eGenesis Provides Clinical Update on EGEN-2784 in Kidney Failure Patients

eGenesis, a biotechnology firm focused on creating human-compatible engineered organs to combat the global organ shortage, has announced updates from its ongoing Expanded Access study of EGEN-2784, a genetically modified porcine kidney aimed at treating kidney failure. Since March 2024, five patients with kidney failure have undergone EGEN-2784 kidney transplants as part of this study in collaboration with the Mass General Brigham health system. Notably, three of these patients have maintained sustained kidney function for over eight months without requiring dialysis. One patient has achieved an impressive 290 days of dialysis independence, setting a new global record for genetically engineered porcine kidney transplants, with the EGEN-2784 kidney continuing to perform effectively.

Additionally, two patients who had EGEN-2784 kidneys functioning for more than eight months later received human donor kidney transplants. This marks the first documented transition from xenotransplantation to allotransplantation and provides significant clinical evidence that receiving a genetically engineered porcine kidney does not hinder the possibility of subsequently receiving a human donor kidney. The journey of one patient, Tim Andrews, is highlighted in a publication in The Lancet, where he achieved 271 days of dialysis independence with an EGEN-2784 kidney before successfully transitioning to a human kidney 82 days after the removal of the xenograft. The human kidney showed immediate functionality, with no signs of clinically significant sensitization or transmission of porcine-derived pathogens.

“These patient outcomes provide important evidence that genetically engineered organs can deliver sustained kidney function, dialysis independence and potentially expand treatment options for patients with kidney failure,” stated Mike Curtis, Ph.D., President and Chief Executive Officer of eGenesis. “Our goal is to make transplantation available to patients when they need it, without being constrained by the limited supply of human donor organs. Together, these experiences provide important clinical evidence as we prepare to advance EGEN-2784 into the Phase 1/2/3 RESTORE study.”

The Expanded Access study forms part of eGenesis’ broader clinical development program assessing the safety and effectiveness of EGEN-2784 and this modality in kidney failure patients. The US Food & Drug Administration (FDA) has authorized eGenesis to launch a Phase 1/2/3 trial (RESTORE) to evaluate EGEN-2784 in 33 patients aged between 50 and 70 years who are on the waiting list for a human donor kidney transplant. eGenesis anticipates starting the RESTORE study in the first quarter of 2027.

EGEN-2784 stands as eGenesis’ primary candidate for kidney transplantation, featuring three classes of genetic modifications aimed at enhancing compatibility and supporting long-term function in human recipients: 1) Elimination of three glycan antigens to prevent hyperacute immune rejection, 2) Insertion of seven human transgenes to modulate immune response, reduce inflammation, improve coagulation compatibility, and regulate complement activation, and 3) Inactivation of endogenous retroviruses in the porcine genome to bolster safety. eGenesis is unique in its approach, being the only company developing organs with all three classes of edits to optimize safety and efficacy.

Dr. Leonardo V. Riella, the medical director for kidney transplantation at MGH, acted as the Principal Investigator, leading the clinical study and the multidisciplinary transplant team. Dr. Tatsuo Kawai, Director of the Legorreta Center for Clinical Transplant Tolerance, served as the lead transplant surgeon, supported by Dr. Nahel Elias, Surgical Director for Kidney Transplantation. “The experience with these patients is providing invaluable insight into the potential role of xenotransplantation for people living with kidney failure,” remarked Dr. Riella. “The sustained periods of dialysis independence are highly encouraging, and the successful transplantation of human donor kidneys following xenotransplantation demonstrates that this approach can preserve future treatment options. These findings provide an important foundation for the next stage of clinical development.”

Over 800,000 Americans and millions globally suffer from end-stage kidney disease (ESKD). While organ transplantation is regarded as the gold standard for enhancing survival and quality of life for those with ESKD, a shortage of organs has resulted in only 28,000 kidney transplants being performed in 2024. For individuals unable to receive transplants, dialysis remains the sole alternative, with a five-year mortality rate of 60%, surpassing the mortality rates of the most prevalent cancers. Genetically engineered porcine-derived kidneys present a hopeful solution to this urgent crisis.

About eGenesis: eGenesis is a clinical-stage biotechnology company focused on developing human-compatible engineered organs to address the severe global organ shortage. The company's proprietary genome engineering platform facilitates extensive, multiplex gene edits to eliminate key biological barriers, incorporate protective human transgenes, and inactivate endogenous retroviruses. EGEN-2784, a genetically engineered porcine kidney, is the company's lead program currently undergoing evaluation in a multi-patient Expanded Access study at MGH. eGenesis is based in Cambridge, MA. For more information, visit www.egenesisbio.com.
Media Contact: Kimberly Ha, eGenesis, kimberly.ha@egenesisbio.com