Methods and compositions for CNS delivery of heparan N-sulfatase
AssigneeShire Human Genetic Therapies, Inc.
InventorsFarah Natoli, Gaozhong Zhu, Jennifer Terew, Yuan Jiang, Jamie Tsung, Zahra Shahrokh, Brian Vernaglia, Jing Pan, Richard Pfeifer, 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 a heparan N-sulfatase (HNS) protein, salt, and a polysorbate surfactant for the treatment of Sanfilippo Syndrome Type A.
Claims43
A method of treating Sanfilippo A Syndrome comprising a step of administering intrathecally to a human subject in need of treatment a formulation comprising a heparan N-sulfatase (HNS) protein at a concentration at or greater than 5 mg/ml at a dose of at least 10 mg, wherein the formulation comprises 5 mM to 50 mM of phosphate.
A method of treating Sanfilippo A Syndrome, comprising a step of administering intrathecally to a subject in need of treatment a formulation comprising heparan N-sulfatase (HNS) protein at a concentration at or greater than 5 mg/ml at a dose of at least 10 mg, salt at a concentration of approximately 0-300 mM, a polysorbate surfactant at a concentration of approximately 0-0.02%, phosphate at a concentration of approximately 5-50 mM, and a pH of approximately 6.0-7.0.
Dependencies are inferred from the claim text.