Gene therapy for mucopolysaccharidosis IIIB

US11723989B2 · kind B2 · utility

Assignee

• The Trustees of the University of Pennsylvania · US

Inventors

• James M. Wilson · Glen Mills, US
• Christian Hinderer · Philadelphia, US
• Juliette Hordeaux · Philadelphia, US

Key dates

• Filing date: Nov 29, 2018
• Grant date: Aug 15, 2023
• Expiry date: Dec 6, 2039

Classification

• Technology area (CPC C): Chemistry; Metallurgy
• CPC primary: C12N2800/22
• WIPO field: Pharmaceuticals
• WIPO sector: Chemistry

Abstract

Provided herein is a recombinant AAV (rAAV) comprising an AAV capsid and a vector genome packaged therein, wherein the vector genome comprises an AAV 5′ inverted terminal repeat (ITR), an engineered nucleic acid sequence encoding a functional human N-acetyl-alpha-glucosaminidase (hNAGLU), a regulatory sequence which direct expression of hNAGLU in a target cell, and an AAV 3′ ITR. Also provided is a pharmaceutical composition comprising a rAAV as described herein in a formulation buffer, and a method of treating a human subject diagnosed with MPS IIIB.