AU2018355237B2 - Antisense oligomers for treatment of non-sense mediated RNA decay based conditions and diseases - Google Patents

Antisense oligomers for treatment of non-sense mediated RNA decay based conditions and diseases

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Publication number
AU2018355237B2
AU2018355237B2 AU2018355237A AU2018355237A AU2018355237B2 AU 2018355237 B2 AU2018355237 B2 AU 2018355237B2 AU 2018355237 A AU2018355237 A AU 2018355237A AU 2018355237 A AU2018355237 A AU 2018355237A AU 2018355237 B2 AU2018355237 B2 AU 2018355237B2
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grch38
exon
intron
mrna
nucleotides
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AU2018355237A1 (en
Inventor
Isabel AZNAREZ
Enxuan Jing
Jacob KACH
Gene Liau
Juergen SCHARNER
Baruch TICHO
Aditya VENKATESH
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Stoke Therapeutics Inc
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Stoke Therapeutics Inc
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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
    • 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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    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/7105—Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
    • A—HUMAN NECESSITIES
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    • C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • C07H21/02—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with ribosyl as saccharide radical
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    • C07H21/04—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with deoxyribosyl as saccharide radical
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    • C07K—PEPTIDES
    • C07K16/00—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies
    • C07K16/40—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against enzymes
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    • C12N5/0602—Vertebrate cells
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
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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
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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
    • C12N2310/3231—Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
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    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/33—Alteration of splicing
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    • C12N2510/00—Genetically modified cells

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AU2018355237A 2017-10-23 2018-10-23 Antisense oligomers for treatment of non-sense mediated RNA decay based conditions and diseases Active AU2018355237B2 (en)

Priority Applications (1)

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AU2025287361A AU2025287361A1 (en) 2017-10-23 2025-12-24 Antisense oligomers for treatment of non-sense mediated RNA decay based conditions and diseases

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US201762575924P 2017-10-23 2017-10-23
US62/575,924 2017-10-23
US201862667200P 2018-05-04 2018-05-04
US62/667,200 2018-05-04
PCT/US2018/057165 WO2019084050A1 (en) 2017-10-23 2018-10-23 ANTISENSE OLIGOMERS FOR THE TREATMENT OF CONDITIONS AND DISEASES BASED ON THE DECLINE OF NON-SENSE MEDIATED MRNA

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AU2018355237B2 true AU2018355237B2 (en) 2025-09-25

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US (2) US20210268667A1 (https=)
EP (2) EP3700570B1 (https=)
JP (2) JP7475272B2 (https=)
KR (2) KR102774070B1 (https=)
CN (2) CN111936163B (https=)
AU (2) AU2018355237B2 (https=)
BR (1) BR112020007881A2 (https=)
CA (1) CA3079729A1 (https=)
DK (1) DK3700570T3 (https=)
ES (1) ES3015767T3 (https=)
FI (1) FI3700570T3 (https=)
GB (2) GB2584204B (https=)
HR (1) HRP20250322T1 (https=)
HU (1) HUE070436T2 (https=)
IL (1) IL274023A (https=)
LT (1) LT3700570T (https=)
PL (1) PL3700570T3 (https=)
PT (1) PT3700570T (https=)
RS (1) RS66633B1 (https=)
SG (1) SG11202003692YA (https=)
SI (1) SI3700570T1 (https=)
SM (1) SMT202500127T1 (https=)
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