15-006 - Reduced Immonogenic protein for lysosomal storage disorders and enzyme replacement therapy

Researchers

Adriano Montano, Alexandra Lee and Catalina Sosa.

Summary

Enzyme Replacement Therapy can be an effective treatment for Mucopolysaccharidosis IVA (Morquio A disease) by reducing the accumulation of glycosaminoglycans. But immune response against the GALNS protein limits it effectiveness. In silico analysis of the cDNA sequences and in vitro testing of re-engineered protein resulted in three mutated GALNS proteins with reduced immunogenicity that exhibited 80 percent or more enzyme activity compared to the wild-type GALNS protein in vitro. Ultimately, a less immunogenic GALNS could remove the two to three year period in which the patient develops tolerance to the protein and eliminate the need for immunosuppressive protocols.

Intellectual Property Status

• U.S. patent pending