA202090584A1
EA202090584A1 EA202090584A EA202090584A EA202090584A1 EA 202090584 A1 EA202090584 A1 EA 202090584A1 EA 202090584 A EA202090584 A EA 202090584A EA 202090584 A EA202090584 A EA 202090584A EA 202090584 A1 EA202090584 A1 EA 202090584A1
Authority
EA
Eurasian Patent Office
Prior art keywords
diseases
treatment
conditions
scn1a
lead
Prior art date
Application number
EA202090584A
Other languages
Russian (ru)
Inventor
Изабель Азнарез
Чжоу Хань
Original Assignee
Стоук Терапьютикс, Инк.
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 Стоук Терапьютикс, Инк. filed Critical Стоук Терапьютикс, Инк.
Priority claimed from PCT/US2018/048031 external-priority patent/WO2019040923A1/en
Publication of EA202090584A1 publication Critical patent/EA202090584A1/en

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    • A—HUMAN NECESSITIES
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    • A61K9/00—Medicinal preparations characterised by special physical form
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    • A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0085—Brain, e.g. brain implants; Spinal cord
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/08—Antiepileptics; Anticonvulsants
    • A61P25/12—Antiepileptics; Anticonvulsants for grand-mal
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    • 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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    • 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
    • C12N15/1138—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 against receptors or cell surface proteins
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/11—Antisense
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    • C12N2320/33—Alteration of splicing
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    • C12Q2600/00—Oligonucleotides characterized by their use
    • C12Q2600/158—Expression markers

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  • Chemical Kinetics & Catalysis (AREA)
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  • Orthopedic Medicine & Surgery (AREA)
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  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)

Abstract

Альтернативные события сплайсинга в гене SCN1A могут приводить к непродуктивным транскриптам мРНК, которые, в свою очередь, могут приводить к аберрантной экспрессии белка, а терапевтические средства, которые могут нацеливаться на альтернативные события сплайсинга в гене SCN1A, могут модулировать уровень экспрессии функциональных белков у пациентов с синдромом Драве и/или ингибировать аберрантную экспрессию белка. Такие терапевтические средства могут быть использованы для лечения состояния, вызванного дефицитом белка SCN1A, SCN8A или SCN5A.Alternative splicing events in the SCN1A gene can lead to unproductive mRNA transcripts, which, in turn, can lead to aberrant protein expression, and therapeutic agents that can target alternative splicing events in the SCN1A gene can modulate the expression level of functional proteins in patients with Drave syndrome and / or inhibit aberrant protein expression. Such therapeutic agents can be used to treat a condition caused by a protein deficiency of SCN1A, SCN8A or SCN5A.

EA202090584A 2018-05-15 2018-08-24 ANTI-SENSE OLIGOMERS FOR TREATMENT OF CONDITIONS AND DISEASES EA202090584A1 (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201862671745P 2018-05-15 2018-05-15
PCT/US2018/048031 WO2019040923A1 (en) 2017-08-25 2018-08-24 Antisense oligomers for treatment of conditions and diseases

Publications (1)

Publication Number Publication Date
EA202090584A1 true EA202090584A1 (en) 2020-06-05

Family

ID=71138816

Family Applications (1)

Application Number Title Priority Date Filing Date
EA202090584A EA202090584A1 (en) 2018-05-15 2018-08-24 ANTI-SENSE OLIGOMERS FOR TREATMENT OF CONDITIONS AND DISEASES

Country Status (1)

Country Link
EA (1) EA202090584A1 (en)

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