EP3806868A4 - Oligomères induisant un saut d'exon pour la dystrophie musculaire - Google Patents
Oligomères induisant un saut d'exon pour la dystrophie musculaire Download PDFInfo
- 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
- muscular dystrophy
- exon skipping
- oligomers
- skipping oligomers
- exon
- 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- Withdrawn
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)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Peptides Or Proteins (AREA)
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 |
---|---|---|---|
EP22214630.0A Pending EP4219717A3 (fr) | 2018-06-13 | 2019-06-12 | Oligomères à saut d'exon pour la dystrophie musculaire |
EP19818767.6A Withdrawn EP3806868A4 (fr) | 2018-06-13 | 2019-06-12 | Oligomères induisant un saut d'exon pour la dystrophie musculaire |
Family Applications Before (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) | EP4219717A3 (fr) |
Citations (2)
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US20120289457A1 (en) * | 2011-05-05 | 2012-11-15 | Avi Biopharma, Inc. | Peptide oligonucleotide conjugates |
WO2017062835A2 (fr) * | 2015-10-09 | 2017-04-13 | Sarepta Therapeutics, Inc. | Compositions et méthodes pour traiter la dystrophie musculaire de duchenne et troubles associés |
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-
2019
- 2019-06-12 EP EP22214630.0A patent/EP4219717A3/fr active Pending
- 2019-06-12 EP EP19818767.6A patent/EP3806868A4/fr not_active Withdrawn
Patent Citations (2)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
US20120289457A1 (en) * | 2011-05-05 | 2012-11-15 | Avi Biopharma, Inc. | Peptide oligonucleotide conjugates |
WO2017062835A2 (fr) * | 2015-10-09 | 2017-04-13 | Sarepta Therapeutics, Inc. | Compositions et méthodes pour traiter la dystrophie musculaire de duchenne et troubles associés |
Non-Patent Citations (2)
Title |
---|
CHARLESTON JAY S. ET AL: "Eteplirsen treatment for Duchenne muscular dystrophy : Exon skipping and dystrophin production", NEUROLOGY, vol. 90, no. 24, 12 June 2018 (2018-06-12), US, pages e2146 - e2154, XP055877433, ISSN: 0028-3878, Retrieved from the Internet <URL:https://n.neurology.org/content/neurology/90/24/e2146.full.pdf> DOI: 10.1212/WNL.0000000000005680 * |
GOEMANS NATHALIE ET AL: "A randomized placebo-controlled phase 3 trial of an antisense oligonucleotide, drisapersen, in Duchenne muscular dystrophy", NEUROMUSCULAR DISORDERS, vol. 28, no. 1, 1 January 2018 (2018-01-01), GB, pages 4 - 15, XP055877283, ISSN: 0960-8966, DOI: 10.1016/j.nmd.2017.10.004 * |
Also Published As
Publication number | Publication date |
---|---|
EP4219717A3 (fr) | 2023-12-20 |
EP3806868A2 (fr) | 2021-04-21 |
EP4219717A2 (fr) | 2023-08-02 |
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Inventor name: BESTWICK, RICHARD Inventor name: ZHOU, MING Inventor name: HANSON, GUNNAR Inventor name: ESTRELLA, NELSA Inventor name: PASSINI, MARCO Inventor name: SCHNELL, FREDERICK, JOSEPH |
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