Competitive EnvironmentThe muted stock response seems likely to reflect the competitive environment for treating urea-cycle disorders where one-and-done approaches, such as gene-therapy and gene-editing, are on the table.
Data LimitationsDosing variability, lack of baseline characteristic data, and lack of patient-level data for some of the key biomarkers complicate efficacy interpretation.
Regulatory UncertaintyDefinite limitations of the current data set (small N, differing protocols) and uncertainty on the regulatory path.