UCSF Team Submits Application for First In Utero Gene Therapy Trial

The trial would treat five fetal patients with a rare lysosomal storage disorder. The agency informed the team that animal testing could be bypassed because the safety profile of the planned vector has already been characterized by other researchers and companies developing gene therapies for children and adults.

Background on the Research The effort builds on work begun more than 25 years ago when researchers tested gene replacement therapy in mice with inherited disorders. Experiments at that time successfully treated mice with hemophilia and tyrosinemia. The team has held discussions with the Food and Drug Administration that the researchers described as promising.

The current application represents the closest approach yet to conducting gene therapy in human fetuses. The proposed trial would mark the first in utero gene therapy study in humans if approved.