Gene therapy for treating peroxisomal disorders

US11793887B2 · kind B2 · utility

Assignees

• The Trustees of the University of Pennsylvania · US
• University of Southern California · US
• The Research Institute of McGill University Health Centre · CA

Inventors

• Nancy E. Braverman · Los Angeles, US
• Catherine Argyriou · Montréal, CA
• Joseph Hacia · Los Angeles, US
• Jean Bennett · Via Enrico Fermi, US
• Junwei Sun · Philadelphia, US
• Ji Yun Song · Spring House, US
• Devin McDougald · Philadelphia, US

Key dates

• Filing date: May 31, 2018
• Grant date: Oct 24, 2023
• Expiry date: Nov 29, 2038

Classification

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

Abstract

Methods and compositions are provided for treatment of peroxisomal biogenesis disorders (PBDs). More particularly, recombinant adeno-associated viruses (rAAV) provided in the form of compositions are used to deliver a nucleic acid encoding human PEX1 to host cells. The rAAVs comprise a AAV capsid, and packaged therein a vector genome comprising an AAV 5′ inverted terminal repeat (ITR) sequence; a promoter; a coding sequence encoding a human PEX1; and an AAV 3′ ITR.