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US 9,220,677 B2Granted
Methods and compositions for CNS delivery of iduronate-2-sulfatase
AssigneeShire Human Genetic Therapies, Inc.
InventorsGaozhong Zhu, Kris Lowe, Zahra Shahrokh, James Christian, Rick Fahrner, Jing Pan, 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 iduronate-2-sulfatase (I2S) protein, salt, and a polysorbate surfactant for the treatment of Hunters Syndrome.
Claims27
A method of treating Hunter Syndrome comprising a step of
- administering intrathecally to a human subject in need of treatment a formulation comprising iduronate-2-sulfatase (I2S) at a concentration of at least 10 mg/ml and no greater than 5 mM phosphate, and wherein the I2S is administered at a dose amount of at least 10 mg.
Dependencies are inferred from the claim text.