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EP3806868A4 - Oligomères induisant un saut d'exon pour la dystrophie musculaire - Google Patents

Oligomères induisant un saut d'exon pour la dystrophie musculaire Download PDF

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Publication number
EP3806868A4
EP3806868A4 EP19818767.6A EP19818767A EP3806868A4 EP 3806868 A4 EP3806868 A4 EP 3806868A4 EP 19818767 A EP19818767 A EP 19818767A EP 3806868 A4 EP3806868 A4 EP 3806868A4
Authority
EP
European Patent Office
Prior art keywords
exon
muscular dystrophy
skipping
oligomers
skipping oligomers
Prior art date
Legal status (The legal status 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 status listed.)
Withdrawn
Application number
EP19818767.6A
Other languages
German (de)
English (en)
Other versions
EP3806868A2 (fr
Inventor
Frederick Joseph SCHNELL
Marco Passini
Neisa ESTRELLA
Gunnar Hanson
Ming Zhou
Richard Bestwick
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Sarepta Therapeutics Inc
Original Assignee
Sarepta Therapeutics Inc
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 Sarepta Therapeutics Inc filed Critical Sarepta Therapeutics Inc
Priority to EP22214630.0A priority Critical patent/EP4219717A3/fr
Priority claimed from PCT/US2019/036764 external-priority patent/WO2019241385A2/fr
Publication of EP3806868A2 publication Critical patent/EP3806868A2/fr
Publication of EP3806868A4 publication Critical patent/EP3806868A4/fr
Withdrawn legal-status Critical Current

Links

Classifications

    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/33—Alteration of splicing

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Physics & Mathematics (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Peptides Or Proteins (AREA)
EP19818767.6A 2018-06-13 2019-06-12 Oligomères induisant un saut d'exon pour la dystrophie musculaire Withdrawn EP3806868A4 (fr)

Priority Applications (1)

Application Number Priority Date Filing Date Title
EP22214630.0A EP4219717A3 (fr) 2018-06-13 2019-06-12 Oligomères à saut d'exon pour la dystrophie musculaire

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201862684615P 2018-06-13 2018-06-13
PCT/US2019/036764 WO2019241385A2 (fr) 2018-06-13 2019-06-12 Oligomères induisant un saut d'exon pour la dystrophie musculaire

Related Child Applications (1)

Application Number Title Priority Date Filing Date
EP22214630.0A Division EP4219717A3 (fr) 2018-06-13 2019-06-12 Oligomères à saut d'exon pour la dystrophie musculaire

Publications (2)

Publication Number Publication Date
EP3806868A2 EP3806868A2 (fr) 2021-04-21
EP3806868A4 true EP3806868A4 (fr) 2022-06-22

Family

ID=74879743

Family Applications (2)

Application Number Title Priority Date Filing Date
EP19818767.6A Withdrawn EP3806868A4 (fr) 2018-06-13 2019-06-12 Oligomères induisant un saut d'exon pour la dystrophie musculaire
EP22214630.0A Pending EP4219717A3 (fr) 2018-06-13 2019-06-12 Oligomères à saut d'exon pour la dystrophie musculaire

Family Applications After (1)

Application Number Title Priority Date Filing Date
EP22214630.0A Pending EP4219717A3 (fr) 2018-06-13 2019-06-12 Oligomères à saut d'exon pour la dystrophie musculaire

Country Status (1)

Country Link
EP (2) EP3806868A4 (fr)

Citations (2)

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Also Published As

Publication number Publication date
EP4219717A2 (fr) 2023-08-02
EP4219717A3 (fr) 2023-12-20
EP3806868A2 (fr) 2021-04-21

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