A groundbreaking new initiative at the University of California, San Francisco (UCSF) is aiming to revolutionize treatment for rare genetic diseases in children using cutting-edge CRISPR technology.
Announced on July 8, the Center for Pediatric CRISPR Cures is a collaboration between Nobel Prize-winning scientist Jennifer Doudna and Dr. Priscilla Chan, co-founder of the Chan Zuckerberg Initiative (CZI). The new center will be supported by a $20 million investment from CZI and aims to bring customized gene-editing therapies to children who currently have few, if any, effective treatment options.
The center plans to launch with a clinical trial involving eight children with rare genetic disorders. These patients will receive individually tailored CRISPR-based therapies that target specific genetic mutations in their bodies—an approach pioneered in a recent case that has become a model for the center’s mission.
That case involved KJ Muldoon, a baby treated at the Children’s Hospital of Philadelphia. He was the first patient to receive a personalized CRISPR therapy administered directly inside his body. The treatment, which used a base-editing technique, successfully replaced a faulty gene that had prevented him from properly metabolizing protein.
“This new center builds on the momentum from that success,” said Doudna, co-discoverer of CRISPR and director of the Innovative Genomics Institute at UC Berkeley. “We want to ensure that CRISPR-based therapies become widely available, especially for rare diseases that likely won’t be addressed by pharmaceutical companies.”
Dr. Chan, a pediatrician who trained at UCSF, has long been committed to improving care for children with rare diseases. Recalling encounters with families carrying binders of research for little-known conditions, she described the center as a step toward equity in care and innovation.
Most rare diseases, roughly 6,000 of which affect 300 million people globally, are genetic—and the majority impact children. The center will focus initially on conditions where CRISPR therapy is most likely to succeed, particularly those involving organs like the liver.
Unlike previous gene therapies that required removing and re-editing cells outside the body, these CRISPR treatments will be delivered directly through injections, potentially speeding up treatment and reducing costs. The team also hopes to streamline the regulatory process by establishing CRISPR as a “platform technology,” meaning future therapies could be approved more quickly with minor adjustments.
The initiative underscores the potential of combining medical innovation, philanthropy, and rapid response to bring life-changing therapies to patients most in need. As Dr. Chan put it, “Every child deserves a chance at a healthy life—and we now have the tools to make that possible.”
