UCSF Broad Stem Cell Center researchers, Drs. Morton J. Cowan, MD, and Jennifer Puck, MD, are advancing a pioneering gene therapy trial for infants with Artemis-deficient Severe Combined Immunodeficiency (SCID). This life-threatening condition leaves children without a functioning immune system, making even minor infections potentially fatal.
The team at UCSF is refining its gene therapy approach, progressing it from an experimental treatment to a licensed, standard-of-care therapy. Dr. Jennifer Puck emphasized the transformative potential of the therapy, which has demonstrated exceptional results. “Our outcome data continues to surpass expectations, allowing most treated infants to discontinue all medications, including immune globulin infusions—a milestone rarely achieved even with matched sibling donor transplants,” said Dr. Puck.
This work has been made possible through the unwavering support and visionary leadership of the UCSF Chancellor and the UCSF Helen Diller Family Comprehensive Cancer Center. Their dedication to fostering innovation in gene therapy and regenerative medicine has been instrumental in advancing this trial, bringing it closer to becoming a standard-of-care treatment and providing renewed hope to families affected by Artemis-deficient SCID.
This trial is part of a collaborative effort to accelerate the development of transformative treatments for rare and severe conditions. Another significant gene therapy trial targeting ADA-deficient SCID is being advanced by Donald Kohn, MD, at the UCLA Broad Stem Cell Research Center.