The invention relates to a method for providing a drug delivery system, in particular for crossing the blood brain barrier (BBB), comprising a virus-like particle (VLP) derived from John Cunningham virus (JCV), a drug delivery system and novel VLP obtainable by said method. The method comprises steps of disassembly of VLP into pentamers and reassembly into VLP.

The invention relates to an enzyme product comprising a beta-galactosidase product. The invention provides that the beta-galactosidase product has an inactive arylsulphatase.

Provided herein are expression cassettes for expressing a transgene in a liver cell, wherein the transgene encodes an ARSA polypeptide. Also provided are methods to treat metachromatic leukodystrophy (MLD). Further provided herein are vectors (e.g., rAAV vectors), viral particles, pharmaceutical compositions and kits for expressing an ARSA polypeptide in an individual in need thereof.

Provided are methods of treating metachromatic leukodystrophy comprising administering to a subject in need of treatment a therapeutically effective amount of recombinant arylsulfatase A enzyme.

The present invention provides, among other things, methods of treatment of Metachromatic Leukodystrophy Disease (MLD) and compositions comprising recombinant arylsulfatase A (ASA) protein using enzyme replacement therapy.

The invention relates to virus like particles (VLP) associated with a lysosomal enzyme or an expression vector encoding a lysosomal enzyme which are used in a method for the treatment of a lysosomal storage disease. The invention also relates to a pharmaceutical composition for use in a method for the treatment of a lysosomal storage disease, to an expression vector encoding a lysosomal enzyme and to a method of associating a VLP with an expression vector encoding a lysosomal enzyme.

Provided are methods of treating metachromatic leukodystrophy comprising administering to a subject in need of treatment a therapeutically effective amount of recombinant arylsulfatase A enzyme.

The present invention relates to a fusion protein between a therapeutic enzyme and an immunoglobulin Fc region, a method thereof, and a composition comprising the fusion protein.

Blends of enzymes of the same enzyme class (same EC number), such as beta-glucuronidase enzyme blends, are provided that exhibit synergistic levels of activity across a range of substrates as compared to the activity levels of each enzyme in the blend individually. The blends also may exhibit a greater effective substrate range, as well as greater effective pH and/or temperature ranges as compared to single enzyme preparations. Predictive methods for determining optimal enzyme blends, methods for preparing enzyme blends, enzyme blend compositions, enzyme blend formulations and methods of using such enzyme blends are provided.

The present invention provides, among other things, improved methods for purifying arylsulfatase A (ASA) protein produced recombinantly for enzyme replacement therapy. The present invention is, in part, based on the surprising discovery that recombinant ASA protein can be purified from unprocessed biological materials, such as, ASA-containing cell culture medium, using a process involving as few as four chromatography columns and only one step of post-chromatographic ultrafiltration/diafiltration.