KJ Muldoon recently made history by becoming the first patient to receive a personalized CRISPR gene therapy, designed specifically for the genetic mutations causing his rare metabolic disease. Now, a new $20 million effort from the Chan Zuckerberg Initiative (CZI) and Innovative Genomics Institute (IGI) endeavors to ensure KJ’s treatment isn’t a one-off.
The Center for Pediatric CRISPR Cures, announced in a July 8 release, will bring together the gene editing expertise of the University of California, Berkeley, with the clinical know-how of its neighbor, the University of California, San Francisco, to develop cures for infants with severe genetic diseases. The $20 million funding for the center comes from a grant from CZI.
"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," Jennifer Doudna, Ph.D., the CRISPR pioneer and 2020 Nobel Laureate who founded IGI, said in the release. "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."
The center’s initial focus will be on genetic diseases affecting immunity and metabolism, with the goal of treating eight patients as a first step towards a long-term goal of establishing “a standardized process for the delivery of this type of therapy so that many more families can access treatment,” CZI and IGI said in the release.
The new initiative will be led by IGI’s Fyodor Urnov, Ph.D., who helped test the safety of KJ’s custom treatment. IGI’s longtime partner, the Danaher Corporation, will also provide manufacturing services to the center through subsidiaries Aldevron and Integrated DNA Technologies, as it did for KJ.
IGI and Danaher have been working together over the past two years to create a “blueprint” for on-demand CRISPR therapies, according to the release. Data from the center’s efforts will also be made available to other institutions who are interested in crafting their own custom CRISPR therapies.
IGI is a collaboration between UC Berkeley, UC San Francisco and UC Davis, and was founded by Doudna in 2015.
KJ's custom treatment and care was led by pediatrician and metabolic disease expert Rebecca Ahrens-Nicklas, M.D., Ph.D., and cardiologist and Verve Therapeutics co-founder Kiran Musunuru, M.D., Ph.D., both of the Children’s Hospital of Philadelphia and the Perelman School of Medicine at the University of Pennsylvania.
KJ was born in August 2024 with a urea cycle disorder called carbamoyl phosphate synthetase 1 (CPS1) deficiency, which is the lack of an enzyme important for processing ammonia during protein digestion. His birth sparked a six-month race to generate a treatment, and while he will likely live with a milder form of the disease for the rest of his life, he has since gone home with his family for the first time.