Ultragenyx reported that its gene therapy candidate for ornithine transcarbamylase (OTC) deficiency met one of two co‑primary endpoints in a Phase 3 study involving 37 patients. The company characterized the readout as a significant efficacy signal in a rare metabolic disorder where therapeutic options are limited. Management framed the result as a validation of their delivery and expression strategy; the outcome will inform regulatory discussions and potential labeling claims. Investors and rare‑disease stakeholders will watch for the second endpoint readout and durability data to judge the therapy’s clinical and commercial potential.