Makana™ Issued Patent in South Korea for Its Genetically-Modified TKO Pig for Use in Xenotransplantation
May 01 2024 - 10:00AM
Business Wire
- Company in discussions with U.S. Food and Drug
Administration regarding first-ever human clinical trial as a
solution to help solve the organ donor shortage crisis
Makana™ Therapeutics, a global leader in the field of
xenotransplantation, was granted an important patent in South Korea
that will catalyze xenotransplantation efforts in that country, the
company announced today. Makana was granted a similar patent in
Europe in 2023.
Makana is working to solve the organ shortage crisis by making
genetically-modified pigs for use as organ donors for human
recipients. The South Korean patent was granted on Makana’s Triple
Knockout pig, or “TKO,” which is a combination of three xenoantigen
gene knockouts in the pig that effectively camouflage the
cross-species grafts from the human recipient’s immune system.
Makana has demonstrated compelling results in
xenotransplantation to date. “Our knockout pigs combined with our
advancements in immunosuppression and patient matching have
resulted in the longest and most consistent preclinical survival
data in the xenotransplantation field,” said Mark Platt, the
company’s President and Chief Executive Officer. “We are very
pleased that the Triple Knockout Pig, which was discovered in the
lab of our founder, Dr. Joe Tector, is being adopted as the ‘gold
standard’ of baseline genetics for xenotransplantation.”
On March 21, 2024, Mass General Hospital announced that it had
transplanted a genetically modified pig kidney into a patient
suffering from End Stage Renal Disease. The procedure was performed
under the expanded access pathway or “compassionate use” which
allows a single patient to receive an investigational medical
product when facing an immediate life-threatening condition. The
pig kidney used in the case contained the baseline TKO genetics
along with other modifications. The earlier reported “compassionate
use” cases of pig heart xenotransplants at the University of
Maryland School of Medicine also incorporated the TKO genetics
discovered by Makana. While compassionate use authorizations may
help a small number of patients, Makana remains focused on pursuing
a clinical trial to help the entire population that suffers from
organ failure.
Platt added, “The Triple Knockout Pig has fundamentally changed
the field and has moved xenotransplantation closer to becoming a
clinically available reality. The organs from our TKO Pig have been
shown to match more than 30% of patients waiting for a kidney
transplant, and likely more than 70% of patients can benefit from
these organs with available pre-transplant treatment. Our team is
working tirelessly to reach our goal of allowing every patient
suffering from organ failure to return to an entirely normal
life.”
According to the National Kidney Foundation, there were 786,000
patients in the U.S. living with kidney failure in 2021. In 2022,
there were approximately 25,000 kidney transplants performed in the
U.S. However there are still an estimated twelve people dying each
day in the U.S. while waiting to receive a life-saving kidney
transplant. Currently there are more than 93,000 people on the U.S.
kidney transplant waitlist. This has been an enduring challenge for
organ transplantation, with the waiting list substantially larger
than the supply of donor organs.
Makana is in discussions with the U.S. Food and Drug
Administration regarding the final steps prior to conducting the
first-ever human clinical trial in kidney xenotransplantation. The
trial is expected to be conducted at the University of Miami. Dr.
Tector, MD, PhD, FACS, a practicing transplant surgeon, heads up
the xenotransplant program at the Miami Transplant Institute (MTI),
a collaboration between Jackson Memorial Hospital and the
University of Miami Health System.
“The TKO pig is widely seen as the preferred genetic profile
that will enter clinical trials,” Platt said. “We’ve been
diligently driving our work to its clinical stage for more than a
decade. We will profoundly improve both the length and quality of
life for our patients, beginning with Patient One.”
About Makana Therapeutics
Founded in 2009, Makana Therapeutics is focused on developing
swine with reduced xenoantigen expression, making human
transplantation of cells, tissues and organs from these animals
possible. Makana's focus on simplified genetics, optimized pig
cloning techniques and careful patient selection is expected to
streamline product development and result in safer more efficacious
products. For more information on Makana, please visit
www.makanatherapeutics.com.
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