On Jul. 8, 2025, the Chan Zuckerberg Initiative (CZI) and the Innovative Genomics Institute (IGI) announced the funding of the Center for Pediatric CRISPR Cures (Center). The Center will use CRISPR-based editing technology to advance cures for severe pediatric genetic diseases and will bridge CRISPR cure design and testing at the University of California, Berkeley (UC Berkeley) with clinical treatment at the University of California, San Francisco (UCSF). 

The new Center will build on recent clinical success in treating ‘Baby KJ’ Muldoon, an infant born with an exceedingly rare metabolic disease, with the first-ever personalized CRISPR gene-editing therapy. The IGI, a joint effort between UC Berkeley, UCSF and UC Davis founded by Nobel laureate Jennifer Doudna, played a critical role in developing and ensuring the safety of this breakthrough therapy.

The IGI team collaborated with the University of Pennsylvania and the Children’s Hospital of Philadelphia caring for Baby KJ, as well as Danaher Corporation, whose subsidiaries Aldevron and Integrated DNA Technologies (IDT) manufactured components of the CRISPR therapy. IGI and Danaher have partnered over the past two years to create a blueprint for developing and delivering on-demand CRISPR therapies, one that could be used by organizations around the world for treating children with life-threatening genetic diseases.

CZI’s support will allow the Center to apply the framework used for Baby KJ to help other children with ultra-rare diseases. The new Center will develop first-in-kind, personalized CRISPR on-demand treatments for children with severe inborn errors of immunity (IEI) and severe metabolic disease, with the initial aim of treating eight patients. This initiative will be the first step to achieve the larger, long-term goal of the Center to establish a standardized process for the delivery of this type of therapy so that many more families can access treatment.

The Center will coordinate the pre-clinical design and safety testing of CRISPR therapies to treat pediatric patients at UCSF’s Medical Center. The established treatment pipeline (pre-clinical, manufacturing, cell product, clinical and regulatory infrastructure) makes this work possible in just three years. The new Center, which will be funded by a $20 million grant from the Chan Zuckerberg Initiative, will demonstrate the strength of collaboration across the University of California and include Doudna, IGI-partnered clinicians at UCSF Dr. Chris Dvorak, Dr. Jennifer Puck, Dr. Irene Chang, and Dr. Brian Shy, and the IGI CRISPR Cures team at UC Berkeley led by Dr. Fyodor Urnov that co-developed the CRISPR therapy for Baby KJ. Dr. Urnov will act as the director of the Center. The Center’s team combines expertise in childhood diseases, CRISPR therapy development, and clinical safety assessment. Danaher subsidiaries IDT, Aldevron, and Cytiva will collaborate to support the delivery of the necessary clinical-grade therapies.