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HLSC-001 is an allogeneic human liver stem cell (HLSC) therapy developed by Unicyte AG, a subsidiary of Fresenius Medical Care. It consists of a novel population of adult stem cells derived from healthy human liver tissue. The therapy is designed to treat early-onset urea cycle disorders (UCD) by providing a source of functional hepatocytes. When injected percutaneously into the liver, HLSCs are expected to differentiate into urea-producing cells, thereby restoring ureagenesis and facilitating ammonia clearance. It is currently being evaluated as a bridging therapy for pediatric patients with enzyme deficiencies such as ornithine transcarbamylase (OTC) and carbamoyl-phosphate synthase 1 (CPS1) prior to liver transplantation.
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