New Pediatric CRISPR Center Launched to Help Children with Genetic Diseases

In a groundbreaking move towards personalized medicine, the Chan Zuckerberg Initiative (CZI) and the Innovative Genomics Institute (IGI) have announced the establishment of the Center for Pediatric CRISPR Cures. This center aims to advance the development of CRISPR-based therapies for children suffering from rare genetic disorders, making strides towards making these innovative treatments widely available.

The launch was officially announced on July 8, 2025, with the center set to leverage CRISPR gene-editing technology in the treatment of severe pediatric genetic diseases. The initiative aims to connect the design and testing of CRISPR cures at the University of California, Berkeley (UC Berkeley) to clinical implementations at the University of California, San Francisco (UCSF).

The center will build on the success observed in treating an infant known as 'Baby KJ' Muldoon, who was born with a uniquely rare metabolic disease. The clinical advancements seen in Baby KJ's personalized CRISPR treatment were made possible through collaborative efforts spearheaded by the IGI, an organization founded by Nobel Prize winner Jennifer Doudna. Not only did Doudna's team at IGI contribute to the creation of Baby KJ's therapy, but they also ensured its safety through rigorous testing. Their partnership with Children's Hospital of Philadelphia and Danaher Corporation played a crucial role in the successful treatment.

“The alignment between IGI’s mission to integrate CRISPR technology into standard care for genetic diseases and CZI’s goal to help scientists conquer all diseases is profound,” remarked Doudna. “The impact of an on-demand CRISPR therapy has been evident for Baby KJ’s family, and together we aspire to provide this life-changing solution for many more children around the globe.”

CZI’s involvement will provide the financial support needed to adapt the therapeutic framework that benefited Baby KJ, enabling the center to assist other children facing ultra-rare diseases. The center intends to create customized CRISPR treatments aimed at addressing severe inborn errors of immunity and metabolic diseases—for an initial group of eight patients. This endeavor signals the beginning of a longer-term vision to standardize CRISPR therapy delivery, thus enhancing access for numerous families.

CZI co-founder and pediatrician Dr. Priscilla Chan expressed her personal connection to the mission, having witnessed the emotional trauma involved when families are informed that their children's illnesses lack clear diagnoses or treatments. “For nearly a decade, CZI has advocated for rare disease communities through the Rare As One Project. This latest initiative strengthens our commitment to bridge research and tangible impacts on the lives of families affected by ultra-rare diseases,” she stated.

The center’s primary focus will be on the pre-clinical design and safety assessments of CRISPR therapies intended for pediatric patients in UCSF's Medical Center. With a well-established treatment pipeline encompassing pre-clinical research, manufacturing, and clinical trials, the center aims to make significant advancements within three years. Funded by a generous $20 million grant from the Chan Zuckerberg Initiative, the new center exemplifies the potential of collaborative efforts across various University of California campuses. Notable figures, including IGI and UCSF clinicians such as Dr. Chris Dvorak, Dr. Jennifer Puck, and Dr. Brian Shy, will be instrumental in directing and executing the center’s mission.

The collaborative team combines knowledge across the fields of childhood diseases, CRISPR development, and clinical safety, promising a robust foundation for this innovative approach. Danaher partners, namely Integrated DNA Technologies (IDT), Aldevron, and Cytiva, will also provide essential support in delivering clinical-grade therapies.

Maintaining confidentiality, the Center for Pediatric CRISPR Cures will ensure that critical data from all drug-enabling studies, regulatory documentation, and clinical trials can be shared with other U.S. academic institutions looking to create their own CRISPR therapies for similar cases. Additionally, IGI and CZI will work closely with patient communities, including those within the Rare As One Network to create strategies for making CRISPR cures more affordable and accessible.

As the center marks a significant leap in pediatric genetic disease treatment, it represents hope for countless families seeking effective solutions for their children’s rare medical conditions. All stakeholders involved are eager to witness the positive transformations that personalized medicine could bring in pediatric care and beyond.