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Getting the latest healthcare news for you
Mayo Clinic is teaming up with a major gene-editing consortium to help cure rare immune disorders in children. The AEGIS project, backed by up to $27.7 million from ARPA-H, will use CRISPR technology to correct genetic mutations in blood-forming stem cells — aiming for one-time treatments that restore immune function. Mayo will serve as one of three clinical trial sites, enrolling children with inborn errors of immunity.
Mayo Clinic has signed on as a clinical collaborator for AEGIS, a five-year, $27.7 million initiative funded by the Advanced Research Projects Agency for Health (ARPA-H). Led by the Innovative Genomics Institute at UC Berkeley, the project brings together a broad consortium of academic, clinical, and industry partners with one ambitious goal: developing affordable, one-time gene-editing therapies for children with rare inherited immune disorders.
The focus is on inborn errors of immunity — a group of over 500 rare genetic conditions that leave children dangerously vulnerable to severe infections, autoimmune disease, and life-threatening complications, often with no cure in sight. Using CRISPR-based technologies, researchers will target disease-causing mutations in blood-forming stem cells to restore normal immune function from the ground up.
By the Numbers:
Why it matters: For children with ultra-rare immune disorders, treatment options are often limited and burdensome. AEGIS aims to build a scalable gene-editing platform that could eventually address hundreds of rare diseases — potentially transforming one-time therapy from a dream into a clinical reality.
