Methods and compositions for CNS delivery of arylsulfatase A
AssigneeTakeda Pharmaceutical Company Limited
InventorsNazila Salamat-Miller, Katherine Taylor, Paul Campolieto, Zahra Shahrokh, Jing Pan, Lawrence Charnas, Teresa Leah Wright, Pericles Calias
Life of this patent
Estimating the term…
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.
Claims6
A method of treating metachromatic leukodystrophy (MLD) comprising a step of administering directly into the cerebrospinal fluid of a subject in need of treatment a formulation comprising an arylsulfatase A (ASA) protein at a concentration at or greater than 10 mg/ml at a dose amount of at least 100 mg, salt, polysorbate, wherein the formulation comprises no greater than 10 mM phosphate, and wherein the formulation has a pH of between 5.5-6.5.
Dependencies are inferred from the claim text.