Viral gene therapy as treatment for cholesterol storage disease or disorder

US12201658B2 · kind B2 · utility

Assignee

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

Inventors

• Charles P. Venditti · Potomac, US
• Randy J. Chandler · Washington, US
• William J. Pavan · Derwood, US

Key dates

• Filing date: Sep 25, 2020
• Grant date: Jan 21, 2025
• Expiry date: Oct 12, 2040

Classification

• Technology area (CPC C): Chemistry; Metallurgy
• CPC primary: C12N2750/14143
• WIPO field: Pharmaceuticals
• WIPO sector: Chemistry

Abstract

Provided herein are compositions and methods for the viral gene therapy (e.g., AAV-directed gene therapy) of cholesterol storage diseases or disorders, such as Niemann-Pick disease, Type C.