ARPA-H Funds Initiative to Develop Affordable Gene-Editing Therapies for Rare Immune Diseases in Children
The Advanced Research Projects Agency for Health (ARPA-H) has allocated up to $27.7 million to a collaborative project led by the Innovative Genomics Institute at the University of California, Berkeley. This initiative, part of the THRIVE program, aims to develop affordable gene-editing therapies for children with inborn errors of immunity (IEI). The project, known as AEGIS, seeks to create a scalable solution by delivering gene editors directly to patients' bone marrow using lipid nanoparticles. This approach could eliminate the need for chemotherapy and stem cell transplantation, which are currently required for genetic therapies. The team includes experts from various institutions, including UCLA, the University of Utah, and Mayo Clinic, and aims to treat 10 children within the next five years. 
