Ethical Framework for n-of-1 Clinical Trials of Genome Editor Therapeutics for Personalized, Single-Dose Therapies

While n-of-1 trials (clinical trials conducted in a single patient) are not new, their use in CRISPR-based trials of rare genetic disease is novel and poised to become a viable option for many patients over the next decade. Existing guidance for n-of-1 trials is largely designed around drug trials and lacks meaningful integration of public perspectives. This project seeks to solicit the perspectives of patients/caregivers, clinicians, scientists, and bioethicists regarding the design and conduct of such trials, and convene these actors to co-develop ethical guidance for their design and conduct.