Compositions and methods for delivering enzymes in enzyme hydrogel formulations are disclosed. More particularly, the present disclosure relates to injectable enzyme hydrogel formulations and delivery of injectable enzyme hydrogel formulations. Also disclosed are methods for GALNS enzyme replacement therapy and lysosomal enzyme replacement therapy.

Compositions and methods for delivering enzymes in enzyme hydrogel formulations are disclosed. More particularly, the present disclosure relates to injectable enzyme hydrogel formulations and delivery of injectable enzyme hydrogel formulations. Also disclosed are methods for GALNS enzyme replacement therapy and lysosomal enzyme replacement therapy.

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.

The present invention relates to a method for the treatment of MPS VI comprising administering an arylsulfatase B by gene therapy to a subject in need thereof, wherein said subject is administered with an arylsulfatase B enzyme replacement therapy (ERT) less frequently than once a week.

Described herein are mammalian cells engineered to express and secrete an ARSB protein and optionally a sialytransferase protein, as well as compositions, implantable devices and device preparations comprising the engineered cells, and methods of making and using the same for treating MPS VI disease.

This invention relates to vectors for delivery of ary lsulfatase B to the eye (e.g., cornea) of a subject and methods of using the same for treatment and pre-vention of corneal clouding and blindness in a subject due to mucopolysaccharidosis VI (MPS-VI) and other MPS VI-associated manifestations in the eye.

Compositions and methods for delivering enzymes in enzyme hydrogel formulations are disclosed. More particularly, the present disclosure relates to injectable enzyme hydrogel formulations and delivery of injectable enzyme hydrogel formulations. Also disclosed are methods for GALNS enzyme replacement therapy and lysosomal enzyme replacement therapy.

Described herein are ARSB fusion proteins, polynucleotides encoding the fusion proteins and mammalian cells genetically modified to express and secrete the fusion proteins, as well as compositions, implantable devices and device preparations comprising the fusion proteins or genetically modified cells secreting the fusion proteins, and methods of making and using the same for treating MPS-6.

The present invention relates to a method, preferably a homology independent targeted integration (HITI), of integrating an exogenous DNA sequence into a genome of a cell comprising contacting the cell with a donor nucleic acid comprising said exogenous DNA sequence, optionally one or more albumin exons, wherein said donor nucleic acid is flanked at 5 and 3 by inverted targeting sequences; a complementary strand oligonucleotide homologous to the targeting sequence and a nuclease that recognizes the targeting sequence, wherein said targeting sequence is located at the 3 end of the albumin gene in a region selected from intron 12, intron 13 and intron 14 of said albumin gene. The invention also relates to systems, vectors and pharmaceutical compositions comprising said donor nucleic acid and/or complementary strand oligonucleotide homologous to the targeting sequence and/or nuclease and to medical uses thereof.