Methods and compositions for CNS delivery of arylsulfatase A
AssigneeShire Human Genetic Therapies, Inc.
InventorsNazila Salamat-Miller, Katherine Taylor, Paul Campolieto, Zahra Shahrokh, Jing Pan, Lawrence Charnas, Teresa Leah Wright, Pericles Calias
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Abstract
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.
Claims19
A method of treating metachromatic leukodystrophy (MLD) disease comprising a step of
- administering intrathecally to a subject in need of treatment a formulation comprising an arylsulfatase A (ASA) protein at a concentration at or greater than 25 mg/mL and at a dose amount of at least 80 mg/kg brain weight, wherein the formulation comprises no greater than 20 mM of phosphate.
The method of 1, wherein the phosphate is present at a concentration no greater than 5 mM.
Dependencies are inferred from the claim text.