Shire Human Genetic Therapies, Inc.

Takeda Pharmaceutical Company Limited

Paul Campolieto

Zahra Shahrokh

Pericles Calias

Jing Pan

Teresa Leah Wright

Lawrence Charnas

Nazila Salamat-Miller

Katherine Taylor

Sujit Basu

Keethkumar Jain

The present invention provides, among other things, compositions and methods for CNS delivery of lysosomal enzymes for effective treatment of lysosomal storage diseases. In some embodiments, the present invention includes a stable formulation for direct CNS intrathecal administration comprising an arylsulfatase A (ASA) protein, salt, and a polysorbate surfactant for the treatment of Metachromatic Leukodystrophy Disease.

Methods and compositions for CNS delivery of arylsulfatase A

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.

Stable formulations for CNS delivery of arylsulfatase A

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Charlottesville, VA, United States of America

Paul Campolieto