Genzyme Corporation

University of Michigan

Carol A Nelson

Craig S Siegel

David J Harris

James A Shayman

Timothy E Weeden

Bruce M Wentworth

Diane P Copeland

Nicholas P Clayton

Ronald K Scheule

Andrew Leger

Seng H Cheng

Seng Cheng

Nicholas Clayton

Bruce Wentworth

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

Disclosed is a novel enantiomeric synthesis ceramide-like inhibitors of UDP-glucose: N-acylsphingosine glucosyltransferase. Also disclosed are novel intermediates formed during the synthesis.

Synthesis of UDP-glucose: N-acylsphingosine glucosyltransferase inhibitors

The present invention provides amino ceramide-like compounds which inhibit glucosyl ceramide (GlyCer) formation by inhibiting the enzyme GlyCer synthase, thereby lowering the level of glycosphingolipids. The compounds of the present invention have improved GlcCer synthase inhibition activity and are therefore useful in therapeutic methods for treating various conditions and diseases associated with altered glycosphingolipid levels.

Amino ceramide-like compounds and therapeutic methods of use

[object Object]

Growing community of inventors

Westford, MA, United States of America

Carol A Nelson