United Therapeutics is developing a gene therapy product called Aurora-GT (endothelial nitric oxide-synthase-enhanced endothelial progenitor cells), which is intended to rebuild blood vessels in the lung. Administering this gene therapy involves isolating a PAH patient’s autologous endothelial progenitor cells, transfecting the cells with the gene for human endothelial NO-synthase, prior to their ex-vivo expansion, and reintroducing them into the same patient.
United Therapeutics is aiming to diversify its pulmonary hypertension (PH) portfolio with a pipeline gene therapy product, Aurora-GT (endothelial nitric oxide-synthase-enhanced endothelial progenitor cells). The company stands out as the only one with a late-phase gene therapy in development. If successful, this therapy could address critical unmet need in the PH market for options beyond symptomatic treatment. Datamonitor Healthcare awaits further developments for Aurora-GT, as Phase II/III clinical trial data are yet to be released.
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