Method for enhancing folding and transport of misfolded glucocerebrosidase

US8937059B2 · kind B2 · utility

Assignees

• The Hospital for Sick Children · CA
• McMaster University · CA

Inventors

• Don J. Mahuran · Toronto, CA
• Michael B. Tropak · Toronto, CA
• Justin D. Buttner · Parramatta, AU
• Jan E. Blanchard · St. Catherines, CA
• Eric Brown · Oakville, CA

Key dates

• Filing date: Apr 17, 2014
• Grant date: Jan 20, 2015
• Expiry date: Apr 17, 2034

Classification

• Technology area (CPC C): Chemistry; Metallurgy
• CPC primary: C12Y302/01045
• WIPO field: Pharmaceuticals
• WIPO sector: Chemistry

Abstract

Therapeutic compositions and methods for treatment of late-onset Gaucher disease are described herein. The compositions comprise compounds having activity as pharmacological chaperones for mutant forms of the beta-glucocerebrosidase. Methods of treatment involve providing therapeutically effective amounts of such compositions to subjects in need thereof.