Genomics development and commercialization specialist firm Synthetic Genomics Inc. (SGI) of La Jolla, CA, and Silver Spring, Maryland-based Lung Biotechnology Inc., a subsidiary of United Therapeutics Corporation, have entered into a multi-year research and development agreement to develop humanized pig organs using synthetic genomic advances. The collaboration will initially focus on developing organs for human patients in need of organ transplants, such as engineered lungs and lung tissues for patients with pulmonary arterial hypertension or other lung diseases.
As part of the agreement SGI will receive royalties and milestone incentives from the development and commercialization of the organs. SGI also announced in May a $50 million equity investment by Lung Biotechnology. Additional financial details were not disclosed.
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 hoping to develop these new methods and advances to create organs that are safe and effective for use in humans.
The project currently awaits construction of a new facility to be located at The Research Triangle Park (RTP) near Durham, Raleigh, and Chapel Hill, North Carolina, home to one of the largest clusters for AgBio, BioTech and life sciences in the U.S. NC BioTech says about 2/3 of NC’s 500+ life science companies are in the Triangle.
According to a report by Laura Oleniacz of the Durham Herald-Sun, the new research and development facility is to be built on 132 acres of land United THerapeutics bought from pharmaceuticals multinational GlaxoSmithKline in 2012 for $17.5 million, with the new development to include renovation of a 150,000 square foot architecturally significant portion of an existing 550 square foot structure known as the Elion-Hitchings Building. The tract purchased from GlaxoSmithKline is contiguous to United Therapeutics’ existing facilities in the park. Ms. Oleniacz reports that construction on the new R&D facility at RTP is targeted to run from 2015 to 2017, but there are still some regulatory and bureaucratic hurdles that need to be negotiated.
Demolition of the unwanted 400,000 square feet of the Elion-Hitchings Building is now complete, including recycling of 78 percent of 38,100 tons of concrete, 7,683 tons of steel, and 215 tons of stainless steel and copper. Part of the new construction 250,000 square foot R&D facility is to house different types of pigs to be utilized in providing lung tissues for transplantation research, incorporating a central hub to simplify food and water delivery to supply the animals. Ms. Oleniacz notes that In 2011 United Theapeutics acquired a Virginia-based Revivicor, a company that focused on genetically engineered pigs to provide, among other things, diabetes treatment and organs and tissues for use in transplant surgery known as “xenografts,” and that while UTC doesn’t have a definitive timeline for completing the project, a company spokesperson indicated that the new, solar powered pathogen-free facility and renovation of the Elion-Hitchings Building will each take about 24 months to execute.
“We are pleased to be partnering with Lung Biotechnology and United Therapeutics to advance organ transplantation,” comments 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, observes that “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.”
The companies note that about 400,000 people die annually from various forms of lung disease including cancer In the United States alone, 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 percent of deaths due to lung failure can be avoided due to the gross shortage of transplantable human lungs. And while 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.
United Therapeutics Corporation
Synthetic Genomics Inc. (SGI)
Lung Biotechnology Inc.
The Durham Herald-sun
Research Triangle Park
Synthetic Genomics Inc. (SGI)