Company Overview

Genetix is the leading gene therapy company for the development of breakthrough treatments for severe genetic disorders. 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 French 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 initiate pivotal studies in both the U.S. and Europe. In addition to ALD, Genetix is conducting Phase 2 trials with its LentiGlobin™ product in Thalassemia, one of the 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.