Our platform, called the Gene Expression Modulation System (GEMS), combines a DNA-binding protein, a customized guide RNA, and any of a wide array of modulator proteins to create a powerful new type of genetic medicine.
Additionally, we hold an exclusive license to the miniature DNA-binding protein called CasMINI, which is the smallest and most deliverable dCas protein to work in human cells. The compact size of CasMINI will enable even greater potential for in vivo delivery and multigene disease treatment.
A modular platform for epigenetic editing
We are using advanced data science, genetic and protein engineering, and functional genomics to expand our toolkit of GEMS components and to optimize the combination of all components into epigenetic therapies.
1MODULATORS
The current universe of modulators is limited and does not address all targets. Therefore, we have built the largest existing library of transcriptional and epigenomic modulators, engineered to be compact and deliverable in any single vector. Our modulator toolkit includes novel activators and repressors to precisely control dosage of targeted genes. It also includes writers and erasers of novel epigenomic modulators — such as histone modifiers, DNA (de)methylators, and chromatin remodelers — to introduce persistent changes to gene expression. Furthermore, with a vast library of modulators, we can design endless combinations of modulators to drive the desired behavior for target genes.
2DNA-BINDING PROTEINS
We are using gene and protein engineering to optimize Cas DNA-binding proteins for increased potency and specificity, decreased immunogenicity, better deliverability, and expandability to new applications.
3GUIDE RNAs
We have established a novel functional screening platform to rapidly identify guide RNAs that enable precise and specific gene targeting in indications of interest and that minimize the potential for off-target effects.
Platform features
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Largest known library of novel modulators
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Exclusive license to CasMINI. A growing library of proprietary, ultracompact and safe Cas proteins, including CasONYX (used in EPI-321), all engineered to work in human cells enabling in vivo gene therapy
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Proprietary computational and experimental methods for designing and engineering guides to enhance on-target efficacy and mitigate off-target binding of GEMS
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Advanced functional and computational genomics technology for the overall design and optimization of effector, modulator, and gRNA constructs
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Multiplex gene modulation, with the possibility of multidirectional gene modulation