Disabling autophagy as a treatment for lysosomal storage diseases

US8536148B2 · kind B2 · utility

Assignee

• The United States of America, as represented by the Secretary, Department of Health and Human Services · US

Inventors

• Nina Raben · Rockville, US
• Cynthia Schreiner · Gowanda, US
• Rebecca Baum · Highland, US
• Shoichi Takikita · Rockville, US
• Tao Xie · Copley, US
• Paul Plotz · Washington, US

Key dates

• Filing date: Sep 2, 2010
• Grant date: Sep 17, 2013
• Expiry date: Sep 2, 2030

Classification

• Technology area (CPC C): Chemistry; Metallurgy
• CPC primary: C12N2310/531
• WIPO field: Pharmaceuticals
• WIPO sector: Chemistry

Abstract

Provided herein are methods of treating lysosomal storage disease, for instance Pompe disease, through inhibition of autophagy. Optionally, treatment is administered as an adjunct to enzyme replacement therapy (ERT).