United Therapeutics Corporation Announces Historic Achievements in its Xenotransplantation Programs

United Therapeutics Corporation (UT) (Nasdaq: UTHR), a public benefit corporation with a purpose to provide a brighter future for patients, announced today that the world's first recipient of an investigational genetically-modified xenotransplanted organ, UT’s UHeart, reached a two-week milestone. University of Maryland School of Medicine (UMSOM) surgeons report continued post-operative cardiovascular improvement in the patient with normal organ function. In addition, the first peer-reviewed publication of a similarly gene-edited investigational xenograft, UT’s UKidney, in a human preclinical model at the University of Alabama at Birmingham Marnix E. Heersink School of Medicine (UAB) was published yesterday in the American Journal of Transplantation.

These major medical milestones come on the heels of the September 2021 historic transplant of UT’s UThymoKidney at New York University Langone Health (NYU). That human preclinical model proved for the first time that UT’s GalSafe pig could, as modified, transcend the most proximate immunological barriers to xenotransplantation. The GalSafe pig was approved by the U.S. Food and Drug Administration (FDA) for human food and as a potential source for biomedical use in December 2020. These achievements rely on UT’s development, through its Revivicor subsidiary, of genetically modified pigs that are designed to provide a supply of organs for people who are unable to receive human organ donations.

Also relevant to UT’s organ development efforts are:

• Drone delivery of organs. UT’s historic October 2021 first-ever delivery of a transplanted lung by electric drone at Toronto General Hospital, demonstrating the feasibility of UT’s goal to deliver its transplantable organs with zero carbon footprint aircraft; and
• Ex-vivo lung perfusion. More than 200 human donor lungs successfully transplanted after being saved from disposal by UT’s subsidiary Lung Bioengineering at its facilities in Silver Spring, Maryland and on the Mayo Clinic campus in Jacksonville, Florida.

“It is enormously gratifying to see these xenotransplantation breakthroughs achieved after working on this for over twenty years,” said Martine A. Rothblatt, Ph.D, Chairperson and Chief Executive Officer. “At UT, we are relentlessly pursuing our goal of producing an unlimited supply of transplantable organs from xenotransplantation, regenerative medicine, and 3D bioprinting technology, and we expect to make additional breakthrough announcements in each of these fields in the coming years. Indeed, we have commenced work on a large clinical-quality organ facility to support upcoming UHeart and UKidney clinical trials.”