United Therapeutics Announces Historic Achievements in its Xenotransplantation Programs

Downtown Silver Spring-based United Therapeutics has reached two historic achievements in its xenotransplantation programs, the company announced.

The world’s first recipient of an investigational genetically-modified pig heart has reached a two-week milestone, according to the company. University of Maryland School of Medicine (UMSOM) surgeons report continued post-operative cardiovascular improvement in the patient with normal organ function.

The patient, David Bennett, was the first recipient of a heart developed by Blacksburg, Va.-based Revivicor Inc., which is a subsidiary of UT, according to Washington Business Journal.

Additionally, the company announced that the first peer-reviewed publication of a similarly gene-edited investigational xenograft — a kidney — in a human preclinical model at the University of Alabama at Birmingham Marnix E. Heersink School of Medicine (UAB), was published this week in the American Journal of Transplantation.

Both the heart and kidney surgeries came after the Food and Drug Administration granted one-time emergency use authorizations, according to WBJ. “UOrgans like the UHeart, UThymoKidney, and UKidney are not approved by the FDA,” the company said. “The UHeart transplant conducted by surgeons at UMSOM and UMMC was authorized by the FDA for the treatment of the individual patient. UT hopes to complete clinical trials for its xeno UOrgan products in the coming years and, if approved by FDA, provide a source of organ transplants for the hundreds of thousands of patients unable to achieve them through the organ transplant list.”

UT also said in the announcement that more than 200 human donor lungs have been successfully transplanted after being saved from disposal by UT’s subsidiary Lung Bioengineering at its facilities in Silver Spring and on the Mayo Clinic campus in Jacksonville, Florida.

“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),” the announcement reads. “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.”

“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, in a statement. “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.”

Dr. Rothblatt noted three key mentors that made key contributions to UT’s recent xenotransplantation successes:

  • Dr. Tom Starzl, who served on UT’s Scientific Advisory Board until his passing in 2017, and taught how the body can be induced to tolerate xenografts;
  • Sir Magdi Yacoub, who continues to serve on UT’s Scientific Advisory Board and guides the company through multiple transplantation technologies; and
  • Dr. Craig Venter, whose team at Synthetic Genomics (now UT’s Exponential Biotherapeutic Engineering group) provided essential porcine gene engineering expertise.

An ethics debate ensued after news about the patient’s past came to light shortly after the transplantation procedure. Bennett was convicted in 1988 of stabbing a man, leaving him paralyzed until he died in 2007. “Edward Shumaker had spent the next 19 years using a wheelchair, before he had a stroke in 2005 and died two years later — one week before his 41st birthday,” The Washington Post reported.

Photo: “File:United Therapeutics 05.jpg” by Farragutful is licensed under CC BY-SA 4.0

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