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Genetix Pharmaceuticals' Adrenoleukodystrophy (ALD) Product Recognized As One of Science Magazine's Top 10 Scientific Breakthroughs of 2009
Date:1/12/2010

ALD Patients Featured in Science Article Now Living Healthy and Normal Lives Due to Groundbreaking Gene Therapy

Cambridge, MA (Vocus) January 12, 2010 -- Genetix Pharmaceuticals, a leader in gene therapy of somatic stem cells, announced today that Science Magazine has honored the ALD gene therapy research recently published by its collaboratiors at National Institute of Health and Medical Research (INSERM), placing it on their list of ten “Scientific Breakthroughs of 2009.” Genetix is developing this gene therapy product in collaboration with Professor Patrick Aubourg and Doctor Nathalie Cartier of INSERM, and planning to file an IND to expand the development in the US, as well as to expand the ongoing trial in France. The results spotlighted in the Science article show that this innovative therapy has been used to stabilize an otherwise devastating disease in two boys called adrenoleukodystrophy (ALD), or Lorenzo’s Oil disease, in addition to providing a safer alternative to previous therapies.

Adrenoleukodystrophy (ALD) affects 1 in 17,000 males, resulting in severe cognitive and neurologic deficits and eventually leading to death if untreated. It currently is treated with allogeneic bone marrow transplantation, often leading to further complications for the afflicted. 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.

Alfred Slanetz, Chief Executive Officer of Genetix, commented, “We are excited and honored that Science Magazine has recognized the ALD gene therapy product. We look forward to further developing this cutting-edge gene therapy in our own clinical trials, and bringing along a therapy that may be curative to this devastating and often terminal disease. I too would like to acknow
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Genetix Pharmaceuticals' Adrenoleukodystrophy (ALD) Product Recognized As One of Science Magazine's Top 10 Scientific Breakthroughs of 2009
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