ASGCT Press Release

ASGCT Members Report Successful Results

Milwaukee, WI - November 6, 2009 - A group of ASGCT Members reported successful results yesterday in the current issue of Science of a Hematopoietic stem cell gene therapy with a lentiviral vector in X-linked adrenoleukodystrophy (ALD), a potentially crippling and fatal disorder that attacks the central nervous system.

The new gene therapy technology allows patients to be their own stem cell donor, replacing the need to find a compatible donor and eliminating the risk of graft versus host disease. The Science paper gives the results of two boys who underwent gene therapy over two years ago to treat their ALD. The gene therapy is successful to date, arresting the progression of ALD in the boys and stabilizing their condition.

"This study is an exciting first step in the use of lentiviral vectors in gene therapy," said Ken Cornetta, MD, ASGCT President, "and represents wonderful news for patients and families dealing with this devastating disease."

ASGCT will be updating the Gene & Cell Therapy section of its homepage (www.asgct.org) with the latest news related to the recent publication.

The American Society of Gene & Cell Therapy (ASGCT) is a professional non-profit medical and scientific organization dedicated to the understanding, development and application of genetic and cellular therapies and the promotion of professional and public education in the field. For more information on ASGCT, visit its website, www.asgct.org.

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