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News from Genzyme May 22, 2007
Genzyme Announces Publication in PNAS Describing Novel Gene
Therapy Approach for Niemann Pick Disease
Date: May 22 2007
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® (imiglucerase for injection) for Gaucher disease,
Fabrazyme® (agalsidase beta) for Fabry disease, Aldurazyme® (laronidase)
for MPS I and Myozyme® (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-1alpha, 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.
http://www.genzyme.com/corp/media/GENZ
PR-052207.asp#TopOfPage
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