FDA Fast Track for UCSF Gene Therapy

FDA has granted a Regenerative Medicine Advanced Therapy designation to a gene therapy developed by University of California San Francisco researchers to treat Artemis-SCID. The designation means the research is considered promising and will be expedited for development and review.

The university says Artemis-SCID is a severe form of immunodeficiency caused by mutations in a specific gene. It says a simple infection can easily kill children who have the disease in their first year of life if it is left untreated.

Even with a bone marrow transplant — the current standard treatment — children with SCID often fail to develop a normal immune system and require repeated infusions to stay alive, the university says.

The clinical trial involves transferring a normal copy of the mutated gene into the patients’ own stem cells. The researchers say early data from the trial are encouraging.