Methods for treating progeroid laminopathies using oligonucleotide analogues targeting human LMNA

US10398721B2 · kind B2 · utility

Assignees

• Sarepta Therapeutics, Inc. · US
• The United States of America, as represented by the Secretary, Department of Health and Human Services · US
• University of Maryland · US
• The Progeria Research Foundation · US

Inventors

• Ryszard Kole · Chapel Hill, US
• Francis S. Collins · Rockville, US
• Michael R. Erdos · Bethesda, US
• Kan Cao · Bowie, US
• Leslie B. Gordon · Foxborough, US

Key dates

• Filing date: Oct 6, 2017
• Grant date: Sep 3, 2019
• Expiry date: Oct 6, 2037

Classification

• Technology area (CPC C): Chemistry; Metallurgy
• CPC primary: C12N2320/33
• WIPO field: Pharmaceuticals
• WIPO sector: Chemistry

Abstract

Provided are methods of treatment in subjects having progeroid diseases and related conditions which rely upon LMNA-targeted antisense oligonucleotides for reducing expression of one or more aberrantly spliced LMNA mRNA isoforms that encode progerin.