Methods and compositions for CNS delivery of iduronate-2-sulfatase
AssigneeShire Human Genetic Therapies, Inc.
InventorsGaozhong Zhu, Kris Lowe, Zahra Shahrokh, James Christian, Richard Fahmer, 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.
Claims30
A method of treating Hunter Syndrome comprising a step of
- administering intrathecally 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 at a dose amount of at least 10 mg.
A method of treating Hunter Syndrome, comprising a step of
- administering intrathecally 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 0-300 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 method of treating Hunter Syndrome comprising a step of
- administering intrathecally 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 in a saline solution at a volume at or greater than 1 ml but less than 15 ml.
Dependencies are inferred from the claim text.