EP 3806868 A4 20220622 - EXON SKIPPING OLIGOMERS FOR MUSCULAR DYSTROPHY
Title (en)
EXON SKIPPING OLIGOMERS FOR MUSCULAR DYSTROPHY
Title (de)
EXON-SKIPPING-OLIGOMERE FÜR MUSKELDYSTROPHIE
Title (fr)
OLIGOMÈRES INDUISANT UN SAUT D'EXON POUR LA DYSTROPHIE MUSCULAIRE
Publication
Application
Priority
- US 201862684615 P 20180613
- US 2019036764 W 20190612
Abstract (en)
[origin: EP4219717A2] Antisense oligomers complementary to a selected target site in the human dystrophin gene to induce exon 51 skipping are described.
IPC 8 full level
C12N 15/113 (2010.01); A61K 31/7088 (2006.01); A61K 31/7125 (2006.01); A61P 21/00 (2006.01)
CPC (source: EP)
C12N 15/113 (2013.01); C12N 2310/11 (2013.01); C12N 2310/3145 (2013.01); C12N 2310/3233 (2013.01); C12N 2310/351 (2013.01); C12N 2310/3513 (2013.01); C12N 2320/33 (2013.01)
Citation (search report)
- [XI] WO 2017062835 A2 20170413 - SAREPTA THERAPEUTICS INC [US]
- [XI] US 2012289457 A1 20121115 - HANSON GUNNAR J [US]
- [A] 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
- [A] 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
- See references of WO 2019241385A2
Designated contracting state (EPC)
AL AT BE BG CH CY CZ DE DK EE ES FI FR GB GR HR HU IE IS IT LI LT LU LV MC MK MT NL NO PL PT RO RS SE SI SK SM TR
DOCDB simple family (publication)
EP 4219717 A2 20230802; EP 4219717 A3 20231220; EP 3806868 A2 20210421; EP 3806868 A4 20220622
DOCDB simple family (application)
EP 22214630 A 20190612; EP 19818767 A 20190612