Drug Development

Enrollment completed in clinical trial for Gaucher disease

Published in Blood Weekly, August 26th, 2004

Transkaryotic Therapies, Inc. (TKTX) has completed enrollment of an open-label phase 1/2 study to evaluate the safety and clinical activity of GA-GCB, its investigational enzyme replacement therapy for the treatment of Gaucher disease.

The study enrolled 12 patients with type I Gaucher disease from several countries. TKT expects to release top-line data in the second half of 2005.

Lead investigator of the study, Ari Zimran, Shaare Zedek Medical Center, Jerusalem, Israel, stated, "I am honored and excited to lead the first Gaucher study evaluating an alternative enzyme replacement therapy for Gaucher patients. Since this therapeutic protein is...

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Source: Blood Weekly (2004-08-26)

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