Genzyme Corporation

Ionis Pharmaceuticals, Inc.

University of Rochester

Bruce M Wentworth

Timothy E Weeden

Carol A Nelson

Nicholas P Clayton

Andrew Leger

Seng H Cheng

Ronald K Scheule

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) for modulating the expression of glycogen synthase. AONs of the present disclosure may be useful in treating diseases associated with the modulation of the expression of the enzyme glycogen synthase, such as Pompe disease. Also provided by the present disclosure are compositions comprising AONs, as well as methods of down regulating mRNA coding for glycogen synthase, methods for reducing glycogen synthase in skeletal and cardiac muscle, and methods for treating Pompe disease.

Inhibiting or downregulating glycogen synthase by creating premature stop codons using antisense oligonucleotides

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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Northborough, MA, United States of America

Bruce M Wentworth