Press Release: Pig Organs to Revolutionize Transplantation Field

[Clark]

http://ir.unither.com/releasedetail.cfm?ReleaseID=845454

Synthetic Genomics Inc. Signs Collaborative Research and Development Agreement with Lung Biotechnology Inc., a Subsidiary of United Therapeutics Corporation, to Develop Humanized Pig Organs to Revolutionize Transplantation Field
Lung Biotechnology Also Makes $50M Equity Investment in SGI

LA JOLLA, Calif. and SILVER SPRING, Md., May 6, 2014 /PRNewswire/ -- Synthetic Genomics Inc. (SGI), a privately held company developing and commercializing genomic driven advancements in a variety of industries, and Lung Biotechnology Inc., a subsidiary of United Therapeutics Corporation (NASDAQ: UTHR), today announced they have entered into a multi-year research and development agreement to develop humanized pig organs using synthetic genomic advances. The collaboration will focus upon developing organs for human patients in need of transplantation, with an initial focus on lung diseases. As part of the agreement SGI will receive royalties and milestone incentives from the development and commercialization of the organs.

SGI is also announcing a $50 million equity investment by Lung Biotechnology. Additional financial details were not disclosed.

"We are pleased to be partnering with Lung Biotechnology and United Therapeutics to advance organ transplantation," said J. Craig Venter, Ph.D., Founder and CEO, SGI. "We believe that our proprietary synthetic genomic tools and technologies, coupled with United Therapeutics' knowledge and advances in regenerative medicine technologies and treatment of lung diseases, should enable us to develop humanized pig organs for safe and effective transplant into humans. We believe this is one of the most exciting and important programs ever undertaken in modern medical science."

Martine Rothblatt, Ph.D., United Therapeutics Chairman and CEO, added, "Our new collaboration with Synthetic Genomics is huge for accelerating our efforts to cure end-stage lung disease. Our combined expertise should enable us to develop an unlimited supply of transplantable organs, potentially helping millions of patients who die from end-stage organ 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 humanized lungs. With the science and technology advances made by the SGI team in recent years, the companies are striving to develop these new methods and advances to create organs that are safe and effective for use in humans. 

In the United States alone, about 400,000 people die annually from various forms of lung disease including cancer, but scarcely 2,000 people are saved with a lung transplant and only about 2,000 are added to the transplant wait list annually. Not even 1% of deaths due to lung failure can be avoided due to the gross shortage of transplantable human lungs. 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.