A University of Pennsylvania and Children’s Hospital of Philadelphia case study highlighted meaningful clinical benefits from an mRNA-based CRISPR base-editing approach in an infant with CPS1 deficiency, but the lead scientist emphasized it was not a traditional cure campaign. Investigators reported that the patient can eat a normal protein-filled diet, with the goal of delaying liver transplant until later. In comments tied to the reported outcomes, Penn’s Kiran Musunuru argued the intervention was an expanded-access IND clinical-care effort rather than a clinical trial or research program, calling it “unambiguously not a cure.” He framed the work as a demonstration of feasibility that has helped accelerate the broader push to get base editing into clinical development. The case also points to ongoing competition in base editing programs, with companies such as Verve Therapeutics and YolTech Therapeutics pursuing their own clinical trajectories.