Thursday, December 13, 2012
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Insulin-like growth factor-2 (IGF-2)
peptide tag to improve enzyme replacement therapies to treat Pompe's disease
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
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.
are human recombinant GAA enzyme replacement therapies marketed by Sanofi's
Corp. unit to treat Pompe's disease (see Muscling up on
Myozyme, page 5).
Published online Dec. 13, 2012
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.
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