Codon-optimized human NPC1 genes for the treatment of Niemann-Pick type C1 deficiency and related conditions

US12116577B2 · 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
• William J. Pavan · Derwood, US
• Randy J. Chandler · Washington, US

Key dates

• Filing date: Jun 20, 2018
• Grant date: Oct 15, 2024
• Expiry date: Jun 16, 2039

Classification

• Technology area (CPC C): Chemistry; Metallurgy
• CPC primary: C12N2799/022
• WIPO field: Biotechnology
• WIPO sector: Chemistry

Abstract

The present disclosure provides compositions for viral gene therapy, e.g. Adeno-Associated virus-directed gene therapy, and methods of using the same for the treatment and/or prevention of cholesterol storage diseases or disorders, such as Niemann-Pick disease, Type C.