CAMBRIDGE, Mass., May 22 /PRNewswire-FirstCall/ -- Genzyme Corp.
(Nasdaq: GENZ) today announced the publication of a new study in The
Proceedings of the National Academy of Sciences describing a novel
preclinical gene therapy approach for treating Niemann-Pick disease.
The study, conducted by a team of Genzyme researchers, demonstrated
that administering gene therapy both systemically and directly into the
brain helped to preserve motor and cognitive functions and significantly
extend lifespan in a mouse model of Niemann-Pick disease. The authors write
that this combination approach may represent a promising clinical strategy
for treating diseases that affect visceral organ systems as well as the
central nervous system. Current treatments which do not cross the
blood-brain barrier, such as enzyme replacement therapy, are limited in
their effectiveness for such disorders with brain involvement.
In the study, Genzyme researchers tested the combination of brain and
systemic injections of adeno-associated viral vectors encoding human acid
sphingomyelinase, the enzyme that is deficient in patients with
Niemann-Pick disease types A & B. This combination approach was compared to
mice treated singly with either of the two modes of delivery, and to mice
that were left untreated.
After 54 weeks of observation, the researchers found that combination
therapy led to preservation of motor and cognitive functions at near normal
levels. The combination-treated group also lived longer and was free of
many of the neurological and physical manifestations of the disease shown
in either of the singly treated groups or the non-treated group.
Importantly, researchers established immune system tolerance to the therapy
by administering it systemically before it was administered to the brain,
which the paper suggests may be an important part of any strategy to apply
these findings to a clinical setting.
The work published today builds on an extensive program at Genzyme to
develop new and next-generation therapies for lysosomal storage disorders.
Genzyme developed and now provides four enzyme replacement therapies for
lysosomal storage disorders including Cerezyme(R) (imiglucerase for
injection) for Gaucher disease, Fabrazyme(R) (agalsidase beta) for Fabry
disease, Aldurazyme(R) (laronidase) for MPS I and Myozyme(R) (alglucosidase
alfa) for Pompe disease. In addition, Genzyme is conducting a Phase 1
clinical trial of a potential enzyme replacement therapy for the treatment
of ASM-deficient Niemann Pick disease. And the company recently completed
enrollment in its ongoing Phase 2 clinical trial testing a novel oral
therapy for Gaucher disease.
In addition to its preclinical work with lysosomal storage disorders,
Genzyme's gene therapy portfolio includes two ongoing clinical trials, the
most advanced of which is examining the safety and effectiveness of locally
delivered Ad2/HIF-1 alpha, an engineered form of the HIF-1 alpha gene. This
experimental therapy is designed to promote the growth of new blood vessels
and improve circulation in the limbs of patients with peripheral arterial
disease. Additionally, Genzyme is enrolling patients in a Phase 1-2
clinical trial of a gene therapy for Parkinson's disease designed to
restore the therapeutic effectiveness of levo-Dopa, the primary treatment
for Parkinson's disease, by enhancing the brain's ability to convert it
into dopamine.
Genzyme is also conducting pre-clinical research using gene therapy in
atrial fibrillation and macular degeneration through partnerships with
Excigen, Inc., and Applied Genetic Technologies Corp., respectively.
About Genzyme
One of the world's leading biotechnology companies, Genzyme is
dedicated to making a major positive impact on the lives of people with
serious diseases. Since 1981, the company has grown from a small start-up
to a diversified enterprise with more than 9,000 employees in locations
spanning the globe and 2006 revenues of $3.2 billion. Genzyme has been
selected by FORTUNE as one of the "100 Best Companies to Work for" in the
United States.
With many established products and services helping patients in nearly
90 countries, Genzyme is a leader in the effort to develop and apply the
most advanced technologies in the life sciences. The company's products and
services are focused on rare inherited disorders, kidney disease,
orthopaedics, cancer, transplant, and diagnostic testing. Genzyme's
commitment to innovation continues today with a substantial development
program focused on these fields, as well as immune disease, infectious
disease, and other areas of unmet medical need.
Genzyme(R), Cerezyme(R), Fabrazyme(R), Aldurazyme(R) and Myozyme(R) are
registered trademarks of Genzyme Corporation. All rights reserved.
Genzyme's press releases and other company information are available at
http://www.genzyme.com and by calling Genzyme's investor information line at
1-800-905-4369 within the United States or 1-703-797-1866 outside the
United States.
Media Contact: Investor Contact:
Dan Quinn Sally Curley
(617) 768-6849 (617) 768-6140
SOURCE Genzyme Corp.
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Related links:
http://www.genzyme.com/
http://www.prnewswire.com/comp/113803.html/
CONTACT:
Media, Dan Quinn, +1-617-768-6849, or
Investors, Sally Curley, +1-617-768-6140, both of Genzyme Corp.
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