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.
Claims7
A stable formulation for intrathecal administration comprising an iduronate-2-sulfatase (I2S) protein, salt, and a polysorbate surfactant.
-22. (canceled)
A stable formulation for intrathecal administration comprising an iduronate-2-sulfatase (I2S) protein at a concentration ranging from approximately 1-300 mg/ml, NaCl at a concentration of approximately 154 mM, polysorbate 20 at a concentration of approximately 0.005%, and a pH of approximately 6.0.
-31. (canceled)
A method of treating Hunters Syndrome comprising a step of
- administering intrathecally to a subject in need of treatment a formulation comprising an iduronate-2-sulfatase (I2S) protein at a concentration ranging from approximately 1-300 mg/ml, NaCl at a concentration of approximately 154 mM, polysorbate 20 at a concentration of approximately 0.005%, and a pH of approximately 6.
-61. (canceled)
Dependencies are inferred from the claim text.