Patent · US Active

Peptide-linked morpholino antisense oligonucleotides for treatment of myotonic dystrophy

US10111962B2 · kind B2 · utility

0Cited by

12References

21Claims

0Family size

Assignee

Genzyme Corporation · US

Inventors

Andrew Leger · Boston, US
Bruce M. Wentworth · Northborough, US
Carol A. Nelson · Westford, US
Timothy Weeden · Waltham, US
Nicholas P. CLAYTON · Sudbury, US
Seng H. Cheng · Wellesley, US

Key dates

Filing date	Sep 24, 2013
Grant date	Oct 30, 2018
Priority date	—
Expiry date	Sep 24, 2033

Classification

Technology area (CPC C)Chemistry; Metallurgy
CPC primaryC12N2320/32
WIPO fieldPharmaceuticals
WIPO sectorChemistry

Abstract

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).

Source: USPTO / EPO open patent data. Objective bibliographic and citation counts.