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BMN 701: GILT GAA for Pompe Disease

Pompe disease, a lysosomal storage disorder, is a progressive degenerative disease of the heart muscle, diaphragm and skeletal muscle. It is caused by a deficiency in the lysosomal enzyme acid alpha glucosidase which leads to the accumulation of glycogen in myocyte lysosomes and results in cell death. The incidence is one in 40,000 births. There are two main forms of Pompe disease: adult onset with an incidence of one in 57,000 births and infantile onset with an incidence of one in 138,000 births.

The key attribute of BMN 701 is its ability to bind to key cell receptors that direct the enzyme to the cell’s lysosome. Instead of trying to engineer cells to make GAA with higher levels of mannose-6 phosphate, which in the case of GAA lowers cell productivity and significantly increases manufacturing costs, BMN 701 takes advantage of the fact that the peptide IGF-2 also binds to the M6P receptor. Every molecule of BMN 701, a fusion of IGF-2 and GAA, can bind the mannose-6 phosphate receptor, be taken up into cells and trafficked to the lysosome where it can degrade the glycogen that causes Pompe disease.