Sticking to its Guns with Gene Therapy, Genzyme To Present Key Findings Within Days
Summary
The world’s largest maker of drugs for genetic diseases has stuck with this field through two decades of ups and downs, and it expects to see important results in coming days on whether it has found an effective approach in people.Then the field ran into a brick wall in 1999 when Arizona teenager Jesse Gelsinger died of a massive inflammatory response in a gene therapy clinical trial.Gene therapy regained some momentum in 2002 when French researchers found that the technique could restore a functioning immune system for infants with severe combined immunodeficiency, sometimes called “bubble boy” disease.Like some other companies, notably Seattle-based Targeted Genetics (NASDAQ: TGEN), it went after what was then considered low-hanging fruit—cystic fibrosis, a deadly lung disease in children and young adults that’s caused by a single faulty gene.If that trial turns out positive, Genzyme will be prepared to advance that program into the final stage of clinical trials—where a competing gene therapy from Sanofi-Aventis has already ventured.