Mayo Clinic Joins $27.7m ARPA-H CRISPR Trial for Children

Mayo Clinic has joined AEGIS, a multi-consortium project funded by up to $27.7 million from the US Advanced Research Projects Agency for Health (ARPA-H) to develop affordable, scalable gene-editing therapies for children with rare inherited immune disorders. Announced on 27 August 2026, the five-year initiative is led by the Innovative Genomics Institute at UC Berkeley and will use CRISPR-based technologies to repair disease-causing errors in blood-forming stem cells.

Inborn errors of immunity comprise more than 500 rare genetic disorders that leave children vulnerable to severe infections, autoimmune disease and other life-threatening complications, many with limited treatment options and no cure. AEGIS aims to develop one-time CRISPR-based treatments that restore normal immune function. The consortium brings together researchers from the Innovative Genomics Institute (UC Berkeley), UCLA, Stanford, University of Utah, Princeton, UC San Diego, Emory, Danaher Corporation and the Immune Deficiency Foundation, among others. Mayo Clinic will serve as one of three clinical sites conducting the planned clinical trials.

Dr Avni Joshi, chair of Mayo Clinic’s Division of Pediatric Allergy and Immunology and lead principal investigator for Mayo, framed the initiative as combining gene editing with new delivery methods to address rare disease at its root cause. Mayo Clinic brings expertise in rare immune disorders, paediatric transplantation and advanced cellular and gene therapies. The AEGIS team will combine CRISPR gene editing with innovative manufacturing and delivery technologies to make precision genetic medicines more accessible and affordable, with the ambition of establishing a scalable framework that could support treatments for hundreds of rare genetic diseases beyond the initial immune disorder targets.

The commercial signal is significant federal validation for the CRISPR gene editing rare disease category, alongside the earlier ARPA-H $175m autonomous stroke robotics funding. Rare immune disorder gene therapy represents a substantial commercial adjacency. Expect CRISPR Therapeutics, Intellia, Beam Therapeutics, Editas Medicine, Prime Medicine and Rocket Pharmaceuticals to sharpen their positioning as ARPA-H validation reshapes the rare disease competitive landscape through 2027.

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