New funding brings personalized CRISPR cures closer to children with rare diseases

Personalized CRISPR cures for children born with rare genetic diseases are now a step closer to being more widely available. Today, the Chan Zuckerberg Initiative (CZI) and the Innovative Genomics Institute (IGI) announced the funding of the Center for Pediatric CRISPR Cures (Center). The Center will use CRISPR-based editing technology to advance cures for severe pediatric genetic diseases and will bridge CRISPR cure design and testing at the University of California, Berkeley (UC Berkeley) with clinical treatment at the University of California, San Francisco (UCSF). 

The new Center will build on recent clinical success in treating 'Baby KJ' Muldoon, an infant born with an exceedingly rare metabolic disease, with the first-ever personalized CRISPR gene-editing therapy. The IGI, a joint effort between UC Berkeley, UCSF and UC Davis founded by Nobel laureate Jennifer Doudna, played a critical role in developing and ensuring the safety of this breakthrough therapy. The IGI team collaborated with the University of Pennsylvania and the Children's Hospital of Philadelphia caring for Baby KJ, as well as Danaher Corporation, whose operating companies Aldevron and Integrated DNA Technologies (IDT) manufactured components of the CRISPR therapy. 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.

"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." 

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 (IEI) and severe metabolic disease, with the initial aim of treating eight patients. This initiative will be the first step to achieve the larger, long-term goal of the Center to establish a standardized process for the delivery of this type of therapy so that many more families can access treatment.

As a pediatrician trained at UCSF, a national referral center for children with rare diseases, I know firsthand the heartbreak of telling parents that we don't understand their child's illness or that we don't know how to treat them. For nearly a decade, CZI has supported rare disease communities through our Rare As One Project and Network, empowering patient-led organizations with the tools and resources they need to drive research and accelerate the search for treatments and cures. This new Center builds on that commitment and aims to bridge research to impact on the lives and futures of families facing ultra-rare diseases."

Dr. Priscilla Chan, co-CEO and co-founder of CZI

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 (pre-clinical, manufacturing, cell product, clinical and regulatory infrastructure) makes this work possible in just three years. The new Center, which will be funded by a $20 million grant from the Chan Zuckerberg Initiative, will demonstrate the strength of collaboration across the University of California and include Doudna, IGI-partnered clinicians at UCSF Dr. Chris Dvorak, Dr. Jennifer Puck, Dr. Irene Chang, and Dr. Brian Shy, and the IGI CRISPR Cures team at UC Berkeley led by Dr Fyodor Urnov that co-developed the CRISPR therapy for Baby KJ. Dr. Urnov will act as the director of the Center. The Center's team combines expertise in childhood diseases, CRISPR therapy development, and clinical safety assessment. Danaher operating companies IDT, Aldevron, and Cytiva will collaborate to support the delivery of the necessary clinical-grade therapies.

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. Additionally, IGI and CZI together will engage with patient communities, including those in the Rare As One Network, and other stakeholders to develop recommendations for making CRISPR cures more affordable and accessible.