Mayo Clinic is joining a $27.7 million initiative from the Advanced Research Projects Agency for Health (ARPA-H) to advance affordable and scalable gene-editing treatments for children with rare inherited immune disorders.
The five-year programme, known as AEGIS, is led by the Innovative Genomics Institute at the University of California, Berkeley, and brings together academic, clinical, nonprofit and industry partners. Mayo Clinic will serve as one of three clinical sites involved in planned trials, helping identify and care for children who could benefit from the 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,” says Avni Joshi, M.D., chair of Mayo Clinic’s Division of Pediatric Allergy and Immunology and the lead principal investigator for Mayo on the project. “Our goal is to free patients from the burden of ongoing treatments, and ultimately improve the lives of patients and families.”
The project focuses on inborn errors of immunity, a group of more than 500 rare genetic disorders that can cause severe infections, autoimmune conditions and other serious complications. Many affected children currently have limited treatment options.
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Researchers will use CRISPR-based gene-editing technologies to target disease-causing mutations in blood-forming stem cells that generate immune cells. The aim is to develop potentially one-time treatments that can restore immune function and provide lasting benefits.
The initiative also seeks to improve the manufacturing and delivery of genetic medicines, making them more accessible and affordable. Researchers hope the approach can eventually support treatments for a wider range of rare genetic diseases.
