Deep Genomics Incorporated

Daniele Merico

Erno Wienholds

Frank Schmitges

Matthew O'Hara

Joao Antonio Lourenco Goncalves

Mark George Ford Sun

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.

Oligonucleotide therapy for Wilson disease

The present disclosure provides compositions and methods for treating a disorder associated with mutations in the CEP290 gene. The disclosure includes synthetic polynucleotides for skipping a reading-frame of a CEP290 pre-RNA, yielding a CEP290 translated product that lacks one or more exons. The disclosure also provides methods of treating patients with the synthetic polynucleotides disclosed herein.

Growing community of inventors

Toronto, Canada

Daniele Merico