Shire Human Genetic Therapies, Inc.

Takeda Pharmaceutical Company Limited

University of Minnesota

Synageva Biopharma Corporation

Lawrence Charnas

Pericles Calias

Zahra Shahrokh

Jing Pan

Teresa Leah Wright

Jan Powell

Thomas Mccauley

Richard Pfeifer

Nazila Salamat-Miller

Katherine Taylor

Paul Campolieto

Michael W Heartlein

Michael F Concino

Paolo Martini

Muthuraman Meiyappan

Dennis Keefe

Serene Josiah

Sujit Basu

Alla Romashko

Brian Felice

Thomas M Luby

Atsushi Asakura

Mayank Verma

Brian Pescatore

Nancy Savioli

Keethkumar Jain

Verma Mayank

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.

CNS delivery of therapeutic agents

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 arylsulfatase A (ASA) protein, salt, and a polysorbate surfactant for the treatment of Metachromatic Leukodystrophy Disease.

Methods and compositions for CNS delivery of arylsulfatase A

The present invention provides, among oilier things, compositions and methods for CNS delivery of lysosomal enzymes (e.g., recombinant human arylsulfatase A (rhASA)) for effective treatment of lysosomal storage diseases (e.g. Metachromatic Leukodystrophy Disease), In some embodiments, the present invention includes a stable formulation for intrathecal administration comprising an ASA protein and a poloxamer, wherein less than 3% of the ASA protein exists in aggregated form.

Stable formulations for CNS delivery of arylsulfatase A

The present invention provides, among other things, methods and compositions for treating muscular dystrophy, in particular, Duchenne muscular dystrophy (DMD). In some embodiments, a method according to the present invention includes administering to an individual who is suffering from or susceptible to DMD an effective amount of an anti-Flt-1 antibody, or antigen binding fragment thereof, such that at least one symptom or feature of DMD is reduced in intensity, severity, or frequency, or has delayed onset.

Anti-FLT-1 antibodies in treating duchenne muscular dystrophy

The present invention provides compositions and methods for effective treatment of a lysosomal acid lipase deficiency (LALD) disease, in particular, Wolman's disease and Cholesteryl Ester Storage Disease (CESD). Among other things, the present invention provides a method of treating developmental impairment or malnutrition in an individual suffering from a lysosomal acid lipase deficiency (LALD) disease, comprising administering to the individual a therapeutic effective amount of a lysosomal acid lipase.

Methods for treating lysosomal acid lipase deficiency

Growing community of inventors

Natick, MA, United States of America

Lawrence Charnas