CRANBURY, N.J., March 20, 2007 /PRNewswire/ -- Amicus Therapeutics, a biopharmaceutical company developing small molecule, orally-administered pharmacological chaperones for the treatment of a range of human genetic diseases, announced today that it will present the results of preclinical studies of Plicera(TM) (isofagomine tartrate, AT2101) for Gaucher disease at the of Medical Genetics (ACMG) annual meeting March 21-25 in Nashville, TN. The data demonstrate the ability of Plicera to increase levels of the target enzyme in cells derived from a patient with the N370S mutation and in mice that express the L444P mutation. The N370S and the L444P are the two most common mutations associated with Gaucher disease. Additional data from both studies will also be presented.
Plicera is designed to selectively bind to and stabilize GCase, the enzyme deficient in Gaucher disease. This deficiency leads to lysosomal accumulation of glucocerebroside inside certain cells, which is believed to cause the various symptoms of Gaucher disease. Plicera facilitates proper trafficking of the enzyme to the lysosomes, the compartments in the cell where it is needed to break down glucocerebroside.
The following is a summary of the preclinical Plicera data being presented at ACMG.
- In vitro exposure to Plicera increased transport of GCase to the lysosomes in cells derived from a patient with the N370S mutation. Once in the lysosome, the enzyme was stable and active for more than 3 days after Plicera was removed. The N370S is the most common mutation associated with Gaucher disease in the Western world. These studies were published in the September 12, 2006 issue of the Proceedings of the National Academy of Sciences (PNAS). - Oral administration of Plicera resulted in a dose-dependent increase of
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