The National Institutes of Health, through the National Center for Advancing Translational Sciences, has awarded Factor Bioscience, a developer of cell-based therapeutics and research tools, 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," said project leader Mark Scott, CSO and director of new model development, Factor Bioscience.
The second project will combine RiboSlice technology with Factor Bioscience'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," said project leader Christopher Rohde, COO and director of high-throughput technologies, Factor Bioscience.
Matt Angel, CEO of Factor Bioscience, added, "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. 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."