New $20M Center Launches to Expand Custom CRISPR Therapies for Rare Pediatric Diseases

In a significant step forward for personalized gene therapy, the Chan Zuckerberg Initiative (CZI) and Innovative Genomics Institute (IGI) have announced the launch of the Center for Pediatric CRISPR Cures. This $20 million initiative aims to build upon the recent success of the first customized CRISPR treatment for a rare genetic disorder, with the goal of developing cures for more infants suffering from severe genetic diseases.

Collaborative Effort to Scale Personalized Gene Therapies

The new center will combine the gene editing expertise of the University of California, Berkeley with the clinical capabilities of the University of California, San Francisco. Led by IGI's Fyodor Urnov, Ph.D., the initiative will initially focus on genetic diseases affecting immunity and metabolism.

Jennifer Doudna, Ph.D., CRISPR pioneer and 2020 Nobel Laureate who founded IGI, emphasized the alignment between IGI's mission and CZI's goals: "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."

Building on Recent Success

The center's creation follows the groundbreaking treatment of KJ Muldoon, an infant born in August 2024 with a rare urea cycle disorder called carbamoyl phosphate synthetase 1 (CPS1) deficiency. KJ became the first patient to receive a personalized CRISPR gene therapy designed specifically for his genetic mutations.

The treatment, developed and administered in just six months, was led by experts from the Children's Hospital of Philadelphia and the Perelman School of Medicine at the University of Pennsylvania. While KJ will likely live with a milder form of the disease, he has since been able to go home with his family for the first time.

Expanding Access and Establishing Standards

The Center for Pediatric CRISPR Cures aims to treat eight patients as an initial step towards establishing a standardized process for delivering personalized gene therapies. This effort is supported by IGI's partnership with the Danaher Corporation, which will provide manufacturing services through its subsidiaries Aldevron and Integrated DNA Technologies.

Data from the center's work will be made available to other institutions interested in developing their own custom CRISPR therapies, potentially accelerating the broader adoption of personalized gene editing treatments for rare genetic disorders.