CAMBRIDGE, Mass. & OXFORD, England — In a landmark step forward for the treatment of liver failure, biotechnology company eGenesis and medical technology firm OrganOx announced that the U.S. Food and Drug Administration (FDA) has cleared their investigational new drug (IND) application to begin clinical trials of a novel therapy for patients with acute-on-chronic liver failure (ACLF).
According to a joint press release issued by the companies on April 15, 2025, the trial will mark the first use in humans of EGEN-5784, a human-compatible, genetically engineered porcine liver developed by eGenesis. The engineered organ will be paired with OrganOx’s extracorporeal liver cross-circulation (ELC) system, forming an innovative perfusion platform designed to temporarily support critically ill patients whose livers are failing and who are not eligible for transplantation.
“This collaboration with eGenesis demonstrates a unique application of our platform metra® technology which can potentially save the lives of patients with acute-on-chronic liver failure who have limited treatment options and face high mortality rates,” said Craig Marshall, Chief Executive Officer of OrganOx, in the announcement.
The Phase 1 trial will enroll up to 20 patients in intensive care units across the U.S., all of whom will have Grade 2 to Grade 3 ACLF with hepatic encephalopathy (Grade 3 or lower) and no transplant eligibility. The study will assess the safety, performance, and potential therapeutic benefit of the EGEN-5784 liver in supporting native liver recovery or acting as a bridge to transplantation.
The announcement highlights the urgent medical need this technology aims to address: an estimated 35,000 patients in the U.S. are hospitalized each year for ACLF, which carries short-term mortality rates of up to 50% due to the lack of viable support options.
“In partnership with OrganOx, we are well positioned to evaluate the potential of this breakthrough technology in patients,” said Michael Curtis, Ph.D., Chief Executive Officer of eGenesis. “The FDA’s clearance of EGEN-5784 in combination with the OrganOx ELC system represents a significant advance towards fulfilling our mission to develop safe and effective human-compatible organs that have the potential to transform the treatment of organ failure and extend patients’ lives.”
The upcoming clinical trial builds on the results of the PERFUSE-2 study, in which four perfusions were successfully completed with decedent recipients, demonstrating the feasibility of the combined system.
The project is the result of an exclusive clinical co-development agreement between eGenesis and OrganOx announced in November 2024, combining eGenesis’ pioneering work in xenotransplantation with OrganOx’s expertise in organ preservation and perfusion.
As the trial moves forward, it could mark a new era in organ support and transplantation—offering a lifeline to patients who currently have none.
