Mayo Clinic is a clinical collaborator on an award of up to $27.7 million from the Advanced Research Projects Agency for Health (ARPA-H) to help develop affordable, scalable gene-editing therapies for children with rare inherited immune disorders.
The project, known as AEGIS, is led by the Innovative Genomics Institute at the University of California, Berkeley, and brings together a consortium of academic, clinical, nonprofit and industry partners.
The five-year initiative aims to advance gene-editing treatments for children with inborn errors of immunity - a group of more than 500 rare genetic disorders that can leave children vulnerable to severe infections, autoimmune disease and other life-threatening complications. Many of these conditions currently have limited treatment options and no cure.
Mayo Clinic will serve as one of three clinical sites participating in the program's planned clinical trials, helping identify, enroll and care for children who may benefit from these investigational therapies.
By combining advances in gene editing with new ways to deliver treatments, we hope to create therapies that not only treat disease but address its root cause. Our goal is to free patients from the burden of ongoing treatments, and ultimately improve the lives of patients and families."
Avni Joshi, M.D., chair of Mayo Clinic's Division of Pediatric Allergy and Immunology and lead principal investigator for Mayo on the project
Gene editing seeks to correct the genetic mutations that cause disease. In AEGIS, researchers will use CRISPR-based technologies to repair disease-causing errors in blood-forming stem cells that give rise to the immune system. The goal is to develop one-time treatments that restore normal immune function and potentially provide lasting benefit for children with these rare disorders.
The award reflects Mayo Clinic's growing leadership in genomic medicine, pediatric immunology and next-generation therapies. As a participating clinical center, Mayo Clinic will help evaluate how emerging gene-editing technologies can be safely integrated into patient care and expanded to reach more children with rare diseases.
Mayo Clinic brings expertise in rare immune disorders, pediatric transplantation, and advanced cellular and gene therapies. As a member of the Primary Immune Deficiency Treatment Consortium and a FACT-accredited transplant center, Mayo will help guide the clinical evaluation of these emerging gene-editing approaches.
The AEGIS team will combine advances in CRISPR gene editing, innovative manufacturing approaches and new delivery technologies to make precision genetic medicines more accessible and affordable for patients with ultra-rare diseases. Researchers hope the work will establish a scalable framework that can accelerate the development of treatments for a broad range of rare immune disorders.
The consortium includes researchers and clinicians from the Innovative Genomics Institute at UC Berkeley, UCLA, Stanford University, the University of Utah, Princeton University, the University of California San Diego, Emory University, Danaher Corporation, the Immune Deficiency Foundation and other partners.
The project aligns with ARPA-H's mission to accelerate breakthroughs that improve health outcomes and with the agency's THRIVE program, which seeks to expand access to transformative genetic medicines. While the initial work will focus on several severe inherited immune disorders, researchers hope the platform could eventually support the development of treatments for hundreds of rare genetic diseases, expanding access to precision therapies for patients who currently have few options.
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