CZI Gives $20 Million to Fund Center for Personalized CRISPR Cures

Rare Daily Staff

The Chan Zuckerberg Initiative has provided $20 million in funding for a new center designed to build on the recent clinical success of a bespoke gene-editing therapy to treat an infant with a rare metabolic disorder.

CZI’s support of The Center for Pediatric CRISPR Cures will allow the Center to apply the framework used to treat the infant known as Baby KJ to help other children with ultra-rare diseases. Fyodor Urnov, who led the team at the Innovative Genomics Institute (IGI) at the University of California, Berkeley—which co-developed the CRISPR therapy for Baby KJ—will serve as director of the new center.

The Center for Pediatric CRISPR Cures will develop first-in-kind, personalized CRISPR on-demand treatments for children with severe inborn errors of immunity and metabolic diseases, with an initial aim of treating eight patients. The center will be powered by IGI, which will design and test treatments. The University of California, San Francisco will provide clinical care for patients treated by the center.

The IGI team collaborated with the University of Pennsylvania, the Children’s Hospital of Philadelphia, and Danaher Corporation—whose subsidiaries Aldevron and Integrated DNA Technologies manufactured components of the CRISPR therapy—in treating the infant. 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.

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.

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—including pre-clinical, manufacturing, cell product, clinical, and regulatory infrastructure—makes this work possible in just three years.

The initiative will be the first step toward the center’s larger, long-term goal of establishing a standardized process for delivering this type of therapy, so that many more families can access treatment.

“There is a natural alignment between the mission of the IGI to make CRISPR gene editing the standard of care for genetic diseases and CZI’s ambitious mission to help scientists cure all diseases,” said Doudna. “We’ve already seen the profound impact that an on-demand CRISPR therapy can have for one family. Now we want to ensure that this approach can scale and be made available for more children around the world.”