Deep Genomics Incorporated

Frank Schmitges

Daniele Merico

Erno Wienholds

Matthew O'Hara

The present disclosure provides antisense oligonucleotides, compositions, and methods that target ATP7B exon 6 or a flanking intron, thereby modulating splicing of ATP7B pre-mRNA to increase the level of ATP7B mRNA molecules having exon 6, e.g., to provide a therapy for Wilson disease. The present disclosure provides an antisense oligonucleotide including a nucleobase sequence at least 70% complementary to an ATP7B target sequence in exon 6, a 5'-flanking intron, a 3&#x2032;-flanking intron, or a combination of exon 6 and the 5&#x2032;-flanking or 3&#x2032;-flanking intron.

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Princeton, NJ, United States of America

Frank Schmitges