Improved method to produce proteins to treat lysosomal storage disorders (SJ-01-0020)

St. Jude Reference #SJ-01-0012


The inventors discovered that proteins produced in insect cells by standard baculovirus expression systems are glycosylated in a unique way, which causes them to be selectively taken up by cells of the reticuloendothelial system, including macrophages. This discovery has particular significance for proteins used to treat lysosomal storage disorders(LSDs) in which the  primary therapeutic target for such proteins are macrophages. Simply by producing such proteins in insect cells, this discovery reveals that they will be targeted for uptake by macrophages.


Lysosomal storage disorder, macrophage, baculovirus, insect cell, protein production

Granted patents or published applications

U.S. Patent Nos. 7,232,670 and 7,241,442

Related scientific references

Bonten et al., American Journal of Human Genetics, Suppl. Vol. 69 (4): Abst.1759 (Oct. 2001);

Bonten EJ, et al., "Targeting macrophages with baculovirus-produced lysosomal enzymes:  implications for enzyme replacement therapy of the glycoprotein storage disorder galactosialidosis", FASEB J. 18(9):  971-973 (June 2004).

Licensing opportunities

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