Stable formulations for CNS delivery of arylsulfatase A

US10660944B2 · kind B2 · utility

Assignee

• SHIRE HUMAN GENETIC THERAPIES, INC. · US

Inventors

• Nazila Salamat-Miller · Arlington, US
• Katherine Taylor · Arlington, US
• Paul Campolieto · Charlottesville, US
• Zahra Shahrokh · Weston, US
• Jing Pan · Newark, US
• Lawrence Charnas · Natick, US
• Teresa Leah Wright · Lexington, US
• Pericles Calias · Melrose, US
• Keethkumar Jain · Watertown, US
• Sujit Basu · Lexington, US

Key dates

• Filing date: Jun 5, 2018
• Grant date: May 26, 2020
• Expiry date: Jun 5, 2038

Classification

• Technology area (CPC C): Chemistry; Metallurgy
• CPC primary: C12Y301/06008
• WIPO field: Pharmaceuticals
• WIPO sector: Chemistry

Abstract

The present invention provides, among oilier things, compositions and methods for CNS delivery of lysosomal enzymes (e.g., recombinant human arylsulfatase A (rhASA)) for effective treatment of lysosomal storage diseases (e.g. Metachromatic Leukodystrophy Disease), In some embodiments, the present invention includes a stable formulation for intrathecal administration comprising an ASA protein and a poloxamer, wherein less than 3% of the ASA protein exists in aggregated form.