Provided herein are compositions, kits and methods related to permitting a human or non-human primate subject to receive multiple doses of recombinant adeno-associate virus (rAAV) vectors. In some aspects, provided herein are methods comprising administering an anti-CD20 antibody, optionally in combination with an mTOR inhibitor, to the subject to permit multiple doses of an rAAV vector to be administered.

Nucleic acid regulatory elements that are able to enhance muscle-specific expression of genes, methods employing these regulatory elements and uses of these elements. Expression cassettes and vectors containing these nucleic acid regulatory elements are also disclosed. The present invention is particularly useful for applications using gene therapy, more particularly muscle-directed gene therapy.

Methods for the production, capturing and purification of recombinant human lysosomal proteins are described. Such recombinant human lysosomal proteins can have high content of mannose-6-phosphate residues. Also described are pharmaceutical compositions comprising such recombinant human lysosomal proteins, as well as methods of treatment and uses of such recombinant human lysosomal proteins.

The invention relates to a kit of parts comprising (i) pharmacological chaperones or a pharmaceutically acceptable salt thereof and (ii) a therapeutic acid-alpha glucosidase (GAA) polypeptide or a nucleic acid molecule encoding a therapeutic GAA polypeptide, wherein said pharmacological chaperones are 1-deoxynojirimycin (DNJ) or a derivative thereof and ambroxol (ABX) or a derivative thereof.

The present invention is related to a dual promoter lentiviral vector and methods of use for the treatment of diseases and disorders, specifically lysosomal storage disorders.

Disclosed herein are methods of treating a toxicity in a subject receiving recombinant viral vector, such as a recombinant adeno-associated viral AAV (rAAV) vector comprising co-administration of an antibiotic and a viral vector. In one embodiment, the antibiotic is a tetracycline or macrolide family member. Also provided herein are compositions comprising an antibiotic and a viral vector.

Recombinant human alpha glucosidase (rhGAA) composition derived from CHO cells that contains a more optimized glycan composition consisting of a higher amount of rhGAA containing N-glycans carrying mannose-6-phosphate (M6P) or bis-M6P than conventional rhGAAs, along with low amount of non-phosphorylated high mannose glycans, and low amount of terminal galactose on complex oligosaccharides. Compositions containing the rhGAA, and methods of use are described.

The disclosure relates, in general, to Glycogen Storage Disease and, in particular, to a method of treating Glycogen Storage Disease and to compounds and compositions suitable for use in such a method.

Provided herein are edible compositions for reducing semen viscosity and/or enhancing semen flavor, and methods of using and preparing such compositions.

A method for treating Pompe disease including administration of recombinant human acid α-glucosidase having optimal glycosylation with mannose-6-phosphate residues in combination with an amount of miglustat effective to maximize tissue uptake of recombinant human acid α-glucosidase while minimizing inhibition of the enzymatic activity of the recombinant human acid α-glucosidase is provided.