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# FDA Fast Track for UCSF Gene Therapy
- URL: https://www.fdaweb.com/fda-fast-track-for-ucsf-gene-therapy/
- Published: 2022-02-16T12:00:00.000Z
- Updated: 2026-09-14T17:32:52.000Z
- Author: David McFarland
- Tags: Biologics, #legacy-id-D5151180

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](https://www.ucsf.edu/news/2022/02/422276/ucsf-gene-therapy-deadly-mutation-fast-tracked-fda-review?ref=fdaweb.com) 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.