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BMN-701

BioMarin Pharmaceutical Inc.

Drug Names(s): BMN 701, ZC-701, BMN701, IGF2-GAA

Description: ZC-701 is a recombinant protein containing ZyStor's proprietary Glycosylation Independent Lysosomal Targeting (GILT) tag fused to acid-alpha-glucosidase (GAA), thereby allowing more efficient delivery of GAA to the lysosome of muscle cells.

This proprietary technology is termed GILT because the peptide tag replaces the M6P carbohydrate as the moiety targeting the lysosome. The GILT-tag is derived from IGF-II, which is a high affinity ligand for the CI-MPR receptor, the same receptor on the cell surface that recognizes M6P-modified proteins. Binding of GILT-tagged therapeutic enzymes to the CI-MPR receptor targets the protein to the lysosome via the endocytic pathway. Thus, the pathway that GILT-tagged proteins take to the lysosome is identical to that taken by M6P-containing proteins, including the initial interaction with the CI-MPR receptor.

Deal Structure: ZC-701 was originally developed by ZyStor Therapeutics.

In August 2010, BioMarin announced that it has acquired ZyStor Therapeutics. Under the terms of the agreement, BioMarin acquired ZyStor for $22 million upfront and up to an additional $93 million if certain development, regulatory and commercial milestones are achieved. There are no royalties owed.


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