Platform

Platform

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.

Platform
GEMS therapies can precisely modulate gene expression both in vivo and ex vivo. They are mutation-agnostic, meaning that one GEMS construct can address most or all genetic subtypes of a disease. And they are modular, allowing us to create an effectively infinite range of combinations that can be tailored to specific therapeutic needs.

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 engineering

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.

The current universe of modulators is limited and does not address all targets. Therefore, we have built the largest existing library of transcriptional and epigenetic modulators, which we are continually expanding with our discovery screening platform and dedicated research efforts to design novel modulators. Our modulator toolkit includes novel activators and repressors to precisely control dosage of targeted genes. It also includes writers and erasers of novel epigenetic 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.

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.

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

  • Largest known library of novel modulators

  • Exclusive license to CasMINI, most compact dCas DNA-binding protein created to work in human cells enabling delivery via a single AAV vector

  • Proprietary computational and experimental methods for designing and engineering CasMINI guides

  • Advanced functional and computational genomics technology for the overall design and optimization of effector, modulator, and gRNA constructs

  • Multiplex and multidirectional gene modulation possible