A new center dedicated to advancing CRISPR-based treatments for severe genetic conditions in children has been launched through a $20 million grant from the Chan Zuckerberg Initiative (CZI) to the Innovative Genomics Institute (IGI). The Center for Pediatric CRISPR Cures was announced on July 8 and brings together the gene-editing expertise of the University of California, Berkeley, and the clinical capabilities of the University of California, San Francisco.
This effort follows the case of KJ Muldoon, who recently became the first patient to receive a personalized CRISPR therapy targeting the mutations responsible for his rare metabolic disorder. KJ was born in August 2024 with carbamoyl phosphate synthetase 1 (CPS1) deficiency, a condition that prevents proper ammonia breakdown during protein digestion. His diagnosis led to a six-month development process for a base-editing therapy that was administered through three injections directly into his body. He has since been discharged home with his family and is expected to live with a milder form of the disease.
The new center aims to build on this approach and expand access to similar individualized CRISPR treatments. Its initial clinical efforts will focus on rare genetic disorders affecting metabolism and the immune system. The plan includes treating eight patients as an early step toward establishing a repeatable process for developing and delivering these therapies. According to the announcement, this process could enable wider access to such treatments for additional patients in the future.
Jennifer Doudna, Ph.D., who founded IGI and was involved in co-developing the CRISPR technology, said in the release, “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.”
The center will be led by Fyodor Urnov, Ph.D., of IGI, who also participated in the safety testing of KJ’s treatment. Manufacturing support for the therapies will be provided by Danaher Corporation through its subsidiaries Aldevron and Integrated DNA Technologies. These companies previously supplied materials for KJ’s treatment.
IGI and Danaher have spent the past two years working together to establish a development model for CRISPR treatments that can be created quickly and on demand. The center’s data and processes will be shared with other institutions interested in building similar therapies. IGI is a partnership among UC Berkeley, UC San Francisco, and UC Davis, and was established in 2015.
KJ’s treatment and care were overseen by physicians at the Children’s Hospital of Philadelphia and the University of Pennsylvania, including Rebecca Ahrens-Nicklas, M.D., Ph.D., and Kiran Musunuru, M.D., Ph.D.
The CRISPR model used in KJ’s case differs from previous FDA-approved therapies for sickle cell disease and beta thalassemia, which require extracting and reinfusing modified cells. Instead, the new method delivers the gene-editing components directly into the body. This approach will be used in the center’s upcoming clinical trial.
Each therapy for the initial group of patients will be reviewed individually by the FDA. Over time, there is a possibility that the CRISPR process could be evaluated as a platform, which may reduce the need for repeated safety testing if only the guide RNA is modified. According to Doudna, most of the guide RNA remains unchanged, with only the targeting segment differing for each therapy.
Additional research is ongoing to support the development of predictive tools that may help assess the effects of specific genetic edits and refine treatment design.
$20 Million Pediatric CRISPR Center Launched to Develop Treatments for Rare Genetic Diseases
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