yet another approach to the issue of hyperacute rejection.... this is a gross over simplification of the issues, but good stuff nonetheless......
Thursday October 7, 10:54 am Eastern Time
Company Press Release
SOURCE: PPL Therapeutics
PPL Therapeutics Wins $2,000,000 ATP Award for its
Xenotransplantation Program
Application of Gene Targeting and Nuclear Transfer Technologies
Promises Supply of Human Compatible Organs
BLACKSBURG, Va., Oct. 7 /PRNewswire/ -- PPL Therapeutics, one of the world's leading companies in the application of
transgenic technology to the production of human proteins for therapeutic and nutritional applications, today announced that it
had won a $2m ATP (Advanced Technology Program) award from the National Institute of Standards and Technology in the
US. The award will be used to fund a three-year research program aimed at reducing the risk of rapid rejection of pig organs
when transplanted into humans. All of the research funded by this award will be done at PPL's US subsidiary, PPL Inc, based
in Blacksburg, VA.
'Xenotransplantation' offers the opportunity to address the current acute shortage of human organs for transplantation, and
presents a considerable market opportunity estimated to be worth $6.5 billion within 10 years of launch. At present, however,
rapid rejection of non-human organs is a major obstacle that needs to be overcome. Having 'knocked out' the alpha-1, 3
galactosyl transferase (A1, 3GT) gene, which is involved in causing hyperacute rejection (HAR) in pig cells, PPL aims to use
these cells to produce cloned pigs with organs more likely to be accepted by human recipients. PPL plans to use its proprietary
pig nuclear transfer technology to generate transgenic pigs containing the genetic modification.
Dr. Ron James, Managing Director of PPL, said: ''Winning this award is an important boost to our xenotransplantation
program, and we believe it is an endorsement by the US authorities of the leading edge nature of PPL technology in this area.
There is a global shortage of organs for transplantation and we believe that xenotransplantation presents an opportunity to meet
this clinical need, benefiting patients and extending our range of therapeutic products. The ATP award will provide the funding
necessary for a key phase of this important work to take place.''
The research program will build on PPL's announcement in July of the birth of the first livestock carrying genetic modifications
introduced using the Company's novel and extremely efficient process for accurately replacing one selected gene with another
('gene targeting'). As a first step in meeting the goals of this program, PPL has already demonstrated that it can disrupt the A1,
3GT gene in pig cells. The challenge is now to use similarly modified cells to make pigs with no A1, 3GT activity, using nuclear
transfer.
The HAR program is only one element of PPL's xenotransplantation research which addresses each of the immunological
reactions that can prevent the success of transplants from animals to humans: hyperacute rejection, delayed xenograft rejection
and chronic rejection.
Notes to Editors:
1. PPL Therapeutics is a biopharmaceutical company which is one of the
world's leaders in the application of transgenic animal technologies
to the development and production of human proteins for therapeutic
and nutritional applications.
2. PPL has a broad pipeline of diverse protein products including AAT,
the Company's lead product, which is currently in Phase II clinical
trials for cystic fibrosis. AAT is also under development for the
treatment of congenital deficiency. PPL's other lead products are
fibrinogen and bile salt stimulated lipase (BSSL).
3. Transgenic production of human proteins involves the introduction of
copies of human DNA into the genetic material of another species.
The resulting transgenic animals express the human gene product
(protein) in the mammary gland allowing its collection and
purification during lactation. The technique offers the opportunity
to produce human proteins economically and in potentially unlimited
quantities.
4. PPL has a world wide exclusive licence from the Roslin Institute to
use the Institute's intellectual property relating to nuclear
transfer (cloning) in the field of production of proteins for
pharmaceutical and nutraceutical use in the milk of ruminant
livestock and rabbits.
5. Xenotransplantation is the transfer of organs from one species to
another. The fundamental problem with transferring organs between
species is rejection by the recipient's immune system, often
triggered by the product of a single gene in the donor cells. PPL's
HAR program aims to knock out the gene which causes one type of
rejection (hyperacute). This will reduce the risk of rejection, and
allow the development of a stock of transgenic animals containing
the genetic modification which can be used as organ donors for
humans.
SOURCE: PPL Therapeutics |
