Technology and Products
LentiGlobin™ hemoglobin gene therapy has demonstrated 100% correction in animal models of sickle cell disease and thalassemia major, the two most prevalent human genetic disorders, with large orphan markets. No current cure exists for thalassemia: patients die young, have major complications, and must wear a pump 12 hours per day. With a worldwide prevalence of over one million patients, the major markets for thalassemia treatment will be Western Europe (25,000 patients), Saudi Arabia and other Arab countries (10,000s), and China/Asia (estimated 100,000s). Sickle cell disease affects 75,000 patients in the US alone, with additional cases in the EU. GENETIX will support clinical centers of excellence in the EU and the US, enabling additional patients with means to travel from all parts of the globe for a cure.
Gene Therapy
The GENETIX Phase I/II clinical trial is among the first clinical trials to be initiated since the shut-down in early 2003 of numerous gene therapy clinical trials by the U.S. Food and Drug Administration and its counterparts in other countries. The shut-down followed adverse results observed in two patients in a French clinical trial against a rare and fatal genetic disease known as severe combined immune deficiency (SCID, or "boy-in-a-bubble" disease). Nevertheless, all ten children who received a therapeutic dose of the gene therapy in that trial are now living at home and are apparently cured of their terminal disease -- the first such outcome in the history of gene therapy -- as reported in a recent issue of the journal Nature Medicine.
The clinical trial investigators along with GENETIX scientists and others have further demonstrated that the adverse results in the French clinical tiral, comprising an unusual lymphoproliferative blood disorder which has persisted in one child, were caused by a combination of biological factors specific to SCID disease and to the particular form of gene therapy used in the clinical trial. These results have contributed to the lifting of the 2003 regulatory bans on a broad category of gene therapy trials. GENETIX believes clinical trials of its LentiGlobin™ hemoglobin gene therapy should be as effective as the French gene therapy, but without the severe adverse effects because of the Company's inclusion of numerous, patented, biological safety enhancements in its gene therapy design.
