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US 2022/0025368 A1Published application
Bispecific antisense oligonucleotides for dystrophin exon skipping
AssigneeBioMarin Technologies B.V.
InventorsJudith Christina Theodora van Deutekom, Nicole Ann Datson
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Abstract
The current invention provides splice-switching compounds with improved characteristics that enhance clinical applicability preferably for treating, ameliorating, preventing, and/or delaying neuromuscular disorders, more specifically DMD.
Claims22
A compound comprising a first and a second antisense oligonucleotide (AON) linked to each other by a linking moiety, wherein said first antisense oligonucleotide (AON) is complementary to or binds to or targets or hybridizes to or overlaps with at least a part of SEQ ID NO:
- 3, and wherein said second antisense oligonucleotide (AON) is complementary to or binds to or targets or hybridizes to or overlaps with at least a part of SEQ ID NO:
- 4, wherein SEQ ID NO:
- 3 and 4 are located within exon 51 of dystrophin pre-mRNA.
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Dependencies are inferred from the claim text.