Mort Cowan, MD, a researcher at the Eli and Edythe Broad Center of Regeneration Medicine and Stem Cell Research at UCSF received a $15M grant from the California Institute for Regenerative Medicine (CIRM), the state’s stem cell agency, to advance 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 funding will enable the team at UCSF to refine their gene therapy approach, progressing it from an experimental treatment to a licensed, standard-of-care therapy. Co-Principal Investigator Jennifer Puck, MD, 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 was 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. The collaborative efforts of CIRM and UCSF are pivotal in accelerating the development of transformative treatments for rare and severe conditions.
This award is one of only two CLIN2 grants awarded in this funding round, underscoring the project’s significance in advancing regenerative medicine. The other grant awarded to Donald Kohn, MD, at the UCLA Broad Stem Cell Research Center, advances his gene therapy trial targeting ADA-deficient SCID.
To learn more about the clinical trial for Artemis-SCID and each of these awards, explore the following articles: