Gene therapy for ceroid lipofuscinoses

US11591614B2 · kind B2 · utility

Assignee

• The Trustees of the University of Pennsylvania · US

Inventors

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

Key dates

• Filing date: May 11, 2018
• Grant date: Feb 28, 2023
• Expiry date: Feb 17, 2040

Classification

• Technology area (CPC C): Chemistry; Metallurgy
• CPC primary: C12Y304/14009
• WIPO field: Pharmaceuticals
• WIPO sector: Chemistry

Abstract

Provided herein are methods and compositions for treatment of Batten disease. Such compositions include a recombinant adeno-associated virus (rAAV), said rAAV comprising an AAV capsid, and a vector genome packaged therein, said vector genome comprising (a) an AAV 5′ inverted terminal repeat (ITR) sequence; (b) a promoter; (c) a CLN2 coding sequence encoding a human TPP1; (d) an AAV 3′ ITR.