The Chan Zuckerberg Initiative (CZI) and the Innovative Genomics Institute (IGI) announced the funding of the new 'Center for Pediatric CRISPR Cures' that will use CRISPR-based editing technology to advance cures for severe pediatric genetic diseases and will bridge CRISPR cure design and testing with clinical treatment.
The new center, which will be funded by a $20 million grant from the Chan Zuckerberg Initiative, will coordinate the preclinical design and safety testing of CRISPR therapies to treat pediatric patients at the University of California, San Francisco’s (UCSF) medical center. The established treatment pipeline makes this work possible in just three years.
The IGI, a joint effort between UC Berkeley, UCSF and UC Davis and founded by Nobel laureate Jennifer Doudna, played a critical role in developing and ensuring the safety of the breakthrough therapy that treated 'Baby KJ' Muldoon, an infant born with a rare metabolic disease.
CZI's support will allow the center to apply the framework used for Baby KJ to help other children with ultra-rare diseases. The new center will develop first-in-kind, personalized CRISPR on-demand treatments for children with severe inborn errors of immunity and severe metabolic disease, with the initial aim of treating eight patients.
With all appropriate confidentiality considerations, the center will ensure that data from all investigative new drug-enabling studies, regulatory documentation, and clinical trials is available to other U.S. academic centers in pediatric disease that wish to design and deploy their own CRISPR-based therapies for similar cases.
Dr. Fyodor Urnov, a co-developer of the CRISPR therapy for Baby KJ, will act as the director of the new center.
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