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Insulin-like growth factor-2 (IGF-2) peptide tag to improve enzyme replacement therapies to treat Pompe's disease

A lysosome-targeting peptide tag may be useful for producing Pompe's disease enzyme replacement therapies that have better tissue uptake than unmodified acid a-glucosidase (GAA). A fusion protein consisting of a portion of IGF-2 linked to recombinant human GAA was expressed in and purified from mammalian cells. In vitro, the resulting BMN-701 (IGF-2-GAA) showed better uptake by rodent myoblasts than unmodified GAA. In a mouse model of Pompe's disease, BMN-701 led to lower levels of glycogen in heart, diaphragm and skeletal muscle than unmodified GAA. BMN-701 is in Phase I/II testing to treat Pompe's disease, with topline data expected 1Q13.
Myozyme and Lumizyme are human recombinant GAA enzyme replacement therapies marketed by Sanofi's Genzyme Corp. unit to treat Pompe's disease (see Muscling up on Myozyme, page 5).

SciBX 5(48); doi:10.1038/scibx.2012.1271
Published online Dec. 13, 2012

Findings patented; licensing status undisclosed

Maga, J.A. et al. J. Biol. Chem.; published online Nov. 27, 2012;
Contact: Jonathan H. LeBowitz, BioMarin Pharmaceuticals Inc., Novato, Calif.