Wednesday, May 15, 2024

eGenesis and OrganOx announce successful use of genetically modified pig liver from human donors

eGenesis a biotechnology company developing human-compatible organs and cells to treat organ failure and focusing on therapeutic applications of isolated organ perfusion. Medical device company OrganOx is pleased to announce that it has successfully completed extracorporeal perfusion of a brain-dead research donor using genetically engineered pig liver. This donor was the first donor enrolled in the ongoing PERFUSE-2 study.

Made possible by the generosity of donor families who seek to help other families through advances in clinical research, this study was conducted in partnership with the University of Pennsylvania Transplant Institute and the Gift of Life Donor Program. Perfusion was performed using an eGenesis liver EGEN-5784 connected to an OrganOx extracorporeal liver cross circulation (ELC) device for circulation of donor blood through the pig liver. Stable blood flow, pressure, pH, and robust bile production were maintained throughout the procedure. No evidence of rejection was identified. Perfusion was selectively stopped at 72 h according to the protocol, and the liver showed normality.

The PERFUSE-2 study is being conducted to assess the feasibility of using this liver perfusion system to support patients suffering from liver failure. In the United States, more than 300,000 patients are hospitalized each year with various forms of liver failure and require acute treatment. Existing liver support options have limited effectiveness, and patients with liver failure face a high risk of mortality. In some patients, human-compatible genetically modified whole pig livers are utilized to support the function of the patient’s decompensated liver, allowing time for the patient’s native liver to recover or undergoing a liver transplant. You may get more time. eGenesis and OrganOX are co-developing this technology and plan to submit an investigational new drug (IND) application to the U.S. Food and Drug Administration (FDA) and begin first-in-human clinical trials in 2024.

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The genetically engineered pig livers used in this study had the same genetics as the pig kidneys used in a landmark preclinical study recently published in the journal Nature . These edits include: (1) Knockout of three genes involved in the synthesis of glycan antigens involved in hyperacute rejection. (2) Insertion of seven human transgenes involved in the regulation of pathways regulating rejection (inflammation, innate immunity, coagulation, complement). (3) Inactivation of endogenous retroviruses in the pig genome.

Dr. Michael Curtis, President and CEO of eGenesis, said: “The donors and their families who made this important medical achievement possible and paved the way for future efforts towards potential solutions for the many patients in need of life-saving liver support. We would like to express our deep gratitude to the authors. This study is not only the first of its kind in the field of xenotransplantation, but also contains important information to advance our IND application.”

“We are very excited about this new study,” said Abraham Shaked, MD, PhD, of the Transplant Institute at the University of Pennsylvania. “The success of this study supports further exploration of organ products developed using advanced genomic engineering to provide new, high-quality treatment options for people affected by organ failure.”

“This is an important milestone on our path towards effective treatments for acute liver decompensation. The combination of OrganOX’s ELC system and eGenesis’ genetically engineered liver is an important milestone in our path to effective treatment of acute liver decompensation. Our goal is to create a treatment that connects the entire body and provides a lifeline to critically ill patients – time to recover their liver or receive a transplant,” said OrganOX’s Chief Medical Officer. said Professor Peter Friend.

SOURCE: Businesswire

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