AEGIS Targets Personalised Editing for Childhood Immune Disorders

Inborn errors of immunity encompass approximately 500 genetic disorders and an estimated 20,000 pathogenic variants, making separate treatment programmes for individual mutations commercially and logistically difficult. AEGIS – Affordable Gene Editing Therapies for Immune System Diseases of Children – will establish a common development, manufacturing and regulatory platform in which the mutation-specific editing component can be rapidly exchanged.

Alexis Komor at UC San Diego will lead base-editor development, while Britt Adamson at Princeton University will develop prime-editing strategies to expand the range of correctable variants. Hyejin Kim and James Dahlman at Emory University and Georgia Tech will develop specialised lipid nanoparticles intended to deliver editors directly to haematopoietic stem cells in the bone marrow, avoiding the collection and ex vivo manipulation of patient cells.

UCLA’s Donald Kohn will lead the planned clinical trial, with additional treatment sites at the University of Utah and Mayo Clinic. The consortium aims to reduce development of an individualised editor to under three months and below $200,000. The delivery approach has been tested in mice and non-human primates, but no patients have yet been treated under AEGIS, and the first target disorders have not been announced.

The news was announced by the Innovative Genomics Institute in a press release on 27 July 2026.