Gene Therapy Soldiers On

Vical Inc.'s naked DNA nonviral delivery technology

Even as gene transfer research endures criticism for inadequate clinical trial monitoring and underreporting of adverse events, the first several treatments are poised to enter final stages of development. Gene therapy for cancers of the skin and kidney, for severe combined immunodeficiency (SCID) or "bubble boy" disease, and for hemophilia B, all have produced promising early clinical trial results, attendees were told during the American Society for Gene Therapy's (ASGT's) third annual meeting, May 31 to June 4 in Denver.

These achievements have "really energized" the field, declared society president Savio L. Woo, director of the Institute for Gene Therapy at Mount Sinai School of Medicine. Then he warned, "Until the Phase III results are in, no one can say for sure that gene therapy works." In his presidential address, Woo noted that of 652 patient adverse events reported to the Food...