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A LEAP forward for stem cell research
LA JOLLA, Calif.—The Sanford-Burnham Medical Research Institute, a world leader in stem cell research, has entered into a collaboration agreement with leading synthetic biology company Intrexon Corp. The partnership will focus on rapidly accelerating induced pluripotent stem cell research by giving world-class researchers access to innovative, proprietary technology platforms that are not widely available.
The agreement grants Sanford-Burnham access to Intrexon's Laser-Enabled Analysis and Processing (LEAP) instrument and RheoSwitch Therapeutic System (RTS). These sophisticated cellular selection and gene regulation technologies, respectively, are proprietary to Intrexon and are not currently available on the market. In exchange, according to the terms of the agreement, Intrexon may gain commercial and intellectual property rights resulting from technological advances made under the auspices of the collaboration.
Induced pluripotent stem cells (iPSCs) are stem cells derived from adult rather than embryonic cells. Sanford-Burnham is currently building the largest collection of iPSCs in the world from individual patients and volunteers.
The LEAP instrument is Intrexon's proprietary automated system that provides high-throughput cell imaging and processing. The instrument images cells right in their plates, where computational software is able to identify cell types based on their shape and chemistry. Undesired cell types can be targeted and eliminated in situ by way of a laser system.
"The laser fires 1,000 shots per second to eliminate undesirable cells without touching cells of interest," says Dr. Fred Koller, vice president of business development at Intrexon and co-inventor of the LEAP instrument. "We have the only technology today that's able to purify adherent cells directly in the dish."
The LEAP instrument will allow greater efficiency of iPSC cultures, and will allow cell lines to be developed much more quickly. Standard methods typically require two plates to generate one cell line.
"The bottleneck for progress has been how many colonies you can generate with limited technicians," says Dr. Yang Liu, manager of the Stem Cell Research Institute at Sanford-Burnham. "Thanks to this collaboration, we will have access to software that will enable high-throughput plates— allowing the possibility of multiple cell lines on a single plate."
Using the LEAP instrument to develop iPSCs promises a vastly improved method because researchers will have no need to take cells out of the well.
"This will allow us to save reagents, while also reducing the risk of contaminations," says Liu.
The LEAP instrument will help researchers to achieve the next step in stem cell development, which is cell differentiation. The typical process of transitioning iPSCs into a desired cell type has about a 10-percent efficiency rate, resulting in an impure culture that contains roughly 10 parts the desired cell type and 90 parts something else. The laser-based processing affording by the LEAP instrument will allow researchers to purify the cells directly in the adherent state, yielding a sample containing only the desired cell type.
"If we can achieve this, we can create and label a pure sample of a desired cell type," says Liu. This is especially exciting for the prospect of growing pure pancreatic and liver cells, among others.
The agreement also provides Sanford-Burnham access to Intrexon's RTS technology, a proprietary ligand-activated biological "switch" that allows researchers to turn gene expression on or off. This will allow Sanford-Burnham scientists to regulate when certain genes are expressed in cells, which has several promising applications with regard to iPSCs.
"We're interested in the RTS technology because it will help us to turn genes on or off in stem cells that have been transplanted," Dr. Evan Y. Snyder, director of Sanford- Burnham's Stem Cell Research Center and Stem Cell and Regenerative Biology Program, said in a media release about the agreement. "For example, it can be used for therapeutic protein expression in stem cells that home to and help eradicate brain tumors."
Sanford-Burnham is a nonprofit medical research institute with locations in California and Florida. It consistently ranks among the top five organizations in the world for its scientific impact in the fields of biology and biochemistry. Sanford-Burnham's Stem Cell Research Center, nicknamed the "Stem Cell Core," provides its resources and services to researchers at nonprofit and for-profit organizations around the world. Sanford-Burnham and its partners are pursuing iPSCs for a variety of prospective uses, including drug development or potentially using culture-born cells to treat disease directly.
Intrexon is a privately held biotechnology company focused on the industrial engineering of synthetic biology.
"We're a private company that's trying to get biotech into new spaces beyond healthcare and agriculture—into animal sciences and the energy sector, for example," says Koller. "Here, we're engaging in a different market, and iPSCs are something we believe in. There are technical hurdles to overcome yet, but we think our technology will be enabling in the effort to create the next wave of therapies."