Biological preparations for medical use; pharmaceutical preparations, namely, recombinant adeno-associated viral (rAAV) vectors for use in gene therapy; Pharmaceutical preparations for the treatment of genetic diseases and disorders and central nervous system (CNS) disorders, namely, neurodegenerative disorders; Pharmaceutical preparations for use in gene therapy; Clinical medical reagents for use in gene therapy; Pharmaceutical preparations for the treatment of genetic disorders and neurodegenerative disorders; Therapeutic pharmaceutical compositions that include recombinant adeno-associated viral (rAAV) vectors to provide a platform for specific expression of biologics with therapeutic effect in gene therapy; Pharmaceutical preparations containing adeno-associated viral (AAV) vectors and nucleic acids for a system delivering genes to biological cells for use in the treatment of diseases, sicknesses, and disorders, namely, lysosomal storage disorders; gene therapy preparation containing genetic materials for sustained protein expression, namely, lysosomal enzyme expression
Pharmaceuticals