Gene Therapy Technology Licensed from National Institute of Health and Medical
Research (INSERM) Featured in November Issue of Science
CAMBRIDGE, Mass.--(Business Wire)--
Genetix Pharmaceuticals, a leader in gene therapy of somatic stem cells,
announced today the company`s clinical program to treat Adrenoleukodystrophy
(ALD). The company is developing a gene therapy product in collaboration with
Professor Patrick Aubourg and Doctor Nathalie Cartier of the National Institute
of Health and Medical Research (INSERM). Genetix is planning to file an IND to
expand the development in the US, as well as to expand the ongoing trial in
France. The current development timeline should make the product commercially
available to patients by 2014. Patrick Aubourg and Nathalie Cartier will become
scientific advisors to Genetix`s ALD clinical programs.
ALD is a devastating disease that afflicts 1/17,000 males with onset of severe
cognitive and neurologic deficits between the ages of 5 and 12, or in adulthood.
It progresses to death within 3-10 years of diagnosis. Many people became
familiar with ALD from the 1992 movie Lorenzo's Oil, which featured a young boy
affected with this disease.
ALD is currently treated with allogeneic bone marrow transplantation when there
is an appropriate donor. Even so, the graft sometimes does not take and there is
Graft Versus Host Disease leading to death in 15% of children and at least 30%
of adults post transplant. For those patients who survive the transplant
procedure, the disease is stabilized and no longer progresses. In 50% of the
patients who survive, Graft Versus Host Disease or delayed hematological
reconstitution delay the time at which disease is stabilized, allowing the
disease to have progressed further before stabilization.
Patrick Aubourg, M.D. of Inserm-University Paris Descartes commented, "We are
delighted to develop this ground breaking gene therapy product together with
Genetix, the leading gene therapy company. Our future close scientific
collaboration is off to a good start. Production of large GMP batches is
underway, which will allow us to rapidly treat the rest of the patients in this
clinical trial. We look forward to working with Genetix to expand the trials and
acquire the pivotal data and regulatory approvals needed to bring this product
to market."
Gene therapy places a healthy gene into the patients` own bone marrow stem
cells. In the first two patients described in the November issue of Science, the
disease has been stabilized. To view the Science article, click on the following
link: http://www.sciencemag.org/cgi/content/abstract/326/5954/818
Genetix`s gene therapy approach has the following advantages over allogeneic
bone marrow transplantation:
* Applicable to all patients and no limitation to have a matched donor;
* No risk of death from Graft Versus Host Disease by using patients` own cells;
* More rapid engraftment/reconstitution of marrow and immune function post
transplant due to perfect match between patients` stem cells and marrow mileu.
To date, in both patients treated, the therapy has been safe and efficacious. As
with all drugs in development, the ongoing clinical trials are designed to
statistically prove safety and efficacy for regulatory approval.
Alfred Slanetz, Chief Executive Officer of Genetix, commented, "We congratulate
our colleagues and collaborators at INSERM on their publication and
accomplishments. Today two of two treated children with ALD, who otherwise would
be severely cognitively impaired and on a road to death, are able to live
healthy and normal lives because of the ALD lentiviral gene therapy. Developing
safe and effective treatments for incurable diseases is what our industry is all
about. Genetix is now committed to developing this product in the most
scientific and ethical way, and to make this product commercially available to
all patients afflicted with this devastating disease."
About Genetix Pharmaceuticals
Genetix is a biopharmaceutical company with operations in Cambridge, MA and
Paris, France. Its products are curative and treat the cause of genetically
based diseases by placing a healthy gene into the patient`s extracted bone
marrow or other somatic stem cells, and transplanting these corrected cells back
into the patient. The Genetix clinical pipeline includes: Lenti-D for
Adrenoleukodystrophy (ALD) now in development, and Lentiglobin for
Thalassemia/Sickle Cell Disease also in clinical development with projected
launch in 2014 and 2015, respectively. The company is a leader in the gene
therapy of somatic stem cells, GMP production and product development. Its
proprietary platform technology is applicable to the development of curative
therapies for numerous important diseases. The company`s unique technology,
production capability and international team enable global reach with modest
infrastructure. As the leading global gene therapy company, Genetix is committed
to developing curative therapies for the world`s patients.
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Genetix Pharmaceuticals
Alfred E. Slanetz, Ph.D, 617-491-5601
President & CEO
aslanetz@genetixpharm.com
OR
LaVoie Group
Lisa Rivero, 978-745-4200 X 106
Director of Media Relations
lrivero@lavoiegroup.com
Copyright Business Wire 2009
