CAMBRIDGE, Mass.—Organ transplantation is held up by one major factor, which is the availability and accessibility of compatible human organs. To be honest, the scarcity of human organs in general is a key bottleneck, beyond the issue of the whether an individual patient might reject an organ.

One company trying to address the global organ shortage is eGenesis, a biotechnology company using gene-editing technologies for the development of safe and effective human-compatible organs, tissues and cells.

Very recently, the company announced the start of a research collaboration with the Duke University School of Medicine in which the two organizations will evaluate gene-edited pancreatic islet cells in non-human primate recipients as a prerequisite to advancing to human clinical trials. This collaboration is in addition to an existing eGenesis partnership with Massachusetts General Hospital, initiated in 2017.

“There are 1.6 million Americans who live with type 1 diabetes and whose quality of life is greatly impacted by the monitoring of their glucose levels and the need for multiple insulin injections on a daily basis,” said Dr. Allan Douglas Kirk, who holds professorships in surgery, pediatrics and immunology at Duke . “With advancements in gene editing technology, there is now the potential of developing and safely transplanting human-compatible xeno-islet cells, which could allow these patients to reduce or eliminate their need for glucose monitoring and insulin injections. The research we will conduct at Duke will help determine whether a minimally-invasive approach into human clinical studies might be possible.”

Added Dr. Michael Curtis, president of research and development of eGenesis: “eGenesis’ mission is to develop human-compatible organs, tissues and cells to alleviate the organ shortage crisis and to improve the health and quality of life of all patients who could benefit from transplant. This collaboration with Duke, a leading transplant center with deep expertise in immunology and diabetes, will accelerate our research and provide validation of our xeno-islet cell program, leading to the evaluation in human clinical trials in patients with type 1 diabetes. We look forward to working with our new colleagues to advance the field of organ, tissue, and cell transplantation.”

The concept of xenotransplantation, or the transplantation of organs, tissue and cells from one species to another, has been explored for several decades, with the pig considered the most suitable donor species for humans. However, challenges related to molecular incompatibilities between species as well as virologic concerns have stymied the advancement of the field. The goal of eGenesis is to advance the field of transplantation and make available safe and reliable xeno organs, tissues and cells to patients in need.

The company uses gene editing technology such as CRISPR to directly address the key virology and immunology hurdles that have impeded xenotransplantation to date. Currently, eGenesis is advancing an initial product toward the clinic for kidney transplant, with the longer-term potential of addressing a broader organ recipient population and expanding the applicability of xenotransplantation into other areas such as cell therapy.