On Nov. 21, 2024, the California Institute for Regenerative Medicine (CIRM) announced it had approved awarding $29.7 million to fund two clinical projects aimed at addressing rare pediatric diseases. 
The awards will support projects in the Agency’s clinical program which provides funding for eligible stem cell and gene therapy-based projects through all stages of clinical trial development. These awards bring the number of CIRM-funded clinical trials to 111. 

Donald B. Kohn, MD, of UCLA was awarded $14 million to support a clinical trial evaluating a gene-edited stem cell therapy for the treatment of Adenosine Deaminase-Deficient Severe Combined Immunodeficiency (ADA-SCID), a rare and life-threatening condition in which babies are born without a functioning immune system.

Another CIRM award aimed to support a potential therapy for a rare pediatric disease. Mort Cowan, MD, of UCSF received a $15 million award to support a clinical trial for Artemis-deficient severe combined immunodeficiency (Artemis-SCID), a life-threatening genetic disorder that primarily affects infants.