The first living recipient of a UThymoKidney, in conjunction
with a heart pump implant, is recovering after a successful
transplant
This transplant builds on two successful UHeart transplants
completed in 2022 and 2023
Please replace the photo for release dated April 24, 2024 with
the accompanying corrected photo.
This press release features multimedia. View
the full release here:
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A) Genetically modified source pig, 16 to
20 weeks old. B) The thymus is then explanted from the source
genetically modified pig, and the thymic tissue is implanted under
its kidney capsule. C) The thymus tissue matures under the kidney
capsule for six to eight weeks. D) The UThymoKidney is procured and
transplanted into human recipient. (Photo: Business Wire)
The release reads:
UNITED THERAPEUTICS ANNOUNCES WORLD’S FIRST
SUCCESSFUL XENOTHYMOKIDNEY TRANSPLANT
The first living recipient of a UThymoKidney, in conjunction
with a heart pump implant, is recovering after a successful
transplant
This transplant builds on two successful UHeart transplants
completed in 2022 and 2023
United Therapeutics Corporation (Nasdaq: UTHR), a public
benefit corporation, today announced the world’s first successful
transplant of a UThymoKidney™, which the company produced, into a
living person on April 12, 2024. This transplant represents several
historic firsts for transplantation:
- The first-ever transplant of a
xenothymokidney into a living human recipient; - The first-ever
combined mechanical heart pump and organ transplant; and - The
first-ever xenotransplant into a living human using only
FDA-approved immunosuppressive medicines
The transplant is the third xenotransplant using United
Therapeutics’ xeno organs, following two successful UHeart™
transplants at the University of Maryland Medicine in 2022 and
2023.
The transplant was authorized by U.S. Food and Drug
Administration (FDA) under the expanded access pathway and
performed by surgeons at NYU Langone Health led by Robert
Montgomery, M.D., DPhil. The patient, a 54-year-old woman from
New Jersey, suffers from heart and kidney failure. The combination
of several chronic medical conditions, coupled with a lack of
available human organs for transplant, prevented her from
qualifying for human heart and kidney transplants.
United Therapeutics’ xenothymokidney, known by the proposed
trade name UThymoKidney, is an investigational-stage xenokidney
from a pig with a single genetic edit, together with tissue from
the same pig’s thymus. The use of the pig’s thymus tissue is
intended to condition the recipient human’s immune system to
recognize the UThymoKidney as “self” and reduce the likelihood of
rejection.
The single genetic modification in the pig is the inactivation,
or “knock-out”, of the gene responsible for the synthesis of
alpha-gal, a sugar on the surface of cells that can cause the
immediate rejection of an organ when transplanted into the human
body. Because tissues from pigs containing this modification do not
contain detectable levels of alpha-gal, United Therapeutics refers
to materials derived from this pig as GalSafe®.
The GalSafe pig was developed by Revivicor, Inc., a subsidiary
of United Therapeutics. In December 2020, this pig line was
approved by the FDA for use as human food or as a potential source
for biomedical purposes, with this being the first investigational
biomedical use in a living human.
“This historic transplant builds on the base of knowledge that
the teams at United Therapeutics and our academic collaborators
have established over the past two decades and demonstrates the
potential utility for xeno organs to revolutionize the way patients
with end-stage organ disease are managed in the future,” said
Leigh Peterson, Ph.D., Executive Vice President, Product
Development & Xenotransplantation at United Therapeutics. “We
look forward to continuing our dialogue with the FDA with the goal
of starting human clinical studies for xenotransplantation in
2025.”
According to the U.S. Health Resources and Services
Administration, around 110,000 Americans are currently waiting for
an organ transplant, and more than 6,000 patients – 17 every day –
die each year before receiving one. More than 89,000 patients are
waiting for kidneys, close to 10,000 for livers, over 3,400 for
hearts, and almost 1,000 for lungs, with many more patients
suffering from end-stage organ failure who are ineligible for the
strict organ transplant waiting list who could benefit from a
readily available supply of organs on demand.
“I am pleased and impressed that decades of research into
expanding the supply of kidneys have resulted in this historic,
successful xenokidney transplant using United Therapeutics' gene
editing and thymokidney technology,” said Dr. Louis
Sullivan, Secretary of the United States Department of Health
and Human Services in President George H.W. Bush’s administration,
member of the United Therapeutics Board of Directors, and Chair of
its Scientific Advisory Board.
“I am so proud of the many scientists and surgeons working with
United Therapeutics on its xenotransplantation programs,” said
Gov. Tommy Thompson, Secretary of the United States
Department of Health and Human Services in President George W.
Bush's administration and member of United Therapeutics' Board of
Directors. “This major breakthrough is a revolutionary step forward
in our quest to create an unlimited supply of transplantable
organs.”
United Therapeutics’ organ manufacturing efforts consist of four
platforms – xenotransplantation, regenerative medicine, 3D organ
bioprinting, and bio-artificial organs - encompassing four
different organs – hearts, kidneys, livers, and lungs. These
groundbreaking programs are intended to address the ongoing
shortage of transplantable organs for patients with end stage organ
disease.
United Therapeutics initiated its xenotransplantation research
work in 2011 and currently employs close to 50 scientists and
support staff advancing xenotransplant science with three different
organ programs: the UHeart xenoheart, the UThymoKidney, a one-gene
modified kidney and thymus, and the UKidney™, a 10-gene modified
kidney. In 2024, the company inaugurated the world’s first
clinical-scale designated pathogen-free facility in Christiansburg,
Virginia to support future clinical xenotransplantation studies
with a capacity of approximately 125 organs per year.
To date, 11 xenotransplantation procedures using United
Therapeutics' UHearts, UThymoKidneys, and UKidneys have been
performed in living and brain-dead human recipients: two living
human recipients of UHearts, one living recipient of a
UThymoKidney, six brain-dead UKidney and UThymoKidney recipients,
and two brain-dead UHeart recipients. United Therapeutics has built
on its history of innovation in xenotransplantation with strong
research collaborations with top academic medical centers including
NYU Langone Health, the University of Maryland Medicine, Johns
Hopkins Medicine, and the University of Alabama at Birmingham.
United Therapeutics is preparing for clinical trials of its
xenokidney, xenothymokidney, and xenoheart products, following
completion of ongoing preclinical studies required by the FDA.
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. Our
public benefit purpose is to provide a brighter future for patients
through the development of novel pharmaceutical therapies; and
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
regarding progress toward developing our organ manufacturing
programs, including our plans to commence clinical trials of one or
more xenotransplantation products in 2025, and our goals of
innovating for the unmet medical needs of our patients and to
benefit our other stakeholders and 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. In particular, our plans to commence
clinical studies of one or more xenotransplantation products in
2025 are subject to regulatory clearance, including the completion
of preclinical studies to the satisfaction of the FDA, and many
other factors that we cannot control. 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 April 24, 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 is a registered trademark of United Therapeutics
Corporation and its subsidiaries.
UHEART, UKIDNEY, and UTHYMOKIDNEY are trademarks of United
Therapeutics Corporation and its subsidiaries.
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version on businesswire.com: https://www.businesswire.com/news/home/20240424110823/en/
Dewey Steadman at (202) 919-4097
https://ir.unither.com/contact-uthr/
Grafico Azioni United Therapeutics (NASDAQ:UTHR)
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