Genzyme Corp. has won orphan drug designation for itsrecombinant protein that replaces the gene product that isdamaged in cystic fibrosis, the company said Thursday.
Working with Tufts University researchers, the Cambridge,Mass., company (NASDAQ:GENZ) has produced the membraneprotein in the milk of transgenic mice. The protein is a chloridechannel that sits in the cell membrane in the airways, allowingthe proper production of mucus. In cystic fibrosis, the proteinis unable to reach the cell surface.
Genzyme wants to use goats to scale up research quantities ofthe protein, which is called the cystic fibrosis transmembraneregulator.
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