CAMBRIDGE, Mass. & WASHINGTON--(BUSINESS WIRE)--Nathalie Cartier-Lacave, M.D., study investigator and a director of research at the National Institute of Health and Medical Research in France (INSERM), and collaborator of Genetix Pharmaceuticals, Inc., today presented additional data from a pilot study treating X-linked adrenoleukodystrophy (ALD) at the American Society of Gene and Cell Therapy’s 13th Annual Meeting. The findings demonstrate continued stable expression of ALD protein and disease stabilization at three years in two ALD patients. Initial data on a third patient show stable expression of the protein at 20 months; however, current data are insufficient to determine disease stabilization. Genetix, an emerging leader in developing breakthrough gene therapies for severe genetic disorders, plans to initiate larger clinical studies in ALD in both the United States and Europe in 2011.
