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Synthetic Genomics expands research and development deal with Lung Biotechnology PBC

Synthetic Genomics (SGI) and Lung Biotechnology PBC, a subsidiary of United Therapeutics Corporation, have expanded their multi-year research and development agreement to develop transplantation-ready pig organs using synthetic genomic advances.

The companies initially focused efforts on lung diseases and will now also include kidney diseases. As part of the agreement, SGI will receive royalties and milestones from the development and commercialization of the organs.

SGI is also announcing an additional $50 million equity investment by Lung Biotechnology. Financial details were not disclosed. Lung Biotechnology previously invested $50 million in SGI preferred stock in April, 2014.

SGI CEO Oliver Fetzer said: "We are excited to expand our collaboration with United Therapeutics with the goal to provide organs to patients in high need.

"The progress on the program to date using SGI’s proprietary cell engineering technology has enhanced our confidence and will now be expanded to an additional transplantable organ. We look forward to deepening the collaboration with our partner United Therapeutics and to combine our expertise to help these patients."

United Therapeutics Chairman and Co-CEO Martine Rothblatt said: "Our expanded collaboration with Synthetic Genomics is significant for applying our growing xenotransplantation science platform to the problem of end-stage renal failure. Our combined expertise will accelerate our efforts to develop an expanded supply of transplantable kidneys, potentially helping tens of thousands of patients suffering from incurable kidney disease."

Using unique DNA design, DNA synthesis and genome editing, as well as genome modification tools, SGI will develop engineered primary pig cells with modified genomes. This work will entail modification of a substantial number of genes at an unprecedented scale and efficiency.

United Therapeutics will leverage its xenotransplantation expertise to implant these engineered cells, generating pig embryos which develop and are born with transplantable organs. With the science and technology advances made by the SGI team over the last years, the companies are striving to develop these new methods and advances to create organs that are safe and effective for use in humans.

Treatments for kidney failure include dialysis and transplantation, and there were more than 600,000 people with end-stage kidney disease under treatment in the United States during 2012. More than 50,000 people are added to the kidney transplant wait list each year with more than 100,000 total awaiting a kidney transplant in the United States. The tremendous shortfall of donor organs, results in only about 17,000 United States kidney transplants annually.

Previous attempts to rectify this shortage with animal organs have failed due to genomic incompatibilities, especially with respect to immune and coagulation systems. The collaboration between Synthetic Genomics and Lung Biotechnology aims to eliminate these genomic incompatibilities.