AEGIS: Affordable Gene Editing Therapies for Immune System Diseases of Children
The team will develop the in vivo gene editing platform to address inborn errors of immunity (approximately 500 disorders), many of which lack curative therapies and represent significant unmet need. The program will advance a novel, rapid, noninvasive patient identification approach to compress the diagnosis-to-treatment timeline. Together, these capabilities are intended to enable more efficient clinical development and support scalable real-world deployment.