CNS delivery of therapeutic agents
AssigneeShire Human Genetic Therapies, Inc.
InventorsPericles Calias, Jing Pan, Jan Powell, Lawrence Charnas, Thomas McCauley, Teresa Leah Wright, Richard Pfeifer, Zahra Shahrokh
Life of this patent
Estimating the term…
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.
Claims6
A method of treating a lysosomal storage disease associated with reduced level or activity of a lysosomal enzyme comprising a step of:
- administering intrathecally to a subject in need of treatment, a composition comprising a replacement enzyme for the lysosomal enzyme at a concentration at or greater than about 5 mg/ml at a dose amount of at least 10 mg, wherein the composition comprises phosphate at a concentration of no greater than 50 mM.
Dependencies are inferred from the claim text.