The Business of Biofabrication

In a significant development for the field of xenotransplantation, two biotech companies have recently received critical regulatory approvals that could reshape the future of organ transplantation. Following early clinical successes and notable setbacks, industry leaders are now advancing clinical trials aimed at addressing the chronic shortage of human organs for transplant.

The concept of xenotransplantation involves using organs, tissues, or cells from one species to address human transplant needs. Pigs have emerged as the preferred donor species due to their physiological similarities with humans. Advances in gene editing techniques, such as CRISPR, have enabled scientists to modify pig organs—by inserting human genes and removing pig genes—in an effort to reduce immune rejection and viral transmission risks.

United Therapeutics

United Therapeutics, the parent company of Revivicor, recently obtained clearance from the U.S. Food and Drug Administration to initiate the first clinical trial for gene-edited pig kidneys. This follows a landmark procedure in January 2022, when a patient with end-stage heart failure received a genetically engineered pig heart under compassionate use. The upcoming kidney trial, set to begin with six patients suffering from end-stage renal disease, is scheduled to launch mid-2025 and could provide a roadmap for broader regulatory approval.

eGenesis

Similarly, eGenesis has made headway with its platform designed to mitigate molecular incompatibilities between pig organs and the human immune system. Building on a compassionate use study approved in December 2024, the company performed its first genetically engineered kidney transplant earlier this year at Massachusetts General Hospital. The transplant, involving an organ with extensive gene edits, has marked a significant clinical milestone. With additional transplants planned later this year, the study will further assess the viability and long-term benefits of these modifications.

Challenges Remain

Despite these advancements, challenges remain. Previous procedures have underscored the complexity of xenotransplantation, with some early recipients of modified organs not surviving the procedure. The mixed outcomes highlight the need for further research and careful monitoring as clinical trials progress.

The upcoming clinical trials by United Therapeutics and eGenesis are closely watched by the medical community, as they offer promising avenues to overcome longstanding immunologic and virologic hurdles. As data from these trials emerge, they could pave the way for a future where xenotransplantation plays a critical role in addressing the global shortage of donor organs.