United Therapeutics Announces the First Comprehensive Review Publication on Xenotransplantation

Review manuscript available in the journal Physiological Reviews

United Therapeutics Corporation (Nasdaq: UTHR), a public benefit corporation, today announced the online availability of a review manuscript titled “Physiological Basis for Xenotransplantation from Genetically-Modified Pigs to Humans: A Review,” in the journal Physiological Reviews. The manuscript is the first detailed overview of the current state of the xenotransplantation industry and, in one place, collects the vast history of the technology used to generate genetically engineered pigs and the research conducted to date on the xenotransplantation of porcine hearts and kidneys.

The manuscript can be found at Physiological Reviews using the following URL: https://doi.org/10.1152/physrev.00041.2023

Xenotransplantation is one of four organ manufacturing platforms being developed by United Therapeutics, the goal of which is to provide an unlimited supply of tolerable, transplantable organs to patients with end-stage organ disease.

“We’ve seen the collective efforts of scientists over the last several decades lead to meaningful scientific advances, from animal cloning to gene editing, that has allowed the industry to enhance the suitability of porcine organs for human use,” said Leigh Peterson, Ph.D., Executive Vice President, Product Development and Xenotransplantation at United Therapeutics and lead author of the manuscript. “We believe the continued progress by United Therapeutics and our scientific collaborators will lead to the regular use of porcine organs to help patients in need.”

The authors of the manuscript are Leigh Peterson, Ph.D.; Sir Magdi Yacoub, OM, FRS; David Ayares, Ph.D; Kazuhiko Yamada, M.D., Ph.D; Daniel Eisenson, M.D.; Bartley Griffith, M.D., FACS, FRCS; Muhammad Mohiuddin, MBBS; Will Eyestone, Ph.D; J. Craig Venter, Ph.D; Ryszard T. Smolenski, M.D., Ph.D, and Martine Rothblatt, Ph.D.

United Therapeutics has three xenotransplantation programs in development with the goal to provide hearts and kidneys to patients in need. The UHeart™ is a heart from 10-gene modified pig, the UKidney is a kidney from a 10 gene-modified kidney, and the UThymoKidney™ is a kidney and thymus from a one gene-modified GalSafe™ pig.

Beyond xenotransplantation, United Therapeutics has six other organ manufacturing programs in the lung, kidney, and liver using three distinct platforms: regenerative medicine, bio-artificial organs, and 3-D organ printing. United Therapeutics announced in January 2024 that the U.S. Food and Drug Administration cleared its miroliverELAP™ therapy for enrollment of a phase 1 study in patients with acute liver failure which is expected to occur in 2024. United Therapeutics believes that this study will be the first human clinical trial of a manufactured organ.

United Therapeutics: Enabling Inspiration

At United Therapeutics, our vision and mission are one. We use our enthusiasm, creativity, and persistence to innovate for the unmet medical needs of our patients and to benefit our other stakeholders. We are bold and unconventional. We have fun, we do good. We are the first publicly-traded biotech or pharmaceutical company to take the form of a public benefit corporation (PBC). Our public benefit purpose is to provide a brighter future for patients through (a) the development of novel pharmaceutical therapies; and (b) technologies that expand the availability of transplantable organs.

You can learn more about what it means to be a PBC here: unither.com/pbc.

Forward-Looking Statements

Statements included in this press release that are not historical in nature are “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking statements include, among others, statements related to our goal of providing an unlimited supply of tolerable, transplantable organs to patients with end-stage organ disease; our expectation that its xenotransplantation efforts, and those of our scientific collaborators, will lead to the regular use of porcine organs to help patients in need; our plan to commence a phase 1 study of miroliverELAP; and our goals of innovating for the unmet medical needs of our patients and to benefit our other stakeholders, furthering our public benefit purpose of developing novel pharmaceutical therapies and technologies that expand the availability of transplantable organs. These forward-looking statements are subject to certain risks and uncertainties, such as those described in our periodic reports filed with the Securities and Exchange Commission, that could cause actual results to differ materially from anticipated results. Consequently, such forward-looking statements are qualified by the cautionary statements, cautionary language and risk factors set forth in our periodic reports and documents filed with the Securities and Exchange Commission, including our most recent Annual Report on Form 10-K, Quarterly Reports on Form 10-Q, and Current Reports on Form 8-K. We claim the protection of the safe harbor contained in the Private Securities Litigation Reform Act of 1995 for forward-looking statements. We are providing this information as of March 26, 2024, and assume no obligation to update or revise the information contained in this press release whether as a result of new information, future events, or any other reason.

GALSAFE, MIROLIVERELAP, UHEART, UKIDNEY, and UTHYMOKIDNEY are trademarks of United Therapeutics Corporation and its subsidiaries.

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