Company targets organ transplant shortage after world’s first successful transplant of genetically edited pig kidney into humans.
Xenotransplantation biotech eGenesis has secured $191 million in Series D financing to advance its lead program focused on developing genetically edited pig kidneys for human transplantation. The funding will enable the Cambridge, MA-based company to move its lead product candidate, EGEN-2784, into first-in-human clinical trials, expand its production capabilities and further its pipeline programs focused on other organs.
Kidney failure, or end-stage renal disease, affects over 800,000 individuals in the US alone, with millions more globally suffering from the condition. Kidney transplantation is widely considered the most effective treatment option, yet the demand for organs far exceeds the available supply. eGenesis says that more than 90,000 people are currently on the kidney waitlist, while only about 25,000 transplants are performed each year, highlighting the need for alternative solutions.
One alternative, xenotransplantation, is defined as the transplantation of living organs, tissues, or cells from one species to another. Pigs have been identified as a good species for xenotransplantation in humans due to their similarity to humans in terms of organ structure and physiology, in addition to the abundance of the species. However, the potential risk of the transmission of viruses between species and organ rejection due to immune system incompatibilities have limited progress in the field.
eGenesis is tackling this challenge by developing human-compatible donor organs using a genome engineering approach, aiming to provide a safer and more effective source of transplantable organs. The company’s technology is designed to address the complex molecular barriers that arise in cross-species transplantation. It incorporates genetic modifications to reduce the risk of organ rejection and prevent viral transmission from donor animals. Preclinical studies have demonstrated promising results, and eGenesis is working on programs for kidney, liver and heart transplants.
Mike Curtis is CEO of eGenesis
In March 2024, the company achieved a significant milestone with the world’s first successful transplant of a genetically modified porcine kidney in a living human patient at Massachusetts General Hospital. The transplanted kidney, known as EGEN-2784, includes three key classes of genetic edits: the removal of certain glycan antigens linked to hyperacute rejection, the addition of seven human genes to regulate immune response and inflammation, and the inactivation of endogenous retroviruses within the porcine genome.
These modifications are designed to make the organ more suitable for human transplantation and reduce the likelihood of rejection. Without the edits, eGenesis says that porcine organs would face immediate rejection by human recipients.
While the company did not provide details on the path to clinical trials, eGenesis CEO Mike Curtis touched on the subject in his interview with Longevity.Technology last year.
“We hear stories all the time of patients who are dying of kidney failure, so there is a population of patients that we could, from a safety, efficacy, and risk-benefit standpoint, support a first trial,” he said. “We’ve now had four meetings with the FDA about our platform, two specifically on our kidney program, so we have good clarity on what the agency is looking for, as far as the first trials go.”
The latest funding round was led by Lux Capital with participation from ARCH Ventures, Khosla Ventures, Farallon Capital Management and Alta Partners. New investors such as DaVita, Eisai Innovation and NATCO Pharmaceuticals also joined the round.
“With this support, we are one step closer to bringing a solution to help patients suffering from organ failure,” said Curtis.
“Decades of progress in cross-species transplantation, accelerated by the evolution of modern genome editing tools and next-generation sequencing, have enabled eGenesis to advance genetically engineered organs to the clinical setting,” said Lux Capital’s Peter Hebert. “What was once science fiction is now science fact. We are thrilled to support eGenesis in advancing this pivotal modality to address one of the most intractable challenges facing medicine today – the global organ shortage crisis.”
READ MORE: Xenotransplantation comes of age
Photo credits: eGenesis and Massachusetts General Hospital