CAMBRIDGE, Mass., July 26 /PRNewswire-FirstCall/-- Transkaryotic
Therapies, Inc. (Nasdaq: TKTX) announced today that it has completed
enrollment of an open-label Phase I/II 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 twelve patients with
Type I Gaucher disease from several countries. TKT expects to release top-
line data in the second half of 2005.
The lead investigator of the study, Professor Ari Zimran of Shaare Zedek
Medical Center in 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 produced in human
cells, I believe it may have important effects in treating the disease."
About Gaucher Disease
Gaucher disease is the most common lysosomal storage disease and is caused
by an inherited deficiency of the enzyme glucocerbrosidase. This deficiency
results in the accumulation of the substrate glucocerbroside in the phagocytic
cells of the liver, spleen and bone marrow. The symptoms of Gaucher disease
range in severity and may include liver and spleen enlargement, bleeding
tendency (due to low platelet counts), fatigue (due to anemia), bone pains and
fractures. Currently, there is one enzyme replacement therapy approved for
the treatment of Gaucher disease.
About TKT
Transkaryotic Therapies, Inc. is a biopharmaceutical company primarily
focused on researching, developing and commercializing treatments for rare
diseases caused by protein deficiencies. Within this focus, the company
markets Replagal(TM), an enzyme replacement therapy for Fabry disease, and is
developing treatments for Hunter syndrome and Gaucher disease. In addition to
its focus on rare diseases, TKT intends to commercialize Dynepo(TM), its Gene-
Activated(R) erythropoietin product for anemia related to kidney disease, in
the European Union. TKT was founded in 1988 and is headquartered in
Cambridge, Massachusetts, with additional operations in Europe, Canada and
Latin America. Additional information about TKT is available on the
company's website at http://www.tktx.com.
This press release contains forward-looking statements regarding the
company's development of GA-GCB for Gaucher disease, as well as statements
containing the words "believes," "anticipates," "plans," "expects,"
"estimates," "intends," "should," "could," "will," "may," and similar
expressions. There are a number of important factors that could cause the
company's actual results to differ materially from those indicated by forward-
looking statements, including, whether GA-GCB will be safe and effective as a
treatment for Gaucher disease; whether TKT will adequately manufacture
adequate supply for, and otherwise complete, clinical trials of GA-GCB;
whether future trials of GA-GCB will be conducted; whether future trials of
GA-GCB will commence on a timely basis; whether the trial described in this
press release will be sufficient for regulatory approval or additional studies
will be required; whether the FDA and equivalent authorities will approve GA-
GCB on a timely basis, or at all; whether if approved, this product will
achieve commercial success; whether competing products will reduce any market
opportunity that may exist for GA-GCB; whether TKT will be successful in
partnering GA-GCB; and other factors set forth under the caption "Risk
Factors" in the company's Current Report on Form 8-K filed April 29, 2004,
which is on file with the Securities and Exchange Commission and which risk
factors are incorporated herein by reference. While the company may elect to
update forward-looking statements at some point in the future, the company
specifically disclaims any obligation to do so, even if its expectations
change.
Gene-Activated(R) is a registered trademark and Replagal(TM) is a
trademark of Transkaryotic Therapies, Inc. Dynepo(TM) is a trademark of
Aventis Pharmaceuticals, Inc.
For More Information Contact:
Justine E. Koenigsberg
Senior Director, Corporate Communications
(617) 349-0271
Daniella M. Lutz
Corporate Communications Manager
(617) 349-0205
SOURCE Transkaryotic Therapies, Inc.
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http://www.tktx.com
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CONTACT:
Justine E. Koenigsberg, Senior Director,
Corporate Communications, +1-617-349-0271, or Daniella M. Lutz,
Corporate Communications Manager, +1-617-349-0205, both of
Transkaryotic Therapies, Inc.
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