Methods and compositions for cns delivery of iduronate-2-sulfatase
AssigneeShire Human Genetic Therapies, Inc.
InventorsGaozhong Zhu, Kris Lowe, Zahra Shahrokh, James Christian, Richard 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.
Claims21
1-61. (canceled)
A method of treating Hunter Syndrome comprising a step of administering intraventricularly (ICV) to a human subject in need of treatment a formulation comprising an iduronate-2-sulfatase (I2S) protein at a concentration of at least 5 mg/ml and no greater than 50 mM phosphate.
A method of treating Hunter Syndrome, comprising a step of administering intraventricularly (ICV) to a human subject in need of treatment a formulation comprising an iduronate-2-sulfatase (I2S) protein at a concentration at or greater than 5 mg/ml, salt at a concentration of approximately 154 mM, a polysorbate surfactant at a concentration of approximately 0-0.02%, and a pH of approximately 5.5-6.5;
- wherein the formulation is administered at a volume of about 1-5 ml.
A pharmaceutical composition for intraventricular administration comprising an iduronate-2-sulfatase (I2S) protein at a concentration of at least 5 mg/ml and no greater than 50 mM phosphate.
Dependencies are inferred from the claim text.