Cns delivery of therapeutic agents
AssigneeTakeda Pharmaceutical Company Limited
InventorsPericles Calias, Jing Pan, Jan Powell, Lawrence Charnas, Thomas McCauley, Teresa Leah Wright, Richard Pfeifer, Zahra Shahrokh
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Abstract
The present invention provides an effective and less invasive approach for direct delivery of therapeutic agents to the central nervous system (CNS). In some embodiments, the present invention provides methods including a step of administering intrathecally to a subject suffering from or susceptible to a lysosomal storage disease associated with reduced level or activity of a lysosomal enzyme, a composition comprising a replacement enzyme for the lysosomal enzyme.
Claims32
A formulation comprising a lysosomal enzyme at a concentration of at least 5 mg/mL and a surfactant, wherein the formulation comprises no greater than 50 mM phosphate, and wherein the formulation is suitable for CNS delivery of the lysosomal enzyme.
A method of treating a lysosomal storage disease associated with reduced level or activity of a lysosomal enzyme comprising delivering a replacement enzyme to the CNS of a subject in need of treatment, wherein the lysosomal enzyme is administered at a concentration of at least 5 mg/mL in a formulation comprising no greater than 50 mM phosphate.
A method of treating a lysosomal storage disease associated with reduced level or activity a lysosomal enzyme comprising delivering a replacement enzyme to the CNS of a subject in need of treatment, wherein the lysosomal enzyme is administered at a dose amount of at least 10 mg in a formulation comprising no greater than 50 mM phosphate.
Dependencies are inferred from the claim text.