Genzyme Corporation

Ionis Pharmaceuticals, Inc.

University of Rochester

Andrew Leger

Bruce M Wentworth

Seng H Cheng

Timothy E Weeden

Carol A Nelson

Nicholas P Clayton

Susan M Freier

C Frank Bennett

Robert A MacLeod

Sanjay K Pandey

Charles A Thornton

Thurman Wheeler

Sanjey K Pandey

The present disclosure relates to antisense oligonucleotides (AONs), such as phosphorodiamidate morpholino oligonucleotides (PMOs). The present disclosure further relates to the conjugation of multiple PMOs to cationic cell penetrating peptides (CPPs) to enhance the uptake of PMOs into skeletal and cardiac muscle cells.

Multiple oligonucleotide moieties on peptide carrier

Provided herein are peptide-linked morpholino (PPMO) antisense oligonucleotides that target the poly CUG repeat tract in the 3' untranslated region of the gene encoding dystrophia myotonica-protein kinase (DMPK) and methods for systemic administration of the same for the treatment of mytonic dystrophy type I (DM1).

Peptide-linked morpholino antisense oligonucleotides for treatment of myotonic dystrophy

Provided herein are methods, compounds, and compositions for reducing expression of a DMPK mRNA and protein in an animal. Also provided herein are methods, compounds, and compositions for preferentially reducing CUGexp DMPK RNA, reducing myotonia or reducing spliceopathy in an animal. Such methods, compounds, and compositions are useful to treat, prevent, delay, or ameliorate type 1 myotonic dystrophy, or a symptom thereof.

Modulation of dystrophia myotonica-protein kinase (DMPK) expression

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Boston, MA, United States of America

Andrew Leger