Chan Zuckerberg Initiative Launches Center for Personalized CRISPR Cures for Rare Pediatric Diseases

SAN FRANCISCO, July 8, 2025 /PRNewswire/ — The Chan Zuckerberg Initiative (CZI) has launched the Center for Pediatric CRISPR Cures, an ambitious new initiative dedicated to developing personalized gene editing therapies for children suffering from rare inherited immune conditions and metabolic diseases. Part of the Innovative Genomics Institute (IGI) at University of California, Berkeley, the center is backed by a $20 million grant from CZI.

This new endeavor builds upon the recent, rapid success in developing a custom CRISPR therapy for “Baby KJ,” an infant with a debilitating metabolic disease. This bespoke treatment, conceived by researchers at the University of Pennsylvania and the Children’s Hospital of Philadelphia (CHOP), used CRISPR base editing to correct his unique genetic mutation. Administered in February, the therapy has restored his ability to break down protein and significantly improved his health, allowing him to return home in June after more than 300 days in the hospital.

Fyodor Urnov, a gene editing scientist at the IGI and leader of the new center, described Baby KJ’s success as a “yes we can” moment, shifting perspectives on the feasibility of rapid, custom gene therapies. The Center for Pediatric CRISPR Cures aims to design and treat eight patients with bespoke therapies over the next three years.

Urnov acknowledges the ambitious timeline and budget, given the traditional complexities of drug development. The goal is to “aggressively walk the walk of CRISPR platformization,” streamlining regulatory review and manufacturing processes by treating gene editing as a versatile platform rather than a series of individual drugs. This approach aims to dramatically reduce the time and cost associated with bringing these life-changing treatments to patients.

Scientists at UC Berkeley will be responsible for designing the gene editing therapies, while doctors at UCSF will handle patient treatment. The center will also leverage a previous partnership between the IGI and Danaher, a life science supply and manufacturing company, which was instrumental in creating Baby KJ’s therapy.