Realizing and Sustaining Personalized In Vivo Gene Editing Therapeutics
The team will develop and expand precision genetic medicine platforms to treat a broad range of rare metabolic and hematologic diseases driven by pathogenic mutations, many of which lack curative therapies and represent significant unmet need. The project will advance innovative clinical trial designs, trailblaze regulatory paths, and leverage a multi-clinic care model to ensure scalability, sustainability, and accessibility.