Methods and compositions for cns delivery of b-galactocerebrosidase
AssigneeShire Human Genetic Therapies, Inc.
InventorsNazila Salamat-Miller, Katherine Taylor, Ken Manning, Gaozhong Zhu, Paul Campolieto, Zahra Shahrokh, Pericles Calias, Thomas McCauley
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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 B-Galactocerebrosidase protein, salt, and a polysorbate surfactant for the treatment of GLD Disease.
Claims51
A stable formulation for intrathecal administration comprising a β-Galactocerebrosidase (GLC) protein, salt, a buffering agent, a stabilizing agent and a polysorbate surfactant.
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The stable formulation of claim 60, wherein the phosphate is present at a concentration no greater than 20 mM.
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A stable formulation for intrathecal administration comprising a β-Galactocerebrosidase (GLC) protein, phosphate at a concentration of approximately 5 mM, NaCl at a concentration of approximately 150 mM, sucrose at a concentration of approximately 1%, polysorbate 20 at a concentration of approximately 0.005%, and a pH of approximately 6.3.
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Dependencies are inferred from the claim text.