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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

ledge the pioneering and steadfast work of Patrick Aubourg and Doctor Nathalie Cartier, with whom we will continue to work closely as our research and development progresses.”

To view the Science article that recognizes the work of Genetix and INSERM, please click on the following link. http://www.sciencemag.org/cgi/content/full/326/5960/1600

About Genetix Pharmaceuticals
Genetix is the leading global gene therapy company developing curative therapies for genetic diseases. The company’s proprietary platform treats the cause of genetic diseases by placing a healthy gene into the patient's extracted bone marrow stem cells, and transplanting these corrected stem cells back into the patient. Genetix's lead clinical product is Lenti-D™ for Adrenoleukodystrophy (ALD), also known as Lorenzo's Oil disease, which is now in clinical development. The results of the ongoing ALD trial, based upon work conducted by the National Institute of Health and Medical Research (INSERM), were named to Science Magazine's "Scientific Breakthroughs of 2009." Genetix has in-licensed the technology from INSERM, and plans to file for pivotal studies in 2010 in both the U.S. and Europe. In addition to ALD, Genetix is conducting Phase I/II trials with its LentiGlobin™ product in Thalassemia/Sickle Cell Disease, the two most prevalent human genetic disorders. To date, all patients receiving the full therapy with both products have been cured and are healthy. The company’s proprietary stem cell processing, GMP manufacturing and gene therapy vector technologies are also applicable to treating other genetic diseases moving forward. Genetix is privately financed by an experienced syndicate of healthcare investors, including TVM Capital, Forbion Capital Partners and Easton Capital. For more information, please visit
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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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