CAMBRIDGE, Mass., Nov. 13, 2012 /PRNewswire/ -- The National Institutes of Health, through the Office of the Director and the National Center for Advancing Translational Sciences, has awarded Factor Bioscience two Small Business Innovation Research (SBIR) grants to accelerate the development of new treatments for Alzheimer's Disease.
The first project will use Factor's patent-pending RiboSlice gene-editing technology to generate rodent models of Alzheimer's. "The unprecedented efficiency of RiboSlice enables the rapid generation of complex, defined mutations in rodent cells. With this technology, we can create models that exhibit exceptional similarity to human disease," commented project leader Mark Scott, Chief Science Officer and Director of New Model Development.
The second project will combine RiboSlice technology with Factor's patent-pending integration-free reprogramming and directed-differentiation technologies to generate a library of human neural cells containing defined mutations in Alzheimer's-associated genes. "This library will enable the first well-controlled high-throughput screens using human neural cells with an Alzheimer's phenotype. We believe that this library has the potential to dramatically accelerate the identification of new Alzheimer's drug candidates by shifting high-content efficacy testing to an earlier stage of the drug-development process," explains project leader Dr. Christopher Rohde, Chief Operating Officer and Director of High-Throughput Technologies.
"Alzheimer's research has been held back by a lack of good models. These models are the critical missing links needed to understand the fundamental biology of Alzheimer's and to develop effective treatments," says Dr. Matt Angel, CEO. "Factor's technologies provide extraordinary control over both the genome and epigenome of cells, allowing us to insert and delete genes, introduce defined mutations, and control cell type. With the support of the National Alzheimer's Plan, we will focus these powerful new methods on Alzheimer's Disease to elucidate and advance new, more effective treatments."
About Factor Bioscience
Founded in 2011, Factor Bioscience develops cell-based therapeutics and research tools using its patent-pending gene-editing, reprogramming, and directed-differentiation technologies. For more information, visit www.factorbio.com, and follow us on Twitter at @factorbio.
This research is supported by the Office of the Director and the National Center for Advancing Translational Sciences, National Institutes of Health, under Award Numbers R43OD016687 and R43TR000644. The content of this press release is solely the responsibility of the authors and does not necessarily represent the official views of the National Institutes of Health.
CEO of Factor Bioscience,