Methods and compositions for CNS delivery of heparan N-sulfatase
AssigneeTakeda Pharmaceutical Company Limited
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.
Claims19
A stable formulation for intrathecal administration comprising a heparan N-sulfatase (HNS) protein at a concentration of at least 10 mg/ml, a polysorbate surfactant, and phosphate at a concentration of no greater than 10 mM.
A stable formulation for intrathecal administration comprising a heparan N-sulfatase (HNS) protein at a concentration of approximately 15 mg/ml, NaCl at a concentration of approximately 100-200 mM, polysorbate 20 at a concentration of approximately 0.02%, phosphate at a concentration of approximately 5 mM, and a pH of approximately 7.0.
A stable formulation for intrathecal administration comprising a heparan N-sulfatase (HNS) protein at a concentration up to approximately 15 mg/ml, NaCl at a concentration of approximately 145 mM, polysorbate 20 at a concentration of approximately 0.02%, phosphate at a concentration of approximately 5 mM, glucose at a concentration of approximately 0.5-10%, and a pH of approximately 7.0.
Dependencies are inferred from the claim text.