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Clean and mean cells
CAMBRIDGE, Mass.—Seeking to replace traditional reprogramming methods with targeted genome engineering, Cellectis bioresearch, a subsidiary of the Cellectis Group and a genome-engineering specialist, has partnered with Stemgent Inc., a developer of cellular reprogramming tools for leading stem cell researchers worldwide.
Both companies have developed extensive experience in stem cells—namely, induced pluripotent stem (iPS) cells. Under an agreement announced March 7, the companies will equip stem cell researchers with tools that generate genome-engineered iPS cells for use in their research and regenerative medicine by marrying Cellectis' genome engineering expertise with Stemgent's work in cellular reprogramming.
Cellectis developed a high-throughput synthesis platform that is capable of synthetizing thousands of such molecules each year: TALEN, which uses synthetic molecules made of a fusion of an engineered transcription factor and an endonuclease. TALEN masters specifity issues to avoid off-target effects and toxicity, and enables researchers to overcome epigenetic modification, like cytosine methylation, that usually abolishes binding and cleavage.
"In addition to the perfect match between the two companies' technologies, the management team from both sides share the same vision on the way of developing future applications using iPS cells," says Jean-Charles Epinat, deputy CEO of Cellectis. "Generating 'clean' iPS cells is a prerequisite for clinical applications involving iPS cells, and thus all clinical applications in regenerative medicine will benefit from this technology."
While traditional reprogramming methods can lead to the integration of unwanted genetic material into the host genome, disrupting a reprogrammed cell's function, Stemgent's proprietary mRNA reprogramming technology addresses the challenges around deriving non-viral, non-integrating, clinically relevant iPS cells for use in regenerative medicine, drug discovery and basic research, says Dr. James Kehler, director of global services at Stemgent.
"We wanted to go beyond the ability to recruit and reprogram patient samples of interest," says Kehler. "We thought there would be a real need for a group to validate drug targets or introduce precise genomic modifications for an iPS line. This partnership struck us as a very rapid way of doing precise genetic studies and a pragmatic approach to working with established leaders in the field."
Stemgent's technology will be integrated into Cellectis' iPS Hub reprogramming platform. The end result will be a "one-stop shop" for patient/donor biopsies to be used for iPS cells by academic and pharma/biotech customers, says Epinat.
"No doubt, in the near future, drug testing will be heavily based on iPS cells," he says. "Cellectis is already offering, for instance, human cardiomyocytes and hepatocytes for drug-toxicity applications and looking to develop additional products for drug screening and testing that reflect the physiopathology of diseases. Regenerative medicine applications are the natural extension, and Cellectis is building capacities first in generating clinical-grade iPS cell lines, which are the starting materials for any future clinical studies."
"I do believe that while in the longer term, regenerative therapies using iPS cells will come to the clinic in a global context, the more ready achievement will be the development of new assays utilizing iPS-derived cells from a range of patients who can add value early in the screening process to help identify off-target toxicities," Kehler concludes.