At ASGCT 2026, Kiran Musunuru cautioned that a high-profile gene-editing case for urea cycle disorder should not be treated as a cure and was not a substitute for controlled clinical evidence. In remarks at the society’s conference, Musunuru described the Baby KJ story as an N-of-1 outcome that aims to reduce a devastating disease to a milder, manageable condition, while emphasizing the limits of what can be concluded without clinical trials. He urged the field to avoid “snake oil” framing and stressed ethical obligations not to mislead families about what is possible, even as gene editing momentum continues in rare disease settings.