Humanized pig organs to revolutionize transplantation

Initial focus: the almost 400,000 people who die from lung disease, including cancer, each year
May 7, 2014

(Credit: iStock)

Genome pioneer J. Craig Venter’s Synthetic Genomics Inc. (SGI) is teaming up with United Therapeutics Corporation subsidiary Lung Biotechnology Inc. to use synthetic genomic advances to develop humanized pig lungs.

The collaboration will focus on creating organs that are safe and effective for use in human patients in need of transplantation, with an initial focus on lung diseases — addressing specifically the urgent need for transplant organs for people with end-stage lung disease.

SGI  plans to use its unique DNA design, DNA synthesis, genome editing, and genome-modification tools to develop engineered primary pig cells with modified genomes. This will involve modifying a substantial number of genes at an unprecedented scale and efficiency, the company says.

400,000 people die annually from lung disease

United Therapeutics will leverage its xenotransplantation (between-species) expertise to implant these engineered cells, generating pig embryos that are born with humanized lungs.

“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,” said said J. Craig Venter, Ph.D., Founder and CEO, SGI. “We believe this is one of the most exciting and important programs ever undertaken in modern medical science.”

“Our new collaboration with Synthetic Genomics is huge for accelerating our efforts to cure end-stage lung disease, Martine Rothblatt, Ph.D., United Therapeutics Chairman and CEO, added. “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.”

More than 35 million Americans are living with chronic lung disease, and almost 400,000 people die from lung disease, including cancer, each year, according to the American Lung Association, due primarily 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.

SGI also announced a $50 million equity investment by Lung Biotechnology. As part of the agreement, SGI will receive royalties and milestone incentives from the development and commercialization of the organs.